Allogeneic hematopoietic cell transplantation for patients with mixed phenotype acute leukemia.

Publication Type:

Journal Article


Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (2016)


Acute biphenotypic leukemia or mixed phenotype acute leukemia (MPAL) is rare and considered high-risk. The optimal treatment and the role of allogeneic hematopoietic stem cell transplant (alloHCT) are unclear. Most prior case series include only modest numbers of transplanted patients. We analyzed the outcome of 95 carefully characterized alloHCT patients with MPAL reported to the Center for International Blood and Marrow Transplant Research (CIBMTR) between 1996 and 2012. The median age was 20 years (range 1- 68). Among the 95 patients, 78 were in first complete remission (CR1) and 17 in CR2. Three-year overall survival (OS) 67% (95% confidence interval [CI] 57-76), leukemia-free survival (LFS) 56% (95% CI 46-66), relapse incidence 29% (95% CI 20-38) and non-relapse mortality 15% (95% CI 9-23) were encouraging. OS was best in younger patients (< 20), but no significant differences were observed between those 20-40 versus and 40 years. A matched-pair analysis showed similar outcomes comparing MPAL cases to 375 acute myelogenous leukemia (AML) or 359 acute lymphoblastic leukemia (ALL) cases. MPAL patients had more acute and a trend for more chronic graft-versus host disease (GVHD). No difference was observed between patients transplanted in CR1 versus CR2. AlloHCT is a promising treatment option for pediatric and adult patients with MPAL with encouraging long-term survival.