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<a href="/topic/sickle-cell-disease/" class="article_btn">Sickle Cell Disease</a>
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<h1 id="main">Investigating bone marrow transplants: A cure for some sickle cell disease patients</h1>
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<!-- article/investigating-bone-marrow-transplants-a-cure-for-some-sickle-cell-disease-patients investigating-bone-marrow-transplants-a-cure-for-some-sickle-cell-disease-patients -->
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<h2 class="lead_text">NIH researcher works to improve transplant success for adults</h2>
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<img src="/images/uploads/main_images/sickle-cell-fitzhugh.jpg" alt="Courtney Fitzhugh, M.D., is working to expand treatment options for sickle cell patients of all ages. ">
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<p><i class="fa fa-camera"></i> Courtney Fitzhugh, M.D., is working to expand treatment options for sickle cell patients of all ages. </p>
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<p class="lead_text">Sickle cell disease (SCD) specialists have performed bone marrow transplants in children since the 1990s. This procedure is the only cure for SCD to date.</p>
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<p class="p-before-blockquote">NIH MedlinePlus magazine spoke with Courtney Fitzhugh, M.D., a Lasker Clinical Research Scholar at the National Heart, Lung, and Blood Institute, about the current and future outlook for this important treatment.</p>
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<blockquote aria-hidden="true">
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<p>"I'm hoping our research can go beyond sickle cell disease to have a bigger impact."</p>
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<span>- Courtney Fitzhugh, M.D.</span></blockquote>
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<h3>What NIH-supported SCD research are you working on? </h3>
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<p>Since the 1990s, to do a bone marrow transplant, a patient had to have a sibling with a complete tissue match and take a medication that suppresses their immune system (Cyclophosphamide), as well as high dose chemotherapy.</p>
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<p>This has been highly successful in children. The problem is, most adults can't tolerate high doses of chemotherapy because their organs are already damaged and they don't have a sibling with an exact tissue match.</p>
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<p>My research involves giving medications to suppress the immune system in adults. We are trying to create a state where instead of completely replacing bone marrow with that of their donors, we are creating a mixture of donor and patient cells because we now know that as low as 20% donor cells is enough to reverse sickle cell disease.</p>
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<p>If we use a donor who is half-matched, we can increase the chance that a patient will have a suitable donor from 10% to 90%. Parents and children can be a half-match.</p>
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<h3>What prompted you to do this research?</h3>
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<p>About 95% of children can be cured if they have a sibling with a complete tissue match. Adults with a sibling who have a tissue match can also survive with immune suppression medication. I wanted to develop a way to have half-match donors contribute bone marrow and for the outcomes to be successful. </p>
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<h3>What challenges do you face when researching SCD?</h3>
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<p>We had a lot of patients referred to us that we couldn't help because they didn't have a donor who was a tissue match. Then when we developed a half-matched protocol, half of the patients eventually rejected the cells, and the disease came back. So I wrote a new protocol in 2017 which has been much more successful so far. </p>
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<h3>What types of changes are you seeing? </h3>
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<p>Bone marrow transplants are not working in 100% of patients. I want to make changes to make them more successful. Also, I'd like to figure out why a transplant works in some patients rather than others and also focus our approach instead of generally suppressing the whole immune system.</p>
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<p>I'm hoping our research can go beyond sickle cell disease to have a bigger impact.</p>
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<h3>What advice do you have for people with SCD? </h3>
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<p>Keep an open mind about new opportunities that are available. There are a lot of resources for care and treatment. Also, talk to your doctor and join a clinical trial in your area.</p>
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<p>There's a lot of hope. It's an exciting time for sickle cell disease research. Not everyone can get a bone marrow transplant. However, there are a lot of studies, drug trials, and therapies. It's a different time now—it's a better time.</p>
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<div class="mt-1 mb-2"><strong>Image credit:</strong> Courtesy of Courtney Fitzhugh </div>
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<p><i class="fa fa-calendar"></i> January 20, 2020</p>
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<li><a href="https://medlineplus.gov/sicklecelldisease.html" target="_blank">MedlinePlus: Sickle Cell Disease</a></li>
|
||
<li><a href="https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease" target="_blank">National Heart, Lung, and Blood Institute: Sickle Cell Disease</a></li>
|
||
<li><a href="https://newsinhealth.nih.gov/2012/04/when-blood-cells-bend" target="_blank">NIH News in Health: When Blood Cells Bend</a></li>
|
||
<li><a href="https://clinicaltrials.gov/ct2/results?cond=sickle+cell+disease" target="_blank">ClinicalTrials.gov: Sickle Cell Disease</a></li>
|
||
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|
||
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<img src="/images/uploads/ad_images/CSC_Digital302x384Ad_rev1.jpg" alt="Cure sickle cell ">
|
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<figcaption class="nih-caption-text"><p>It's time to rewrite the<br />
|
||
story of sickle cell.</p>
|
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|
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<div><a class="usa-external_link-alt" href="http://curesickle.org/?utm_source=nih%20medlineplus%20magazine&utm_medium=banner&utm_campaign=nhlbi-winter-20" target="_blank">Visit Cure Sickle Cell</a></div>
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|
||
<p><strong><span class="ff-purple">The number one disease among newborn babies </span></strong>is sickle cell disease.</p>
|
||
|
||
|
||
<small><strong>Source:</strong> Centers for Disease Control and Prevention</small>
|
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<div class="explore_link"><a href="https://medlineplus.gov/sicklecelldisease.html" class="usa-external_link" target="_blank">Sickle Cell Disease</a><a href="https://medlineplus.gov/blooddisorders.html" class="usa-external_link" target="_blank">Blood Disorders</a><a href="https://medlineplus.gov/geneticdisorders.html" class="usa-external_link" target="_blank">Genetic Disorders</a></div><div class="explore_link"><a href="https://medlineplus.gov/bonemarrowtransplantation.html" class="usa-external_link" target="_blank">Bone Marrow Transplantation</a><a href="https://medlineplus.gov/cancerchemotherapy.html" class="usa-external_link" target="_blank">Chemotherapy</a> </div>
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