Effect of emicizumab prophylaxis on bone and joint health markers in people with haemophilia A without factor VIII inhibitors in the HAVEN 3 study.

Autor: Kiialainen A; F. Hoffmann-La Roche Ltd, Basel, Switzerland., Niggli M; F. Hoffmann-La Roche Ltd, Basel, Switzerland., Kempton CL; Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory, Emory University School of Medicine, Atlanta, Georgia, USA., Castaman G; Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy., Chang T; Genentech, Inc., South San Francisco, California, USA.; Graphite Bio, Inc., South San Francisco, California, USA., Paz-Priel I; Genentech, Inc., South San Francisco, California, USA.; Spark Therapeutics, Inc., Philadelphia, Pennsylvania, USA., Adamkewicz JI; Genentech, Inc., South San Francisco, California, USA., Levy GG; Genentech, Inc., South San Francisco, California, USA.; Graphite Bio, Inc., South San Francisco, California, USA.
Jazyk: angličtina
Zdroj: Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2022 Nov; Vol. 28 (6), pp. 1033-1043. Date of Electronic Publication: 2022 Jul 29.
DOI: 10.1111/hae.14642
Abstrakt: Introduction: Emicizumab prophylaxis significantly reduces bleeding events; however, the associated impact on bone/joint health is unknown.
Aim: To explore the effect of emicizumab prophylaxis on bone/joint health in people with haemophilia A (PwHA) without FVIII inhibitors enrolled in HAVEN 3 (NCT02847637).
Methods: Haemophilia joint health scores (HJHS; v2.1) were evaluated at baseline and Weeks 49 and 97 in PwHA receiving emicizumab (n = 134), and at baseline and Weeks 49, 73 and 97 in PwHA who switched to emicizumab after 24 weeks of no prophylaxis (n = 17). Bone and joint biomarkers were measured in 117 PwHA at baseline and at Weeks 13, 25, 49 and 73.
Results: HJHS was lower for PwHA who were previously on FVIII prophylaxis, aged <40 years or had no target joints at baseline compared with PwHA who were receiving no prophylaxis, aged ≥40 years or with target joints. Clinically significant mean (95% confidence interval) improvements from baseline of -2.13 (-3.96, -.29) in HJHS joint-specific domains were observed at Week 49 in PwHA with at least one target joint at study entry (n = 71); these changes were maintained through Week 97. Improvements in HJHS from baseline were also observed for PwHA aged 12-39 years. Biomarkers of bone resorption/formation, cartilage degradation/synthesis, and inflammation did not change significantly during emicizumab prophylaxis.
Conclusions: Clinically relevant improvements in HJHS were observed in younger PwHA and those with target joints after 48 weeks of emicizumab in HAVEN 3. Biomarkers of bone/joint health did not show significant changes during 72 weeks of emicizumab prophylaxis.
(© 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd.)
Databáze: MEDLINE
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