A significant proportion of patients with hemophilia B have less than optimal quality of life regardless of the disease severity, according to a new study published in Haemophilia.

“Unmet needs remain as joint disease is present in a proportion of patients and include improved treatment algorithms to prevent adverse outcomes also in nonsevere [hemophilia B],” the authors said.

The proportion of patients showing less than optimal quality of life was particularly high among patients taking inhibitors or those with severe disease, with only 13% and 47% reporting no problems in any dimension in the EuroQol-5 Dimension questionnaire, respectively.

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Patients taking inhibitors reported lower quality of life in all domains compared to those without inhibitors. At least half mentioned problems with mobility, pain/discomfort, and anxiety/depression, while a lower proportion also experienced problems with self-care and usual activities.

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Patients with the severe disease showed a similar pattern to those taking inhibitors, albeit the proportion of patients reporting problems in each domain was lower, and patients with nonsevere disease mainly reported no problems in any of the dimensions.

The researchers found a similar pattern between patients with moderate and mild disease, although those with moderate hemophilia B had reported more problems in the pain/discomfort domain.

Patients taking inhibitors and those with severe disease also tended to report lower EuroQol-Visual Analogue Scale (EQ-VAS) scores than patients with the nonsevere disease. The authors found negative correlations between the EQ-VAS and the level sum score summary scores within each severity group, as well as with the composite joint score. Moreover, the authors found a decline in EQ-VAS scores with patients’ age.

The B-Natural study enrolled an international cohort of 224 individuals with factor IX deficiency. About half (50.9%) of participants had moderate disease, followed by severe (30.4%) and mild (18.8%) disease. Twenty-nine participants were receiving inhibitors or had a history of inhibitors.


Berntorp E, LeBeau P, Ragni M V., et al. Quality of life in a large multinational haemophilia B cohort (the B‐Natural study) – unmet needs remain. Haemophilia. Published online March 8, 2022. doi:10.1111/hae.14525