Hemlibra outperforms ITI in controlling bleeding in hemophilia A children
Using approved therapy for prevention also shown to improve life quality
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- Treatment with Hemlibra for severe hemophilia A in children with inhibitors offers better bleeding control than immune tolerance induction, a new study demonstrates.
- Hemlibra use led to improved quality of life and lower medication costs compared with ITI.
- The researchers noted, however, that Hemlibra does not eradicate inhibitors, unlike ITI.
Preventive treatment with Hemlibra (emicizumab) is associated with better bleeding control and lower medication costs compared with immune tolerance induction, or ITI, among children with severe hemophilia A with inhibitors, a new study in China found.
The researchers noted that use of the injection therapy Hemlibra also led to improved quality of life among the children.
Both ITI and Hemlibra are among a number of treatment strategies widely used in hemophilia A. ITI treatment aims to reeducate the immune system to prevent the formation of inhibitors, neutralizing antibodies that target blood clotting factors in the body. Hemlibra, for its part, mimics the action of FVIII — the blood clotting protein that’s missing in people with hemophilia A — but does not eliminate inhibitors.
“[Hemlibra] prophylaxis showed favorable outcomes regarding bleeding control, [quality of life] enhancement, and medication cost compared to ITI,” the researchers wrote. “These findings suggest that [Hemlibra] prophylaxis is a valuable therapeutic option, particularly for patients prioritizing immediate bleeding control over inhibitor eradication, or in settings where intensive ITI protocols are not feasible.”
The study, “Emicizumab Prophylaxis versus Immune Tolerance Induction in Children with Severe Hemophilia A and Inhibitors: A Retrospective Comparison of Bleeding Control, Quality of Life, and Cost,” was published in the journal Research and Practice in Thrombosis and Haemostasis.
Hemophilia A is caused by mutations in the F8 gene that lead to a deficiency of the blood-clotting factor FVIII. Without enough working FVIII, blood doesn’t clot properly, increasing the risk of symptoms that include excessive and prolonged bleeding.
Over 80% of children treated with ITI
Standard hemophilia treatment involves preventive (prophylactic) factor replacement therapy, in which a functional form of FVIII is infused regularly into the bloodstream to prevent bleeding. However, some patients develop inhibitors, or neutralizing antibodies against the provided clotting factor, which can stop these therapies from working as effectively.
For patients with inhibitors, treatment may involve frequent high-dose FVIII to essentially reeducate the immune system and eradicate inhibitors. Hemlibra, an antibody-based therapy that mimics FVIII activity, is approved for patients with hemophilia A regardless of inhibitor status.
There is limited evidence, however, comparing Hemlibra and immune tolerance induction in children with severe hemophilia A with inhibitors.
To learn more, researchers conducted a retrospective study involving 140 children with hemophilia A who received either Hemlibra or ITI at Beijing Children’s Hospital between 2020 and 2024. The children’s median age was 6.3.
Most children were treated with ITI (82.9%), at a medium (100 U/kg daily) or low dose (50 IU/kg every other day), for a median of 2.4 years. The remaining children were treated with Hemlibra for a median of 3.7 years, at a median loading dose of 2.3 mg/kg weekly, for the first four weeks, and a maintenance dose of 5.4 mg/kg monthly. Among these children, five had previously received ITI.
The children had similar levels of inhibitors before and after starting treatment with either ITI or Hemlibr. They also had similar total and joint annualized bleeding rates, or the projected number of bleeding episodes over one year.
Bleeding rates dropped from over 35% to 0% with Hemlibra
Overall, Hemlibra prophylaxis was generally associated with no bleeding events at all study phases, from initial treatment response through up to three years of treatment. This was significantly lower than that of children treated with ITI, the researchers noted.
Within the ITI group, those receiving a medium dose showed significantly better bleeding control than those treated with a low dose (median of up to 2.1 vs. up to 10.6 bleeding events), the data showed.
With prolonged treatment, bleeding rates in the ITI group progressively declined, and the therapeutic advantage of Hemlibra over ITI reduced, according to the researchers.
Both treatments significantly reduced the proportion of target joints, defined as problematic joints with chronic bleeding. Rates declined from 23.7% to 9.2% with low-dose ITI, and from 35% to 7.5% with medium-dose ITI. With Hemlibra, the rates dropped from 35.7% to 0%.
Overall, the proportion of patients with no bleeding events significantly increased in the Hemlibra group (7.2% at baseline vs. 85.7% after treatment) and in the medium-dose ITI group (22.5% vs. 47.5%). A similar trend was seen in the low-dose ITI group, but it was not statistically significant, per the researchers.
Significantly lower treatment costs seen with Hemlibra
Although all treatments also increased parents’ and children’s reported quality of life, the effects were significantly greater for children treated with Hemlibra than those receiving ITI, according to the researchers. Hemlibra was also associated with a significantly lower injection frequency, the team noted.
The study also showed lower treatment costs from treatment initiation to treatment response with Hemlibra.
“Our findings demonstrate that [Hemlibra] prophylaxis is associated with improved bleeding control … greater [quality of life] improvement … and significantly lower medication cost compared to ITI regimens, when evaluated without considering inhibitor eradication as an endpoint,” the researchers wrote. However, the team noted that Hemlibra’s inability to eliminate inhibitors remains a treatment limitation.
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