Donor stem cell transplant for serious blood disorders in children
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a special type of donor stem cell transplant (with donor T-cells removed) to treat serious non-cancer blood problems in children. It may help by resetting the bone marrow so the body can make healthy blood cells again.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your child) have a serious blood disorder like severe sickle cell disease, thalassemia major, or certain bone marrow/autoimmune blood failures
- There must be donor and patient HLA matching: a very close genetic match across key blood/immune markers (5/10 matching)
- The transplant center must find the child’s heart, lungs, kidneys, and liver results are good enough for transplant
- Your child must be well enough to participate (performance score at least 50, depending on age)
- If you have had heavy long-term transfusions for a blood condition, liver tests may be required—and significant liver scarring/hepatitis can exclude you
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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