Emicizumab is efficacious in people with hemophilia A with comorbidities aged ≥50 years: analysis of 4 phase III trials

Víctor Jiménez-Yuste; Johannes Oldenburg; Eunice Tzeng; Elise Lim; Fabian Sanabria; Johnny Mahlangu

doi:10.1016/j.rpth.2024.102405

Emicizumab is efficacious in people with hemophilia A with comorbidities aged ≥50 years: analysis of 4 phase III trials

Res Pract Thromb Haemost. 2024 Apr 10;8(3):102405. doi: 10.1016/j.rpth.2024.102405. eCollection 2024 Mar.

Authors

Víctor Jiménez-Yuste¹, Johannes Oldenburg², Eunice Tzeng³, Elise Lim³, Fabian Sanabria⁴, Johnny Mahlangu⁵

Affiliations

¹ Hematology Department, Hospital Universitario La Paz, IdiPAZ, Autónoma University, Madrid, Spain.
² Institute of Experimental Haematology and Transfusion Medicine, University of Bonn, Bonn, Germany.
³ Genentech, Inc., South San Francisco, California, USA.
⁴ F. Hoffmann-La Roche Ltd, Basel, Switzerland.
⁵ Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa.

Abstract

Background: The treatment of older people with hemophilia A (HA) can be complicated by comorbidities.

Objectives: This post hoc analysis evaluates the efficacy and safety of emicizumab in people with HA aged ≥50 years with cardiovascular (CV) risk factors or HIV and/or hepatitis C virus (HCV) infection.

Methods: The HAVEN 1 (NCT02622321), HAVEN 3 (NCT02847637), HAVEN 4 (NCT03020160), and STASEY (NCT03191799) studies enrolled adults/adolescents with severe HA. Participants were categorized as having a comorbidity if they had any CV risk factors (including history of CV disease, hypertension, diabetes, hyperlipidemia, prior stroke, or obesity), HIV, and/or HCV infection. Efficacy and safety outcomes were compared by age (<50 vs ≥50 years).

Results: Of 504 participants at data cutoff, 408 were aged <50 years and 96 were aged ≥50 years. In people with HA aged <50 years, 26.7% had ≥1 CV risk factor and 29.4% had HIV and/or HCV infection. In people with HA aged ≥50 years, 72.9% had ≥1 CV risk factor and 74.0% had HIV and/or HCV infection. The mean (95% CI) annualized bleed rate for treated bleeds was 1.29 (0.07-6.06) for people with HA aged <50 years and 1.82 (0.19-6.93) for people with HA aged ≥50 years. No significant differences in annualized bleed rates were observed for those with comorbidities compared with those without. Safety outcomes were similar regardless of age.

Conclusion: This pooled analysis suggests that emicizumab efficacy and safety in people with HA aged ≥50 years with CV and HIV/HCV comorbidities were consistent with those in people with HA aged <50 years enrolled in the HAVEN 1, 3, and 4 and STASEY studies.

Keywords: comorbidities; comorbidity; emicizumab; hemophilia A; prophylaxis.