Item type |
itemtype_ver1(1) |
公開日 |
2024-03-06 |
タイトル |
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タイトル |
Phase 3 Trial of Concizumab in Hemophilia with Inhibitors |
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言語 |
en |
著者 |
Matsushita, Tadashi
Shapiro, Amy
Abraham, Aby
Angchaisuksiri, Pantep
Castaman, Giancarlo
Cepo, Katarina
d’Oiron, Roseline
Frei-Jones, Melissa
Goh, Ai-Sim
Haaning, Jesper
Hald Jacobsen, Sanja
Mahlangu, Johnny
Mathias, Mary
Nogami, Keiji
Skovgaard Rasmussen, Josephine
Stasyshyn, Oleksandra
Tran, Huyen
Vilchevska, Kateryna
Villarreal Martinez, Laura
Windyga, Jerzy
You, Chur Woo
Zozulya, Nadezhda
Zulfikar, Bulent
Jiménez-Yuste, Victor
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アクセス権 |
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アクセス権 |
open access |
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アクセス権URI |
http://purl.org/coar/access_right/c_abf2 |
権利 |
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言語 |
en |
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権利情報 |
“From [New England Journal of Medicine, T. Matsushita, A. Shapiro, A. Abraham, P. Angchaisuksiri, G. Castaman, K. Cepo, R. d’Oiron, M. Frei‑Jones, A.-S. Goh, J. Haaning, S. Hald Jacobsen, J. Mahlangu, M. Mathias, K. Nogami, J. Skovgaard Rasmussen, O. Stasyshyn, H. Tran, K. Vilchevska, L. Villarreal Martinez, J. Windyga, C.W. You, N. Zozulya, B. Zulfikar, and V. Jiménez‑Yuste, for the explorer7 Investigators, Phase 3 Trial of Concizumab in Hemophilia with Inhibitors, 389, 783-794. Copyright © (2023)] Massachusetts Medical Society. Reprinted with permission. |
内容記述 |
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内容記述タイプ |
Abstract |
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内容記述 |
Background: Concizumab is an anti–tissue factor pathway inhibitor monoclonal antibody designed to achieve hemostasis in all hemophilia types, with subcutaneous administration. A previous trial of concizumab (explorer4) established proof of concept in patients with hemophilia A or B with inhibitors. Methods: We conducted the explorer7 trial to assess the safety and efficacy of concizumab in patients with hemophilia A or B with inhibitors. Patients were randomly assigned in a 1:2 ratio to receive no prophylaxis for at least 24 weeks (group 1) or concizumab prophylaxis for at least 32 weeks (group 2) or were nonrandomly assigned to receive concizumab prophylaxis for at least 24 weeks (groups 3 and 4). After a treatment pause due to nonfatal thromboembolic events in three patients receiving concizumab, including one from the explorer7 trial, concizumab therapy was restarted with a loading dose of 1.0 mg per kilogram of body weight, followed by 0.2 mg per kilogram daily (potentially adjusted on the basis of concizumab plasma concentration as measured at week 4). The primary end-point analysis compared treated spontaneous and traumatic bleeding episodes in group 1 and group 2. Safety, patient-reported outcomes, and pharmacokinetics and pharmacodynamics were also assessed. Results: Of 133 enrolled patients, 19 were randomly assigned to group 1 and 33 to group 2; the remaining 81 were assigned to groups 3 and 4. The estimated mean annualized bleeding rate in group 1 was 11.8 episodes (95% confidence interval [CI], 7.0 to 19.9), as compared with 1.7 episodes (95% CI, 1.0 to 2.9) in group 2 (rate ratio, 0.14 [95% CI, 0.07 to 0.29]; P<0.001). The overall median annualized bleeding rate for patients receiving concizumab (groups 2, 3, and 4) was 0 episodes. No thromboembolic events were reported after concizumab therapy was restarted. The plasma concentrations of concizumab remained stable over time. Conclusions: Among patients with hemophilia A or B with inhibitors, the annualized bleeding rate was lower with concizumab prophylaxis than with no prophylaxis. (Funded by Novo Nordisk; explorer7 ClinicalTrials.gov number, NCT04083781.) |
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言語 |
en |
出版者 |
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出版者 |
Massachusetts Medical Society |
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言語 |
en |
言語 |
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言語 |
eng |
資源タイプ |
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資源タイプ識別子 |
http://purl.org/coar/resource_type/c_6501 |
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資源タイプ |
journal article |
出版タイプ |
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出版タイプ |
VoR |
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出版タイプResource |
http://purl.org/coar/version/c_970fb48d4fbd8a85 |
関連情報 |
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関連タイプ |
isVersionOf |
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識別子タイプ |
DOI |
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関連識別子 |
https://doi.org/10.1056/NEJMoa2216455 |
収録物識別子 |
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収録物識別子タイプ |
PISSN |
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収録物識別子 |
0028-4793 |
書誌情報 |
en : New England Journal of Medicine
巻 389,
号 9,
p. 783-794,
発行日 2023-08-31
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