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. 2014 Jan;12 Suppl 1(Suppl 1):s330-6.
doi: 10.2450/2012.0091-12. Epub 2012 Nov 20.

Outcome in moderate haemophilia

Ingrid den Uijl  1 , Douwe Biesma  2 , Diederick Grobbee  3 , Kathelijn Fischer  1
Affiliations

Outcome in moderate haemophilia

Ingrid den Uijl et al. Blood Transfus. 2014 Jan.

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.

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Figures

Figure 1
Figure 1
Distribution of residual clotting factor activity of patients with moderate haemophilia (n=75).
Figure 2
Figure 2
Percentage of patients with a history of prophylaxis according to age at first joint bleed.
Figure 3
Figure 3
Health-related quality of life, as measured by the SF-36, according to baseline factor activity. The solid black line represents the male population in The Netherlands.
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