The World Federation of Hemophilia World Bleeding Disorders Registry: Insights from the First 10,000 Patients

Authors: Coffin, D; Gouider, E; Konkle, B; Hermans, C; Lambert, C; Diop, S; Ayoub, E; Tootoonchian, E; Youttananukorn, T; Dakik, P; Pereira, T; Iorio, A; Pierce, GF.

Affiliations: World Federat Hemophilia, 1425 Rene Levesque Blvd W Bureau 1200, Montreal, PQ H3G 1T7, Canada. Fac Med Tunis, Hemophilia Ctr Aziza Othmana, Serv Hematol Biol, Tunis, Tunisia.] Bloodworks Northwest, Washington Ctr Bleeding Disorders, Seattle, WA USA. Catholic Univ Louvain, Dept Internal Med, Louvain La Neuve, Belgium. Clin Univ St Luc, Div Hematol, Haemostasis & Thrombosis Unit, Brussels, Belgium. Univ Cheikh Anta Diop, Natl Blood Transfus Ctr, Dept Hematol, Dakar, Senegal. McMaster Univ, Dept Clin Epidemiol & Biostat, Hamilton, ON, Canada. McMaster Univ, Dept Med, Hamilton, ON, Canada.

Publication: Research and Practice in Thrombosis and Haemostasis. 2023. 7

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 se-lection bias.