Researchers Question Health-Related Quality of Life for Youth, Young Men with Hemophilia

Researchers at several Canadian institutions investigated the tendencies of health-related quality of life (HRQoL) among teenagers and young adults with hemophilia A (HA) and found that young adults demonstrated more joint disease and worse HRQoL when physical function and pain were considered.

The research paper, “Generic and disease-specific quality of life among youth and young men with Hemophilia in Canada,” published in BMC Hematology, provides new information for the selection of HRQoL parameters for future hemophilia clinical trials.

The objective assessment of patient status and outcomes of different therapeutic strategies is usually achieved by measurement of bleeding rates, joint and musculoskeletal status, pain, and physical and social function. Health-related quality of life (HRQoL) assessment aims to measure the impact of a disease and chosen treatment on the patient. Such tools basically consist of self-administered questionnaires. At the present, there are very few published studies on long-term trends of HRQoL in hemophilia.

HRQoL in hemophilia may be not only be determined by change in physical status, but also “influenced by common social events such as educational, vocational or relational changes associated with transition from adolescence to adulthood” the authors wrote. The study enrolled 13 youth (ages 12.9-17.9 years) and 33 young adults (ages 18.4-28.7 years) with moderate (7 %) and severe (93 %) HA, from six treatment centers in Canada.

According to results, the youth had minimal joint damage, when compared to young adults. HRQoL scores for SF-36 Physical Component Summary (PCS) and Mental Component Summary (MCS), for both age groups, were comparable to published Canadian norms. But the differences for Physical Functioning and Bodily Pain were clinically meaningful and statistically significant —  young adults presented declining function when compared to the youth. Lifelong prophylaxis treatment correlated positively with better PCS scores, but was also associated with worse MCS scores.

The study concluded: “Comparatively little is known about factors that might influence longitudinal patterns of HRQoL in patients on a stable treatment regimen. The current results form the foundation for a longitudinal study to examine the impact of biological factors and life events on the HRQoL of youth and young adults with hemophilia followed prospectively for 3 years.”