Can We Improve on Prednisone and Rituximab for Immune TTP? an Analysis of Alternatives to the Standard of Care from the Ustma TTP Retrospective Registry

• Published in: Blood 2024-11, Vol.144, p.142-142
• Main Authors: Kaufman, Adrienne, Mazepa, Marshall, Evans, Michael D
• Format: Article
• Language: English
• ISSN: 0006-4971
• DOI: 10.1182/blood-2024-209162

Introduction: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening disorder caused by an autoantibody-mediated deficiency of ADAMTS13, for which the goal of treatment with immunosuppression is to induce long term remission. Currently, the standard of care (SOC), corticosteroids and rituximab (RTX), fails to induce long term remission in many patients. Therefore, we aimed to assess whether alternative regimens for treatment of iTTP episodes improved upon or were less efficacious in inducing relapse-free survival (RFS) compared to SOC, using data from the United States Thrombotic Microangiopathies (USTMA) Consortium iTTP Registry. Methods: The study cohort included participants with iTTP from the USTMA retrospective registry from 1990 to 2020. Methods of selecting confirmed iTTP participants from the registry as previously been described (Chaturvedi et al Blood 2022). Briefly, iTTP diagnosis was confirmed by the presence of ADAMTS13 deficiency, severe thrombocytopenia, and microangiopathic hemolytic anemia. Participants were followed for iTTP diagnosis or first contact after iTTP diagnosis until death or last contact. Treatment for all participants included daily plasma exchange until a normal platelet count was sustained for at least 2 consecutive days. We excluded participants who were followed for