The efficacy and safety of caplacizumab in Japanese patients with immune-mediated thrombotic thrombocytopenic purpura: an open-label phase 2/3 study
Abstract Caplacizumab is an anti-von Willebrand factor humanized single-variable-domain immunoglobulin fragment whose efficacy and safety in immune-mediated thrombotic thrombocytopenia purpura (iTTP) have been demonstrated in international studies. This prospective, open-label phase 2/3 study evaluated caplacizumab 10 mg administered daily during plasma exchange and for 30 days afterward, in combination with immunosuppressive treatment, in Japanese adults with a clinical diagnosis of iTTP (new or recurrent). The primary endpoint was prevention of iTTP recurrence; key secondary endpoints included time to platelet count response, time to organ damage normalization, and safety. Among 21 treated patients, 1 of 15 (6.7%) evaluable patients developed iTTP recurrence. Median time to normalization was 2.79 days for platelet count and 2.65 days for organ damage markers (n = 15). Treatment-emergent adverse events (TEAEs) were mostly mild to moderate in severity; the most frequently reported caplacizumab-related TEAEs were increased alanine aminotransferase, epistaxis, and gastrointestinal hemorrhage (all in 9.5% of patients). At least one bleeding event was reported in 7 of 21 patients (33%). Caplacizumab was effective in Japanese patients with iTTP, with a low rate of iTTP recurrence, rapid normalization of platelet counts and organ damage markers, and no unexpected TEAEs. Trial registration: ClinicalTrials.gov identifier, NCT04074187..
| Medienart: |
Artikel |
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| Erscheinungsjahr: |
2022 |
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| Erschienen: |
2022 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:117 |
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| Enthalten in: |
International journal of hematology - 117(2022), 3 vom: 24. Nov., Seite 366-377 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Miyakawa, Yoshitaka [VerfasserIn] |
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| Links: |
Volltext [lizenzpflichtig] |
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| Themen: |
ADAMTS13 |
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| Anmerkungen: |
© The Author(s) 2022 |
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| doi: |
10.1007/s12185-022-03495-6 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
OLC213419894X |
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| 245 | 1 | 0 | |a The efficacy and safety of caplacizumab in Japanese patients with immune-mediated thrombotic thrombocytopenic purpura: an open-label phase 2/3 study |
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| 520 | |a Abstract Caplacizumab is an anti-von Willebrand factor humanized single-variable-domain immunoglobulin fragment whose efficacy and safety in immune-mediated thrombotic thrombocytopenia purpura (iTTP) have been demonstrated in international studies. This prospective, open-label phase 2/3 study evaluated caplacizumab 10 mg administered daily during plasma exchange and for 30 days afterward, in combination with immunosuppressive treatment, in Japanese adults with a clinical diagnosis of iTTP (new or recurrent). The primary endpoint was prevention of iTTP recurrence; key secondary endpoints included time to platelet count response, time to organ damage normalization, and safety. Among 21 treated patients, 1 of 15 (6.7%) evaluable patients developed iTTP recurrence. Median time to normalization was 2.79 days for platelet count and 2.65 days for organ damage markers (n = 15). Treatment-emergent adverse events (TEAEs) were mostly mild to moderate in severity; the most frequently reported caplacizumab-related TEAEs were increased alanine aminotransferase, epistaxis, and gastrointestinal hemorrhage (all in 9.5% of patients). At least one bleeding event was reported in 7 of 21 patients (33%). Caplacizumab was effective in Japanese patients with iTTP, with a low rate of iTTP recurrence, rapid normalization of platelet counts and organ damage markers, and no unexpected TEAEs. Trial registration: ClinicalTrials.gov identifier, NCT04074187. | ||
| 650 | 4 | |a ADAMTS13 | |
| 650 | 4 | |a Caplacizumab | |
| 650 | 4 | |a Single-domain antibody | |
| 650 | 4 | |a Thrombotic thrombocytopenic purpura | |
| 650 | 4 | |a Von Willebrand factor inhibitor | |
| 700 | 1 | |a Imada, Kazunori |4 aut | |
| 700 | 1 | |a Ichikawa, Satoshi |4 aut | |
| 700 | 1 | |a Uchiyama, Hitoji |4 aut | |
| 700 | 1 | |a Ueda, Yasunori |4 aut | |
| 700 | 1 | |a Yonezawa, Akihito |4 aut | |
| 700 | 1 | |a Fujitani, Shigeki |4 aut | |
| 700 | 1 | |a Ogawa, Yoshiyuki |4 aut | |
| 700 | 1 | |a Matsushita, Tadashi |4 aut | |
| 700 | 1 | |a Asakura, Hidesaku |4 aut | |
| 700 | 1 | |a Nishio, Kenji |4 aut | |
| 700 | 1 | |a Suzuki, Kodai |4 aut | |
| 700 | 1 | |a Hashimoto, Yasuhiro |4 aut | |
| 700 | 1 | |a Murakami, Hidenori |4 aut | |
| 700 | 1 | |a Tahara, Sayaka |4 aut | |
| 700 | 1 | |a Tanaka, Tomoyuki |4 aut | |
| 700 | 1 | |a Matsumoto, Masanori |0 (orcid)0000-0002-7243-3126 |4 aut | |
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