Among patients with hemophilia A who receive primary prophylaxis, quality of life (QoL) may be lower compared with the general population, according to research published in Haemophilia.

Among patients with severe hemophilia, prophylaxis with clotting factor replacement therapy is the standard treatment, though patients vary in when they initiate therapy. In the optimal scenario, patients will receive prophylaxis prior to the development of clinically evident joint disease, because prophylaxis cannot reverse joint damage.

Primary prophylaxis is defined as treatment initiated prior to the second clinically evident large joint bleed, and where the patient is aged 3 years. Secondary and tertiary prophylaxis refer to treatment initiated after 2 or more bleeds and after the onset of joint disease, respectively; prophylaxis may also be used intermittently. Each of these forms of treatment is distinct from on-demand treatment, where each bleeding episode is treated separately.

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Previous research suggests that on-demand treatment leads to worse morbidity and QoL. However, there is limited evidence as to whether use of primary prophylaxis leads to improved morbidity and QoL compared with other forms of treatment, or with the general population. For this study, researchers evaluated QoL and functional variables among patients with severe hemophilia A treated with primary prophylaxis and who had not received inhibitors with several groups: a) patients receiving on-demand treatment or secondary prophylaxis, b) the general population, and c) a population of patients with osteoarthritis.

All patient data were obtained from the Cost of Haemophilia in Europe: A Socioeconomic Survey (CHESS) database. Overall, 166 patients (median age, 24 years) who received primary prophylaxis and 349 patients (median age, 26 years) who received on-demand treatment or secondary prophylaxis were included.

In the primary prophylaxis group, 39% of the patients’ clinicians reported any target joint, and the median number of bleeds per year was 1; 89 patients (54%) had no pain as reported by clinicians, while 63 patients (38%) had mild pain and 14 patients (8%) had moderate pain. Severe pain was not reported in this patient group.

In the secondary prophylaxis/on-demand treatment group, 216 of the patients’ (62%) clinicians reported any target joint, and the median number of bleeds per year was 2; 141 patients (40%) had no pain as reported by clinicians, while 140 patients (40%) had mild pain, 61 patients (17%) had moderate pain, and 7 patients (2%) had severe pain.

Although anxiety and depression levels were similar between the primary prophylaxis and on-demand treatment groups, patients in the primary prophylaxis group had lower employment and productivity than that seen in the general population. Presenteeism, defined as attending work with an impairment, was similar between the primary prophylaxis group and those with osteoarthritis, who were on average older and had higher rates of pain and joint damage.

Disclosures: Some authors have declared affiliations with or received funding from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.


O’Hara S, Castro FA, Black J, et al. Disease burden and remaining unmet need in patients with haemophilia A treated with primary prophylaxis. Haemophilia. Published online October 20, 2020. doi:10.1111/hae.14171