Questionnaires Offer Insight on Hemophilia Patients’ Quality of Life

Self-reported health outcomes of hemophilia patients provide useful and clinically valuable data on the efficacy of treatments, a population-based study shows.

The study, “Real-world utilities and health-related quality-of-life data in hemophilia patients in France and the United Kingdom,” was published in the journal Patient Preference and Adherence.

Hemophilia can have a severe impact on patients’ quality of life, affecting their physical, psychological, social, and economic well-being.

Bleeding episodes can limit patients’ activities, either because of mobility or functional impairments or long-term, painful damage to the joints. As such, patients with more severe disease are more likely to have poorer quality of life compared to those with mild-moderate disease.

This highlights the importance of patient monitoring to ensure adequate long-term care without unnecessarily increasing treatment-associated economic burden.

U.K. and French researchers, in collaboration with researchers from the pharmaceutical company Novo Nordisk, conducted a study to gather data that could provide real-world evidence of hemophilia patients’ quality of life.

The study recruited 122 hemophilia patients in France and 62 in the U.K., age 12 or older. They were asked to respond to a series of questionnaires that assessed their perspectives on their health outcomes.

The majority reported that their disease was severe, including 71% in the French patient group and 69% in the UK group. All except one participant with a mild condition (20 patients) were taking hemophilia therapies in an on-demand regimen; 71% of the moderate group (34 patients) were also in this regimen.

In contrast, the majority (77%) of those with more severe disease (130 patients) were also taking prophylactic (preventive) treatment.

Both groups of patients showed similar health-related quality of life as determined by the results of the EQ-5D and Short-Form Health Survey (SF-36 version) standardized questionnaires.

As expected, patients who had more severe disease were also those who showed poorer health-related quality of life. Also, those who reported having more than two target joints, higher frequency of joint pain, and history of joint surgery had significantly poorer questionnaire scores.

“Results of this study reinforce the importance of appropriate treatment to limit the physical burden and long-term joint damage associated with hemophilia,” researchers said.

The team believes that the data collected in this study “could be used to convey the burden of hemophilia and its management,” as well as aid in the decision-making process “around potential treatment benefit by providing evidence to support economic modeling efforts.”