Presentation (CTI)

OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening hematological disorder characterized by intravascular hemolysis, bone marrow failure, and thrombosis, which substantially impair patients’ quality of life. In China, delayed diagnosis, fragmented referrals, care inequities, and high financial burdens shape how patients seek care and continue treatment. However, evidence remains limited on how structural barriers and patient-level factors interact across the PNH care pathway.
METHODS: This qualitative study used semi-structured online interviews (June-December 2023) with 12 adults with confirmed classical PNH. Participants were recruited through a national advocacy group using purposive and quota sampling until thematic saturation. Data were analyzed using framework analysis, informed by the COM-B model to examine capability-, opportunity-, and motivation-related influences on patient engagement.
RESULTS: Analysis identified 3 overarching themes and 15 subthemes. Patients’ capability to engage with care was shaped by health literacy, self-management strategies, and physical limitations. These factors interacted with external conditions, including fragmented referral and follow-up arrangements, inequities in access to specialist services, financial burden, and variable support networks. Motivation was further influenced by stigma, self-efficacy, perceived risk, and emotional burden. Together, these factors shaped how patients sought diagnosis, navigated treatment, and remained engaged in long-term management across the care pathway.
CONCLUSIONS: Patient engagement in PNH was shaped by the interaction between structural barriers and patient-level factors across the care pathway. External constraints often intensified patients’ physical and emotional challenges, limiting timely help-seeking, continuity of care, and long-term disease management. Improving PNH care will require coordinated strategies that combine patient education, peer and family support, care navigation, and stronger financial protection and service coordination. These findings highlight the importance of addressing both health system barriers and patient-side challenges to support more equitable diagnosis, treatment, and follow-up for people living with PNH.