Pediatric Blood & Marrow Transplantation Center
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Margaret MacMillan, M.D., M.Sc.

Professor, Division of Pediatric Blood and Marrow Transplantation

Dr. MacMillan

“I’m part of an extraordinary team who is dedicated to providing exceptional care to patients and their families.

We have the optimal research environment allowing innovative BMT therapies to be brought directly from the lab to patients, giving them the best chance for a cure."

Dr. MacMillan’s research focuses on developing new therapeutic strategies to reduce the early side effects of blood and marrow transplantation. She is an expert in the diagnosis, prevention and treatment of acute graft-versus-host disease (GVHD) and a leader in clinical trial design to study novel agents for GVHD. Dr. MacMillan is an international known expert in hematopoietic cell transplantation for patients with Fanconi anemia. She is the co-director of the University of Minnesota Fanconi Anemia Comprehensive Care Program, which follows the largest number of Fanconi anemia patients in the world. Dr. MacMillan is also the Clinical Medical Director of the Pediatric Blood and Marrow Transplant Program at the University of Minnesota. She serves on many national committees including the American Society of Blood and Marrow Transplantation where she is the director-at-large.


Medical School: University of Toronto
Masters of Science: University of Toronto
Residency: University of Toronto (Pediatrics)
Fellowships: University of Toronto School of Medicine (Hematology/Oncology and Blood and Marrow Transplantation); University of Minnesota (Blood and Marrow Transplantation)

Clinical & Research Interests

Dr. MacMillan's research focuses on the development and implementation of novel therapeutic strategies for preventing the early toxicities of allogeneic hematopoietic stem cell transplantation. She is the principal investigator of many Phase I and II clinical trials in the field of blood and marrow transplantation. Along with a team of basic scientists and cellular therapy experts at the University of Minnesota, she is studying the safety and efficacy of inducible T regulatory cells (iTregs) to prevent and treat graft-versus-host disease. Dr. MacMillan is the principal investigator of the world’s first clinical trial to prevent GVHD with iTregs. Dr. MacMillan, along with other members of the care team, has devised novel approaches to transplanting patients with Fanconi anemia, greatly improving the outcomes for these patients.

Publications and Awards

A Selection of Recent Publications and Awards


Graft versus Host Disease: From Basics to the Clinic. Guinan EC, MacMillan ML. In: Smith FO, Reaman G, Racadio J. (Eds) Hematopoietic Cell Transplantation in Children with Cancer. Spring 2014.

Haematopoietic cell transplantation for acute leukaemia and advanced myelodysplastic syndrome in Fanconi anaemia. Mitchell R, Wagner JE, Hirsch B, DeFor TE, Zierhut H, MacMillan ML. Br J Haematol. 2014 Feb.

Analysis of risk factors influencing outcomes after cord blood transplantation in children with juvenile myelomonocytic leukemia: a EUROCORD, EBMT, EWOG-MDS, CIBMTR study. Locatelli F, Crotta A, Ruggeri A, Eapen M, Wagner JE, Macmillan ML, Zecca M, Kurtzberg J, Bonfim C, Vora A, Díaz de Heredia C, Teague L, Stein J, O'Brien TA, Bittencourt H, Madureira A, Strahm B, Peters C, Niemeyer C, Gluckman E, Rocha V. Blood. 2013 Sep.

What predicts high risk acute graft-versus-host disease (GVHD) at onset?: identification of those at highest risk by a novel acute GVHD risk score. MacMillan ML, DeFor TE, Weisdorf DJ. Br J Haematol. 2012 Jun

Cellular therapy for Fanconi anemia: the past, present, and future. MacMillan ML, Hughes MR, Agarwal S, Daley GQ. Biol Blood Marrow Transplant. 2011 Jan

The best endpoint for acute GVHD treatment trials. MacMillan ML, DeFor TE, Weisdorf DJ. Blood. 2010 Jul

Management of acute graft-versus-host disease in children. Carpenter PA, MacMillan ML. Pediatr Clin North Am. 2010 Feb

Haematopoeitic cell transplantation for Fanconi anaemia - when and how? MacMillan ML, Wagner JE. Br J Haematol. 2010 Apr

Transplant outcomes in bone marrow failure syndromes and hemoglobinopathies. MacMillan ML, Walters MC, Gluckman E. Semin Hematol. 2010 Jan

Diagnosis of myelodysplastic syndrome among a cohort of 119 patients with Fanconi anemia: morphologic and cytogenetic characteristics. Cioc AM, Wagner JE, MacMillan ML, DeFor T, Hirsch B. Am J Clin Pathol. 2010 Jan

Endpoints for clinical trials testing treatment of acute graft-versus-host disease: a joint statement. Martin PJ, Bachier CR, Klingemann HG, McCarthy PL, Szabolcs P, Uberti JP, Schuster MW, Weisdorf D, Chao NJ, Kebriaei P, Shpall EJ, Macmillan ML, Soiffer RJ. Biol Blood Marrow Transplant. 2009 Jul

Where is the start line? MacMillan ML. Blood. 2009 Mar

Acute graft-versus-host disease after unrelated donor umbilical cord blood transplantation: analysis of risk factors. MacMillan ML, Weisdorf DJ, Brunstein CG, Cao Q, DeFor TE, Verneris MR, Blazar BR, Wagner JE.
Blood. 2009 Mar

Click here to see more of Dr. MacMillan’s work which has been published by academic and research journals.


Best Doctors in America (2008-2013)
Best Abstracts Award, Center for International Blood and Marrow Transplant Research (2009)

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Journey Clinic - Pediatric Blood and Marrow Transplantation Center

2450 Riverside Avenue
Minneapolis, MN 55454
612-365-1000 TTY: 612-672-7300

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