Clinical outcome of moderate haemophilia compared with severe and mild haemophilia.
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 bleeds, and 68% reported joint impairment.


CONCLUSIONS
Although outcome in moderate haemophilia is generally in between severe and mild haemophilia, moderate haemophilia patients reported a substantial burden of disease, and for more than 25% of patients with moderate haemophilia long term prophylaxis was implemented because of frequent bleeds.
den Uijl
I E M
IE
Van Creveldkliniek, Department of Haematology, UMCU, Itrecht, The Netherlands.
Fischer
K
K
Van Der Bom
J G
JG
Grobbee
D E
DE
Rosendaal
F R
FR
Plug
I
I
D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002648:Child; D002675:Child, Preschool; D017281:Cost of Illness; D004185:Disability Evaluation; D004363:Drug Utilization / Q000706:statistics & numerical data; D004651:Employment / Q000706:statistics & numerical data; D006395:Hemarthrosis / Q000453:epidemiology / Q000209:etiology / Q000517:prevention & control; D006467:Hemophilia A / Q000150:complications / Q000453:epidemiology / Q000534:rehabilitation*; D006470:Hemorrhage / Q000453:epidemiology / Q000209:etiology / Q000517:prevention & control; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D009426:Netherlands / Q000453:epidemiology; D011379:Prognosis; D011788:Quality of Life; D012720:Severity of Illness Index; D012959:Socioeconomic Factors; D055815:Young Adult