Real-world data of the use and experience of caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura : Case series
Copyright © 2023 Elsevier Ltd. All rights reserved..
PURPOSE: Caplacizumab 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.
METHODS: We 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.
RESULTS: Patients' 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).
CONCLUSION: We 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.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:62 |
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| Enthalten in: |
Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis - 62(2023), 3 vom: 15. Juni, Seite 103722 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Albanell-Fernández, Marta [VerfasserIn] |
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| Links: |
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| Themen: |
2R27AB6766 |
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| Anmerkungen: |
Date Completed 09.06.2023 Date Revised 09.06.2023 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1016/j.transci.2023.103722 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM356725723 |
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| 100 | 1 | |a Albanell-Fernández, Marta |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Real-world data of the use and experience of caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura |b Case series |
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| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Copyright © 2023 Elsevier Ltd. All rights reserved. | ||
| 520 | |a PURPOSE: Caplacizumab 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 | ||
| 520 | |a METHODS: We 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 | ||
| 520 | |a RESULTS: Patients' 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) | ||
| 520 | |a CONCLUSION: We 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 | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Review | |
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| 650 | 4 | |a Acquired thrombotic thrombocytopenic purpura | |
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