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category-thalassemia-patients tag-agranulocytosis tag-blood-disorders tag-chelators tag-deferasirox tag-health-professionals tag-hemoglobin tag-iron-overload tag-monotherapy tag-novel-developments tag-oxygen tag-serum-creatinine tag-synergy tag-thalassemia" itemtype="https://schema.org/CreativeWork" itemscope> <div class="inside-article"> <div class="featured-image page-header-image-single "> <img width="1200" height="628" src="https://mutation.blog/archive/wp-content/uploads/2024/09/banner-5-1-09-min-scaled-e1727522866186.jpg" class="attachment-full size-full" alt="" itemprop="image" decoding="async" fetchpriority="high" /> </div> <header class="entry-header"> <h1 class="entry-title" itemprop="headline">Advancements in Iron Chelation Therapy for Thalassemia Patients</h1> <div class="entry-meta"> <span class="posted-on"><time class="updated" datetime="2024-09-28T16:57:54+05:30" itemprop="dateModified">September 28, 2024</time><time class="entry-date published" datetime="2024-09-28T14:47:10+05:30" itemprop="datePublished">September 28, 2024</time></span> <span class="byline">by <span class="author vcard" itemprop="author" itemtype="https://schema.org/Person" itemscope><a class="url fn n" href="https://mutation.blog/archive/author/mutation/" title="View all posts by mutation" rel="author" itemprop="url"><span class="author-name" itemprop="name">mutation</span></a></span></span> </div> </header> <div class="entry-content" itemprop="text"> <h3><b>Introduction</b></h3> <p><span style="font-weight: 400;">Thalassemia is one of the blood disorders passed on to the offspring in which the human body fails to make enough hemoglobin for proper oxygen uptake. The treatment mostly involves frequent blood transfusions to treat anemia. On the negative side, there are repercussions associated with regular transfusions: iron burden. If unchecked, excess iron may settle within different organs, creating complications such as liver cirrhosis, heart disorders, and endocrine malfunction. These past years have been imperative for the role that iron chelating therapy has played in the management of iron overload in thalassemia patients. Novel developments significantly improved the safety, efficacy, and convenience of iron chelation treatment, bringing renewed hope to both patients and health professionals.</span></p> <h3><b>Initial Treatments and Historical Context</b></h3> <p><span style="font-weight: 400;">In the 1960s, iron chelation therapy was pioneered by the introduction of deferoxamine (DFO). DFO is introduced through subcutaneous or intravenous perfusion in a way that efficiently binds excess iron and promotes its excretion via urine and feces. While this treatment was effective, regular infusions, which sometimes caused pain, and expensive charges created hindrances to patients for long-term compliance and quality of life. Consequently, oral chelators were developed to provide an alternative that is more convenient to be used by patients.</span></p> <h3><b>Development of Oral Chelators</b></h3> <p><span style="font-weight: 400;">Deferiprone (DFP) was the first of the newer generation of oral iron chelators that were introduced following the drawbacks of DFO. DFP was more easily administered than SOT because it was an oral agent, compared to the prior painful injections of the latter. These trials showed that it is capable of reducing serum ferritin levels and, best of all, enhancing the cardiac iron load, therefore expanding the armamentarium for the treatment of patients with iron overload. Nevertheless, constant unexpected issues were found in DFP implementation. It was accompanied by side effects like gastrointestinal disturbances and, in some cases, agranulocytosis that required constant monitoring, not being too rare.</span></p> <p></div></div> <div style="background: #f7f7f7;border: 1px solid rgba(0, 0, 0, 0.07);"> <div style="padding: 30px;"><div class="Adblock-main"> <div class="Adblock-head"> <h2>Yearwise Publication Trend on <b>“<a href="https://mutation.blog/publication-trends/index/thalassemia patients" target="_blank" title="thalassemia patients - yearwise publication trends">thalassemia patients</a>”</b></h2> </div> </div><div class="results-container"><div class="chart-block" style="padding:15px;"> <div class="left"> <div id="results" class="results"></div> </div> <div class="right"> <div class="chart-container"><canvas id="publicationChart"></canvas></div> </div> <div class="keywordsdiv"> <div style="text-align:center;"><b>Find publication trends on relevant topics</b> </div> <span class="gp-icon icon-tags"><svg viewBox="0 0 512 512" aria-hidden="true" xmlns="http://www.w3.org/2000/svg" width="1em" height="1em"><path d="M20 39.5c-8.836 0-16 7.163-16 16v176c0 4.243 1.686 8.313 4.687 11.314l224 224c6.248 6.248 16.378 6.248 22.626 0l176-176c6.244-6.244 6.25-16.364.013-22.615l-223.5-224A15.999 15.999 0 00196.5 39.5H20zm56 96c0-13.255 10.745-24 24-24s24 10.745 24 24-10.745 24-24 24-24-10.745-24-24z"></path><path d="M259.515 43.015c4.686-4.687 12.284-4.687 16.97 0l228 228c4.686 4.686 4.686 12.284 0 16.97l-180 180c-4.686 4.687-12.284 4.687-16.97 0-4.686-4.686-4.686-12.284 0-16.97L479.029 279.5 259.515 59.985c-4.686-4.686-4.686-12.284 0-16.97z"></path></svg></span> <span id="keyword-stats"></span> </div> </div></div></div><div class="inside-article"><style> table { margin: 0 0 1.5em; 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if (!statistics || Object.keys(statistics).length === 0) { resultsContainer.innerHTML = '<p>No data found.</p>'; return; } var tableHTML = `<div class='pub-scroll'> <table class='tablediv' border='1' cellspacing='0' cellpadding='0'> <tr> <th>Year</th> <th>Publication Count</th> </tr>`; Object.entries(statistics).sort(([yearA], [yearB]) => yearB - yearA).forEach(([year, count]) => { const displayCount = count === 0 ? 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DFX fixes iron and increases the level of iron expelled from the body through the feces. Clinical investigations have found DFX useful in the prevention and treatment of overload both in the liver and in the heart, which makes DFX all-embracing in handling thalassemia patients. This makes their once-a-day use of the drug easy for the patients to deal with, hence improving their standard of living. Recent years have shown cohorts proven to be safe and confirm what was rightfully cited as mild gastrointestinal symptoms and a non-progressive rise in serum creatinine.</span></p> <h3><b>Crossover and Maintenance of Treatment Strategies</b></h3> <p><span style="font-weight: 400;">The efficacy of different chelation therapies has been investigated, especially regarding the comparison between monotherapy and combined therapy regimens. </span><span style="font-weight: 400;">Among these, a fairly controlled trial was carried out comparing the sequential, year-long administration of DFO in conjunction with DFP and DFP only for thalassemia major patients. </span><span style="font-weight: 400;">The analysis revealed that the combination therapy had a substantial effect on the reduction of serum ferritin levels in patients as compared to those on DFP monotherapy; however, no differences in terms of survival and SIDAE were observed. This study also stressed the importance of synergy in using chelators as a nearly perfect method of treatment with the fewest side effects because the side effects can be minimized while improving the efficiency when chelators are combined.</span></p> <p><span style="font-weight: 400;">A further crucial investigation focused on the administration of both DFO and DFP in a cohort of patients with cardiac iron burden. To my knowledge, combined therapy was proven to be more effective than DFO therapy in reducing myocardial iron. These findings are rather important given that cardiologic events are among the main causes of death in TM patients. Thus, successful clearance of DIF and the possibility of managing cardiac iron load with combination therapy have become decisive achievements, providing more favorable outcomes for the patients.</span></p> <h3><b>Improvements in Supervisory and Personalized Treatment</b></h3> <p><span style="font-weight: 400;">Cardiovascular magnetic resonance, particularly T2* CMR, has recently enhanced the method of monitoring iron overload among individuals with thalassemia. CMR enables the precise measurement of cardiac and liver iron concentrations without the need for invasive techniques, thus enabling early modifications of these patients’ therapy. This individualization of treatment guarantees that the patient gets the optimal dose and regimen of the drug, and, therefore, rare side effects appear out of iron toxicity or the toxic effects of the chelating agents.</span></p> <p></div></div> <div style="background: #f7f7f7;border: 1px solid rgba(0, 0, 0, 0.07);"> <div style="padding: 30px;"><div class="Adblock-main"> <div class="Adblock-head"> <h2>Recent Publications on <b>“<a href="https://mutation.blog/recent-publications/index/thalassemia patients" target="_blank" rel="noopener" title="thalassemia patients - yearwise publication list">thalassemia patients</a>”</b></h2> </div> </div> <div class="pb-main"><div class="article-scroll"><div id="results_recent" class="results"></div></div><div class="keywordsdiv" style="margin: 0px 15px;margin-top:20px;"> <div style="text-align:center;"><b>Find publications on relevant topics</b> </div> <span class="gp-icon icon-tags"><svg viewBox="0 0 512 512" aria-hidden="true" xmlns="http://www.w3.org/2000/svg" width="1em" height="1em"><path d="M20 39.5c-8.836 0-16 7.163-16 16v176c0 4.243 1.686 8.313 4.687 11.314l224 224c6.248 6.248 16.378 6.248 22.626 0l176-176c6.244-6.244 6.25-16.364.013-22.615l-223.5-224A15.999 15.999 0 00196.5 39.5H20zm56 96c0-13.255 10.745-24 24-24s24 10.745 24 24-10.745 24-24 24-24-10.745-24-24z"></path><path d="M259.515 43.015c4.686-4.687 12.284-4.687 16.97 0l228 228c4.686 4.686 4.686 12.284 0 16.97l-180 180c-4.686 4.687-12.284 4.687-16.97 0-4.686-4.686-4.686-12.284 0-16.97L479.029 279.5 259.515 59.985c-4.686-4.686-4.686-12.284 0-16.97z"></path></svg></span> <span id="keyword-papers"></span> </div></div></div><div class="inside-article"> <style> .pb-main{ border: solid 1px #ccc; border-top: none; margin-bottom: 20px; padding-bottom: 25px; background:#fff; } .author-main { border: solid 1px #ccc; border-top: none; margin-bottom: 20px; padding-bottom: 25px; background:#fff; } .publication-block { padding: 10px; margin-bottom: 10px; background-color: #f9f9f9; text-align: left; background: #FFF; border-bottom: solid 1px #ccc; margin-left: 15px; margin-right: 15px; } .publication-block h3 { margin: 0 0 10px; color: #000!important; } .publication-block a { font-size: 16px !important; line-height: 1em; font-weight: 600; text-transform: none; color: #000; padding: 0px; } .publication-block a:hover{ color: #227cdc; text-decoration:underline; } .article-scroll { max-height: 445px; overflow-y: auto; overflow-x: hidden; } ::-webkit-scrollbar-track { -webkit-box-shadow: inset 0 0 6px rgba(0,0,0,0.3); background-color: #efefef; border-radius:30px; } ::-webkit-scrollbar { width: 6px; background-color: #efefef; border-radius:30px; } ::-webkit-scrollbar-thumb { background-color: #ababab; 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publicationBlock.innerHTML = publicationHTML; resultsContainer.appendChild(publicationBlock); }); } function displayKeywordPapers(keywords) { var resultsContainer = document.getElementById('keyword-papers'); resultsContainer.innerHTML = ''; if (!keywords || keywords.length === 0) { resultsContainer.innerHTML = '<p>No data found.</p>'; return; } var keywordHTML = ''; keywords.forEach((key, index) => { let key_replace = key.replace(/ /g, '-'); key_replace = key_replace.toLowerCase(); keywordHTML += `<a href="https://mutation.blog/recent-publications/index/${key_replace}" target="_blank" title="${key} - publication list">${key}</a>`; if (index < keywords.length - 1) { keywordHTML += ', '; } }); resultsContainer.innerHTML = keywordHTML; } // Call the function with the PHP data var recent_papers = [ { "title": "Red Cell Alloimmunisation Among Sickle Cell Disease and Thalassemia Patients Following Rh- and K-Matched Red Cell Transfusion in Southwestern Saudi Arabia: A Multicenter Study.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38947563", "publishedDate": "2024" }, { "title": "Optimizing the management of inherited blood disorders in a changing market: Findings from the AMCP Market Insights Program.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38953485", "publishedDate": "2024" }, { "title": "Awareness and practical evaluation of correct use of iron chelators; a study to track the ambiguities of thalassemia patients on their medications in Iran.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38872196", "publishedDate": "2024" }, { "title": "Prenatal Diagnosis of Cystic Fibrosis by Celocentesis.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38927598", "publishedDate": "2024" }, { "title": "Catalase, Glutathione Peroxidase, and Peroxiredoxin 2 in Erythrocyte Cytosol and Membrane in Hereditary Spherocytosis, Sickle Cell Disease, and \u03b2-Thalassemia.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38929068", "publishedDate": "2024" }, { "title": "How I treat iron overload in adult myelodysplastic syndrome.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38941618", "publishedDate": "2024" }, { "title": "Early Detection of Renal Complication in Children With Sickle Cell Disease: A Single Center Prospective Study.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/39114188", "publishedDate": "2024" }, { "title": "Factors Associated With Quality of Life Among Adolescent With Beta Thalassemia in Indonesia: A Cross-Sectional Study.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/39109385", "publishedDate": "2024" }, { "title": "Routine antenatal molecular testing for \u03b1-thalassemia at a tertiary referral hospital in China: ten years of experience.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38894721", "publishedDate": "2024" }, { "title": "Endoscopic manifestation of intestinal transplant-associated microangiopathy after stem cell transplantation.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38649868", "publishedDate": "2024" }, { "title": "Multiparametric cardiac magnetic resonance in patients with thalassemia intermedia: new insights from the E-MIOT network.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38683500", "publishedDate": "2024" }, { "title": "Differential effects of iron chelators on iron burden and long-term morbidity and mortality outcomes in a large cohort of transfusion-dependent \u03b2-thalassemia patients who remained on the same monotherapy over 10\u00a0years.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38820707", "publishedDate": "2024" }, { "title": "Survival of transfused red blood cells from a donor with alpha-thalassemia trait in a recipient with sickle cell disease.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38693059", "publishedDate": "2024" }, { "title": "COVID-19 presenting as severe acute hepatitis in a pediatric patient with thalassemia minor: A case report.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38799536", "publishedDate": "2024" }, { "title": "Modified lentiviral globin gene therapy for pediatric \u03b2\/\u03b2 transfusion-dependent \u03b2-thalassemia: A single-center, single-arm pilot trial.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38759653", "publishedDate": "2024" }, { "title": "Clinical Practice Patterns in Sickle Cell Disease Treatment: Disease-Modifying and Potentially Curative Therapies.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38718300", "publishedDate": "2024" }, { "title": "Elevating fetal hemoglobin - recently discovered regulators and mechanisms.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38728575", "publishedDate": "2024" }, { "title": "Iron chelation therapy for children with transfusion-dependent \u03b2-thalassemia: How young is too young?", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38753107", "publishedDate": "2024" }, { "title": "New Born Screening of Hemoglobinopathies in a Center Tunisian Population.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38748601", "publishedDate": "2024" }, { "title": "Nutritional and Body Composition Changes in Paediatric \u03b2-Thalassemia Patients Undergoing Hematopoietic Stem Cell Transplantation: A Retrospective Study Using Bioelectrical Impedance Analysis.", "url": "https:\/\/pubmed.ncbi.nlm.nih.gov\/38751668", "publishedDate": "2024" } ]; var keywordsArray = ["Thalassemia","Iron Chelation Therapy","Deferoxamine","Deferiprone","Deferasirox","Iron Overload","Cardiac Iron Load","Combination Therapy","Personalized Medicine"]; displayResults_recent(recent_papers); displayKeywordPapers(keywordsArray); // function stripslashes(str) { // if (typeof str === 'string') { // return str.replace(/\/g, ''); // } // } </script></p> <h3><b>Current and Emerging Work and Research Directions</b></h3> <p><span style="font-weight: 400;">Iron chelation therapy is an evolving field of science, and research work in progress is aiming at providing better treatment with higher potencies and safety factors, along with newer approaches. The studies are being conducted for the development of oral chelators with improved pharmacokinetics and minimum side effects. Furthermore, genetic and biomarker-based treatments are searched in experiments to enhance the efficiency of iron chelation therapy and ensure the administration of the right treatment to each patient in conformity with their genetic profile and disease symptoms.</span></p> <p><span style="font-weight: 400;">One of the most promising directions in the area is the research of multitarget chelators that would combine the action of several chelants. At the moment, scientists use chelators working on certain routes through which iron is expelled from the organism and rely on synergistic effects to increase the efficacy of chelators, decrease the frequency and application dosage to minimize side effects, and increase patient compliance.</span></p> <h3><b>Conclusion</b></h3> <p><span style="font-weight: 400;">The progress in the treatment of iron chelation for patients with thalassemia over the past few decades has brought substantive changes in controlling cases of iron involvement that have consequently boosted the life duration of the patients and given them a better quality of life. The history of iron chelation therapy can be divided into four distinct generations, starting with the painful and time-consuming IV deferoxamine therapy and ending with the efficient and comfortable oral agents deferiprone and deferasirox. There is hope for even better forms of treatment and personalization in the future, given the never-ending innovations and research. Looking ahead, the goal of enhancing the quality of life for patients suffering from thalassemia through efficient handling of iron overload shall always remain unchallenged.</span></p> <p></p> <h3><b>References</b></h3> <ol> <li>Maggio, A., Vitrano, A., Capra, M., Cuccia, L., Gagliardotto, F., Filosa, A., Romeo, M.A., Magnano, C., Caruso, V., Argento, C. and Gerardi, C., 2009. <a href="https://onlinelibrary.wiley.com/doi/abs/10.1111/j.1365-2141.2009.07609.x">Long‐term sequential deferiprone–deferoxamine versus deferiprone alone for thalassaemia major patients: a randomized clinical trial.</a> <i>British journal of haematology</i>, <i>145</i>(2), pp.245-254.</li> <li>Galanello, R. and Origa, R., 2008. <a href="https://www.tandfonline.com/doi/abs/10.1586/17512433.1.2.231">Once-daily oral deferasirox for the treatment of transfusional iron overload.</a> <i>Expert Review of Clinical Pharmacology</i>, <i>1</i>(2), pp.231-240.</li> <li>Cappellini, M.D., Cohen, A., Piga, A., Bejaoui, M., Perrotta, S., Agaoglu, L., Aydinok, Y., Kattamis, A., Kilinc, Y., Porter, J. and Capra, M., 2006. <a href="https://ashpublications.org/blood/article/107/9/3455/133482/A-phase-3-study-of-deferasirox-ICL670-a-once-daily">A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with β-thalassemia.</a> <i>Blood</i>, <i>107</i>(9), pp.3455-3462.</li> <li>Neufeld, E.J., 2006. <a href="https://ashpublications.org/blood/article/107/9/3436/133443/Oral-chelators-deferasirox-and-deferiprone-for">Oral chelators deferasirox and deferiprone for transfusional iron overload in thalassemia major: new data, new questions.</a> <i>Blood</i>, <i>107</i>(9), pp.3436-3441.</li> <li>Anderson, L.J., Westwood, M.A., Holden, S., Davis, B., Prescott, E., Wonke, B., Porter, J.B., Malcolm Walker, J. and Pennell, D.J., 2004. <a href="https://onlinelibrary.wiley.com/doi/abs/10.1111/j.1365-2141.2004.05202.x">Myocardial iron clearance during reversal of siderotic cardiomyopathy with intravenous desferrioxamine: a prospective study using T2* cardiovascular magnetic resonance.</a> <i>British journal of haematology</i>, <i>127</i>(3), pp.348-355.</li> <li>Tanner, M.A., Galanello, R., Dessi, C., Smith, G.C., Westwood, M.A., Agus, A., Roughton, M., Assomull, R., Nair, S.V., Walker, J.M. and Pennell, D.J., 2007. <a href="https://www.ahajournals.org/doi/full/10.1161/CIRCULATIONAHA.106.648790">A randomized, placebo-controlled, double-blind trial of the effect of combined therapy with deferoxamine and deferiprone on myocardial iron in thalassemia major using cardiovascular magnetic resonance.</a> <i>Circulation</i>, <i>115</i>(14), pp.1876-1884.</li> <li>Borgna-Pignatti, C., Cappellini, M.D., De Stefano, P., Del Vecchio, G.C., Forni, G.L., Gamberini, M.R., Ghilardi, R., Piga, A., Romeo, M.A., Zhao, H. and Cnaan, A., 2006. <a href="https://ashpublications.org/blood/article/107/9/3733/133488/Cardiac-morbidity-and-mortality-in-deferoxamine-or">Cardiac morbidity and mortality in deferoxamine-or deferiprone-treated patients with thalassemia major.</a> <i>Blood</i>, <i>107</i>(9), pp.3733-3737.</li> </ol> <p></div></div> <div style="background: #f7f7f7;border: 1px solid rgba(0, 0, 0, 0.07);"> <div style="padding: 30px;"><div class="Adblock-main"> <div class="Adblock-head"> <h2>Top Experts on “<b style="color:#000;font-size:22px;">thalassemia patients</b>“</h2> </div> </div><div class="author-main"><div id="results_author"></div><div style="text-align: center;"><a class="register-button" href="https://mutation.blog/expert-search" target="_blank" rel="noopener">Find experts on any field</a></div></div><div class="inside-article" style="background: none;border: none;box-shadow: none;margin-top: -70px;"> <style> .author-block { padding: 15px; 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