Allogeneic Hematopoietic Cell Transplantation for Patients with Mixed Phenotype Acute Leukemia

Reinhold Munker, Ruta Brazauskas, Hai Lin Wang, Marcos de Lima, Hanna J. Khoury, Robert Peter Gale, Richard T. Maziarz, Brenda M. Sandmaier, Daniel Weisdorf, Wael Saber

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31 Scopus citations


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

Original languageEnglish (US)
Pages (from-to)1024-1029
Number of pages6
JournalBiology of Blood and Marrow Transplantation
Issue number6
StatePublished - Jun 1 2016

Bibliographical note

Funding Information:
The CIBMTR is supported by Public Health Service grant/cooperative agreement U24-CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID) ; a Grant/Cooperative Agreement 5U10HL069294 from NHLBI and NCI ; a contract HHSH250201200016C with Health Resources and Services Administration (HRSA/DHHS) ; 2 grants N00014-13-1-0039 and N00014-14-1-0028 from the Office of Naval Research ; and grants from * Actinium Pharmaceuticals ; Allos Therapeutics, Inc. ; * Amgen, Inc. ; anonymous donation to the Medical College of Wisconsin ; Ariad ; Be the Match Foundation ; * Blue Cross and Blue Shield Association ; * Celgene Corporation ; Chimerix, Inc. ; Fred Hutchinson Cancer Research Center ; Fresenius-Biotech North America, Inc. ; * Gamida Cell Teva Joint Venture Ltd. ; Genentech, Inc. ; * Gentium SpA ; Genzyme Corporation ; GlaxoSmithKline ; Health Research, Inc. ; Roswell Park Cancer Institute ; HistoGenetics, Inc. ; Incyte Corporation ; Jeff Gordon Children's Foundation ; Kiadis Pharma ; The Leukemia & Lymphoma Society ; Medac GmbH ; The Medical College of Wisconsin ; Merck & Co, Inc. ; Millennium: The Takeda Oncology Co. ; * Milliman USA, Inc. ; * Miltenyi Biotec, Inc. ; National Marrow Donor Program ; Onyx Pharmaceuticals ; Optum Healthcare Solutions, Inc. ; Osiris Therapeutics, Inc. ; Otsuka America Pharmaceutical, Inc. ; Perkin Elmer, Inc. ; * Remedy Informatics ; * Sanofi US ; Seattle Genetics ; Sigma-Tau Pharmaceuticals ; Soligenix, Inc. ; St. Baldrick's Foundation ; StemCyte , A Global Cord Blood Therapeutics Co. ; Stemsoft Software, Inc. ; Swedish Orphan Biovitrum ; * Tarix Pharmaceuticals ; * TerumoBCT ; * Teva Neuroscience, Inc. ; * THERAKOS, Inc. ; University of Minnesota ; University of Utah ; and * Wellpoint, Inc. . The views expressed in this article do not reflect the official policy or position of the National Institute of Health, the Department of the Navy, the Department of Defense, Health Resources and Services Administration or any other agency of the US Government.

Publisher Copyright:
© 2016 The American Society for Blood and Marrow Transplantation.


  • Acute biphenotypic leukemia
  • Allogeneic transplantation
  • Mixed phenotype leukemia


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