Khanji C, Nuabor W, Kim HK, Yubin SA, Kim R, Njue A. Systematic literature review of the humanistic burden of hemophilia A and B. Poster presented at the ISPOR Europe 2024; November 17, 2024. Barcelona, Spain. [abstract] Value Health. 2024 Dec; 27(12 Supplement):S613. doi: 10.1016/j.jval.2024.10.3082

OBJECTIVES: Hemophilia causes substantial clinical burden, which can lead to reduced health-related quality of life (HRQOL) in patients. This systematic literature review aimed to examine the humanistic burden of hemophilia A or B, with or without inhibitors in patients ≥ 12 years and their caregivers.

METHODS: Searches were conducted in Embase, MEDLINE, and MEDLINE In-Process for English-language articles published from November 2012 through November 2022. Abstracts and full texts were screened by one researcher with a 10% quality check by another researcher.

RESULTS: Screening 6,057 citations yielded 42 observational studies for inclusion. The studies were heterogenous, utilizing 25 different HRQOL scales. Patients with hemophilia A or B experienced poor HRQOL. Severe disease and inhibitor status had a negative impact on HRQOL. For example, the mean predicted EQ-5D-5L score for patients with hemophilia A or B without inhibitors was 13% worse for patients with severe versus mild disease (−0.105). Globally, patients with hemophilia A or B with inhibitors had worse mean EQ-5D scores (0.7075) than those without inhibitors (hemophilia A, 0.7453; hemophilia B, 0.7407). Severe hemophilia A had a negative impact on work productivity. Hemophilia A or B had minimal to moderate negative effect on functional abilities, with mean Hemophilia Activities List scores ranging from 60 to 99. Caregivers of children with hemophilia A or B with inhibitors had significantly poorer HRQOL compared with those providing care to children without inhibitors based on the care-related HRQOL instrument and a novel disease-specific questionnaire. Decreased infusion frequency appeared to reduce the practical burden and emotional distress of caregiving.

CONCLUSIONS: Patients with hemophilia A or B and their caregivers experience poor HRQOL and functionality, especially those with inhibitors, and severe disease negatively affected HRQOL.