Ingrid den Uijl1, Douwe Biesma2, Diederick Grobbee3, Kathelijn Fischer1. 1. van Creveldkliniek, Department of Haematology, Medical University Centre Utrecht, Utrecht, The Netherlands Julius Center for Health Sciences and Primary Care, Medical University Centre Utrecht, Utrecht, The Netherlands. 2. Department of Haematology, Medical University Centre Utrecht, Utrecht, The Netherlands. 3. Julius Center for Health Sciences and Primary Care, Medical University Centre Utrecht, Utrecht, The Netherlands.
Abstract
BACKGROUND: Moderate haemophilia is the rarest form of haemophilia. This study aims to assess short- and long-term outcome, including its association with treatment, in patients with moderate haemophilia. MATERIAL AND METHODS: Seventy-five patients with moderate haemophilia (1-5% factor VIII/ factor IX activity), without a history of inhibitors, treated at the van Creveldkliniek, Utrecht (NL) were included in the study. Life-long data on bleeding and treatment were collected. Joints were evaluated using the Haemophilia Joint Health Score. Adults completed questionnaires on activity (HAL) and quality of life (SF-36, EQ5D). RESULTS: The median age of the patients was 37 years (IQR 23-52 years) and haemophilia A was diagnosed in 89%. Bleeding frequency was low: the median annual bleeding rate was 2.0 bleeds/ year (IQR 0.8-3.7 bleeds/year), including a median of 0 joint bleeds/year (IQR 0.8-3.7 bleeds/year). Joint function was good: 82% scored<10 out of 126 points of the Haemophilia Joint Health Score (HJHS). Nevertheless, 29% of patients with moderate haemophilia had a history of prophylaxis, because of a high bleeding frequency. Median age at first joint bleed was 4.8 years (IQR 3.5-8.5). Use of prophylaxis was more associated with age at first joint bleed (P<0.01) than with baseline factor activity (P=0.12). Most patients (52%) who suffered their first joint bleed before the age of 5 years required prophylaxis later in life. DISCUSSION: The majority of patients with moderate haemophilia have few bleeds and complications; however, a considerable subset of patients with a more severe bleeding pattern need prophylactic treatment. These latter patients may be identified by the onset of joint bleeding before the age of 5 years.
BACKGROUND: Moderate haemophilia is the rarest form of haemophilia. This study aims to assess short- and long-term outcome, including its association with treatment, in patients with moderate haemophilia. MATERIAL AND METHODS: Seventy-five patients with moderate haemophilia (1-5% factor VIII/ factor IX activity), without a history of inhibitors, treated at the van Creveldkliniek, Utrecht (NL) were included in the study. Life-long data on bleeding and treatment were collected. Joints were evaluated using the Haemophilia Joint Health Score. Adults completed questionnaires on activity (HAL) and quality of life (SF-36, EQ5D). RESULTS: The median age of the patients was 37 years (IQR 23-52 years) and haemophilia A was diagnosed in 89%. Bleeding frequency was low: the median annual bleeding rate was 2.0 bleeds/ year (IQR 0.8-3.7 bleeds/year), including a median of 0 joint bleeds/year (IQR 0.8-3.7 bleeds/year). Joint function was good: 82% scored<10 out of 126 points of the Haemophilia Joint Health Score (HJHS). Nevertheless, 29% of patients with moderate haemophilia had a history of prophylaxis, because of a high bleeding frequency. Median age at first joint bleed was 4.8 years (IQR 3.5-8.5). Use of prophylaxis was more associated with age at first joint bleed (P<0.01) than with baseline factor activity (P=0.12). Most patients (52%) who suffered their first joint bleed before the age of 5 years required prophylaxis later in life. DISCUSSION: The majority of patients with moderate haemophilia have few bleeds and complications; however, a considerable subset of patients with a more severe bleeding pattern need prophylactic treatment. These latter patients may be identified by the onset of joint bleeding before the age of 5 years.
Authors: K Fischer; J G van der Bom; E P Mauser-Bunschoten; G Roosendaal; F J Beek; P de Kleijn; D E Grobbee; H M van den Berg Journal: Thromb Haemost Date: 2000-12 Impact factor: 5.249
Authors: P Hilliard; S Funk; N Zourikian; B-M Bergstrom; C S Bradley; M McLimont; M Manco-Johnson; P Petrini; M van den Berg; B M Feldman Journal: Haemophilia Date: 2006-09 Impact factor: 4.287
Authors: Kathelijn Fischer; Johanna G van der Bom; Eveline P Mauser-Bunschoten; Goris Roosendaal; Robert Prejs; Piet de Kleijn; Diederick E Grobbee; Marijke van den Berg Journal: Blood Date: 2002-04-01 Impact factor: 22.113
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