Treatment satisfaction and limitations in haemophilia A, with a focus on factor VIII product storage conditions: patients’ perspectives and challenges

Abstract

Introduction: Prophylaxis with factor VIII (FVIII) concentrates is the mainstay of treatment for haemophilia A; however, patients’ perceptions of treatment can negatively affect compliance and treatment outcomes. Aim: The aim of this study was to explore treatment limitations and satisfaction in haemophilia A from the patient’s perspective, with a focus on product storage characteristics. Methods: Patients with haemophilia A in Slovenia completed a survey focussing on treatment satisfaction and understanding of FVIII product storage conditions. Patient satisfaction with product storage was analysed using the Kano model. Results: There were 63 survey respondents in total (note: not all patients answered all questions). Most patients were aged > 18 years (93.6%), had severe haemophilia A (68.3%) and were on FVIII prophylaxis (73.8%). The mean (median) number of bleeds in the previous 12 months was 4.9 (3.0). Missed, forgotten or delayed doses were reported by 46.4% of all patients and by 53.3% of patients on prophylaxis. Most patients believed FVIII products should be stored at ≤ 25 °C (90.7%) or only in a refrigerator (53.7%). Additionally, 76.4% of patients reported limitations in daily activities (especially travel) due to product storage requirements. Survey results and Kano model analysis indicated that treatment satisfaction may be improved by a product that can be stored at > 25 °C. Conclusion: This survey highlights patient perspectives regarding treatment satisfaction and limitations in haemophilia A, with a focus on product storage conditions. Perceived product storage requirements limit the daily activities of many patients (especially travel). Ongoing patient education is necessary to improve treatment satisfaction, compliance, treatment outcomes and overall health.