Safety, efficacy of Hemlibra for Hem A confirmed in review study
Researchers found high rate of patient satisfaction in 28 published studies
Hemlibra (emicizumab-KXWH) safely reduces bleeding episodes in people with hemophilia A without inhibitors, resulting in a high rate of patient satisfaction, according to a review of published studies.
“The success of [Hemlibra] underscores the potential for innovative therapeutic approaches to significantly improve patient outcomes and quality of life in rare bleeding disorders,” researchers wrote.
The study, “Efficacy, safety and satisfaction of using emicizumab in hemophilia A patients without factor VIII inhibitors: A systematic review,” was published in Hematology, Transfusion and Cell Therapy.
Studies support efficacy of Hemlibra in reducing bleeding episodes
Hemophilia A is a genetic disease marked by a deficiency or dysfunction of the blood-clotting protein factor VIII (FVIII), which increases the risk of spontaneous or prolonged bleeding.
Replacement therapy is the standard treatment for hemophilia A, which involves supplying the patient with a lab-made version of FVIII to restore blood clotting. Up to 40% of patients, however, can develop antibodies against the therapy, called inhibitors, reducing its effectiveness. Treatment requires regular infusions into the bloodstream.
Hemlibra is an antibody-based therapy designed to mimic the action of FVIII. Given as an under-the-skin, or subcutaneous, injection, it’s approved to prevent or reduce bleeds in hemophilia A patients, with or without FVIII inhibitors.
In this report, researchers in Brazil conducted a systematic review of 28 published studies to provide an overview of the current evidence supporting Hemlibra’s ability to prevent bleeds in hemophilia A patients without inhibitors.
All studies reviewed provided evidence supporting the efficacy of Hemlibra in reducing bleeding episodes in this patient population. For example, a multicenter open-label study reported significant reductions in the annualized bleeding rate (ABR), or the number of bleeds per year, for treated bleeding events. An ABR of 1.3 was reported for patients who received biweekly Hemlibra and and an ABR of 0.7 for monthly doses.
Retrospective study shows reduction in chronic pain
These findings were corroborated by an adult retrospective study that collected data from the past and showed a significant reduction in chronic pain and improved overall health after one year of Hemlibra treatment. Patients reported a high satisfaction rate, with a mean score of 9.1 out of 10. None of the patients indicated that they would return to a previous treatment.
High treatment satisfaction scores were also found in a cross-sectional study that looked at data collected at one point in time. Most patients expressed contentment with Hemlibra’s efficacy and the reduced need for frequent infusions.
These results consistently demonstrate the superior efficacy of [Hemlibra] in preventing both overall and joint-specific bleeding episodes in pediatric populations.
A prospective study that followed a larger number of patients, from birth to 70 year old, over time reported a median ABR of 0.91. More than half (53.8%) of the participants were free of bleeding events during the observation period. Likewise, in another prospective study involving children, Hemlibra significantly reduced mean ABR from 2.41 to 1.11. There was also a decrease in joint bleeds.
“These results consistently demonstrate the superior efficacy of [Hemlibra] in preventing both overall and joint-specific bleeding episodes in pediatric populations,” the team wrote.
The reviewed studies collectively confirmed a favorable safety profile of Hemlibra. Most adverse events were minor, such as injection-site reactions, bruising, and a common cold, with no reports of blood-clotting events or the development of antibodies against Hemlibra. Similarly, adverse events were minimal in children, with no life-threatening bleeds or blood-clotting complications. One child developed anti-Hemlibra antibodies without a loss in clinical efficacy, “indicating the rarity and limited clinical impact of such occurrences,” the team wrote.
“The insights gained from this systematic review provide valuable guidance for clinicians and researchers, emphasizing the importance of continued evaluation and optimization of hemophilia A therapies,” the researchers concluded. “Future research should focus on long-term, real-world studies to monitor the sustained efficacy and safety of [Hemlibra] over extended periods.”
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