1. Management of immune thrombotic thrombocytopenic purpura without therapeutic plasma exchange.
- Author
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Kühne L, Knöbl P, Eller K, Thaler J, Sperr WR, Gleixner K, Osterholt T, Kaufeld J, Menne J, Buxhofer-Ausch V, Mühlfeld A, Seelow E, Schreiber A, Todorova P, Cukoski S, Jabs WJ, Özcan F, Gäckler A, Schönfelder K, Seibert FS, Westhoff T, Schwenger V, Eichenauer DA, Völker LA, and Brinkkoetter PT
- Subjects
- Humans, Female, Male, Retrospective Studies, Adult, Middle Aged, Platelet Count, Aged, Young Adult, Adolescent, ADAMTS13 Protein blood, Purpura, Thrombocytopenic, Idiopathic therapy, Purpura, Thrombocytopenic, Idiopathic blood, Plasma Exchange, Single-Domain Antibodies therapeutic use, Purpura, Thrombotic Thrombocytopenic therapy, Purpura, Thrombotic Thrombocytopenic blood
- Abstract
Abstract: Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening autoimmune disorder caused by a disintegrin and metalloproteinase with thrombospondin type 1 motif, member 13 (ADAMTS13) deficiency. Caplacizumab, an anti-von Willebrand factor nanobody, is approved for iTTP treatment, reducing the need for therapeutic plasma exchange (TPE) and improving platelet count recovery and survival. We conducted a retrospective study on 42 acute iTTP cases in Austria and Germany, treated with a modified regimen aimed at avoiding TPE if platelet count increased after the first caplacizumab dose. Baseline characteristics and patient outcomes were compared with a control group of 59 patients with iTTP receiving frontline treatment with TPE, caplacizumab, and immunosuppression. The main outcome was the time to platelet count normalization. Secondary outcomes included clinical response, exacerbation, refractory iTTP, iTTP-related deaths, and the time to platelet count doubling. The median time to platelet count normalization was similar between the 2 cohorts (3 and 4 days; P = .31). There were no significant differences in clinical response, exacerbations, refractoriness, iTTP-related deaths, or time to platelet count doubling, reflecting the short-term treatment response. Four patients did not respond to the first caplacizumab dose, and TPE was subsequently initiated. Cytomegalovirus infection, HIV/hepatitis B virus coinfection, an ovarian teratoma with associated antiplatelet antibodies, and multiple platelet transfusions before the correct diagnosis may have impeded the immediate treatment response in these patients. In conclusion, caplacizumab and immunosuppression alone, without TPE, rapidly controlled thrombotic microangiopathy and achieved a sustained clinical response in iTTP. Our study provides a basis for TPE-free iTTP management in experienced centers via shared decision-making between patients and treating physicians., (© 2024 American Society of Hematology. Published by Elsevier Inc. All rights are reserved, including those for text and data mining, AI training, and similar technologies.)
- Published
- 2024
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