Clinical outcome of moderate haemophilia compared with severe and mild haemophilia

Haemophilia : the Official Journal of the World Federation of Hemophilia
I E M Den UijlI Plug

Abstract

Information on outcome and treatment of patients with moderate haemophilia is scarce. In this study, we compared self-reported burden of disease in moderate haemophilia to severe and mild haemophilia. A nationwide questionnaire on bleeding pattern, treatment, impairment and quality of life was sent to 1567 Dutch patients with haemophilia. Out of 1066 respondents (response rate: 68%), 16% had moderate, 44% severe and 39% mild haemophilia. Median age was 36 years. Although overall outcome in moderate haemophilia was in between severe and mild haemophilia, moderate haemophilia patients did report a substantial burden of disease. The majority of patients with moderate haemophilia (73%) reported bleeds in the previous year; and a considerable proportion of moderate patients reported joint impairment (43%), chronic pain (15%), needed orthopaedic aids (24%) or were unemployed because of disability (27%). Within the group of moderate haemophilia patients, a large variation in bleeding pattern and outcome was observed. A quarter of patients with moderate haemophilia reported a more severe phenotype and intermittent use of prophylaxis. These patients reported frequent bleeding, with a median of eight bleeds per year, including two joint ...Continue Reading

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Citations

Dec 10, 2013·Hematology·Neil Josephson
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Feb 2, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·Madhvi RajpurkarMarilyn Manco-Johnson
Feb 18, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·Alban Fouasson-ChaillouxMarc Dauty
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Mar 3, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·María García-DasíSantiago Bonanad
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