Study Finds Safe Treatment for Children with Severe Hemophilia A with Inhibitors

Researchers have found combining immune tolerance induction (ITI) with Hemlibra (emicizumab, Roche) is a feasible and safe way of treating children with severe hemophilia A. Hemlibra is a nonfactor replacement therapy to treat hemophilia A patients with or without inhibitors; however, it does not help rid a person of factor VIII (FVIII) inhibitors against FVIII treatments that neutralize their effectiveness. ITI, in which FVIII is given regularly over a period of time until the body is trained to recognize the product without reacting against it, is the only effective strategy for removing inhibitors and restoring a normal response to FVIII replacement therapy.

In the study, seven children between 21 months and 12 years old were started on a combination of ITI with FVIII infusions three times a week plus Hemlibra, and they were followed for a median time of 35 weeks. Results showed treatment for three of the seven led to either a complete clearing of inhibitors or a drop to unmeasurable levels. Three patients (43 percent) also had no bleeding events, and nine bleeding events were reported among the other four during follow-up. No adverse effects such as blood clots were reported. Six children underwent surgery during the study with no major complications or excess bleedings during or after. Length of hospital stays also decreased, changing from an average of three days to seven days to one day to two days.

According to the researchers, their work is the first to demonstrate “immune tolerance induction while on emicizumab (Hemlibra) prophylaxis is a feasible approach in pediatric hemophilia A patients with inhibitors.”