Study of genetic and blood features in Upshaw-Schulman
Part of Blood & lymphatic clinical trials.
This study looks at how genetic changes (ADAMTS13 gene mutations) and blood test results relate to symptoms and treatment needs in Upshaw-Schulman syndrome. It may help doctors better predict disease behavior and personalize care.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your blood test shows very low ADAMTS13 activity (10% or less) on at least two checks.
- Your tests do not show an anti-ADAMTS13 immune blocker (inhibitor) on those repeat checks at least one month apart.
- You are a family member of someone with confirmed or suspected Upshaw-Schulman.
- You have genetic testing of the ADAMTS13 gene showing one or more mutations, or
- You have had a trial of infusion treatment where ADAMTS13 activity fully returned after fresh frozen plasma (FFP), and the levels fell with a half-life of about 2–4 days.
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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