Haplo transplant for severe bone marrow failure using donor cells
Treatments studied
Part of Blood & lymphatic clinical trials.
This trial tests a stem cell transplant from a closely matched but not fully matched family donor (haploidentical), with chemotherapy after the transplant to help prevent graft-versus-host disease (GVHD). It may help people with severe aplastic anemia, certain MDS after transformation, or PNH when other treatments have not worked or are not available.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are age 4 to 60, and you (or your parent/guardian) can understand and agree to the study
- You have severe aplastic anemia with very low bone marrow activity (or it changed into MDS meeting specific lab/pathology limits), or you have PNH that did not respond to eculizumab/ravulizumab or you cannot access it
- Your condition is related to ongoing blood problems such as low red blood cell and/or platelet levels or low neutrophils (or severe PNH symptoms like serious clots or painful hemolysis crises)
- You have at least one haploidentical related donor (a family donor with at least a 5/10 HLA match), plus a backup donor option if the first transplant fails
- No “wrong-match” immune antibodies: you must not have detectable HLA antibodies against mismatched parts from the haplo donor
- Important health limits: you cannot have certain major organ function problems (like very weak heart function, severe liver/kidney limits, or very low lung DLCO) or an uncontrolled serious infection
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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