Marrow Stem Cell Defects Treatment Options | UCSF Benioff Children's Hospitals

<!DOCTYPE html>     <html class="no-js" lang="en">  <head> <link rel="stylesheet" type="text/css" href=/assets/ucsf/main.css?v=40182074ecb56dad04debd94fedfc468 /> <link rel="stylesheet" type="text/css" href=/assets/bch/main.css?v=754aa12d0b3de52563c5716af1d66516 />  <script type="text/javascript" src="https://cdn.cookielaw.org/consent/69de606f-159b-41ad-82da-d7a76950873b/OtAutoBlock.js" ></script> <script src="https://cdn.cookielaw.org/scripttemplates/otSDKStub.js" type="text/javascript" charset="UTF-8" data-domain-script="69de606f-159b-41ad-82da-d7a76950873b" ></script> <script type="text/javascript"> function OptanonWrapper() { } </script> <script type="text/javascript"> function OptanonWrapper() { // Check if banner div is available if (document.getElementById('onetrust-banner-sdk')) { // Get the buttons and container var acceptBtn = document.getElementById("onetrust-accept-btn-handler"); var declineBtn = document.getElementById("onetrust-reject-all-handler"); var cookieSettingsBtn = document.getElementById("onetrust-pc-btn-handler"); var btnContainer = document.getElementById("onetrust-button-group"); // Set the order you want below... btnContainer.append(acceptBtn, cookieSettingsBtn, declineBtn) } } </script>   <script src="https://datalayer.ucsfhealth.org/ucsf-data-layer.js" defer></script>  <script src="https://datalayer.ucsfhealth.org/ucsfbenioffchildrens-marketing-data-layer.js" defer></script>  <script type="text/plain" class="optanon-category-C0007" src="https://assets.adobedtm.com/db5f13cb240b/b1130ce1ceae/launch-db8f55f3e131.min.js" defer></script> <title>Marrow Stem Cell Defects Treatment Options | UCSF Benioff Children's Hospitals</title> <link href="/-/media/project/ucsf/ucsf-bch/header/favicon-32x32.png?rev=7da17355eec44d65b26747967816d41b" rel="shortcut icon" /> <link rel="canonical" href="https://www.ucsfbenioffchildrens.org/treatments/marrow-stem-cell-defects-treatment-options" /> <meta content="Patient Education" name="mainNavName" /> <meta content="false" name="includeBackgroundCircles" /> <meta property="og:description" content="Treatments for marrow failure syndromes include bone marrow transplant, often with medicines that help the transplant work better. 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Read more." /><meta property="twitter:creator" content="@UCSFChildrens" /><meta property="twitter:title" content="Marrow Stem Cell Defects Treatment Options" /><meta property="twitter:card" content="summary_large_image" /> <meta name="viewport" content="width=device-width, initial-scale=1"/> <link rel="manifest" href="/manifest.json" /> <meta name="format-detection" content="telephone=no"> <script type="application/ld+json"> {"@context":"http://schema.org","@type":"Article","description":"Treatments for marrow failure syndromes include bone marrow transplant, often with medicines that help the transplant work better. Read more.","image":"https://www.ucsfbenioffchildrens.org/-/media/project/ucsf/ucsf-bch/images/treatment/hero/marrow-stem-cell-defects-treatment-options-2x.jpg?rev=200054722613458b94974a232b53d690","url":"https://www.ucsfbenioffchildrens.org/treatments/marrow-stem-cell-defects-treatment-options","author":{"@type":"Organization","name":"UCSF Health"},"dateCreated":"2021-03-10T19:05:12+00:00","dateModified":"2021-06-16T14:37:46+00:00","headline":"Marrow Stem Cell Defects Treatment Options","publisher":{"@type":"Organization","name":"UCSF Health","logo":{"@type":"ImageObject","url":"https://www.ucsfbenioffchildrens.org/-/media/project/ucsf/ucsf-health/ezgifcom-gif-maker.png?rev=b49aee14cf934fb086632358c341cf97"}},"articleBody":"At UCSF Benioff Children's Hospitals we use bone marrow transplants from closely matched healthy donors to treat children with a variety of marrow failure disorders, inherited blood disorders and immunosuppressive diseases.\nA BMT can result in 80 percent to 90 percent disease-free survival in children with:\n\n Primary red cell aplasia\n Severe congenital neutropenia or Kostmann's syndrome\n Red cell aplasia or diamond-blackfan syndrome\n Amegakaryocytic thrombocytopenia\n Severe aplastic anemia\n Fanconi's anemia\n\nA BMT from a matched relative can cure 75 percent to 90 percent of children with thalassemia major or sickle cell disease.\nA BMT offers the only known cure – and therefore treatment of choice – for children with most inherited severe immunodeficiency diseases, such as Wiskott-Aldrich disease, Chediak-Higashi syndrome and chronic granulomatous disease. Up to 90 percent are cured by a transplant from a matched donor. \nThe goals of this protocol are to determine:\n\n The efficacy and toxicity of a fludarabine-based conditioning regimen in children with marrow failure syndromes, hemoglobinopathies or severe immunodeficiency diseases other than severe combined immunodeficiency disease (SCID)\n The pharmacokinetics of an intravenous (IV) preparation of busulfan in children undergoing BMT\n\nImproving engraftment\nTwo of the major causes of failure of BMT for these diseases are:\n\n Graft rejection due to an inadequate preparative regimen\n Organ damages caused by the conditioning regimen\n\nThe standard conditioning regimens for all of these non-malignant disorders, except for severe aplastic anemia or Fanconi's anemia, consists of busulfan, cyclophosphamide and anti-thymocyte globulin. Busulfan is an alkylating agent that until recently could only be taken by mouth. A limitation of using busulfan in children is the significant variability in its absorption and clearance. Techniques are available for monitoring the pharmacology of busulfan that have improved its efficacy to reduce graft rejection. However, the use of busulfan in combination with cyclophosphamide also is associated with an increased incidence of liver damage in the form of veno-occlusive disease and bladder damage resulting in hemorrhagic cystitis. The good news is that an IV preparation of busulfan has become commercially available which should improve the administration and pharmacokinetics in children and may reduce these negative side effects. To learn more, please see Busulfan Pharmacokinetics.\nUsing Fludarabine\nRecently, a chemotherapy drug called fludarabine has been used in conditioning regimens, replacing cyclophosphamide as a potent immunosuppressive agent. Fludarabine is a purine analogue that has major effects on resting and activated T and B lymphocytes. It has been associated with minimal side effects, such as nausea, vomiting and neurotoxicity.\nFludarabine has primarily been associated with opportunistic infections, called lymphopenia, and there have been a limited number of reports of autoimmune hemolytic anemia or thrombocytopenia. Fludarabine has been used in combination with busulfan or cyclophosphamide to prepare children with a variety of marrow stem cell disorders. It has been used alone in a limited number of patients with Fanconi's anemia.\nEligibility\nPatients with the following are eligible for this protocol if they have a closely matched related or unrelated donor available:\n\n Severe aplastic anemia who are unresponsive to immunosuppressive therapy within three months follow-up and have an alternative matched unrelated donor\n Congenital marrow failure syndromes, including:\n \n Primary red cell aplasia or diamond-blackfan syndrome\n \n Congenital neutropenia or Kostmann's syndrome\n Amegakaryocytic thrombocytopenia\n Other congenital and acquired cytopenias unresponsive to conventional therapy\n \n \n Inherited blood disorders, including:\n \n Thalassemia major\n Sickle cell anemia\n \n \n Severe immunodeficiency disease, including:\n \n Chediak-Higashi disease\n Wiskott-Aldrich Syndrome\n Combined immunodeficiency disease or Nezelof's\n Hyper IgM syndrome\n Bare lymphocyte syndrome\n Chronic granulomatous disease\n Other stem cell defects, such as osteopetrosis\n Chronic myelogenous leukemia in the first chronic phase\n Acute myelogenous leukemia in first remission\n Myelodysplastic syndrome\n \n \n","articleSection":"Treatment"} </script> </head> <body class="default-device bodyclass"> <a href="#content" class="skip-to-content">Skip to Main Content</a> <div id="wrapper">  <header> <div id="header" class="container-fluid"> <div class="component rich-text ie-warning-banner"> <div class="component-content"> <p> Looks like you’re visiting Benioff Children's Hospitals on Internet Explorer. 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href="/refer-a-patient">Refer a Patient</a></li> </ul> <div class="mobile-nav-footer"> </div> </div> </div> </div> </div> </div> </div> </div> </header>   <main> <div id="content" class="container"> <div class="component fiftyfifty-hero"> <div class="component-content"> <div class="fiftyfifty-hero-image"> <img src="/-/media/project/ucsf/ucsf-bch/images/treatment/hero/marrow-stem-cell-defects-treatment-options-2x.jpg?h=1112&iar=0&w=1668&rev=200054722613458b94974a232b53d690&hash=79E0FB981961AB6F394444C9BF5F876E" alt="marrow-stem-cell-defects-treatment-options-2x" width="1668" height="1112" /> </div> <div class="fiftyfifty-hero-content"> <a href="/treatments" class="fiftyfifty-hero-content-breadcrumb">Treatments A-Z </a> <h1 class=""> <span>Marrow</span> <span>Stem</span> <span>Cell</span> <span>Defects</span> <span>Treatment</span> <span>Options</span> </h1> </div> </div> </div><div class="component share-print-email container clinic-share"> <div class="component-content"> <div class="share-component"> <a aria-label="Print Page" href="#" rel="nofollow" class="icon-ui-print" title="Print"></a> <a aria-label="Share Page" data-email-body="Thought you might want to see this:" class="icon-ui-share" data-email-body="Thought you might want to see this:" data-email-subject="Take a look at this link from UCSF"></a> </div> <div class="clearfix"></div> </div> </div><div class="component article-intro column-layout col-2-66-33 container"> <div class="component-content col-2-80-20"> <div class="article-content left-col"> <div class="font-larger article-text rich-text"> <p>At UCSF Benioff Children's Hospitals we use bone marrow transplants from closely matched healthy donors to treat children with a variety of marrow failure disorders, inherited blood disorders and immunosuppressive diseases.</p> <p>A BMT can result in 80 percent to 90 percent disease-free survival in children with:</p> <ul> <li>Primary red cell aplasia</li> <li>Severe congenital neutropenia or Kostmann's syndrome</li> <li>Red cell aplasia or diamond-blackfan syndrome</li> <li>Amegakaryocytic thrombocytopenia</li> <li>Severe aplastic anemia</li> <li>Fanconi's anemia</li> </ul> <p>A BMT from a matched relative can cure 75 percent to 90 percent of children with thalassemia major or sickle cell disease.</p> <p>A BMT offers the only known cure – and therefore treatment of choice – for children with most inherited severe immunodeficiency diseases, such as Wiskott-Aldrich disease, Chediak-Higashi syndrome and chronic granulomatous disease. Up to 90 percent are cured by a transplant from a matched donor. </p> <p>The goals of this protocol are to determine:</p> <ul> <li>The efficacy and toxicity of a fludarabine-based conditioning regimen in children with marrow failure syndromes, hemoglobinopathies or severe immunodeficiency diseases other than severe combined immunodeficiency disease (SCID)</li> <li>The pharmacokinetics of an intravenous (IV) preparation of <a href="/education/busulfan-pharmacokinetics">busulfan </a>in children undergoing BMT</li> </ul> <h2>Improving engraftment</h2> <p>Two of the major causes of failure of BMT for these diseases are:</p> <ul> <li>Graft rejection due to an inadequate preparative regimen</li> <li>Organ damages caused by the <a href="/education/conditioning-regimen-for-a-bone-marrow-transplant-bmt">conditioning regimen</a></li> </ul> <p>The standard conditioning regimens for all of these non-malignant disorders, except for severe aplastic anemia or Fanconi's anemia, consists of busulfan, cyclophosphamide and anti-thymocyte globulin. <a href="/education/busulfan-pharmacokinetics">Busulfan</a> is an alkylating agent that until recently could only be taken by mouth. A limitation of using busulfan in children is the significant variability in its absorption and clearance. Techniques are available for monitoring the pharmacology of busulfan that have improved its efficacy to reduce graft rejection. However, the use of busulfan in combination with cyclophosphamide also is associated with an increased incidence of liver damage in the form of veno-occlusive disease and bladder damage resulting in hemorrhagic cystitis. The good news is that an IV preparation of busulfan has become commercially available which should improve the administration and pharmacokinetics in children and may reduce these negative side effects. To learn more, please see <a href="/education/busulfan-pharmacokinetics">Busulfan Pharmacokinetics</a>.</p> <h2>Using Fludarabine</h2> <p>Recently, a chemotherapy drug called fludarabine has been used in <a href="/education/conditioning-regimen-for-a-bone-marrow-transplant-bmt">conditioning regimens</a>, replacing cyclophosphamide as a potent immunosuppressive agent. Fludarabine is a purine analogue that has major effects on resting and activated T and B lymphocytes. It has been associated with minimal side effects, such as nausea, vomiting and neurotoxicity.</p> <p>Fludarabine has primarily been associated with opportunistic infections, called lymphopenia, and there have been a limited number of reports of autoimmune hemolytic anemia or thrombocytopenia. Fludarabine has been used in combination with busulfan or cyclophosphamide to prepare children with a variety of marrow stem cell disorders. It has been used alone in a limited number of patients with Fanconi's anemia.</p> <h2>Eligibility</h2> <p>Patients with the following are eligible for this protocol if they have a closely matched <a href="/education/types-of-pediatric-bone-marrow-transplant-bmt-donors">related</a> or <a href="/education/unrelated-bone-marrow-transplant-bmt-donor-program">unrelated</a> donor available:</p> <ul> <li>Severe aplastic anemia who are unresponsive to immunosuppressive therapy within three months follow-up and have an alternative matched unrelated donor</li> <li>Congenital marrow failure syndromes, including: <ul> <li>Primary red cell aplasia or diamond-blackfan syndrome </li> <li>Congenital neutropenia or Kostmann's syndrome</li> <li>Amegakaryocytic thrombocytopenia</li> <li>Other congenital and acquired cytopenias unresponsive to conventional therapy</li> </ul> </li> <li>Inherited blood disorders, including: <ul> <li>Thalassemia major</li> <li>Sickle cell anemia</li> </ul> </li> <li>Severe immunodeficiency disease, including: <ul> <li>Chediak-Higashi disease</li> <li>Wiskott-Aldrich Syndrome</li> <li>Combined immunodeficiency disease or Nezelof's</li> <li>Hyper IgM syndrome</li> <li>Bare lymphocyte syndrome</li> <li>Chronic granulomatous disease</li> <li>Other stem cell defects, such as osteopetrosis</li> <li>Chronic myelogenous leukemia in the first chronic phase</li> <li>Acute myelogenous leukemia in first remission</li> <li>Myelodysplastic syndrome</li> </ul> </li> </ul> </div> </div> <div class="article-info right-col"> </div> </div> </div> <div class="component content container"> <div class="component-content"> <div class="page-content"> <div class="component column-layout col-2-66-33 col-xs-12 col-2-80-20"> <div class="component-content"> <div class="left-col"> <div class="component disclaimer col-xs-12"> <div class="component-content"> <div class="field-ucsf-bch-text"><p><em>UCSF Benioff Children's Hospitals medical specialists have reviewed this information. It is for educational purposes only and is not intended to replace the advice of your child's doctor or other health care provider. We encourage you to discuss any questions or concerns you may have with your child's provider.</em></p></div> </div> </div> </div> <div class="right-col"> </div> </div> </div> <div class="component related-conditions col-xs-12"> <div class="component-content"> <div> <h2 class="heading1 title-anchor"> Related conditions </h2> </div> <div> <div class="meta-links"> <div class="component-content"> <ul class="use-arrow-icon vertical-sort "> <li class=""> <p class="font-smaller"> <a href="/conditions/aplastic-anemia" target="_blank"> Aplastic anemia </a> </p> </li> <li class=""> <p class="font-smaller"> <a href="/conditions/ewings-sarcoma" target="_blank"> Ewing's sarcoma </a> </p> </li> <li class=""> <p class="font-smaller"> <a href="/conditions/hodgkins-lymphoma" target="_blank"> Hodgkin's lymphoma </a> </p> </li> <li class=""> <p class="font-smaller"> <a href="/conditions/leukemia" target="_blank"> Leukemia </a> </p> </li> <li class=""> <p class="font-smaller"> <a href="/conditions/neuroblastoma" target="_blank"> Neuroblastoma </a> </p> </li> <li class=""> <p class="font-smaller"> <a href="/conditions/neutropenia" target="_blank"> Neutropenia </a> </p> </li> <li class=""> <p class="font-smaller"> <a href="/conditions/non-hodgkins-lymphoma" target="_blank"> Non-Hodgkin's lymphoma </a> </p> </li> <li class=""> <p class="font-smaller"> <a href="/conditions/osteosarcoma-bone-cancer-in-children" target="_blank"> Osteosarcoma </a> </p> </li> <li class=""> <p class="font-smaller"> <a href="/conditions/sickle-cell-disease" target="_blank"> Sickle cell disease </a> </p> </li> </ul> </div> </div> </div> </div> </div> <div class="component numbered-clinic-rollup col-xs-12"> <div class="component-content"> <h2 class="heading1">Clinics we work with (1)</h2> <div class="cards"> <div class="numbered-clinic-card"> <div class="numbered-clinic-card-first"> <div class="numbered-clinic-card-first-image"> <img loading="lazy" src="/-/media/project/ucsf/ucsf-bch/images/clinic/numbered-clinic-card/blood-and-marrow-transplant-clinic-320x320-2x.jpg?rev=1f44e5e064aa4042875be187ef616cbc&hash=6F4814B39935F7AB743C3B2F18FA960E" alt="blood-and-marrow-transplant-clinic-320x320-2x" width="640" height="640"> <span>3</span> </div> <h2 class="numbered-clinic-card-header first">Blood & Marrow Transplant (BMT) Clinic</h2> </div> <div class="numbered-clinic-card-second"> <h2 class="numbered-clinic-card-header second">Blood & Marrow Transplant (BMT) Clinic</h2> <div class="numbered-clinic-card-second-locations"> <a href="/clinics/blood-and-marrow-transplant-bmt-clinic" target="_self" title="Blood & Marrow Transplant (BMT) Clinic"></a> <span><p>San Francisco / Oakland</p></span> </div> </div> </div> </div> </div> </div><div class="component doctor-results col-xs-12"> <div class="component-content"> <h2 class="heading1 title-anchor"> Our experts </h2> <div class="doctor-results-wrapper custom-scrollbar-container"> <ul> <li> <a href="/providers/dr-morton-j-cowan"> <img loading="lazy" src="/-/media/project/ucsf/ucsf-bch/images/provider/card/dr-mort-cowan-md-103-320x320-2x.jpg?h=526&w=526&rev=07d9150aa59948e5b1d628548c20a00f&hash=46BE86079C1622398534595AB939DA92" alt="dr-mort-cowan-md-103-320x320-2x" width="640" height="640"> <div> <h3 class="doctor-name"> Morton J. Cowan </h3> <p class="font-larger doctor-class"> MD </p> <span class="doctor-simple-title"> Pediatric immunologist and bone marrow transplant specialist </span> </div> </a> </li> <li> <a href="/providers/dr-christopher-dvorak"> <img loading="lazy" src="/-/media/project/ucsf/ucsf-bch/images/provider/card/dr-chris-dvorak-md-106-320x320-2x.jpg?h=526&w=526&rev=842c2f60a19d43c69629ac97d0892f80&hash=F7FCEADD632B99556B0548FCF76104DF" alt="dr-chris-dvorak-md-106-320x320-2x" width="640" height="640"> <div> <h3 class="doctor-name"> Christopher Dvorak </h3> <p class="font-larger doctor-class"> MD </p> <span class="doctor-simple-title"> Pediatric hematologist-oncologist </span> </div> </a> </li> <li> <a href="/providers/dr-robert-e-goldsby"> <img loading="lazy" src="/-/media/project/ucsf/ucsf-bch/images/provider/card/dr-robert-goldsby-md-104-320x320-2x.jpg?h=526&w=526&rev=47237b0c5d40457eb4fd941f2f34b4f7&hash=0EC12BB650473A1633AD2F41A696AD1C" alt="" width="640" height="640"> <div> <h3 class="doctor-name"> Robert E. Goldsby </h3> <p class="font-larger doctor-class"> MD </p> <span class="doctor-simple-title"> Pediatric hematologist-oncologist </span> </div> </a> </li> <li> <a href="/providers/dr-james-huang"> <img loading="lazy" src="/-/media/project/ucsf/ucsf-bch/images/provider/card/dr-james-huang-md-88685-320x320-2x.jpg?h=526&w=526&rev=3fca57cf7f0c4aa49258dc448e736150&hash=C62A327FBE6BBFEB79F1EBB67FDE8788" alt="dr-james-huang-md-88685-320x320-2x" width="640" height="640"> <div> <h3 class="doctor-name"> James Huang </h3> <p class="font-larger doctor-class"> MD </p> <span class="doctor-simple-title"> Pediatric hematologist </span> </div> </a> </li> </ul> </div> </div> </div><div class="component article-cards cards-component col-xs-12"> <div class="component-content"> <div> <h2 class="heading1 title-anchor"> Recommended reading </h2> <div class="view-all"> </div> </div> <div class="cards"> <div class="card"> <div class="component-content"> <div class="card-content"> <a href="/education/conditioning-regimen-for-a-bone-marrow-transplant-bmt" class="phantom-link" title="Conditioning Regimen for a Bone Marrow Transplant (BMT)"></a> <img loading="lazy" src="/-/media/project/ucsf/ucsf-bch/images/education/hero/conditioning-regimen-for-a-bmt-2x.jpg?h=376&w=974&rev=4dfa0e2ce127497d9e3c1057493ee770&hash=5466D13643EF68549A012F57C7E0BF7B" alt="conditioning-regimen-for-a-bmt-2x" width="2880" height="1112" /> <p class="font-larger"> Conditioning Regimen for a Bone Marrow Transplant (BMT) </p> <p> Getting ready for a bone marrow transplant (BMT) may include chemotherapy and radiation. 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