Study of Immunosuppression, Caplacizumab, and Plasma Infusion Without Plasma Exchange in Adults With Immune-Mediated Thrombotic Thrombocytopenic Purpura

What this Trial Is About

Immune thrombotic thrombocytopenic purpura iTTP is a serious condition caused by a severe deficiency of ADAMTS13, leading to the buildup of large von Willebrand factor multimers and formation of blood vessel clots. The current standard treatment combines daily plasma exchange PEX, immunosuppressive drugs, and caplacizumab, which has shown to reduce death, complications, and the number of PEX sessions. However, PEX is invasive, time-consuming, and linked to complications, so researchers are exploring treatments without PEX to ease patient care. This study aims to evaluate the safety and effectiveness of a regimen without PEX using plasma infusion, corticosteroidsrituximab, and caplacizumab. The study replaces daily PEX with daily plasma infusions at a dose of 15 mL per kilogram of body weight, using different types of plasma such as quarantine fresh frozen plasma or inactivated plasma. This PEX-free regimen is combined with immunosuppression using corticosteroids and rituximab alongside the anti-adhesive drug caplacizumab. This approach is being tested as a single experimental treatment group for adults diagnosed with iTTP. Participants will be monitored for 30 days after starting plasma therapy to evaluate treatment outcomes. Researchers will assess the proportion of participants who experience death, treatment failure, disease worsening, or low ADAMTS13 activity below 20%. The study includes clinical and laboratory evaluations to track safety and effectiveness. The overall goal is to determine if this PEX-free regimen can provide a feasible alternative treatment for iTTP with fewer complications.

Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura