OBJECTIVES: Hemophilia A is a congenital bleeding disorder disease that is characterized by missing or defective factor VIII. The objective of the present study was to determine the clinical value, economic value, patient value and social value of prophylaxis in adult patients with hemophilia A compared with on-demand.

METHODS: Using Cochrane methodology, a systematic literature review was conducted in PubMed, EMBASE, CNKI, WanFang and VIP (Search Period: June 2002-June 2022). Studies published in Chinese/English reporting on value of prophylactic treatment in adult hemophilia A patients were included. The following search terms were used for identifying relevant literature, ‘Hemophilia A’, ‘prophyla*’, ‘safety’, ‘efficacy’, ‘cost-effectiveness’, ‘cost-utility’, ‘quality of life’, ‘QALY’, ‘utility’ and ‘productivity loss’. At the same time, the guidelines or documents of value framework, which were established by health technology assessment agencies around the world, were searched.

RESULTS: The literature search yielded 2,485 citations. After reviewing the identified citations, 89 studies were included. The value of prophylaxis was summarized into four aspects, which contains clinical value, economic value, patient value and social value. In the clinical value (n=44), prophylaxis could effectively reduce the number of bleeding episodes, protect joint function, and reduce the disability rate compared with on-demand. In the economic value (n=29), prophylaxis was demonstrated to be cost-effective compared with on-demand. In the patient value (n=7), prophylaxis could obtain higher social participation and improve the quality of life compared with on-demand. In the social value (n=9), prophylaxis could enable patients to return to normal work, school and life compared with on-demand.

CONCLUSIONS: The review indicated that the core value of prophylaxis in adult patients with hemophilia A. Future research is needed to construct a value-based Multi-Criteria Decision Analysis framework approved by experts, which provides assessment basis for decision making by multiple stakeholders.