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
Notice bibliographique
Résumé
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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Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,005 | 0,004 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,001 | 0,002 |
| Bibliométrie | 0,000 | 0,001 |
| Études des sciences et des technologies | 0,000 | 0,001 |
| Communication savante | 0,001 | 0,001 |
| Science ouverte | 0,001 | 0,000 |
| Intégrité de la recherche | 0,001 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,001 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».