OBJECTIVES : Adherence is an important determinant in cost-effective analyses, which often assume perfect adherence when establishing value. For PwH, high rates (>50%) of nonadherence to prescribed regimens of prophylactic replacement factor can limit its effectiveness, compromising joints and restricting physical activity. The HemACTIVE study provided insight on how PwH define treatment satisfaction and its impact on adherence.

METHODS : Persons with moderate/severe hemophilia A, aged 2–65 years (caregivers for PwH <18y)from EU and North America, received a 25-minute, IRB-approved, web-based questionnaire. Reasons for treatment satisfaction were not assessed in Germany.

RESULTS : 275 PwH were enrolled (n=39 France, n=60 Italy, n=25 Germany, n=110 US, n=41 Canada; 29% children/caregivers; 61% severe hemophilia). Most were satisfied with current treatment, with varying degrees of satisfaction (24% extremely satisfied, 41% very satisfied, 32% satisfied, 4% unsatisfied). PwH were more likely to cite tolerability (minimal side effects: 67%; comfort: 62%) and/or efficacy (symptom minimization: 62%) as reasons for treatment satisfaction than its impact on freedom from restrictions (ability to treat whenever needed: 46%; ability to participate in activities: 44%). Strict adherence to prescribed regimens improved with degree of treatment satisfaction: 18% vs 36% vs 41% vs 52% for unsatisfied, satisfied, very satisfied, and extremely satisfied, respectively; PwH who were unsatisfied with their treatment were more likely to add infusions (27% vs 23% vs 15% vs 8%) or frequently miss scheduled infusions (18% vs. 4% vs 4% vs 2%). Occasional missed infusions were similar across PwH regardless of treatment satisfaction (range: 28%-36%).

CONCLUSIONS : Treatment adherence appears to be linked to treatment satisfaction--which PwH are more likely to define according to tolerability and/or efficacy of a product than impact on quality of life. By redefining treatment satisfaction to encompass freedom and flexibility in lifestyle, there is an opportunity to improve adherence, thus improving value of treatment.