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
Bibliographic record
Abstract
Abstract 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. Table. Patient-Reported Outcomes at Year 3 of the SPINART Study US Patients Non-US Patients Prophylaxis (n=14) On-Demand (n=21) Prophylaxis (n=28) On-Demand (n=21) McGill Pain Questionnaire, last 4 weeks' pain rating,* mean ± SD –16.6±26.4 9.1±23.8 –17.4±22.2 –7.6±24.2 Activities List, n (%) More physical activities over the past 4 weeks 1 (7.1) 3 (14.3) 16 (57.1) 5 (23.8) Currently unrestricted activities 3 (21.4) 6 (28.6) 17 (60.7) 5 (23.8) Treatment satisfaction, n (%) Very or extremely satisfied 5 (35.7) 6 (28.5) 22 (78.5) 12 (57.1) Will continue current regimen 6 (42.9) 7 (33.3) 22 (78.6) 13 (61.9) Healthcare resource utilization, n Contact with any health care professional 152 289 149 419 Joint-related surgeries 1 4 7 10 *Change from baseline to year 3. 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. Disclosures Pocoski: Bayer HealthCare Pharmaceuticals: Employment. Kim:Bayer: Consultancy; CSL Behring: Consultancy; Novo Nordisk: Consultancy; Baxter: Consultancy; Biogen: Consultancy. Humphries:Bayer HealthCare Pharmaceuticals: Employment. Hong:Bayer HealthCare: Employment.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.005 | 0.004 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.002 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.001 | 0.001 |
| Insufficient payload (model declined to judge) | 0.001 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".