Systematic Literature Review of the Efficacy and Safety of Treatments in Congenital Thrombotic Thrombocytopenic Purpura
Despite preventive blood therapy, patients with rare congenital TTP still experience breakthrough clots and organ injury, highlighting unmet needs.
PURPOSE: Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare, life-threatening thrombotic microangiopathy arising from an inherited deficiency in ADAMTS13 enzyme activity. CONCLUSION: The available evidence highlights the persistent disease burden of cTTP, with patients experiencing acute events and organ damage despite prophylactic PBT.
What the study was
- Study design
- Review
- Population
- PURPOSE: Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare, life-threatening thromboti
- Category
- Drug Development
- Maturity
- Exploratory
- Journal
- Journal of blood medicine
Why it surfaced
high triage score
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