Novel Hemophilia A Treatment Method Sees Early Success
New approach involves treating patients with a high dose factor VIII concentrates along with emicizumab, a modification of the standard immune tolerance induction.
A new method of treatment for patients with hemophilia A, known as Atlanta Protocol, has shown early success at the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta. The method includes high dose factor VIII concentrates, along with emicizumab, a modification of the standard immune tolerance induction (ITI), according to a press release from Emory University.
The study, published in Haemophilia, included 7 of the total 200 patients with hemophilia A in the Children’s Healthcare center, of whom approximately 30% with severe disease typically develop inhibitors. The participants, ranging from 21 months to 12 years of age, were started on the novel combination of ITI with FVIII infusions 3 times per week and emicizumab. They were followed for an average of 35 weeks.
The researchers found that 3 of the 7 patients were completely rid of the inhibitor or the inhibitor was reduced to an unmeasurable level. There were minimal bleeding events and no adverse events, such as blood clots, according to the release. Six of the patients underwent surgery during this time and hospital stays decreased from the average stay of 3-7 days to 1-2 days.
Hemophilia is a rare bleeding and clotting disorder affecting 1 in 5000 males, and approximately 20,000 people in the United States have hemophilia A or B. Approximately one-third of patients with severe hemophilia A will develop antibodies directed against FVIII, called inhibitors, limiting the ability to treat hemophilia. For these patients, the immune system produces antibodies that inhibit clot formation by destroying the clotting factor before it has a chance to stop or prevent bleeding. Inhibitors make treating bleeds more difficult and do not allow for the standard treatment for preventing bleeds with replacement FVIII concentrates.
The study authors noted that the findings are valuable, because they demonstrate that ITI is safe and feasible when in combination with emicizumab. As a result of this study, 2 clinical trials will launch in fall 2019 to address safety and efficacy using an observational and prospective study design, according to the release.
The Atlanta Protocol will be offered to all hemophilia patients at the Aflac Cancer and Blood Disorders Center and will be shared with other pediatric hospitals and institutions across the United States.
- Pediatric hematologists introduce a novel way to treat pediatric hemophilia A patients [press release]. Korschun, Holly. Medical Express website. Published August 29, 2019. https://medicalxpress.com/news/2019-08-pediatric-hematologists-hemophilia-patients.html. Accessed September 4, 2019.