Health‐related quality of life and health status in adolescent and adult people with haemophilia A without factor VIII inhibitors—A non‐interventional study

Abstract

Introduction Real‐world data on health‐related outcomes in persons with haemophilia A (PwHA) can provide useful information for improving patient care. The global, non‐interventional study (NIS; NCT02476942) prospectively collected high‐quality data in PwHA, including those without factor VIII (FVIII) inhibitors treated according to local routine clinical practice. Aim To report health‐related quality of life (HRQoL) and health status of adult/adolescent PwHA without FVIII inhibitors. Methods Participants were PwHA without FVIII inhibitors age ≥12 years; they remained on existing episodic treatment or prophylaxis. HRQoL was assessed by Haemophilia Quality of Life Questionnaire for Adults (Haem‐A‐QoL) or Haemophilia‐Specific Quality of Life Assessment for Children and Adolescents Short Form (Haemo‐QoL‐SF II). Health status was assessed through EuroQol 5‐Dimensions 5‐Levels (EQ‐5D‐5L) index utility score and visual analogue scale (EQ‐VAS). Results Ninety‐four participants enrolled; median age was 34.0 years (range 12–76). Forty‐five received episodic treatment and 49 received prophylaxis for a median time of 27.7 weeks and 30.4 weeks, respectively. Mean (standard deviation) baseline Haem‐A‐QoL total scores were 40.1 (17.0) for the episodic group and 26.6 (14.6) for the prophylaxis group, indicating impairments in HRQoL, which remained consistent over time. Mean EQ‐5D‐5L IUS scores were similar between treatment regimens (0.8 episodic; 0.9 prophylaxis) and consistent over time. The mean EQ‐VAS scores were similar between treatment regimens, and lower on days when bleeding occurred (79.0 vs 85.0 for episodic treatment; 77.0 vs 82.0 for prophylaxis, respectively). Conclusions Adult and adolescent PwHA without FVIII inhibitors had HRQoL impairments regardless of whether they were treated with episodic or prophylactic standard care with FVIII.