Study of bone marrow failure causes over time
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This study looks at the natural history (how a condition progresses) in people with acquired or inherited bone marrow failure syndromes, including related lung and liver findings in some cases. It helps researchers understand underlying causes—sometimes linked to gene or telomere changes—and how symptoms evolve, which may guide future care.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are age 2 or older
- You have a diagnosis of bone marrow failure or “ineffective blood-making,” or are being evaluated for it
- You (or your legal representative) can understand the study and agree in writing
- You can travel to the NIH Clinical Center (NIH CC) for consultation and testing
- If you are in the special “cohort 3” group: you have a known family gene change in a telomere-related gene OR certain blood/liver/lung findings plus a family history
- If you are in “cohort 5”: you are a first-degree family member of someone with an inherited bone marrow failure syndrome and you can provide a blood or cheek swab (or skin cells) sample
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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