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Comparable Long-Term Survival After Unrelated and HLA-Matched Sibling Donor Hematopoietic Stem Cell Transplantations for Acute Leukemia in Children Younger Than 18 Months

From the Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin; Medical College of Wisconsin, Milwaukee, WI; New York Blood Center; Columbia University, New York, NY; Cincinnati Children's Hospital and Medical Center, Cincinnati, OH; Duke University Medical Center, Durham, NC; Utah Blood and Marrow Transplant Program, Salt Lake City, UT; University of Minnesota, Minneapolis, MN; Hospital Materno-Infantil, Barcelona, Spain; Hospital for Sick Children, Toronto, Ontario, Canada

Address reprint requests to Mary Eapen, MD, Statistical Center, Center for Blood and Marrow Transplant Research, Medical College of Wisconsin, 8701 Watertown Plank Rd, Milwaukee, WI 53226; e-mail: meapen{at}mail.mcw.edu

PURPOSE: To describe outcomes after unrelated donor stem cell transplantation (HCT) in children (< 18 months at diagnosis) with acute leukemia and compare these with outcomes after human leukocyte antigen (HLA)-matched sibling donor HCT.

PATIENTS AND METHODS: We compared the results of unrelated donor HCT with bone marrow (n = 85) or cord blood grafts (n = 81) and HLA-matched sibling donor HCT with bone marrow grafts (n = 101) for acute myeloid or acute lymphoblastic leukemia using Cox proportional hazards models. Unrelated donor HCT recipients were younger, more likely to have MLL gene rearrangement, to have advanced leukemia, and to receive irradiation before HCT.

RESULTS: Treatment-related mortality rates were 6%, 15%, and 31% after matched sibling, unrelated donor bone marrow, and cord blood HCT, respectively. Risks of relapse, overall and leukemia-free survival were significantly associated with disease status at transplantation. Though leukemia recurrence was lowest after unrelated donor HCT in first clinical remission (CR), overall survival, and leukemia-free survival rates were similar after matched sibling and unrelated donor HCT, after adjustment for disease status. Relapse, overall and leukemia-free survival did not differ by graft type (bone marrow v cord blood) or type of leukemia. Three-year probabilities of leukemia-free survival were 49% and 54% after HLA-matched sibling and unrelated donor transplantation in first CR, respectively. Corresponding rates for those with advanced leukemia were 20% and 30%.

CONCLUSION: Unrelated donor HCT should be considered for infants with acute leukemia in first CR using the same eligibility criteria as are currently used for those with HLA matched sibling donors.

Supported by Public Health Service Grant U24-CA76518 from the National Cancer Institute, the National Institute of Allergy and Infectious Diseases, and the National Heart, Lung and Blood Institute, and a Clinical Research Career Development Award from the American Society of Clinical Oncology (ME).

Authors' disclosures of potential conflicts of interest and author contributions are found at the end of this article.

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