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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...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2022
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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. |
format | Online Article Text |
id | pubmed-8902287 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | John Wiley and Sons Inc. |
record_format | MEDLINE/PubMed |
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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