Humanistic burden of problem joints for children and adults with haemophilia.

Abstract

The "problem joint" (PJ) concept was developed to address patient-centric needs for a more holistic assessment of joint morbidity for people with haemophilia (PwH). To quantify the humanistic burden of PJs in PwH to further support validation of the PJ outcome measure. Multivariable regression models evaluated the relationship between PJs and health-related quality of life (HRQoL, EQ-5D-5L) and overall work productivity loss (WPL) using data from the 'Cost of HaEmophilia: a Socioeconomic Survey' population studies (adults: CHESS II, CHESS US+; children/adolescents: CHESS-Paeds). Covariates included were haemophilia severity, age, comorbidities and education. The CHESS II sample included 292 and 134 PwH for HRQoL and WPL analyses, mean age 38.6 years (39% ≥1 PJ, 61% none). CHESS US+ included 345 and 239 PwH for HRQoL and WPL, mean age 35 years (43% ≥1 PJ, 57% none). CHESS-Paeds included 198 PwH aged 4-17 (HRQoL only), mean age 11.5 years (19% ≥1 PJ, 81% none). In CHESS II and CHESS US+, presence of PJs was associated with worse HRQoL (Both p<.001). Few CHESS-Paeds participants had PJs, with no significant correlation with HRQoL. In CHESS II, upper body PJs were significantly correlated to WPL (p<.05). In CHESS US+, having ≥1 PJ or upper and lower body PJs were significantly correlated to WPL (vs. none; both p<.05). This study has shown a meaningful burden of PJs on PwH, which should be considered in clinical and health policy assessments of joint health.