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Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5)

BACKGROUND: Emicizumab is a subcutaneously administered humanized, bispecific, monoclonal antibody approved for prophylaxis in people with hemophilia A. METHODS: HAVEN 5 (NCT03315455) is a randomized, open‐label, phase 3 study of individuals aged ≥12 years with severe hemophilia A without factor VII...

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Autores principales: Yang, Renchi, Wang, Shujie, Wang, Xuefeng, Sun, Jing, Chuansumrit, Ampaiwan, Zhou, Jianfeng, Schmitt, Christophe, Hsu, Wanling, Xu, Jeffrey, Li, Lindong, Chang, Tiffany, Zhao, Xielan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8902287/
https://www.ncbi.nlm.nih.gov/pubmed/35284778
http://dx.doi.org/10.1002/rth2.12670
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author Yang, Renchi
Wang, Shujie
Wang, Xuefeng
Sun, Jing
Chuansumrit, Ampaiwan
Zhou, Jianfeng
Schmitt, Christophe
Hsu, Wanling
Xu, Jeffrey
Li, Lindong
Chang, Tiffany
Zhao, Xielan
author_facet Yang, Renchi
Wang, Shujie
Wang, Xuefeng
Sun, Jing
Chuansumrit, Ampaiwan
Zhou, Jianfeng
Schmitt, Christophe
Hsu, Wanling
Xu, Jeffrey
Li, Lindong
Chang, Tiffany
Zhao, Xielan
author_sort Yang, Renchi
collection PubMed
description BACKGROUND: Emicizumab is a subcutaneously administered humanized, bispecific, monoclonal antibody approved for prophylaxis in people with hemophilia A. METHODS: HAVEN 5 (NCT03315455) is a randomized, open‐label, phase 3 study of individuals aged ≥12 years with severe hemophilia A without factor VIII (FVIII) inhibitors, or hemophilia A of any severity with FVIII inhibitors, across the Asia‐Pacific region. Participants were randomly assigned (2:2:1) to receive emicizumab 1.5 mg/kg once weekly (arm A), emicizumab 6 mg/kg every 4 weeks (arm B), or no prophylaxis (arm C). The primary end point was annualized bleeding rate (ABR) for treated bleeds; ABRs were compared between people receiving emicizumab prophylaxis versus those with no prophylaxis. Secondary end points included ABR for treated target joint bleeds. Safety was also evaluated. RESULTS: From April 26, 2018, to January 4, 2019, 70 of 76 screened participants were enrolled and randomized (arm A, n = 29; arm B, n = 27; arm C, n = 14). ABRs (95% confidence interval) for treated bleeds and treated target joint bleeds, respectively, were: arm A, 1.0 (0.53‐1.85) and 0.4 (0.18‐1.09); arm B, 1.0 (0.50‐1.84) and 0.3 (0.12‐0.85); arm C, 27.0 (13.29‐54.91) and 8.6 (3.15‐23.42). The most common adverse event, upper respiratory tract infection, was reported for 14 of 56 (25.0%; emicizumab) and 2 of 14 (14.3%; no prophylaxis) participants. No thrombotic events, thrombotic microangiopathies, or deaths were reported. CONCLUSION: Emicizumab 1.5 mg/kg once weekly and 6 mg/kg every 4 weeks demonstrated bleed control in this study population, was well tolerated, and could improve use of prophylaxis in people with hemophilia A.
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spelling pubmed-89022872022-03-11 Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5) Yang, Renchi Wang, Shujie Wang, Xuefeng Sun, Jing Chuansumrit, Ampaiwan Zhou, Jianfeng Schmitt, Christophe Hsu, Wanling Xu, Jeffrey Li, Lindong Chang, Tiffany Zhao, Xielan Res Pract Thromb Haemost Original Articles BACKGROUND: Emicizumab is a subcutaneously administered humanized, bispecific, monoclonal antibody approved for prophylaxis in people with hemophilia A. METHODS: HAVEN 5 (NCT03315455) is a randomized, open‐label, phase 3 study of individuals aged ≥12 years with severe hemophilia A without factor VIII (FVIII) inhibitors, or hemophilia A of any severity with FVIII inhibitors, across the Asia‐Pacific region. Participants were randomly assigned (2:2:1) to receive emicizumab 1.5 mg/kg once weekly (arm A), emicizumab 6 mg/kg every 4 weeks (arm B), or no prophylaxis (arm C). The primary end point was annualized bleeding rate (ABR) for treated bleeds; ABRs were compared between people receiving emicizumab prophylaxis versus those with no prophylaxis. Secondary end points included ABR for treated target joint bleeds. Safety was also evaluated. RESULTS: From April 26, 2018, to January 4, 2019, 70 of 76 screened participants were enrolled and randomized (arm A, n = 29; arm B, n = 27; arm C, n = 14). ABRs (95% confidence interval) for treated bleeds and treated target joint bleeds, respectively, were: arm A, 1.0 (0.53‐1.85) and 0.4 (0.18‐1.09); arm B, 1.0 (0.50‐1.84) and 0.3 (0.12‐0.85); arm C, 27.0 (13.29‐54.91) and 8.6 (3.15‐23.42). The most common adverse event, upper respiratory tract infection, was reported for 14 of 56 (25.0%; emicizumab) and 2 of 14 (14.3%; no prophylaxis) participants. No thrombotic events, thrombotic microangiopathies, or deaths were reported. CONCLUSION: Emicizumab 1.5 mg/kg once weekly and 6 mg/kg every 4 weeks demonstrated bleed control in this study population, was well tolerated, and could improve use of prophylaxis in people with hemophilia A. John Wiley and Sons Inc. 2022-03-07 /pmc/articles/PMC8902287/ /pubmed/35284778 http://dx.doi.org/10.1002/rth2.12670 Text en © 2022 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis (ISTH). https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
spellingShingle Original Articles
Yang, Renchi
Wang, Shujie
Wang, Xuefeng
Sun, Jing
Chuansumrit, Ampaiwan
Zhou, Jianfeng
Schmitt, Christophe
Hsu, Wanling
Xu, Jeffrey
Li, Lindong
Chang, Tiffany
Zhao, Xielan
Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5)
title Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5)
title_full Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5)
title_fullStr Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5)
title_full_unstemmed Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5)
title_short Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5)
title_sort prophylactic emicizumab for hemophilia a in the asia‐pacific region: a randomized study (haven 5)
topic Original Articles
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8902287/
https://www.ncbi.nlm.nih.gov/pubmed/35284778
http://dx.doi.org/10.1002/rth2.12670
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