The World Federation of Hemophilia World Bleeding Disorders Registry: insights from the first 10,000 patients.

ABSTRACT

Background: The prevalence of hemophilia varies globally, with close to 100% of patients diagnosed in high-income countries and as low as 12% diagnosed in lower-income countries. These inequalities in the care of people with hemophilia exist across various care indicators. Objectives: This analysis aims to describe the clinical care outcomes of patients in the World Bleeding Disorders Registry (WBDR). Methods: In 2018, the World Federation of Hemophilia developed a global registry, the WBDR, to permit hemophilia treatment centers to collect clinical data, monitor patient care longitudinally, and identify gaps in management and treatment. Results: As of July 18, 2022, 10,276 people with hemophilia were enrolled from 87 hemophilia treatment centers in 40 countries. Nearly half (49%, n = 5084) of patients had severe hemophilia; 99% were male, 85% had hemophilia A, and 67% were from low-middle-income countries. Globally, the age of diagnosis for people with severe hemophilia has improved considerably over the last 50 years, from 82 months (∼7 years) for those born before 1980 to 11 months for those born after 2010, and most prominently, among people with severe hemophilia in low- and low-middle-income countries, the age of diagnosis improved from 418 months (∼35 years) for those born before 1970 to 12 months for those born after 2010. Overall, the age of diagnosis of people with hemophilia in low- and low-middle-income countries is delayed by 3 decades compared to patients in upper-middle-income countries and by 4 decades compared to patients in high-income countries. Conclusion: Data reveal large treatment and care disparities between socioeconomic groups, showing improvements when prophylaxis is initiated to prevent bleeding. Overall, care provided in low-income countries lags behind high-income countries by up to 40 years. Limitations in the interpretation of data include risk of survival and selection bias.