Half-Matched stem cell transplant offers new hope for rare blood diseases
NCT ID NCT03520647
First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 27 times
Summary
This study tests whether a stem cell transplant using blood stem cells from a half-matched family member (haploidentical donor) can safely treat severe aplastic anemia, a related bone marrow disorder (MDS), and a blood cell disease (PNH). Participants receive chemotherapy and a one-time radiation dose before the transplant, then take drugs to prevent graft-versus-host disease (GVHD). The main goal is to see how many patients are alive and free of moderate or severe chronic GVHD one year after transplant. The study enrolls patients aged 4 to 60 and their donors aged 4 to 75.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Study contacts
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Contact
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Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
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Conditions
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