Journal of blood medicine
Systematic Literature Review of the Efficacy and Safety of Treatments in Congenital Thrombotic Thrombocytopenic Purpura
By Kaur H, Gupta A, Berkley A and 1 others.
Abstract
Purpose
Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare, life-threatening thrombotic microangiopathy arising from an inherited deficiency in ADAMTS13 enzyme activity. A systematic literature review was conducted to review and evaluate the clinical efficacy, safety, and tolerability of current and emerging treatments for cTTP.
Methods
Literature searches were completed in January 2024. Searches were performed in accordance with PRISMA guidelines to identify studies reporting treatments in patients with cTTP published at any time since database inception. Outcomes of interest included the rates of acute and subacute cTTP events, other disease manifestations, long-term comorbidities, organ damage, mortality, and treatment-related adverse events.
