Emicizumab Demonstrates Efficacy in Treatment of Hemophilia

Emicizumab (Hemlibra; Genentech) prophylaxis has been associated with improved quality of life (QOL) and decreases in bleeding episodes in patients with hemophilia A, according a study recently published in Clinical and Applied Thrombosis and Haemostasis.

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Hemophilia A, a rare, X-linked, recessive bleeding disorder, is also known as factor VIII (FVIII) deficiency. Patients lacking FVIII can experience a plethora of dangerous bleeding episodes—such as intracranial hemorrhaging, bleeding of the joints and gastrointestinal tract, and more—and the severity of these events can be affected by a patient’s plasma levels. A common treatment for FVIII deficiency comes in the form of FVIII replacement as prophylaxis, which is performed intravenously several times a week. However, this approach is not always effective, as some patients still experience uncontrolled bleeding and require a bypassing agent.

Emicizumab is a humanized recombinant bispecific monoclonal antibody. It was created as a potential frontline treatment option with or without inhibitors for patients with hemophilia A and works to repair the function of missing activated FVIII regardless of a patient’s levels of FVIII. The aim of emicizumab treatment is to elude the complications and inefficacies that can arise from intravenous therapy with FVIII.

Prophylaxis can provide multiple benefits for patients, such as improved QOL and reductions in joint bleeding, discomfort, and pain; however, this treatment can be costly and is unavailable in some countries. The current study sought to investigate the efficacy of emicizumab in the resource-limited country, Pakistan, which receives emicizumab prophylaxis donated by the World Federation of Hemophilia. The assessment evaluated outcomes related to QOL, safety, and bleeding in patients with hemophilia A with and without inhibitors.

Data were gathered prospectively from patients with hemophilia A from January 2022 until June 2023 through the Hemophilia Welfare Society Karachi, Pakistan. Male patients with severe hemophilia A who had prior histories of intracranial bleeding, intra-abdominal bleeding, pseudo tumors, and an annual bleeding rate of 8 or above were included. Dosing of emicizumab began at 3 mg/kg in the initial 4 weeks and then transitioned to a maintenance dose of 6 mg/kg/month thereafter. Every 3 months, the Hemophilia Joint Health Score (HJHS), Functional Independence Score in Hemophilia (FISH), and the EuroQol five-dimensional questionnaire (EQ-5D-5L), which assesses self-care, mobility, anxiety/depression, pain/discomfort, and usual activities, were utilized to evaluate patients’ joints and QOL.

Thirty-six male patients with hemophilia A were included, 19 of whom were inhibitor positive and 17, inhibitor negative. Following treatment initiation, joint evaluations revealed a significant reduction in average FISH and HJHS scores (pre- vs post treatment: FISH, 16 vs 9; HJHS, 10 vs 4; P < .057). Improvements were seen in this realm after every 3 months of emicizumab.

Results from patients’ EQ-5D-5L index scores reported a 63.3% improvement in mobility (P = .00), 66.6% in self-care (P = .009), and 70% in patients’ abilities to handle their daily activities (P = .00). Additionally, researchers witnessed a significant 58.6% improvement in patients’ experiences with anxiety/depression (P = .00).

"This improvement indicated a significant reduction in joint hemorrhages and an associated increase in patients’ joint mobility,” Manira Borhany, MD, and lead investigator wrote. “Importantly, this outcome was statistically significant (P < .05), reinforcing the therapeutic benefits of emicizumab on joint health. The positive impact on both quality-of-life domains and joints health underscores the significance of this therapeutic intervention for enhancing the well-being and clinical outcomes of patients with hemophilia A.”

Reference

Borhany M, Arshad A, Qureshi H, Nadeem R, Jamal A, Khan RA. Emicizumab prophylaxis in patients with severe hemophilia A: Insights from a resource limited country. Clin Appl Thromb Hemost. 2024;30:10760296231224357. doi:10.1177/10760296231224357