Hematopoietic Stem Cell Transplantation to Eliminate a Factor VIII Inhibitor

In a patient with hemophilia A, HSCT failed to block the immune response that led to development of factor VIII inhibitor.

As many as one third of patients with hemophilia become resistant to the hemostatic benefits of factor VIII therapy when they develop inhibitors to factor VIII-containing products. Frequent administration of factor VIII concentrates to induce immune tolerance in such patients often reduces inhibitor titers and reestablishes responsiveness to factor replacement therapy. However, patients who fail to achieve immune tolerance have few other options.

U.K. investigators now report a novel approach to this problem: hematopoietic stem cell transplantation (HSCT). In a 22-year-old male with severe hemophilia A (who was first discovered at age 2 to produce factor VIII inhibitor), multiple regimens — including several attempts at tolerance induction a…

Reviewing Author

David Green, MD, PhD

Disclosures

EquityNovartis

David Green, MD, PhD

Disclosures

EquityNovartis