The Health-Related Quality of Life scores and joint health in children and young adults with hemophilia.

Abstract

Background: Patients with hemophilia should be evaluated for joint health and overall health in their visits. The aims of this study were to evaluate joint health and health-related quality of life (HRQoL) in patients with mild, moderate, and severe hemophilia; determine which patient groups to focus on and whether there are any neglected patient groups.

Methods: This was a single-center, cross-sectional study. Patients were evaluated by ultrasonography (Hemophilia Early Arthropathy Detection with Ultrasound [HEAD-US]), physical examination (Hemophilia Joint Health Score version 2.1 [HJHS-2.1]), and HRQoL scales (EQ-5D/EQ-VAS and Haemo-QoL).

Results: Thirty-nine patients with regular follow-up were evaluated for a total of 234 joints. When hemophilia severity was compared with the HEAD-US and HJHS-2.1, a significant difference was found between severe and non-severe hemophilia. On the other hand, when patients' total HEAD-US scores were compared with total HJHS-2.1 scores, no statistically significant correlations were found; only a statistically significant but negligible correlation was detected when HEAD-US and HJHS-2.1 scores were examined at joint level. No significant difference was found when mild, moderate or severe hemophilia were compared with the HRQoL scores. Also, HEAD-US scores and HRQoL scores were not correlated, showing that the HRQoL score did not change whether the patient has arthropathy or not.

Conclusion: Despite recent advances in treatment options for hemophilia, arthropathy in patients with severe hemophilia remains challenging. For the follow-up of pediatric hemophilia, the HEAD-US and HJHS should be used together because their correlation was weak. Although patients with severe hemophilia are at higher risk in terms of arthropathy, patients with mild/moderate hemophilia should not be ignored because their HRQoL is not different from that of severe hemophilia.