What type of study is this?

This is a Case Report study (also classified as: Cohort Study, Systematic Review).

What question did this study set out to answer?

This scoping review analyzes the safety and efficacy of caplacizumab in treating pediatric immune thrombotic thrombocytopenic purpura.

December 8, 2025BloodOpen Access

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Scoping review shows caplacizumab improves platelet counts in pediatric iTTP, suggesting efficacy and a favorable safety profile.

This scoping review analyzes the safety and efficacy of caplacizumab in treating pediatric immune thrombotic thrombocytopenic purpura.
Conducted a scoping review following PRISMA guidelines.
Searched PubMed and Scopus for studies on caplacizumab in children with iTTP.
Included case reports, case series, and cohort studies involving pediatric patients with confirmed iTTP.
Included 15 publications covering 36 pediatric cases.
Median treatment duration was 33.5 days, with 80% achieving rapid platelet recovery.
All patients survived with few adverse events reported, indicating a favorable safety profile.

Al‐Mashdali et al. (2025) studied this question.

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