Although the effects of prophylaxis in mild to moderate hemophilia are unclear due to the variability in bleeding risk between patients, there is a subpopulation of patients with a more severe phenotype of moderate hemophilia that includes joint bleeds and impairment, and most of these individuals use prophylaxis at some point.
The authors emphasized that hemophilia is generally underrecognized in women, although symptoms and complications of the disease are similar to those observed in male patients. Heavy menstrual bleeding and increased bleeding risk in pregnancy and postnatally confer additional challenges in women with hemophilia.
A recent roundtable discussion that included healthcare professionals and patients revealed a misconception among some providers that women are “just genetic carriers” and do not have an increased bleeding risk. Other findings demonstrated barriers to treatment access for women with hemophilia.2,3
“I think that most people associate hemophilia in their heads with a male disorder and don’t understand genetics well, hence the bleeding tendencies of carrier women may sometimes go underrecognized,” explained Dr Von Drygalski. “Certainly, carrier women or women with hemophilia are at the same risk of bleeding as hemophilic males at similarly low factor levels.”
Treatment recommendations for these women are the same as for men with similar factor levels, and it is also advisable for women of reproductive age to “involve Ob/Gyn to control menorrhagia or find the optimal solutions for menstrual bleed control, such as oral contraceptives, intrauterine devices, tranexamic acid, desmopressin, or other hormonal or interventional measure – it’s a combined effort,” she stated.
In an analysis of data from the PROBE study, participants with mild to moderate hemophilia demonstrated significantly reduced health-related quality of life as well as more frequent reports of acute and chronic pain compared to those with no bleeding disorder.3 In addition, results from the P-FiQ and B-HERO-S studies showed that many of these patients experience anxiety and depression (51%) and relationship issues associated with the disease (81%-94%), along with difficulty gaining access to hemophilia treatment centers (17%) and factor products (54%).4
Taken together, these findings underscore the importance of learning more about the unique needs of this population and optimizing strategies for clinical management.
- Rajpurkar M, Forsyth A, Manco-Johnson M. Current challenges for men and women with mild-to-moderate haemophilia. Haemophilia. 2021;27(Suppl 1):5-7. doi:10.1111/hae.14193
- Walsh C, Boggio L, Brown-Jones L, et al. Identified unmet needs and proposed solutions in mild-to-moderate haemophilia: a summary of opinions from a roundtable of haemophilia experts. Haemophilia. 2021;27(Suppl 1):25-32. doi:10.1111/hae.14168
- Non-severe haemophilia: is it benign? – insights from the PROBE study. Haemophilia. 2021;27(Suppl 1):17-24. doi:10.1111/hae.14105
- Impact of haemophilia on patients with mild-to-moderate disease: results from the P-FiQ and B-HERO-S studies. Haemophilia. 2021;27(Suppl 1):8-16. doi:10.1111/hae.14251