Regional Differences in Patient-Reported Outcomes in a Study of Adult Prophylaxis Versus on-Demand Treatment with Bayer’s Sucrose-Formulated Recombinant Factor VIII: 3-Year Data from Spinart

Introduction: The SPINART study compared routine prophylaxis vs on-demand treatment using Bayer's sucrose-formulated recombinant factor VIII (rFVIII-FS) in adolescents and adults with severe hemophilia A. Primary results at the end of the first year of the study showed that routine prophylaxis was significantly more efficacious than on-demand treatment in decreasing bleeding episodes. Assessment of health-related quality of life from a patient perspective is an important aspect of evaluating hemophilia treatment effectiveness. Here we report patient-reported outcomes (PROs), including assessment of pain, activity levels, treatment satisfaction, and healthcare resource utilization as well as regional differences (US vs non-US patients) in these PROs after 3 years of treatment with rFVIII-FS.

Methods: SPINART, a 3-year, randomized, controlled, open-label, multinational study, enrolled previously treated patients aged 12–50 years with severe hemophilia A with no inhibitors, no prophylaxis for >12 consecutive months in the past 5 years, and 6–24 documented bleeding events or treatments in the previous 6 months. Patients were treated with rFVIII-FS, either on-demand therapy or prophylaxis (25 IU/kg 3 times weekly, with dose escalation of 5 IU/kg permitted at years 1 and 2). PRO measures included the Short-Form McGill Pain Questionnaire (SF-MPQ), Treatment Regimen Satisfaction Questionnaire, Activities List Questionnaire, and Healthcare Resource Utilization Questionnaire. The Activities List and SF-MPQ were completed at baseline and at years 1, 2, and 3. Treatment satisfaction was assessed at baseline and at months 3, 6, 12, 18, 24, 30, and 36. The Healthcare Resource Utilization Questionnaire was filled out weekly via electronic patient diary. Changes from baseline to year 3 were summarized using descriptive statistics. Subgroup analyses based on region (US vs non-US [Argentina, Bulgaria, and Romania]) were also performed. Data shown are for the intent-to-treat (ITT) population.

Results: The ITT population consisted of84 patients (42 prophylaxis, 42 on demand). PRO results analyzed by region at year 3 are shown in the Table. Marked reduction from baseline to year 3 was observed in SF-MPQ scores for the last 4 weeks' pain rating for both US and non-US patients receiving prophylaxis compared with on-demand treatment. Activities List outcomes showed a lower proportion of US patients compared with non-US patients reporting increased physical activities and unrestricted activities at the end of the 3-year study. Overall, there was a trend toward more physical activity and fewer restrictions with prophylaxis compared with on-demand treatment. Treatment satisfaction was lower in US patients compared with non-US patients at the final study visit. Healthcare resource utilization was slightly lower for the US population compared with the non-US population; overall, the prophylaxis group reported lower healthcare resource utilization compared with the on-demand treatment group.

Conclusions: Results of the 3-year SPINART study showed that patients with hemophilia A receiving rFVIII-FS prophylaxis were more active, experienced less pain, utilized fewer healthcare resources, and had better treatment satisfaction compared with those receiving on-demand treatment. Subanalyses based on region showed that non-US patients were more active and had better treatment satisfaction at year 3 compared with their US counterparts. The larger improvement in non-US patients compared with US patients may be explained by the greater degree of hemarthropathy observed at baseline in non-US patients.