Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 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.

PubMed Disclaimer

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.
See this image and copyright information in PMC

Comment in

References

    1. Report on the Global Survey. World federation of Haemophilia; 2008. [Accessed on: 27/01/2011]. Available at: www1.wfh.org/publications/files/pdf-1429.pdf.
    1. Ahlberg A. Haemophilia in Sweden. VII. Incidence, treatment and prophylaxis of arthropathy and other musculo-skeletal manifestations of haemophilia A and B. Acta Orthop Scand. 1965;77(Suppl):3–132. - PubMed
    1. Plug I, van der Bom JG, Peters M, et al. Thirty years of hemophilia treatment in the Netherlands, 1972–2001. Blood. 2004;104:3494–500. - PubMed
    1. den Uijl IEM, Fischer K, van der Bom JG, et al. Clinical outcome of moderate haemophilia compared with severe and mild haemophilia. Haemophilia. 2009;15:83–90. - PubMed
    1. Lindvall K, Colstrup L, Wollter IM, et al. Compliance with treatment and understanding of own disease in patients with severe and moderate haemophilia. Haemophilia. 2006;12:47–51. - PubMed

Publication types

MeSH terms