Allogeneic peripheral blood stem cell transplantation for active hemophagocytic lymphohistiocytosis

Hemophagocytic lymphohistiocytosis (HLH) is a lethal disorder of immune dysregulation. Successful allogeneic BMT is the only therapy that has produced long-term disease-free survival. However, for patients with active HLH the outcome of BMT remains poor when compared with those transplanted in remission. We describe a patient with active, recurrent HLH treated with high-dose chemotherapy and related allogeneic peripheral blood stem cell (PBSC) transplantation. Hematologic recovery was rapid and the post-transplant course was uncomplicated. The patient is alive and well 21/2 years after transplant. Allogeneic PBSC transplantation may deserve further evaluation as an alternative source of stem cells for transplantation in patients with active HLH.