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. 2021 Nov;27(6):1037-1044.
doi: 10.1111/hae.14403. Epub 2021 Sep 4.

Women and girls with haemophilia receiving care at specialized haemophilia treatment centres in the United States

Connie H Miller  1   2 , J Michael Soucie  1   2 , Vanessa R Byams  1 , Amanda B Payne  1 , Robert F Sidonio Jr  3 , Tyler W Buckner  4 , Christopher J Bean  1
Affiliations

Women and girls with haemophilia receiving care at specialized haemophilia treatment centres in the United States

Connie H Miller et al. Haemophilia. 2021 Nov.

Abstract

Introduction: Females may have haemophilia with the same factor VIII (FVIII) or factor IX (FIX) levels as affected males. Characterization of females with haemophilia would be useful for health care planning to meet their unique needs. Federally-funded haemophilia treatment centres (HTCs) in the United States contribute data on all individuals with bleeding disorders receiving care to the Population Profile (HTC PP) component of the Community Counts Public Health Surveillance of Bleeding Disorders project.

Aims: To estimate the number of females with haemophilia receiving care at HTCs in the United States and compare their characteristics with those of males with haemophilia.

Methods: HTC PP data collected on people receiving care at an HTC from January 2012 through September 2020 with haemophilia A and B were evaluated by sex for demographic and clinical characteristics.

Results: A factor level < 40% was reported for 23,196 males (97.8%) and 1667 females (47.6%) attending HTCs; 51 (.48%) severe, 79 (1.4%) moderate, and 1537 (17.9%) mild haemophilia patients were female. Females were older, more often White, and less often non-Hispanic than males. Females were less likely to have history of HIV or HCV infection, even among those with severe disease, but twice as likely to have infection status unknown. Females with mild haemophilia were more often uninsured than males.

Conclusions: Females with severe or moderate haemophilia are uncommon, even in specialized care centres; however, almost one in five patients with mild haemophilia was female, indicating needs for specialized care based on factor level and history for affected females.

Keywords: factor IX; factor VIII; haemophilia A; haemophilia B.

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Conflict of interest statement

DISCLOSURES

CHM, JMS, and ABP designed the study, conducted the research, analyzed the data and wrote the paper. VRB, RFS, TWB, and CJB conducted the research and wrote the paper. RLS has participated as a paid consultant to Bayer, Biomarin, uniQure, Spark, Novo Nordisk, Genentech/Roche, Octapharma, Takeda, Catalyst, Guardian Therapeutics, Pfizer, Hema Biologics, and Sanofi/Sobi and has investigator initiated grants from Octapharma, Genetech, Grifols, and Takeda. TWB has acted as a paid consultant to BioMarin, Tremeau Pharmaceuticals, and uniQure and has participated in advisory boards with Takeda, Pfizer, CSL Behring, Genentech/Roche, Spark, Novo Nordisk, Spark, and Bayer. All other authors stated that they had no interests which might be perceived as posing a conflict or bias.

Figures

Figure 1.
Figure 1.
Distribution of factor levels for females attending U.S. hemophilia treatment centers
Figure 2.
Figure 2.
Mean and 95% confidence limits for number of visits during the study period (2012-2020) by sex and severity
See this image and copyright information in PMC

References

    1. Miller CH, Bean CJ. Genetic causes of haemophilia in women and girls. Haemophilia. 2021;27:e164–e179. 10.1111/hae.14186. - DOI - PMC - PubMed
    1. Byams VR, Kouides PA, Kulkarni R, et al. Surveillance of female patients with inherited bleeding disorders in United States Haemophilia Treatment Centres. Haemophilia. 2011;17(Suppl. 1):6–13. - PMC - PubMed
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    1. Manco-Johnson MJ, Byams VR, Recht M, et al. Community Counts: Evolution of a national surveillance system for bleeding disorders. Am J Hematol. 2018;93:e137–e140. - PMC - PubMed
    1. National Center on Birth Defects and Developmental Disabilities. Centers for Disease Control and Prevention. HTC Population Profile Technical Notes. 2020. https://www.cdc.gov/ncbddd/hemophilia/communitycounts/data-reports/2020-....

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