A new study has found that 1 year of emicizumab (Hemlibra®) therapy in adult patients with severe hemophilia A without inhibitors achieves high rates of patient satisfaction, as measured by pharmacist consultations and EQ-5D-3L survey scores.

The study, published in Haemophilia, assessed perceived clinical evolution, quality of life, and treatment satisfaction.

“Since the approval of emicizumab for [people with severe hemophilia A] without inhibitor, new consultations with pharmacists have been set up to support patients: at emicizumab therapy introduction (P1) to discuss the characteristics and administration of the drug, 2 months later (P2) to assess the skills acquired by patients, and after 1 year of treatment (P3),” the authors wrote. “This last meeting focuses on the quality of life, evolution, and satisfaction of patients.”

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The research team conducted an observational, retrospective study at a single center in Paris, France. Thirty-eight adult male patients undergoing emicizumab therapy between June 2020 and March 2021 were included, and they participated in 3 consultations with pharmacists to assess the evolution of their clinical perceptions.

Aspects assessed included general state of health, frequency of spontaneous bleeds, chronic pain, consumption of painkillers, mobility, sick leave, physical activity, post-traumatic bleeds, and the ability to identify bleeds.

The results revealed improvements in the perceived clinical parameters after 1 year of treatment with emicizumab compared with standard of care, as well as improvements in general health state and reductions in the perceived frequency of bleeds. Although chronic pain was persistent in this cohort, its intensity decreased overall as measured by the visual analog scale.

The authors caution that the 1-year treatment period is limited, and the sample size was relatively small. However, after 1 year of emicizumab therapy, no patient wanted to return to their previous treatment regimen. The average patient satisfaction rating for the therapy was 9.1 out of 10.


Oka G, Roussel-Robert V, Levivien C, Lopez I, Pieragostini R. Assessment of the clinical perception, quality of life and satisfaction of patients with severe congenital haemophilia A without inhibitor after 1 year of emicizumab therapy. Haemophilia. Published online February 7, 2023. doi:10.1111/hae.14755