Findings from the XTEND-Kids Study: Efanesoctocog Alfa for Severe Haemophilia A in Children-Reference-Cited by-同舟云学术

Findings from the XTEND-Kids Study: Efanesoctocog Alfa for Severe Haemophilia A in Children

Published:2023 Issue:2 Volume:19 Page:2
ISSN:2752-5481
Container-title:Oncology & Haematology
language:en
Short-container-title:

Author:

Simoneau Jillian,Weyand Angela C, ,

Abstract

Data from the XTEND-Kids study presented at the 2023 International Society on Thrombosis and Haemostasis Congress showed that once-weekly prophylaxis with efanesoctocog alfa dosed at 50 IU/kg body weight was well tolerated and provided highly effective bleed protection and treatment in children <12 years of age with severe haemophilia A. These findings are consistent with the results of the XTEND-1 trial, which evaluated the efficacy of efanesoctocog alfa in both adults and adolescents.

Funder

This article is published under the Creative Commons Attribution Non-commercial License.

Publisher

Touch Medical Media, Ltd.

Reference13 articles.

1. Knobe K, Berntorp E. Haemophilia and joint disease: Pathophysiology, evaluation, and management. J Comorb. 2011;1:51–9. DOI: 10.15256/joc.2011.1.2.

2. Weyand AC, Pipe SW. New therapies for hemophilia. Blood. 2019;133:389–98. DOI: 10.1182/blood-2018-08-872291.

3. Cho S, Perry AM, Cheng AM, et al. Advances in hemophilia A management. Adv Pediatr. 2022;69:133–47. DOI: 10.1016/j.yapd.2022.03.009.

4. Callaghan MU, Negrier C, Paz-Priel I, et al. Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies. Blood. 2021;137:2231–42. DOI: 10.1182/blood.2020009217.

5. Sanofi. Press release: FDA approves once-weekly ALTUVIIIO™, a new class of factor VIII therapy for hemophilia a that offers significant bleed protection. 2023. Available at: www.sanofi.com/en/media-room/press-releases/2023/2023-02-23-21-00-00-2614759 (Date last accessed: 23 February 2023).