Patients with severe hemophilia A treated prophylactically with recombinant factor VIII Fc fusion protein (rFVIIIFc) experienced less pain, or even no pain, after 28 to 32 weeks of treatment, according to the results of a new study published in Therapeutic Advances in Hematology.
The authors presented a post hoc analysis of patient-reported outcomes from the A-LONG (NCT01181128) study, a phase 3 open-label, multicenter, partially randomized trial on the use of rFVIIIFc in adults and adolescents with severe hemophilia A.
“Our analysis showed that rFVIIIFc individualized prophylaxis resulted in significantly fewer patients reporting painful swellings and instances of joint pain at end of study vs baseline as assessed by the Haem-A-QoL [Hemophilia-specific Quality of Life questionnaire],” the authors wrote. “A numerical benefit in the proportion of patients who experienced no pain during movement at the end of treatment vs baseline was also observed.”
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rFVIIIFc is an approved, extended half-life factor VIII replacement therapy that was demonstrated to be safe and effective in 2 prior phase 3 studies. In this study, 164 patients with severe hemophilia received individualized prophylactic treatment with rFVIIIFc for a median of 28 to 32 weeks.
The participants completed the Haem-A-QoL and EuroQoL 5-dimension-3-level questionnaires at baseline and at the end of the study. The questionnaires assessed relevant symptoms of hemophilia that affected their quality of life, such as painful swellings, joint pain, pain in movement, and impairments in physical functioning.
The results showed that a significantly larger percentage of patients reported no painful swellings, joint pain, or pain on movement at the end of the study than at baseline. Furthermore, more patients reported no pain or discomfort at the end of the study than at baseline, and the proportion of patients who experienced no pain or discomfort and had 0 bleeds during the study was larger than that of patients without pain or discomfort and at least 1 bleed.
The authors emphasize the importance of pain control in the quality of life in patients with hemophilia, and the data suggest that rFVIIIFc could be a key component of effective patient management.
Pasi J, Hermans C, Hakimi Z, et al. Improvement in pain-related quality of life in patients with hemophilia A treated with rFVIIIFc individualized prophylaxis: post hoc analysis from the A-LONG study. Ther Adv Hematol. 2022;13. doi:10.1177/20406207221079482