Real‐world data suggests that patients with hemophilia A with inhibitors had lower health-related quality of life (HRQOL) while receiving standard therapy, according to an international study.
“The objective of this analysis was to characterize disease‐specific HRQOL, overall health status and the effect of bleeding on health status,” wrote, of the University of the Witwatersrand in Johannesburg, South Africa, and colleagues. The study was published in .
The researchers conducted a prospective, noninterventional study of 103 patients aged 12 years and older with hemophilia A who resided in several different countries, including Australia, Japan, South Africa, and the United States, among others.
The majority of participants (n = 75) received episodic treatment at study enrollment, while others (n = 28) received prophylactic-based therapy. Patients were treated with standard therapy, based on local institutional practice.
HRQOL outcome data were collected in adult and adolescent participants using the Haemophilia Quality of Life Questionnaire for Adults and the Haemophilia‐specific Quality of Life Questionnaire for Children Short Form. Other validated instruments were used to measure additional health‐related outcomes.
After analysis, the researchers found that HRQOL scores revealed impaired quality of life in adult and adolescent participants treated with both episodic and prophylactic regimens. The mean scores in the majority of HRQOL domains showed impairments occurring on average “sometimes” to “often,” the researchers reported.
Adults had highest scores, correlated with greatest impairments, in sports and leisure. Similarly, adolescents reported greatest impairment in the sports and school domain.
“These health‐related outcomes may result from a combination of poor bleed control and treatment burden,” the researchers wrote. “Compliance with prophylactic treatment was low, likely reflecting the high burden associated with standard therapies.”
The researchers acknowledged a key limitation of the study was participant dropout. As a result, some time-related data may be incomplete.
“These [data] demonstrate that patients with hemophilia A with inhibitors have impaired HRQOL, despite standard treatment, and that more effective treatment options are needed,” the researchers concluded.
The study was funded by F. Hoffmann-La Roche. The authors reported financial affiliations with Baxalta, Bayer, CSL Behring, Kaketsuken, Novo Nordisk, Pfizer, and several others.
SOURCE: Mahlangu J et al. Haemophilia. 2019 Apr 24. .