A study of variations in the reported haemophilia B prevalence around the world

Abstract

The objectives of this article were to study the reported prevalence of haemophilia B (HB) on a country-by-country basis and to analyse whether the prevalence of HB varied by national economy. The prevalence of HB is the proportion of diagnosed, reported cases of HB in a population at a specific point of time. We collected data on the HB prevalence for 105 countries from the World Federation of Hemophilia annual global surveys. Our results showed that the HB prevalence varied considerably among countries, even among the wealthiest of countries. The HB prevalence (per 100 000 males) for the highest income countries was 2.69 ± 1.61 (mean ± SD), whereas the prevalence for the rest of the world was 1.20 ± 1.33 (mean ± SD). Ireland had the highest reported HB prevalence of 8.07 per 100 000 males. There was a strong trend of increasing HB prevalence (per 100 000 males) over time. Prevalence data reported from the WFH compared well with prevalence data from the literature. The WFH annual global surveys have some limitations, but they are the best available source of worldwide haemophilia data. Prevalence data are extremely valuable information for the planning efforts of national healthcare agencies in setting priorities and allocating resources for the treatment of HB.

Population pharmacokinetics of plasma-derived factor IX in adult patients with haemophilia B: implications for dosing in prophylaxis

“…The prevalence of haemophilia B is estimated at approximately 1 in 30 000 males [33]. Of these, only 30-40% have severe disease [34] and would be eligible for a PK study.…”

Mutation Spectrum and Genotype–Phenotype Analyses in a Pakistani Cohort With Hemophilia B

“…9 Figures for prevalence of HB in Pakistan, reported annually by WFH since 1998, are very inconsistent, ranging from 2.02 in 2000 to 0.06 in 2003, the reason being lack of a centralized national registry. 10 To date, only 1 study has been performed on genetic analysis of patients with HB from Pakistan, identifying 11 different mutations. 11 The current study broadens spectrum of mutations in Pakistani patients with HB.…”

STALing B cell responses with CD22

“…Hemophilia A affects approximately 1 in 10,000 males, who must be continuously treated with recombinant human FVIII (rhFVIII) (termed replacement therapy) to prevent uncontrolled bleeding (17). Approximately 20%-30% of patients receiving replacement therapy develop anti-FVIII antibodies that preclude further treatment with rhFVIII (18). Immune tolerance induction (ITI), which requires repeated administration of highdose FVIII over a period of months to years, is the current treatment for development of anti-FVIII Abs (19).…”