Association of Patient-Reported Outcomes with Hemophilia A Inhibitor Status and Treatment Product Type.

Objectives

We compared patient-reported outcomes (PROs) in persons with hemophilia A (PwHA) by inhibitor status and prescribed treatment products.

Methods

Hematology Utilization Group VIII study enrolled PwHA aged ≥ 2 years to collect PRO data via surveys. A clinical chart review documented the hemophilic severity, inhibitor level and treatment regimen. PROs were compared across inhibitor status and prescribed treatment products.

Results

Among 85 enrolled PwHA, 9 (10.6%) had active inhibitors, 22 (25.9%) had tolerized inhibitors, and 54 (63.5%) had no inhibitors. The no-inhibitor group was significantly older (mean: 29.3 ± 13.5 years) than the tolerized (16.3 ± 9.5 years) and active inhibitor (21.9 ± 19.1 years; p = 0.001) groups. A larger proportion of participants with active inhibitors (66.7%) and no inhibitors (53.7%) reported having bleeds in the previous month compared to those with tolerized inhibitors (22.7%, p = 0.02). After covariate adjustment for age and hemophilia severity, the tolerized inhibitor group showed the lowest estimated number of joint bleeds compared to those of the no inhibitor and active inhibitor groups (p = 0.08), and the highest EQ-5D index score (p = 0.09). Emicizumab users reported significantly fewer bleeds in the previous months than those who were prescribed standard or extended half-life factor VIII (33.3% vs. 58.6%, 64.3%, p = 0.04).

Conclusions

Participants with active inhibitors experienced joint bleeding rates similar to those of participants without inhibitors, likely attributable to emicizumab use. Tolerized participants reported the fewest joint bleeds and highest quality-of-life scores, potentially reflecting younger age and possible greater prophylaxis adherence. Emicizumab was associated with lower bleed rates compared to standard or extended half-life factor VIII products.