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src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F217c874543101839aacf463159b863d6e797d4cf-1200x800.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="object-contain" loading="lazy"/></noscript></span></a></div><div class="border-t-gray-100 border-t-2 flex-auto py-2 "><span class="text-sm text-gray-500 py-2">July 26th 2024</span><div class="py-3"><a class="lg:text-[20px] text-[19px] font-[500]" href="/view/hemophilia-a-gene-therapy-superior-to-standard-of-care-in-phase-3-trial">Hemophilia A Gene Therapy Superior to Standard of Care in Phase 3 Trial</a></div><div class="pb-2"><div><span class="text-md "><span class="mr-1 italic">By </span><a class="mr-1 text-sky-800 hover:text-primary" href="/authors/laura-joszt"><i>Laura Joszt, MA</i></a></span></div></div><a href="/view/hemophilia-a-gene-therapy-superior-to-standard-of-care-in-phase-3-trial"><a href="/view/hemophilia-a-gene-therapy-superior-to-standard-of-care-in-phase-3-trial"><p class="mt-1 text-gray-800 text-[13px] line-clamp-6 text-hidden">The gene therapy giroctocogene fitelparvovec demonstrated superiority compared with the standard of care, routine prophylaxis, in hemophilia A treatment.</p></a></a></div></div><div class="flex-wrap w-[60%] flex "><a class="flex w-[50%] flex-col sm:pl-[35px] pl-[20px] pb-[35px] " href="/view/field-study-highlights-differences-in-factor-ix-variant-lab-results"><div class="w-full lg:h-[134px] h-[100px] relative bg-default-logo-background "><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:absolute;top:0;left:0;bottom:0;right:0"><img alt="" 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/_next/image?url=https%3A%2F%2Fd36ai2hkxl16us.cloudfront.net%2Fthoughtindustries%2Fimage%2Fupload%2Fv1%2Fcourse-uploads%2Fd2afb412-3c3e-4f73-b3d6-2388078622a3%2Fjr9xakbtsqhw-PTCE_ASH-CML_Webinar_110624v2_800x385.png&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fd36ai2hkxl16us.cloudfront.net%2Fthoughtindustries%2Fimage%2Fupload%2Fv1%2Fcourse-uploads%2Fd2afb412-3c3e-4f73-b3d6-2388078622a3%2Fjr9xakbtsqhw-PTCE_ASH-CML_Webinar_110624v2_800x385.png&w=3840&q=75" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="object-cover" loading="lazy"/></noscript></span></div><div class="jsx-2060643579 content-wrap"><span class="jsx-2060643579 py-3 text-sm text-gray-500 text-left"><i class="jsx-2060643579">February 17th 2025</i></span><div class="jsx-2060643579 w-full h-fit mb-4"><p class="jsx-2060643579 text-[18px] font-bold text-[#000000] text-left line-clamp-3 text-hidden">ASH Annual Meeting Coverage: Highlighting the Recent Updates in TKI Use in the Treatment of CML – Insights and Application for Managed Care</p></div><div class="jsx-2060643579 text-left text-[12px] text-[#000] py-6">1.0 Credit / Oncology, Hematology, Hematologic Cancer</div></div></div></a></div></div></div><div class="flex items-center space-x-4 border-0 border-b border-secondary select-none py-1"><h2 class="text-3xl text-primary">More News</h2></div><div class="flex flex-wrap w-full pb-4"><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-1"></div></div></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/chess-ii-hemophilia-severity-correlates-with-economic-burden-patient-qol"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/fc94481ccae12164191743e9de26a7a092c8bbda-4032x2688.jpg?fit=crop&auto=format" alt="Blood Cells - Катерина Євтехова - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">June 7th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/chess-ii-hemophilia-severity-correlates-with-economic-burden-patient-qol">CHESS II: Hemophilia Severity Correlates With Economic Burden, Patient QOL</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ajmc-contributor">AJMC Contributor</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/chess-ii-hemophilia-severity-correlates-with-economic-burden-patient-qol"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The data come from the observational Cost of Hemophilia in Europe: A Socioeconomic Survey II (CHESS II) study of 288 Spanish patients with hemophilia A and B, which showed certain differences between disease subtypes but overall similar trends in disease impact. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/simoctocog-alfa-effective-safe-in-children-with-severe-hemophilia-a"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/2650a1a6477fcbaeb67ffcc0a0ac413d8d8c5413-1200x800.jpg?fit=crop&auto=format" alt="Hemophilia test | Image Credit: jarun011-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">May 29th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/simoctocog-alfa-effective-safe-in-children-with-severe-hemophilia-a">Simoctocog Alfa Effective, Safe in Children With Severe Hemophilia A</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kimberly-rath-pharmd">Kimberly Rath, PharmD</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/simoctocog-alfa-effective-safe-in-children-with-severe-hemophilia-a"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Recent studies reveal that simoctocog alfa (Nuwiq) offers effective prophylaxis and treatment for severe hemophilia A in children, with low inhibitor rates and strong long-term safety and efficacy.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex md:hidden justify-center items-center"></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/hemophilia-not-linked-with-cognitive-and-behavioral-outcomes-in-children"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/188457277fb64f51834f286c25d090a63e15c19f-1280x838.jpg?fit=crop&auto=format" alt="Children in school | Image credit: Vasyl - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">May 24th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/hemophilia-not-linked-with-cognitive-and-behavioral-outcomes-in-children">Hemophilia Not Linked With Cognitive and Behavioral Outcomes in Children</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jared-kaltwasser">Jared Kaltwasser</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/hemophilia-not-linked-with-cognitive-and-behavioral-outcomes-in-children"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">However, investigators said there is some evidence suggesting attention-deficit/hyperactivity disorder may be more common in certain age groups of children with hemophilia. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/enhancing-outpatient-hemophilia-care-may-improve-health-outcomes-and-costs"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/217c874543101839aacf463159b863d6e797d4cf-1200x800.jpg?fit=crop&auto=format" alt="Red blood drop with double helix | Image Credit: Елена Бутусова-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">May 18th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/enhancing-outpatient-hemophilia-care-may-improve-health-outcomes-and-costs">Enhancing Outpatient Hemophilia Care May Improve Health Outcomes and Costs</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ajmc-contributor">AJMC Contributor</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/enhancing-outpatient-hemophilia-care-may-improve-health-outcomes-and-costs"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Prospective data from patients attending a rural practice in West Virginia between 2016 and 2023 show that a quality improvement program that followed guideline recommendations and was tailored to specific patient needs reduced preventable bleeds and lowered costs. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/bleeds-and-resource-use-in-hemophilia-b-retrospective-observational-study"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/5421358c8f117ee86126333e135f08cf1e61ded6-778x382.png?fit=crop&auto=format" alt="Bleeds and Resource Use in Hemophilia B: Retrospective Observational Study" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">May 15th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/bleeds-and-resource-use-in-hemophilia-b-retrospective-observational-study">Bleeds and Resource Use in Hemophilia B: Retrospective Observational Study</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/lisa-young-msc">Lisa Young, MSc</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/lu-ban-phd">Lu Ban, PhD</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/yong-chen-phd-mhs">Yong Chen, PhD, MHS</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/patrick-f-fogarty-md">Patrick F. Fogarty, MD</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/bleeds-and-resource-use-in-hemophilia-b-retrospective-observational-study"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">This real-world US study describes individuals with hemophilia B who experience bleeds despite factor replacement therapy and quantifies the associated comorbidity and health care resource utilization burden.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/health-equity-accessibility-essential-to-supporting-women-affected-by-hemophilia"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/0b9e22627d4997ce065e1351d195f7b2b1c3fab2-1280x853.jpg?fit=crop&auto=format" alt="Worried young woman | Image Credit: © SB Arts Media - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">May 10th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/health-equity-accessibility-essential-to-supporting-women-affected-by-hemophilia">Health Equity, Accessibility Essential to Supporting Women Affected by Hemophilia</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/luke-halpern">Luke Halpern</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/health-equity-accessibility-essential-to-supporting-women-affected-by-hemophilia"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Due to often being overlooked as only carriers of hemophilia, the review authors sought to highlight the needs of female caretakers for patients with the disorder and the possibility of women themselves being affected by it.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-7"></div></div></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/coexistence-of-hiv-hemophilia-may-lead-to-increased-presence-of-coronary-artery-stenosis"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/65df11129c2a3f1c5c5a9b61b48d71e7b0e4bac4-1200x724.jpg?fit=crop&auto=format" alt="heartbeats | Image Credit: Robert - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">May 8th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/coexistence-of-hiv-hemophilia-may-lead-to-increased-presence-of-coronary-artery-stenosis">Coexistence of HIV, Hemophilia May Lead to Increased Presence of Coronary Artery Stenosis</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/luke-halpern">Luke Halpern</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/coexistence-of-hiv-hemophilia-may-lead-to-increased-presence-of-coronary-artery-stenosis"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">In patients living with HIV, the cardiovascular risk factors that come with infection may lead to an increased prevalence of coronary artery stenosis, especially in those who also have hemophilia. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/review-one-shot-etranacogene-dezaparvovec-represents-game-changing-gene-therapy-for-hemophilia-b"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/3c98541952c09018560f227b5e95b66881df5a38-1200x862.jpg?fit=crop&auto=format" alt="DNA helix inside blood drop | Image Credit: © Елена Бутусова - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">May 3rd 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/review-one-shot-etranacogene-dezaparvovec-represents-game-changing-gene-therapy-for-hemophilia-b">Review: One-Shot Etranacogene Dezaparvovec Represents Game-Changing Gene Therapy for Hemophilia B </a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/gayle-turim-dickstein">Gayle Turim Dickstein</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/review-one-shot-etranacogene-dezaparvovec-represents-game-changing-gene-therapy-for-hemophilia-b"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">More than 20 years of gene therapy clinical research have led to etranacogene dezaparvovec’s use in hemophilia B, according to authors of a recent review—and it takes its place in a line of current and potentially many future genomic medicines for a range of diseases.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex md:hidden justify-center items-center"></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/fda-approves-one-time-gene-therapy-fidanacogene-elaparvovec-for-hemophilia-b"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/15a072b02809d2bef4cba64f5c0025e91778dba3-1200x724.jpg?fit=crop&auto=format" alt="Concept of FDA approved | Image credit: wladimir1804-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">April 26th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/fda-approves-one-time-gene-therapy-fidanacogene-elaparvovec-for-hemophilia-b">FDA Approves One-Time Gene Therapy Fidanacogene Elaparvovec for Hemophilia B</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/fda-approves-one-time-gene-therapy-fidanacogene-elaparvovec-for-hemophilia-b"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The FDA has approved fidanacogene elaparvovec-dzkt (Beqvez; Pfizer), a gene therapy administered in a one-time dose, for certain patients with moderate to severe hemophilia B.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/males-with-hemophilia-a-report-physical-mental-burdens-of-disease-regardless-of-severity"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/6f43cbdd2b9fcd3e1d4e9fd8b5d19497c3bbfd93-1200x724.jpg?fit=crop&auto=format" alt="HemophiliaA | Image Credit: MQ-Illustrations - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">April 25th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/males-with-hemophilia-a-report-physical-mental-burdens-of-disease-regardless-of-severity">Males With Hemophilia A Report Physical, Mental Burdens of Disease Regardless of Severity </a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ajmc-contributor">AJMC Contributor</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/males-with-hemophilia-a-report-physical-mental-burdens-of-disease-regardless-of-severity"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Self-reported data from adult and pediatric males with hemophilia A show that burden of the disease persists regardless of severity, highlighting a need for improved prophylactic treatment. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/long-term-data-demonstrate-emicizumab-safety-efficacy-in-hemophilia-a"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/8b6d6bfddb9ec23cd720c11eb43232ddab0b6b56-1200x724.jpg?fit=crop&auto=format" alt="Hemophilia defined | Image Credit: VitezslavVylicil - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">April 16th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/long-term-data-demonstrate-emicizumab-safety-efficacy-in-hemophilia-a">Long-Term Data Demonstrate Emicizumab Safety, Efficacy in Hemophilia A</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jared-kaltwasser">Jared Kaltwasser</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/long-term-data-demonstrate-emicizumab-safety-efficacy-in-hemophilia-a"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The therapy bridges activated factors IX and X to mimic the function of missing or deficient factor VIII. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/study-suggests-walking-10k-steps-daily-improves-qol-in-severe-hemophilia"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/b3f1ac1ace915f7bfc868c9bf52aafc758633ed5-1200x724.jpg?fit=crop&auto=format" alt="Hemophilia | Image Credit: lexiconimages - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">April 12th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/study-suggests-walking-10k-steps-daily-improves-qol-in-severe-hemophilia">Study Suggests Walking 10K Steps Daily Improves QOL in Severe Hemophilia</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ajmc-contributor">AJMC Contributor</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/study-suggests-walking-10k-steps-daily-improves-qol-in-severe-hemophilia"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Among a small group of patients with severe hemophilia, researchers found that getting in more than 10,000 steps each day was linked to significant improvements in perceived physical health and quality of life (QOL). </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-13"></div></div></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/clot-formation-fibrinolysis-assays-may-help-show-important-differences-between-hemophilia-a-treatments"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/fc94481ccae12164191743e9de26a7a092c8bbda-4032x2688.jpg?fit=crop&auto=format" alt="Blood Cells | Image Credit: © Катерина Євтехова - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">April 6th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/clot-formation-fibrinolysis-assays-may-help-show-important-differences-between-hemophilia-a-treatments">Clot Formation, Fibrinolysis Assays May Help Show Important Differences Between Hemophilia A Treatments</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ajmc-contributor">AJMC Contributor</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/clot-formation-fibrinolysis-assays-may-help-show-important-differences-between-hemophilia-a-treatments"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Researchers found that in vitro clot formulation and fibrinolysis assays were able to determine the hemostatic effects of different replacement agents, as well as identify distinct pharmacodynamic responses across the different mechanisms of action.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/small-study-shows-safety-efficacy-of-prophylactic-rix-fp-in-previously-untreated-hemophilia-b"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/0b8d3f149e5bd89faae2e47de1137df7687fc077-6016x4016.jpg?fit=crop&auto=format" alt="blood samples | Image credit: Daniel CHETRONI – stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">April 5th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/small-study-shows-safety-efficacy-of-prophylactic-rix-fp-in-previously-untreated-hemophilia-b">Small Study Shows Safety, Efficacy of Prophylactic rIX-FP in Previously Untreated Hemophilia B</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ajmc-contributor">AJMC Contributor</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/small-study-shows-safety-efficacy-of-prophylactic-rix-fp-in-previously-untreated-hemophilia-b"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Findings from this new study demonstrate the safety and efficacy of rIX-FP among a group of treatment-naive patients often characterized by young age and particular risk for severe bleeding.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/breaking-barriers-in-bleeding-disorders-experts-call-for-more-data-on-women"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/66537d52b0de62e5fcf7d4f21233f4df827a0812-6720x4480.jpg?fit=crop&auto=format" alt="Menstruation Period Accessories - Atlas - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">January 8th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/breaking-barriers-in-bleeding-disorders-experts-call-for-more-data-on-women">Breaking Barriers in Bleeding Disorders: Experts Call for More Data on Women</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/giuliana-grossi">Giuliana Grossi</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/breaking-barriers-in-bleeding-disorders-experts-call-for-more-data-on-women"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">“These autosomal diseases, from an inheritance standpoint, affect men and women equally, but because of their impact on the gynecological system and menses, women are often affected much more than men," hematologist Donna DiMichele, MD, explains.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/subcutaneous-octa101-trial-in-severe-hemophilia-a-halted-due-to-safety-concerns"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/d3df9a2fb06b8f89a68e8a842898ddfd46bdb9b7-7200x4050.jpg?fit=crop&auto=format" alt="Red Blood Cells - Design Cells stock.adobe.com.jpeg" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">November 24th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/subcutaneous-octa101-trial-in-severe-hemophilia-a-halted-due-to-safety-concerns">Subcutaneous OCTA101 Trial in Severe Hemophilia A Halted Due to Safety Concerns</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/giuliana-grossi">Giuliana Grossi</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/subcutaneous-octa101-trial-in-severe-hemophilia-a-halted-due-to-safety-concerns"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">In a trial assessing subcutaneous OCTA101 in patients with severe hemophilia A, promising results in pharmacokinetics and efficacy were overshadowed by safety concerns. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex md:hidden justify-center items-center"></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/study-reveals-racial-disparities-in-immune-tolerance-induction-treatment-for-hemophilia"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/92dbe8284521d4c53b69b2ba67499be16723a7b7-6720x4480.jpg?fit=crop&auto=format" alt="Professional Nurse Preparing Treatment - Viacheslav Yakobchuk - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">November 14th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/study-reveals-racial-disparities-in-immune-tolerance-induction-treatment-for-hemophilia">Study Reveals Racial Disparities in Immune Tolerance Induction Treatment for Hemophilia</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/giuliana-grossi">Giuliana Grossi</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/study-reveals-racial-disparities-in-immune-tolerance-induction-treatment-for-hemophilia"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">New research highlights racial disparities in hemophilia care, revealing that Black and Hispanic patients are significantly less likely to receive immune tolerance induction treatment compared with White patients, even after considering clinical factors. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/etranacogene-dezaparvovec-gene-therapy-outperforms-fix-products-in-hemophilia-b"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/8b89e01eaccdc9b40b4e32843a4ec865559bd897-4394x2920.jpg?fit=crop&auto=format" alt="Genetic engineering and gene manipulation concept - andrianocz - stock.adobe.com.jpeg" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">November 1st 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/etranacogene-dezaparvovec-gene-therapy-outperforms-fix-products-in-hemophilia-b">Etranacogene Dezaparvovec Gene Therapy Outperforms FIX Products in Hemophilia B</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/giuliana-grossi">Giuliana Grossi</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/etranacogene-dezaparvovec-gene-therapy-outperforms-fix-products-in-hemophilia-b"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The findings from a recent study unveiled notable distinctions in bleeding rates between etranacogene dezaparvovec and the standard prophylactic factor IX (FIX) products among individuals diagnosed with hemophilia B.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-19"></div></div></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/study-identifies-core-instruments-monitoring-physical-function-hemophilia"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/33fa833c4c1c0b50074aae1a59a4dc95c0569ec8-5237x3491.jpg?fit=crop&auto=format" alt="Asian doctor woman image credit Nattakorn stock.adobe.com.jpeg" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 28th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/study-identifies-core-instruments-monitoring-physical-function-hemophilia">Study Identifies Core Instruments for Monitoring Physical Function in Hemophilia</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/giuliana-grossi">Giuliana Grossi</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/study-identifies-core-instruments-monitoring-physical-function-hemophilia"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The study identified practical instruments for monitoring physical function in people with hemophilia by pinpointing 11 potential markers and establishing performance-based tests for 5 of these activities. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/annexa-i-andexanet-alfa-shows-efficacy-safety-for-factor-xa-reversal-in-acute-ich"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/061c1f0ebeb8cd0695346ce474234ebc136899c1-4472x3354.jpg?fit=crop&auto=format" alt="Brain illustration | Image credit: jolygon - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 27th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/annexa-i-andexanet-alfa-shows-efficacy-safety-for-factor-xa-reversal-in-acute-ich">ANNEXA-I: Andexanet Alfa Shows Efficacy, Safety for Factor Xa Reversal in Acute ICH</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/annexa-i-andexanet-alfa-shows-efficacy-safety-for-factor-xa-reversal-in-acute-ich"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The phase 4 ANNEXA-I trial was stopped early after showing superior hemostatic efficacy and the capability to limit potentially life-threatening intracerebral hemorrhage (ICH) compared with usual care in patients taking oral factor Xa (FXa) inhibitors.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/damoctocog-alfa-pegol-demonstrates-safety-efficacy-hemophilia-a"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/dd11b67cc7da3a16e114741e20dcf510de5608ae-5472x3648.jpg?fit=crop&auto=format" alt="Set IV Fluid | Image Credit: Trsakaoe stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 26th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/damoctocog-alfa-pegol-demonstrates-safety-efficacy-hemophilia-a">Damoctocog Alfa Pegol Demonstrates Safety and Efficacy in Hemophilia A</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/giuliana-grossi">Giuliana Grossi</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/damoctocog-alfa-pegol-demonstrates-safety-efficacy-hemophilia-a"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">While clinical trials offer controlled data, real-world studies like HEM-POWR bridge gaps, confirming damoctocog alfa pegol’s effectiveness and tolerance, establishing it as a valuable hemophilia A treatment option.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/evaluating-who-hemophilia-treatment-patient-safety-access-challenges"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/d3df9a2fb06b8f89a68e8a842898ddfd46bdb9b7-7200x4050.jpg?fit=crop&auto=format" alt=" Red Blood Cells | Image Credit: Design Cells - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 12th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/evaluating-who-hemophilia-treatment-patient-safety-access-challenges">Evaluating WHO's Hemophilia Treatment Choices: Patient Safety, Access Challenges</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/giuliana-grossi">Giuliana Grossi</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/evaluating-who-hemophilia-treatment-patient-safety-access-challenges"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A commentary questions the characterization of cryoprecipitate, which is not pathogen-reduced, as an alternative modality for treating hemophilia, despite its substantial risk of transmitting blood-borne pathogens to patients.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/what-we-re-reading-rising-breast-cancer-rates-pharmacists-walkout-school-blood-drives-on-hold"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/703de894e882d8a6da8c19964dc1ca71b969d30c-2000x1046.png?fit=crop&auto=format" alt="What We’re Reading: Rising Breast Cancer Rates; Pharmacists Walkout Over Working Conditions; School Blood Drives on Hold " width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 11th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/what-we-re-reading-rising-breast-cancer-rates-pharmacists-walkout-school-blood-drives-on-hold">What We’re Reading: Rising Breast Cancer Rates; Pharmacists Walkout; School Blood Drives on Hold </a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ajmc-staff">AJMC Staff</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/what-we-re-reading-rising-breast-cancer-rates-pharmacists-walkout-school-blood-drives-on-hold"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Advances in treatments and early detection methods have led to improved survival rates; staffing shortages and rising demands take their toll; the US blood supply is under increasing strain.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/the-framework-for-embracing-gene-therapy-in-hemophilia-treatment"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/4b2bc54cca4ba227fe614fd2d68fc80f98380fdc-5500x2402.jpg?fit=crop&auto=format" alt="Doctor using digital technology | Image credit: otello-stpdc - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">September 30th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/the-framework-for-embracing-gene-therapy-in-hemophilia-treatment">The Framework for Embracing Gene Therapy in Hemophilia Treatment</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/giuliana-grossi">Giuliana Grossi</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/the-framework-for-embracing-gene-therapy-in-hemophilia-treatment"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Although gene therapy is anticipated to bring positive changes for people with hemophilia, choosing to receive gene therapy will require empowerment, participation, and shared decision-making, investigators write.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-25"></div></div></div><div class="jsx-ad50481d5ee26850 flex md:hidden justify-center items-center"></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/altuviiio-supplemental-biologics-license-application-severe-hemophilia-a-in-children-accepted-by-fda"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/bf7ba1f7a9ab295ce5b7e6c41d3188a5ae4bff4a-3840x2560.webp?fit=crop&auto=format" alt="Image credit: Катерина Євтехова | stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">September 29th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/altuviiio-supplemental-biologics-license-application-severe-hemophilia-a-in-children-accepted-by-fda">Altuviiio sBLA for Severe Hemophilia A in Children Accepted by FDA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ashley-gallagher">Ashley Gallagher</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/altuviiio-supplemental-biologics-license-application-severe-hemophilia-a-in-children-accepted-by-fda"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The supplemental biologics license application (sBLA) is supported by the results of the XTEND-Kids study, demonstrating a once-weekly 50 IU/kg dose of antihemophilic factor (recombinant) provides effective bleed protection in children with hemophilia A and can be used across clinical scenarios.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/hemophilia-survival-rates-improve-black-men-still-fall-10-years-behind"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/7546f59dab571ccfc4dd43262d974af9c0529b8b-5349x3566.jpg?fit=crop&auto=format" alt="Blood Transfusion | Image credit: smolaw11 – stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">September 23rd 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/hemophilia-survival-rates-improve-black-men-still-fall-10-years-behind">Hemophilia Survival Rates Improve, Black Men Still Fall 10 Years Behind</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/giuliana-grossi">Giuliana Grossi</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/hemophilia-survival-rates-improve-black-men-still-fall-10-years-behind"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The median age of death remained consistently 10 years lower for Black and Hispanic males in comparison to White males, underscoring a critical area for further attention.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/study-prevalence-subclinical-synovial-proliferation-hemophilia-a"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/e873c2b56b84e258fdc08bfd3868c785052a2954-5760x3840.jpg?fit=crop&auto=format" alt="Doctor Conducting Ultrasound | Image credit: Africa Studio – stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">September 14th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/study-prevalence-subclinical-synovial-proliferation-hemophilia-a">Study Reveals Prevalence of Subclinical Synovial Proliferation in Hemophilia A</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/giuliana-grossi">Giuliana Grossi</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/study-prevalence-subclinical-synovial-proliferation-hemophilia-a"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The findings highlight the critical role that subclinical synovial proliferation plays in the progression of hemophilic arthropathy.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/hemophilia-carriers-reproductive-decisions-linked-to-childhood-trauma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/e9347a24c69fa631d7b1aa8e39d17d8af992c982-8256x5504.jpg?fit=crop&auto=format" alt="Hemophilia Carriers' Reproductive Decisions Linked to Childhood Trauma" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">September 9th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/hemophilia-carriers-reproductive-decisions-linked-to-childhood-trauma">Hemophilia Carriers' Reproductive Decisions Linked to Childhood Trauma</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/giuliana-grossi">Giuliana Grossi</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/hemophilia-carriers-reproductive-decisions-linked-to-childhood-trauma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A study found that negative experiences and concern for future children's health drive hemophilia carriers to choose prenatal tests, with cognitive factors playing a role.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/patients-clinicians-need-hemophilia-gene-therapy-shared-decision-making-tool"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/d589b7a471e940893711d41e38fed21ce52e7c97-1000x666.jpg?fit=crop&auto=format" alt="patient and doctor sitting down talking" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">August 23rd 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/patients-clinicians-need-hemophilia-gene-therapy-shared-decision-making-tool">Patients, Clinicians Need Hemophilia Gene Therapy Shared Decision-Making Tool</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/victoria-johnson">Victoria Johnson</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/patients-clinicians-need-hemophilia-gene-therapy-shared-decision-making-tool"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Clinicians and nurses who treat patients with hemophilia with a gene therapy identified important factors and considerations needed in a potential shared decision making tool.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/national-hemophilia-foundation-updates-name-to-national-bleeding-disorders-foundation"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/f4043ab8939f3dba14645deff229a71d8fe1c542-4425x1327.png?fit=crop&auto=format" alt="National Hemophilia Foundation Updates Name to National Bleeding Disorders Foundation" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">August 18th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/national-hemophilia-foundation-updates-name-to-national-bleeding-disorders-foundation">National Hemophilia Foundation Updates Name to National Bleeding Disorders Foundation</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ajmc-staff">AJMC Staff</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/national-hemophilia-foundation-updates-name-to-national-bleeding-disorders-foundation"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A 75-year-old foundation updates its name to reflect a more comprehensive mission. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div></div><div class="w-full text-center flex justify-center 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Pfizer), a gene therapy administered in a one-time dose, for certain patients with moderate to severe hemophilia B.","contentCategory":{"name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory"},"factCheckAuthors":null,"_id":"1a1dd513-c337-4f26-a951-9cd50f8ed0c8","published":"2024-04-26T11:48:56.981Z","updatedOn":null,"authors":[{"displayName":"Rose McNulty","url":"rose-mcnulty"}],"thumbnail":{"_type":"mainImage","alt":"Concept of FDA approved | Image credit: wladimir1804-stock.adobe.com","asset":{"_ref":"image-15a072b02809d2bef4cba64f5c0025e91778dba3-1200x724-jpg","_type":"reference"}}},{"title":"Males With Hemophilia A Report Physical, Mental Burdens of Disease Regardless of Severity ","thumbnail":{"_type":"mainImage","alt":"HemophiliaA | Image Credit: MQ-Illustrations - stock.adobe.com","asset":{"_ref":"image-6f43cbdd2b9fcd3e1d4e9fd8b5d19497c3bbfd93-1200x724-jpg","_type":"reference"}},"summary":"Self-reported data from adult and pediatric males with hemophilia A show that burden of the disease persists regardless of severity, highlighting a need for improved prophylactic treatment. ","updatedOn":null,"authors":[{"displayName":"AJMC Contributor","url":"ajmc-contributor"}],"_id":"40b022eb-3767-4508-a68a-f3ee386fbcee","published":"2024-04-25T18:10:20.451Z","contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"factCheckAuthors":null,"url":"males-with-hemophilia-a-report-physical-mental-burdens-of-disease-regardless-of-severity"},{"published":"2024-04-16T14:30:19.406Z","contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"authors":[{"displayName":"Jared Kaltwasser","url":"jared-kaltwasser"}],"factCheckAuthors":null,"_id":"53daf201-7486-41fe-9065-710d4c7a657d","title":"Long-Term Data Demonstrate Emicizumab Safety, Efficacy in Hemophilia A","thumbnail":{"alt":"Hemophilia defined | Image Credit: VitezslavVylicil - 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Atlas - stock.adobe.com","asset":{"_type":"reference","_ref":"image-66537d52b0de62e5fcf7d4f21233f4df827a0812-6720x4480-jpg"}},"summary":"“These autosomal diseases, from an inheritance standpoint, affect men and women equally, but because of their impact on the gynecological system and menses, women are often affected much more than men,\" hematologist Donna DiMichele, MD, explains."},{"contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"authors":[{"displayName":"Giuliana Grossi","url":"giuliana-grossi"}],"factCheckAuthors":null,"_id":"76f94cc5-78e1-46e8-aa50-611188d17908","thumbnail":{"_type":"mainImage","alt":"Red Blood Cells - Design Cells stock.adobe.com.jpeg","asset":{"_ref":"image-d3df9a2fb06b8f89a68e8a842898ddfd46bdb9b7-7200x4050-jpg","_type":"reference"}},"updatedOn":null,"published":"2023-11-24T16:27:00.000Z","title":"Subcutaneous OCTA101 Trial in Severe Hemophilia A Halted Due to Safety Concerns","url":"subcutaneous-octa101-trial-in-severe-hemophilia-a-halted-due-to-safety-concerns","summary":"In a trial assessing subcutaneous OCTA101 in patients with severe hemophilia A, promising results in pharmacokinetics and efficacy were overshadowed by safety concerns. "},{"_id":"ae70bca2-52df-42b5-9c16-19d5547dcbfb","title":"Study Reveals Racial Disparities in Immune Tolerance Induction Treatment for Hemophilia","url":"study-reveals-racial-disparities-in-immune-tolerance-induction-treatment-for-hemophilia","thumbnail":{"_type":"mainImage","alt":"Professional Nurse Preparing Treatment - Viacheslav Yakobchuk - stock.adobe.com","asset":{"_ref":"image-92dbe8284521d4c53b69b2ba67499be16723a7b7-6720x4480-jpg","_type":"reference"}},"contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"summary":"New research highlights racial disparities in hemophilia care, revealing that Black and Hispanic patients are significantly less likely to receive immune tolerance induction treatment compared with White patients, even after considering clinical factors.\n\n\n","published":"2023-11-14T22:25:04.428Z","updatedOn":null,"authors":[{"displayName":"Giuliana Grossi","url":"giuliana-grossi"}],"factCheckAuthors":null},{"contentCategory":{"_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles"},"authors":[{"displayName":"Giuliana Grossi","url":"giuliana-grossi"}],"_id":"906865a5-a6ea-4699-85c7-9fb664e7f358","title":"Etranacogene Dezaparvovec Gene Therapy Outperforms FIX Products in Hemophilia B","url":"etranacogene-dezaparvovec-gene-therapy-outperforms-fix-products-in-hemophilia-b","thumbnail":{"_type":"mainImage","alt":"Genetic engineering and gene manipulation concept - andrianocz - stock.adobe.com.jpeg","asset":{"_ref":"image-8b89e01eaccdc9b40b4e32843a4ec865559bd897-4394x2920-jpg","_type":"reference"}},"summary":"The findings from a recent study unveiled notable distinctions in bleeding rates between etranacogene dezaparvovec and the standard prophylactic factor IX (FIX) products among individuals diagnosed with hemophilia B.","published":"2023-11-01T17:02:34.821Z","factCheckAuthors":null,"updatedOn":null},{"url":"study-identifies-core-instruments-monitoring-physical-function-hemophilia","thumbnail":{"alt":"Asian doctor woman image credit Nattakorn stock.adobe.com.jpeg","asset":{"_ref":"image-33fa833c4c1c0b50074aae1a59a4dc95c0569ec8-5237x3491-jpg","_type":"reference"},"_type":"mainImage"},"published":"2023-10-28T13:00:00.000Z","updatedOn":null,"factCheckAuthors":null,"_id":"c033465a-353e-49b4-a963-2e782d19fa71","title":"Study Identifies Core Instruments for Monitoring Physical Function in Hemophilia","summary":"The study identified practical instruments for monitoring physical function in people with hemophilia by pinpointing 11 potential markers and establishing performance-based tests for 5 of these activities. \n\n","contentCategory":{"name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory"},"authors":[{"displayName":"Giuliana Grossi","url":"giuliana-grossi"}]},{"url":"annexa-i-andexanet-alfa-shows-efficacy-safety-for-factor-xa-reversal-in-acute-ich","published":"2023-10-27T13:00:00.000Z","updatedOn":null,"factCheckAuthors":null,"_id":"8ecca6ec-d727-4d79-8d71-5fc103be9e6d","title":"ANNEXA-I: Andexanet Alfa Shows Efficacy, Safety for Factor Xa Reversal in Acute ICH","thumbnail":{"alt":"Brain illustration | Image credit: jolygon - stock.adobe.com","caption":"Brain illustration | Image credit: jolygon - stock.adobe.com","asset":{"_ref":"image-061c1f0ebeb8cd0695346ce474234ebc136899c1-4472x3354-jpg","_type":"reference"},"_type":"mainImage"},"summary":"The phase 4 ANNEXA-I trial was stopped early after showing superior hemostatic efficacy and the capability to limit potentially life-threatening intracerebral hemorrhage (ICH) compared with usual care in patients taking oral factor Xa (FXa) inhibitors.","contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"authors":[{"displayName":"Rose McNulty","url":"rose-mcnulty"}]},{"_id":"ac337fd8-165b-4117-adb7-b312870b81a7","url":"damoctocog-alfa-pegol-demonstrates-safety-efficacy-hemophilia-a","thumbnail":{"_type":"mainImage","alt":"Set IV Fluid | Image Credit: Trsakaoe stock.adobe.com","asset":{"_ref":"image-dd11b67cc7da3a16e114741e20dcf510de5608ae-5472x3648-jpg","_type":"reference"}},"authors":[{"displayName":"Giuliana Grossi","url":"giuliana-grossi"}],"factCheckAuthors":null,"title":"Damoctocog Alfa Pegol Demonstrates Safety and Efficacy in Hemophilia A","summary":"While clinical trials offer controlled data, real-world studies like HEM-POWR bridge gaps, confirming damoctocog alfa pegol’s effectiveness and tolerance, establishing it as a valuable hemophilia A treatment option.","published":"2023-10-26T21:26:00.000Z","updatedOn":null,"contentCategory":{"_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z"}},{"_id":"14f4aa24-47ef-4c90-b9b8-42b7b34f4a19","url":"evaluating-who-hemophilia-treatment-patient-safety-access-challenges","thumbnail":{"_type":"mainImage","alt":" Red Blood Cells | Image Credit: Design Cells - stock.adobe.com","asset":{"_ref":"image-d3df9a2fb06b8f89a68e8a842898ddfd46bdb9b7-7200x4050-jpg","_type":"reference"}},"authors":[{"displayName":"Giuliana Grossi","url":"giuliana-grossi"}],"factCheckAuthors":null,"title":"Evaluating WHO's Hemophilia Treatment Choices: Patient Safety, Access Challenges","summary":"A commentary questions the characterization of cryoprecipitate, which is not pathogen-reduced, as an alternative modality for treating hemophilia, despite its substantial risk of transmitting blood-borne pathogens to patients.","published":"2023-10-12T12:20:00.000Z","updatedOn":null,"contentCategory":{"name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory"}},{"thumbnail":{"_type":"mainImage","alt":"What We’re Reading: Rising Breast Cancer Rates; Pharmacists Walkout Over Working Conditions; School Blood Drives on Hold ","asset":{"_ref":"image-703de894e882d8a6da8c19964dc1ca71b969d30c-2000x1046-png","_type":"reference"}},"summary":"Advances in treatments and early detection methods have led to improved survival rates; staffing shortages and rising demands take their toll; the US blood supply is under increasing 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anticipated to bring positive changes for people with hemophilia, choosing to receive gene therapy will require empowerment, participation, and shared decision-making, investigators write.","published":"2023-09-30T13:30:00.000Z","contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"factCheckAuthors":null,"title":"The Framework for Embracing Gene Therapy in Hemophilia Treatment","updatedOn":null,"authors":[{"displayName":"Giuliana Grossi","url":"giuliana-grossi"}]},{"authors":[{"displayName":"Ashley Gallagher","url":"ashley-gallagher"}],"factCheckAuthors":null,"_id":"e94dc263-c32e-4f04-878e-801ea2599430","title":"Altuviiio sBLA for Severe Hemophilia A in Children Accepted by FDA","summary":"The supplemental biologics license application (sBLA) is supported by the results of the XTEND-Kids study, demonstrating a once-weekly 50 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","_key":"13dd59239e81"},{"_key":"3711baf450301","_type":"span","marks":["em"],"text":"The American Journal of Managed Care"},{"_type":"span","marks":["superscript"],"text":"Ⓡ","_key":"f8784b2b4839"},{"_type":"span","marks":[],"text":", Sarah Wall, MD, assistant professor in the Division of Hematology at The Ohio State University Comprehensive Cancer Center, emphasizes the need to study treatments for different age groups, particularly focusing on older adults with hematologic disorders. Even the FDA has stressed this due to disease variations and social dynamics, she explains.","_key":"c1efc7ec63de"}],"_type":"block","style":"normal"},{"_key":"99683868a797","markDefs":[],"children":[{"_type":"span","marks":["strong","underline"],"text":"Transcript","_key":"7698ddb1dedb0"}],"_type":"block","style":"normal"},{"_key":"327204a1838d","markDefs":[],"children":[{"marks":["strong"],"text":"What are some unique struggles that older adults with hematologic disorders face?","_key":"b403ce26544a0","_type":"span"}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"_key":"177c7784b1100","_type":"span","marks":[],"text":"This is a really important one, and it's one that has had a lot of light shed on it recently by a number of organizations. I think one really important one, in particular, is the FDA has actually issued guidance that it's really important that we study treatments and focusing on those cohorts, in part because of some of the things we just talked about, that the diseases may behave differently."}],"_type":"block","style":"normal","_key":"7b2dfa6e64c3"},{"children":[{"_type":"span","marks":[],"text":"But there's such a difference in social dynamics and engagement with the environment around them for older adults vs younger adults because the things that may often be overlooked are related to financial toxicity. Finances, for older patients, without making an overgeneralization, finances are often very different for older patients when they may be on a fixed income.","_key":"72e4948e0ea30"}],"_type":"block","style":"normal","_key":"03db098031ef","markDefs":[]},{"markDefs":[],"children":[{"text":"The payers are often very different for older patients when we start getting into Medicare coverage. And then, the other things that come into play that are less tangible, even, are things like where you've set up your support system, and that will differ by family. But a lot of older patients are in a setting where they've got kids or grandkids or lifelong friends and family who are around them and supporting them.","_key":"1f5738af63b80","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"abf1686624ab"},{"_key":"d0e3ae27f5af","markDefs":[],"children":[{"_type":"span","marks":[],"text":"And so depending on how far away they live from a treatment center, having to uproot themselves from all of that support can be very taxing and actually sometimes make it prohibitive to get the best treatment that they could. Then there are costs with all of that, too. Gas money, hotel money, all of those things play a role and those can often be really big stressors when you also add in that piece of potentially going on a fixed income or having to dip into savings that were supposed to last well into retirement.","_key":"41a07a3aef190"}],"_type":"block","style":"normal"}],"articleType":"News","contentCategory":{"_createdAt":"2020-04-03T20:03:44Z","_rev":"Yw6MEKZDMdk6hC2JCPjfiB","_type":"contentCategory","name":"Videos","_id":"ee14ccb3-3542-4414-9046-927be1198c76","_updatedAt":"2020-04-03T20:03:44Z"},"factCheckAuthorMapping":null,"internalTag":["Hematology","Geriatric"],"authorMapping":[{"_createdAt":"2022-11-16T19:50:07Z","_type":"author","_updatedAt":"2022-11-16T19:50:07Z","firstName":"Justina","lastName":"Petrullo","displayName":"Justina Petrullo","_rev":"lyPumz4JwtPETh8Y1IKXNg","_id":"28299f59-f977-41f8-b68a-647156265fd3","url":{"current":"justina-petrullo","_type":"slug"}},{"_rev":"FS1VyU7oCYHHQjBpgCp3g5","_updatedAt":"2025-02-13T15:16:17Z","url":{"current":"giuliana-grossi","_type":"slug"},"firstName":"Giuliana","displayName":"Giuliana Grossi","_createdAt":"2023-06-23T19:52:20Z","_id":"8e597e29-c3ad-470c-8174-00c81b563f81","profileImage":{"_type":"mainImage","alt":"Giuliana Grossi","asset":{"_ref":"image-5dc1da24786c43a7f87e4ea0a7ae3880e6cee39f-430x430-png","_type":"reference"}},"lastName":"Grossi","authorType":"author","_type":"author"}],"summary":"Older patients often face financial challenges on fixed incomes with different payer systems like Medicare, Sarah Wall, MD, The Ohio State University Comprehensive Cancer Center, says. Accessing treatment centers far away is difficult due to support system disruptions, leading to increased stress and financial strain.","is_visible":true,"_rev":"iLKhIuRBI5WmRf3jsZOYgq"},{"summary":"In hemophilia cases where there is uncertainty, especially when your factor level is near the 50% cutoff, genetic testing becomes important, Ragni says.","_type":"article","ExcludeFromPubMedXML":false,"_rev":"I69RE849GmtXmLZyCWYYQR","articleType":"News","_id":"ef7a9c10-080f-4aec-b6af-85608f71f47d","published":"2023-09-06T14:45:20.399Z","body":[{"_key":"cb66c9f0c34a","_type":"video","videoID":"6336676998112","disableAutoPlayVideo":false,"source":"brightcove"},{"_key":"5cc5e38042a7","markDefs":[{"_type":"link","href":"https://www.ajmc.com/topic/hemophilia","_key":"a04347cac708","nofollow":false,"blank":true}],"children":[{"_type":"span","marks":[],"text":"Women with mild ","_key":"3acfe369b8890"},{"_type":"span","marks":["a04347cac708"],"text":"hemophilia","_key":"a935c5e49ec0"},{"_type":"span","marks":[],"text":" who carry the gene are often overlooked when it comes to symptoms and treatment options, noted Margaret Ragni, MD, MPH, professor of medicine and clinical and translational research, Division of Hematology/Oncology, director of the Hemophilia Center of Western PA, and American Society of Hematology expert. In this interview with our Center on Health Equity \u0026 Access, she explains why genetic testing is crucial for this population.","_key":"33932a12032b"}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"marks":["strong","underline"],"text":"Transcription","_key":"87e848393456","_type":"span"}],"_type":"block","style":"normal","_key":"e64af088f387"},{"children":[{"_type":"span","marks":["strong"],"text":"Is it common practice to test potential carriers or women who are related to those with hemophilia?","_key":"f19f710b5149"}],"_type":"block","style":"normal","_key":"2f49f2daf3f5","markDefs":[]},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"We will know that you are a carrier if you're the daughter of a patient with hemophilia, a man with hemophilia. If you are a female relative of someone who has hemophilia, like your nephew, your uncle, a daughter of a woman who's a carrier, we only know that you have a 50% chance of being a carrier. So that's where testing is critical.","_key":"af66ac6a14420"}],"_type":"block","style":"normal","_key":"07e8fdb5b434"},{"children":[{"marks":[],"text":"But I would argue testing is critical even if we know you have hemophilia because what the testing will establish is what your factor level is. And in people in whom there's equivocation and we're not sure, we usually use this 50% cutoff, because that's the low range of normal, but you can still be a carrier with a normal level—so we do genotyping.","_key":"6f24feee07250","_type":"span"}],"_type":"block","style":"normal","_key":"047dd65ce47f","markDefs":[]},{"_type":"block","style":"normal","_key":"c2dbe80b6322","markDefs":[],"children":[{"_type":"span","marks":[],"text":"Now, it's become much more common to do genetic testing. If we already know you're an obligate carrier—that is, that you are a carrier—what we really need is your [factor] level. We don't need to do genotyping because we already know it in your son or your father. But many times that's not known. So, in general, if it's not known in the family, we'll do the whole thing.","_key":"301d330344fe0"}]},{"_type":"block","style":"normal","_key":"ef4b2ccce27f","markDefs":[],"children":[{"_key":"4da76cb036cd0","_type":"span","marks":[],"text":"But the reason that 50% cutoff is important: [It's] not just for diagnosing a carrier, but also for decisions about how to manage your bleeding, and it's not a clear-cut science. Generally, if you're less than 0.5, you can't have an epidural. They won't let you unless you have treatment that brings your level higher and then you need longer treatment, you may be at greater risk for bleeding, all those sorts of things."}]},{"style":"normal","_key":"df5380cec5c5","markDefs":[],"children":[{"_key":"1484ea3177f30","_type":"span","marks":[],"text":"And here's another problem: Some women who are carriers don't even know that they're at risk of being carriers, because, don't forget, back 30 years ago, we had the AIDS epidemic. People didn't even want to talk about it or someone died in the family. So again, it was a hush-hush, we didn't talk about it. And so women were the last to know, and certainly their doctors who were not hemostasis doctors, they’re pediatricians, they’re good upstanding internists or gynecologists, don't think of those things. Why would they?"}],"_type":"block"},{"_type":"block","style":"normal","_key":"c71c3160206b","markDefs":[],"children":[{"_type":"span","marks":[],"text":"It's sort of one of those issues when a carrier's first symptom, and most common symptom, is heavy menstrual bleeding, you would think that in the realm of things, you would ask the question, “Do you have any other kind of bleeding? What else is going on, is anybody a bleeder in your family?” Those 2 questions could really pave the way for a lot more things.","_key":"841a8c086a6f0"}]},{"style":"normal","_key":"cd29deeb9ddf","markDefs":[],"children":[{"_type":"span","marks":[],"text":"But let's just suppose now we have established you are a carrier. One of the problems that can happen is we know during pregnancy, for example, your [factor] level may go up, and that's true for hemophilia A, but not for hemophilia B.","_key":"3f94410788870"}],"_type":"block"},{"_key":"4ce42710fa2a","markDefs":[],"children":[{"_type":"span","marks":[],"text":"By the way, the other thing I forgot to tell you is, there is now a major movement afoot not to classify them as carriers but as hemophilia A and B. 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He is also chair of the Medical and Scientific Advisory Council to the National Hemophilia Foundation.","_key":"40647d1670c60"}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"_key":"064f03f76107","_type":"span","marks":[],"text":""}],"_type":"block","style":"normal","_key":"0a00f586cdf2"},{"markDefs":[],"children":[{"_type":"span","marks":["strong","underline"],"text":"Transcript","_key":"6ad4bdfdad240"}],"_type":"block","style":"normal","_key":"54168647a332"},{"style":"normal","_key":"fd853fcb7d51","markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"6ecf360f69df0"}],"_type":"block"},{"_key":"c528f6f1db14","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Can you describe some of the treatment options for hemophilia or other rare blood conditions that are not approved yet, but are anticipated for FDA approval within the next year or so?","_key":"2ff6fb7420fc0"}],"_type":"block","style":"normal"},{"_key":"6088c0e30149","markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"7de84b0441e80"}],"_type":"block","style":"normal"},{"_type":"block","style":"normal","_key":"2c9d50cf0716","markDefs":[],"children":[{"_key":"298c87aed17b0","_type":"span","marks":[],"text":"The foundational problem of patients’ hemophilia is they're at risk for bleeding and particularly severe bleeding into not just soft tissues, but primarily into joints. We call these hemarthroses. Not only do these have acute problems of severe pain and debility, but they can induce an arthropathy that is progressive over the lifespan. Without the ability to prevent joint bleeds, the destiny for a patient with severe hemophilia is that they will end up with a severe hemophilic arthropathy with loss of range of motion, chronic pain and severe disability. What we've been doing for decades is to replace the missing clotting factor. If you have factor VIII deficiency, we want to give you factor VIII, if you have factor VIIII deficiency, we want to give you clotting factor VIIII. In the early days going back to the 1970s, we were able to fractionate those clotting factors from human plasma donors. But since the 1990s, we've had availability of a recombinant version of both factor VIII and factor VIIII. That platform has allowed us to do bioengineering of the molecules and try to improve some of the characteristics of the protein."}]},{"children":[{"text":"","_key":"12b379baacb40","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"251d0f599e82","markDefs":[]},{"_type":"block","style":"normal","_key":"c7219492c5f3","markDefs":[],"children":[{"_type":"span","marks":[],"text":"The most effective innovation has actually been to modulate the pharmacokinetics of the factor VIII or factor VIIII. We now have what are called extended half-life factors, which lasts longer in circulation after infusion. All the factor concentrates have to be delivered intravenously. Even with the extended half-life agents, the half-life is still short enough that, on average, patients have to dose about every other day with factor VIII products, maybe as long as once a week with the longer acting factor VIIII products. That's still a tremendous burden, if you think about from early childhood, all the way through the rest of your life, to have to rely on an IV access.","_key":"b87b475e26c90"}]},{"markDefs":[],"children":[{"text":"","_key":"a597547846f80","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"8ca92074133b"},{"children":[{"_type":"span","marks":[],"text":"One of the greatest innovations in my time caring for patients has been the availability of a of a new substitution therapy for factor VIII. Now, this only works for hemophilia A, but this is a bispecific antibody. It's a monoclonal antibody, it's been humanized, and it substitutes for the clotting function of factor VIII in the body. The beauty of this molecule is it's highly bioavailable, so you can give it subcutaneously, and because it's a monoclonal antibody, it lasts in circulation a really long time, about 30 days. The dosing regimens with this bispecific antibody called emicizumab is once a week, once every 2 weeks, or even once every 4 weeks. The other beauty of this molecule is it doesn't look anything like factor VIII, but it substitutes for it. About 30% of our patients with severe hemophilia develop antibodies directed against the factory molecule and then we can't use that form of therapy anymore. This bispecific antibody, because it doesn't look like factor VIII, is not neutralized by those inhibitor antibodies. This is a prophylactic therapy that can be used whether or not you have a history of an inhibitor. It's greatly revolutionized the care for patients. We can use it all the way from infants all the way through adulthood. This lower burden of administration has really transformed the lives for patients.","_key":"7bfccc26831b0"}],"_type":"block","style":"normal","_key":"e494bc153304","markDefs":[]},{"style":"normal","_key":"3baaa4c56ce4","markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"f7858f1938950"}],"_type":"block"},{"_key":"48be5a7fcb95","markDefs":[],"children":[{"_key":"83c21b45cfb70","_type":"span","marks":[],"text":"There's a whole other category of what we call hemostatic rebalancing agents, which is coming in the pipeline. If you think about your blood clotting system, as procoagulant on one side of the balance and anticoagulants on the other, when you have hemophilia, you're missing an anticoagulant. So, it tilts the balance so that you have a bleeding disorder. We rebalance with procoagulant, like factor VIII or factor VIIII, but we can also rebalance by targeting the natural anticoagulants. There's 3 natural anticoagulants in our blood clotting system, antithrombin, activated protein C, and something called tissue factor pathway inhibitor. It turns out that each of those natural anticoagulants has a biologic target that's being investigated in the clinics. By either neutralizing those functions or knocking down their activity or the levels, it shows a rebalancing of the hemostatic system. We've explored this now in hemophilia, and it looks like these can be effective prophylactic agents, even without giving factor VIII or factor VIIII. "}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"18f07bb1523d"}],"_type":"block","style":"normal","_key":"160343f4d691"},{"_type":"block","style":"normal","_key":"1c4d3940c6bd","markDefs":[],"children":[{"_type":"span","marks":[],"text":"The other beauty of this class of agents, because they work on the natural anticoagulants, they are effective for patients with hemophilia A, hemophilia B, with or without inhibitors. It’s like a cross-platform type of therapy. I think if these get approved, these will also have the advantages of subcutaneous delivery, steady state hemostatic effect, and long duration, so the intensity of the burden of administration will be quite low. ","_key":"3d2995cf13b1"}]},{"_key":"41847b79d0f4","markDefs":[],"children":[{"_type":"span","marks":[],"text":"What I had an opportunity to talk about today was the imminent advent of gene therapy for the hemophilias. If we think about the concept of hemophilia as a monogenic disease, you just have a mutation that's blocking you from expressing factor VIII or factor VIIII. It seems like a nice model, if we could just give you a new functional copy of the factor VIII or factor VIIII gene, and then your own body would be able to make the protein and hopefully, that would restore your blood clotting. We've realized that now, with the current platform of gene therapy, the trick is how do you get the gene, or what we call the transgene, into an organ that's capable of making the clotting factors? What we've settled on is a viral vector delivery system that uses a nonpathogenic virus called the adeno-associated virus. That becomes the sort of packaging for what we want to deliver, which is a copy of the factor VIII or factor VIIII gene. This goes into a patient through a short intravenous infusion, as short as 1 to 3 hours in the outpatient setting. The viral vector homes to the liver, gets taken up by the liver cells, and then the vector particle gets directed to the nucleus where it delivers the payload, and with this case, the factor VIII or factor VIIII gene. Then, the patient's own liver cells start to make the factor VIII or the factor VIIII protein. ","_key":"721e603a651f"}],"_type":"block","style":"normal"},{"style":"normal","_key":"ee6edb4f9f3e","markDefs":[],"children":[{"_key":"ae735dbc5a8a","_type":"span","marks":[],"text":""}],"_type":"block"},{"_key":"95aad4f3b5db","markDefs":[],"children":[{"marks":[],"text":"This has been effective. It works in the vast majority of patients who have received this. There are some challenges that we're working out through the clinical trial processes, but we have long-term correction of blood clotting in patients with hemophilia A as long as 5 years out from this treatment, and as long as 10 years out for hemophilia B. This is a potentially transformative therapy. You no longer have to do prophylaxis with clotting factors, and we've been able to achieve levels that, for the vast majority of the recipients, they don't have any breakthrough bleeding events. I'm excited there's a platform that is before the FDA right now for both hemophilia A as well as for hemophilia B, and I fully expect that next year, we should have products that we'll be delivering through commercial gene therapy in our clinics.","_key":"c059619677c8","_type":"span"}],"_type":"block","style":"normal"}],"link":"/view/dr-steven-pipe-what-to-watch-for-in-the-hemophilia-therapy-pipeline","factCheckAuthorMapping":null,"url":{"current":"dr-steven-pipe-what-to-watch-for-in-the-hemophilia-therapy-pipeline","_type":"slug"},"taxonomyMapping":[{"_updatedAt":"2023-11-03T18:11:36Z","parent":{"_ref":"297fa3d1-5216-46eb-bf51-66c5f77c3c8a","_type":"reference"},"_rev":"rs7eCk0lrr8LMiZP9GB6iV","usedForRecommendations":false,"_id":"47e9ca45-1456-4468-b2ed-1ceb0d1cba59","_type":"taxonomy","name":"Hemophilia","identifier":"hemophilia","perKeywordMapping":["Hematology","Health Equity Diversity \u0026 Inclusion"],"cmeType":"per","_createdAt":"2022-07-28T19:50:48Z"},{"_updatedAt":"2023-10-30T17:48:32Z","identifier":"interviews","_createdAt":"2020-03-30T19:16:54Z","sortOrder":3,"_rev":"xqhc4WIQcOa1P2rns6bqkh","_type":"taxonomy","name":"Interviews","_id":"2d909731-8acf-4e3d-8962-d59b0da85320"}],"updatedOn":null},{"internalTag":["hemophilia","incidence"],"_rev":"olkRnUyLjUnSRjixIezy2l","seoTag":["hemophilia","steven pipe","incidence"],"body":[{"markDefs":[],"children":[{"_key":"9ae2a962d432","_type":"span","marks":[],"text":""}],"_type":"block","style":"normal","_key":"3b27c185049c"},{"_type":"video","videoID":"6316430380112","source":"brightcove","_key":"dc62ce6bb8e8"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"About 1 in 5000 males are born with hemophilia, said Steven W. Pipe, MD, a professor of pediatrics and pathology at the University of Michigan, Ann Arbor and medical director of the Pediatric Hemophilia and Coagulation Disorders Program","_key":"083511e34954"}],"_type":"block","style":"normal","_key":"8731603fa4bd"},{"style":"normal","_key":"56a2e7d3af10","markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"81bbcde1dfa2"}],"_type":"block"},{"children":[{"_type":"span","marks":["strong","underline"],"text":"Transcript","_key":"d5a30fa52b6e"}],"_type":"block","style":"normal","_key":"f43bcad0801d","markDefs":[]},{"style":"normal","_key":"517e4000382b","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"What are some current trends in hemophilia incidence?","_key":"33a3023a452d0"}],"_type":"block"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"48c37170f5ad0"}],"_type":"block","style":"normal","_key":"98fbdbe82a3a"},{"_key":"755bd125a43e","markDefs":[],"children":[{"_type":"span","marks":[],"text":"Hemophilia is a congenital disorder. It's a defective gene for factor VIII, which is hemophilia A or defective gene for factor VIIII, which is hemophilia B. The incidence of hemophilia A and B has been quite static for decades, and it's probably because we've improved the health of carriers as well as the population itself. So, we really don't see any significant changes going forward from what we've been used to seeing over the last few years. There's 30,000-33,000 patients with hemophilia in the US, about 85% of those have hemophilia A, so a factor VIII deficiency; the others are hemophilia B. Just to give you some perspective, it's about 1 in 5000 male births affected by hemophilia. And of those, almost 50% of them are going to have the severe form of the condition.","_key":"05adffaf244f0"}],"_type":"block","style":"normal"}],"link":"/view/dr-steven-pipe-on-current-trends-in-hemophilia-incidence","published":"2022-12-02T20:45:00.000Z","title":"Dr Steven Pipe on Current Trends in Hemophilia Incidence","url":{"current":"dr-steven-pipe-on-current-trends-in-hemophilia-incidence","_type":"slug"},"_createdAt":"2022-11-30T20:30:38Z","authorMapping":[{"biography":[{"_key":"038a30bd95f3","markDefs":[],"children":[{"_type":"span","marks":[],"text":"Hayden is an associate editor for ","_key":"607fdc2dfcbb0"},{"_type":"span","marks":["em"],"text":"The American Journal of Managed Care","_key":"e52dc3c8644d"},{"_type":"span","marks":[],"text":"® (","_key":"594c03a8d658"},{"_type":"span","marks":["em"],"text":"AJMC","_key":"a496fbfb2459"},{"marks":[],"text":"®). She joined the AJMC team in 2021, where she produces written and video content covering multiple disease states.","_key":"421f44e793ed","_type":"span"}],"_type":"block","style":"normal"},{"markDefs":[{"_type":"link","href":"https://www.linkedin.com/in/hayden-e-klein-40b8b1173/","_key":"b0bea7fd1873","nofollow":true,"blank":true}],"children":[{"_type":"span","marks":[],"text":"She has a BA in journalism \u0026 media studies from Rutgers University. You can connect with Hayden on ","_key":"3c55bddd8456"},{"_key":"607fdc2dfcbb1","_type":"span","marks":["b0bea7fd1873"],"text":"LinkedIn"},{"text":".","_key":"607fdc2dfcbb2","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"0c154c4abedd"}],"profileImage":{"alt":"Hayden E. Klein","asset":{"_ref":"image-9a2a017c03e16e188b88d86afed2b268b4b00214-430x430-png","_type":"reference"},"_type":"mainImage"},"lastName":"Klein","authorType":"author","displayName":"Hayden E. 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What will it mean for the health care system if more than one of these expensive therapies is approved in the next 1 to 2 years?","_key":"d6f65e0a6ec10"}]},{"_key":"44ad375a6a6e","markDefs":[],"children":[{"marks":[],"text":"Yeah, that's an interesting thought. When you think of the cell and gene products, you know, I saw 4 therapies for hemophilia A in the pipeline and 2 therapies for hemophilia B. They all have the potential to come to market between now and 2024. All of these products have similar labels upon approval—use limited to moderate to severe disease—and they're really comparable in safety and efficacy. 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I saw some ","_key":"e6f1d2564d430","_type":"span"},{"text":"stats in the ","_key":"e6f1d2564d431","_type":"span","marks":["ef45477e11d8"]},{"_key":"e6f1d2564d432","_type":"span","marks":["ef45477e11d8","em"],"text":"New England Journal of Medicine"},{"marks":[],"text":"; there was an 84% decrease in bleeds and a 99% decrease in factor use for BioMarin's hemophilia product.","_key":"e6f1d2564d433","_type":"span"}],"_type":"block","style":"normal","_key":"155ececc9ac1"},{"_key":"77a8cd1ea6f7","markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"981de44c3fae0"}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"_key":"03065f518c9a0","_type":"span","marks":[],"text":"So, I think they're efficacious. 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