Erica Mamauag, MD, Finds That FVIII May Be Unnecessary in Tolerized Hemophilia A

Dr. Mamauag, of the University of Pittsburgh Medical Center in Pennsylvania, spoke with Heme Today to outline her poster at the 2024 American Society of Pediatric Hematology/Oncology Conference titled, “Prophylactic FVIII Infusions May Be Unnecessary in Tolerized Hemophilia A Patients on Emicizumab.”

“In Pittsburgh, we have quite a large hemophilia population, with the Amish community just hours away. In recent years, we’ve had great success in their outcomes because of the development of emicizumab,” Dr. Mamauag explained.

She noted that factor VIII (FVIII) infusions are used to prevent bleeding in patients with hemophilia, but repeated infusions can lead to the development of inhibitors.
“The way to get around inhibitors is to give small doses of FVIII very frequently, but we don’t know if we need to continue doing that for patients who are receiving emicizumab prophylaxis,” Dr. Mamauag added.

“Our study looked at this exact question: ‘Do we need to continue giving these patients FVIII routinely to prevent the production or recurrence of FVIII inhibitors in those who have been tolerized?’” she said.

According to Dr. Mamauag, only one patient in approximately 70 had recurrence of inhibitors while undergoing treatment with emicizumab. She acknowledged that “these are small numbers and limited by the new practice of sending inhibitors on those patients on emicizumab, but it is promising for the future that these patients may not need to receive prophylactic small doses of FVIII.”