Barriers to prophylaxis treatment among haemophilia A patients and caregivers in Shandong Province, China: a descriptive qualitative study

Abstract

Background: As one of the most common rare diseases, haemophilia A poses a great burden to human society. Prophylaxis treatment is considered optimal for patients to prevent uncontrolled bleeding and reduce severity of injury. However, little is known about the use of prophylaxis treatment among haemophilia A patients in China, especially barriers that predispose them to low or non-adherence. Objective: In this study, we explored barriers that were experienced or perceived by haemophilia A patients and their caregivers who have initiated prophylaxis treatment in Shandong province in China. Methods: Primarily inspired by the AAAQ framework, we conducted 19 one-to-one interviews and 3 focus groups (18 interviewees in total). Data were analysed using a descriptive phenomenology approach and further discussed through inductive analysis. Results: Results revealed that haemophilia A patients and their caregivers struggled with nine intertwined barriers, including lack of health services for early diagnosis; inadequate supportive services for prophylaxis treatment in primary care; difficulties to obtain FⅧ concentrate at locals; financial concern associated with prophylaxis treatment; insufficient information regarding self-infusion and disease management; lack of a clarified role of primary care nurses as an acceptable care provider for infusing FVIII concentrate; lack of trust for newly developed techniques of infusion except gene therapy; safety concern associated with different product types; and poor user experience of domestic-produced infusion devices. Resulted from an inductive analysis, three clusters of underlying causes of barriers were observed: poor primary health care, inadequate social support, and failed to be patient-centred, which meanwhile highlighted opportunities for service providers and policy makers to better target and proactively design interventions towards improving prophylaxis treatment adherence in patients with haemophilia A. Conclusions: Although this study was conducted in the Chinese context, the findings still could serve as a good reference for other nations when actions are needed to protect patients affected by rare diseases.