New transplant prep aims to cut complications in bone marrow failure patients
NCT ID NCT04965597
First seen Nov 01, 2025 · Last updated Apr 28, 2026 · Updated 30 times
Summary
This study tested a combination of three drugs (treosulfan, fludarabine, and rabbit antithymocyte globulin) given before a bone marrow or blood stem cell transplant to people with bone marrow failure diseases. The goal was to see if this drug mix could reduce serious complications like graft-versus-host disease and transplant failure. The study included 40 patients aged 1 to 49 years with conditions such as Diamond-Blackfan anemia or Shwachman-Diamond syndrome. While the transplant may improve blood cell counts, patients typically need lifelong monitoring and sometimes ongoing medication, so this is not a cure.
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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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Contacts and locations
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Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
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Cohen Children's Hospital of NY
Queens, New York, 11040, United States
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Duke University Medical Center
Durham, North Carolina, 27705, United States
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Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109, United States
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Johns Hopkins University
Baltimore, Maryland, 21231, United States
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MD Anderson Cancer Center
Houston, Texas, 77030, United States
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Medical College of Wisconsin/Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Oregon Health & Science University
Portland, Oregon, 97239, United States
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Primary Children's/University of Utah
Salt Lake City, Utah, 84113, United States
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Rady Children's Hospital/UCSD
San Diego, California, 92123, United States
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Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14203, United States
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St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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University of California San Francisco
San Francisco, California, 94143, United States
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University of Michigan Medical Center
Ann Arbor, Michigan, 48109, United States
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
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Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
Conditions
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