Choosing the Optimal Treatment for Hemophilia A

Recombinant Factor VIII, the preferred treatment for hemophilia A, was found less effective than Factor VIII derived from human plasma with von Willebrand factor in hemophilia patients.

Since the 1980s, recombinant factor VIII, which is derived from a hamster cell line, has become the commonly used treatment partly because of a large amount of patients who died after a blood supply was tainted with hepatitis C virus (HCV) and HIV.

“Patients and families have been afraid of plasma-derived products even though they have been safe for decades,” said researcher Mindy Simpson.

In fact, there has been no transmitted cases of HCV or HIV in hemophilia factor products since the 1990s.

A study titled the Survey of Inhibitors in Plasma-Products Exposed Toddlers (SIPPET) is the first randomized, controlled study to examine the associations between inhibitors and the 2 first-line treatments for hemophilia A.

“Who is developing inhibitors, and why?” Simpson said. “This study is the first to try to sort out the answer to those questions regarding the factor treatment options.”

SIPPET enrolled 251 previously untreated young children from 42 centers in 14 countries on 5 continents between 2010 and 2015. The results of the study revealed that Recombinant Factor VIII was associated with an 87% higher likelihood of a patient developing inhibitors.

Inhibitors occur in about 30% of patients and is a major issue in managing hemophilia A, the authors noted. Recombinant Factor VIII becomes ineffective once inhibitors appear in high titers, requiring the patient to move onto more expensive and less effective treatments.

“Families will want to have a discussion with their physicians about how this study might impact the treatment options," Dr. Simpson said.