Mental Health, Work Productivity, and Quality of Life in People with Severe Haemophilia A Receiving Prophylaxis: Findings from the CHESS Data Platform

Newer therapeutic options for people with severe haemophilia A (PwSHA), in addition to improved clinical and patient-reported outcomes (PROs), have offered more personalised treatment regimens. This analysis explored mental health, work productivity, and health-related quality of life (HRQoL) among PwSHA in Europe receiving a prophylactic treatment regimen. The Cost of Haemophilia: a Socio-economic Survey (CHESS) study is a retrospective cross-sectional study of adult men with haemophilia in Europe. We analysed data from CHESS participants with severe haemophilia A and no factor VIII (FVIII) inhibitors who received emicizumab or FVIII replacement prophylaxis. Data are from patient questionnaires and their treating health care providers. This analysis focused on PROs, including productivity and activity impairment via the Work Productivity and Activity Impairment, HRQoL via the EQ-5D-5L, and anxiety via the 7-item General Anxiety Disorder questionnaire (GAD-7) and depression via the 8-item Patient Health Questionnaire (PHQ-8). SHA treatment and clinical characteristics were also collected, including bleeding events, joint health, and chronic pain. All findings were analysed descriptively. A total of 350 PwSHA met the inclusion criteria, 94 (27%) of whom provided PROs. Most (68%; n = 64) were receiving emicizumab (FVIII prophylaxis, 32%; n = 30). Clinical characteristics were generally comparable between emicizumab and FVIII prophylaxis groups, including reported chronic pain (63% and 70%) and problem joints (61% and 63%), with on-demand FVIII use for the treatment of breakthrough bleeding events more commonly reported in the FVIII prophylaxis group (34% vs. 56%). Overall, HRQoL showed comparable EQ-5D-5L scores between the treatment groups, with a marginally higher score in the emicizumab group (0.71 vs. 0.69) compared with the FVIII prophylaxis group. Anxiety and depression scores were both numerically lower in the emicizumab group, suggesting a lower burden of disease (anxiety 7-item General Anxiety Disorder questionnaire GAD-7 mean scores, 6.0 vs. 7.3; depression PHQ-8 mean scores, 6.8 vs. 7.8). Employed PwSHA in the emicizumab group reported a lower impact of SHA on their work impairment (31% vs. 50%), and only 19% (vs. 33%) of the emicizumab group required assistance with daily activities. More PwSHA receiving FVIII prophylaxis reported a negative impact of SHA on their ability to participate in social activities (70% vs. 56%) and on their physical activity (57% vs. 44%). Patients receiving emicizumab prophylaxis appeared to have more favourable mental health, work productivity, and HRQoL-related outcomes than those receiving FVIII prophylaxis. These findings were observed in the context of comparable clinical characteristics between emicizumab and FVIII prophylaxis despite evidence of a more complex treatment history for the emicizumab group. This analysis has limitations, including a lack of adjustment for confounding factors.