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

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.

Figure 1

Mean annualized bleed rates (ABRs) for treated bleeds by age group and comorbidity. Ninety-five percent CI was derived from exact Poisson distribution. N/n represents the number of participants. Data from the one participant with HIV infection only have been omitted. CI, confidence interval; CV, cardiovascular; HCV, hepatitis C virus; People with HA, people with hemophilia A.

Figure 2

Mean annualized bleed rates (ABRs) for treated joint and target joint bleeds by age group and comorbidity. Data from the one participant with HIV infection only have been omitted. CI, confidence interval; CV, cardiovascular; HCV, hepatitis C virus; People with HA, people with hemophilia A.