Patients with hemophilia A who have inhibitors may experience impaired health-related quality of life (HRQoL) when receiving standard treatment, according to a study published in Haemophilia.
The global, prospective, noninterventional study (ClinicalTrials.gov Identifier: NCT02476942) reported the HRQoL and health status over the course of 25 weeks in adult and adolescent patients with hemophilia A who had inhibitors. Patients with hemophilia A were at least 12 years old, had a history of high‐titer factor VIII, and remained on their usual treatment.
Health‐related outcomes were quantified at 7 time points with the Hemophilia Quality of Life Questionnaire for Adults (Hem‐A‐QoL), Hemophilia‐specific Quality of Life Questionnaire for Children Short Form (Hemo‐QoL SF), EuroQol 5‐Dimensions 5‐Level (EQ‐5D‐5L) index utility score (IUS), and visual analogue scale (EQ‐VAS).
In total, 103 individuals enrolled and received either episodic (75 patients) or prophylactic (28 patients) treatment. Median age was 31 years (range, 12‐75). Participants were observed for a median duration of 26 weeks (range, 4‐70).
The mean Hem‐A‐QoL scores for adults indicated impairments and were similar across time. The scores were comparable between the episodic (67 patients) and prophylactic (19 patients) treatment groups. At week 25, the total score was 52.6 (± 17.0) in the episodic group and 54.3 (± 21.5) in the prophylaxis group.
For adolescents, the Hemo‐QoL SF scores for both regimens varied over time and appeared to be lower in the episodic (8 patients) treatment group compared with the prophylactic (9 patients) treatment group; however, the authors cautioned that this may be due to the small sample sizes. At week 25, the total score was 57.1 (± 21.0) in the episodic group and 22.1 (± 7.0) in the prophylaxis group.
Overall, the mean IUS and EQ‐VAS scores were similar between the treatment groups (0.72 and 0.69 in episodic and prophylaxis groups, respectively) and remained consistent over time. The mean proportion of missed work and school days was 16% (95% CI, 10%‐22%) and 23% (95% CI, 14%‐32%), respectively, and these did not vary between the episodic and prophylaxis groups.
The authors concluded that more effective treatment options are needed for patients with hemophilia A who have inhibitors.
Disclosures: Some authors have declared affiliations with the pharmaceutical industry. Please refer to the original study for a full list of disclosures.
1. Mahlangu J, Oldenburg J, Callaghan MU, et al. Health‐related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non‐interventional study (NIS) [published online April 24, 2019]. Haemophilia. doi:10.1111/hae.13731