Real-world data of the use and experience of caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura: Case series |
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| Institution: | 1. Pharmacy Service, Division of Medicines, Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain;2. Apheresis & Cellular Therapy Unit, Department of Hemotherapy and Hemostasis, ICMHO, Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain;3. August Pi i Sunyer Biomedical Research Institute (IDIBAPS), Barcelona, Spain;4. Department of Pharmacology, Toxicology and Therapeutic Chemistry. School of Pharmacy. University of Barcelona, Spain;5. Hemostasis and Erythropathology Laboratory, Pathology Department. Biomedical Diagnosis Center (CDB), Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain;1. Department of Hematology, Faculty of Medicine, King Abdulaziz University, Jeddah, Saudi Arabia;2. Blood Transfusion Services, King Abdulaziz University Hospital, Jeddah, Saudi Arabia;3. Special Infectious Agents Unit BSL3, King Fahd Medical Research Center and Department of Medical Laboratory Sciences, Faculty of Applied Medical Sciences, King Abdulaziz University, Jeddah, Saudi Arabia;1. Department of Laboratory Medicine and Pathology, University of Alberta, Edmonton, Canada;2. Innovation and Portfolio Management, Canadian Blood Services, Edmonton;1. SAINBIOSE INSERM U1049, Université de Saint-Etienne, Saint-Etienne, France;2. CPER INSERM U1100 and Université de Tours, and CHRU de Tours, Tours, France;1. Cell manipulation laboratory, Immunohaematology and Transfusion Service, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy;2. Biostatistics and clinical trial Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy;3. Immunohaematology and Transfusion Service, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy |
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| Abstract: | PurposeCaplacizumab was licensed for acquired thrombotic thrombocytopenic purpura (aTTP) based on prospective controlled trials. Real-world evidence is crucial in rare diseases. We aim to describe a patient population with aTTP, receiving caplacizumab in a real-world setting, reporting their outcomes, including safety and tolerability, and contrasting them with a historical cohort from our center.MethodsWe describe data collected retrospectively from 2012 to 2022 for 16 patients with aTTP (8 received caplacizumab and 8 the historical standard-of-care). Patients' characteristics and outcomes were compared between groups.ResultsPatients’ demographic and baseline characteristics were similar in both groups. Caplacizumab led to a rapid normalization of the platelet count of 3.5 (IQR, 2–6) versus 16 (IQR, 9.5–23.5) days in the historical cohort: (p = .002). The median number of plasma exchanges and the length of days requiring them, between the caplacizumab group versus the historical cohort, was 6 (IQR, 6–10) versus 19.5 (IQR, 12.5–29.5) plasma exchanges (p = .006); and 9 (IQR, 8.5–13.5) versus 22 (15–31) days (p = .049), respectively. There were no refractory cases in the caplacizumab group in comparison with 37.5 % in the historical cohort. None of patients treated with caplacizumab experienced a recurrence after 1081 (IQR, 511–3125) days of follow-up. Safety was in line with data reported in clinical trials, with mild adverse events (mostly grade≤2).ConclusionWe provided real-world evidence in the treatment of aTTP, confirming the results obtained in clinical trials. Caplacizumab reduced the time to platelet count recovery and the number and length of plasma exchanges. |
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| Keywords: | Caplacizumab Plasma exchange Platelet count Acquired thrombotic thrombocytopenic purpura ADAMTS13 Real-world evidence |
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