OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, clonal, complement-mediated hemolytic anemia with protean manifestations. PNH and a series of complications have brought heavy economic burden on patients and their families, however, there are few studies on the economic burden of PNH patients in China. This study aimed to evaluate the economic burden of patients with PNH in China.

METHODS: The data was conducted by Shandong University in collaboration with the PNH China from May to June 2023, and patients with PNH who were clinically diagnosed for one year or more were included in the survey. Direct medical expenses, direct non-medical expenses and indirect costs were investigated. Descriptive analyses was conducted to analyze the annual average of various costs.

RESULTS: A valid sample size of 303 PNH patients was analyzed. The annual direct medical cost per capita was ¥45,779, while the direct non-medical cost per capita was ¥13,628, and the indirect cost per capita was ¥46,738. There was 43.7% of patients were jobless or unemployed, and 93.5% of them were unemployed due to having PNH. The average annual direct medical cost of patients with comorbidities was ¥58,942, which was more than twice that of patients without comorbidities. The average annual out-of-pocket comorbidities related medical costs for patients with combined thrombosis and kidney injury were ¥33,000 and ¥15,900, respectively. 57.8% of patients spent more than 40% of their total annual household income on direct medical care, which means catastrophic household health expenditures were incurred.

CONCLUSIONS: The economic burden of disease for Chinese PNH patients is high, especially in terms of hospitalization costs, transportation costs, and indirect costs. The existence of comorbidities is the main reason for the high economic burden of PNH patients.