title: Outcomes of Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults with Chronic Myeloid Leukemia: A CIBMTR Cohort Analysis.
abstract: Chronic myeloid leukemia (CML) in children and young adults is uncommon. Young patients have long life expectancies and low morbidity with hematopoietic cell transplantation (HCT). Prolonged tyrosine kinase inhibitor (TKI) use may cause significant morbidity. In addition, indication for HCT in patients in the first chronic phase is not established. We hence retrospectively evaluated outcomes in 449 CML patients with early disease receiving myeloablative HCT reported to the CIBMTR. We analyzed various factors affecting outcome, specifically the effect of age and pre-HCT TKI in pediatric patients (age < 18 years, n = 177) and young adults (age 18 to 29 years, n = 272) with the goal of identifying prognostic factors. Post-HCT probability rates of 5-year overall survival (OS) and leukemia-free survival (LFS) were 75% and 59%, respectively. Rates of OS and LFS were 76% and 57% in <18-year and 74% and 60% in 18- to 29-year group, respectively, by univariate analysis (P = .1 and = .6). Five-year rates of OS for HLA matched sibling donor (MSD) and bone marrow (BM) stem cell source were 83% and 80%, respectively. In multivariate analysis there was no effect of age (<18 versus 18 to 29) or pre-HCT TKI therapy on OS, LFS, transplant related mortality, or relapse. Favorable factors for OS were MSD (P < .001) and recent HCT (2003 to 2010; P = .04). LFS was superior with MSD (P < .001), BM as graft source (P = .001), and performance scores > 90 (P = .03) compared with unrelated or mismatched peripheral blood stem cells donors and recipients with lower performance scores. Older age was associated with increased incidence of chronic graft-versus-host disease (P = .0002). In the current era, HCT outcomes are similar in young patients and children with early CML, and best outcomes are achieved with BM grafts and MSD.
authors.affiliation: Department of Pediatrics-Hematology, Oncology and Stem Cell Transplantation, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.
authors.forename: Sonali
authors.initials: S
authors.lastname: Chaudhury
authors.affiliation: CIBMTR (Center for International Blood and Marrow Transplant Research), Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Rodney
authors.initials: R
authors.lastname: Sparapani
authors.affiliation: CIBMTR (Center for International Blood and Marrow Transplant Research), Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Zhen-Huan
authors.initials: ZH
authors.lastname: Hu
authors.affiliation: Department of Blood & Marrow Transplantation, H. Lee Moffitt Cancer Center & Research Institute, Tampa, Florida.
authors.forename: Taiga
authors.initials: T
authors.lastname: Nishihori
authors.affiliation: Division of Hematology, Oncology and Blood & Marrow Transplantation, Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, California.
authors.forename: Hisham
authors.initials: H
authors.lastname: Abdel-Azim
authors.affiliation: Bone Marrow and Stem Cell Transplantation, Icahn School of Medicine at Mount Sinai, New York, New York.
authors.forename: Adriana
authors.initials: A
authors.lastname: Malone
authors.affiliation: Division of Therapeutic Immunology, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden; Centre for Clinical Research Sormland, Uppsala University, Uppsala, Sweden.
authors.forename: Richard
authors.initials: R
authors.lastname: Olsson
authors.affiliation: CIBMTR (Center for International Blood and Marrow Transplant Research), Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin; Froedtert Memorial Lutheran Hospital, Milwaukee, Wisconsin.
authors.forename: Mehdi
authors.initials: M
authors.lastname: Hamadani
authors.affiliation: Cumming School of Medicine, Tom Baker Cancer Centre, University of Calgary, Calgary, Alberta, Canada.
authors.forename: Andrew
authors.initials: A
authors.lastname: Daly
authors.affiliation: Interdisciplinary Clinic for Stem Cell Transplantation, University Cancer Centre Hamburg, Hamburg, Germany.
authors.forename: Ulrike
authors.initials: U
authors.lastname: Bacher
authors.affiliation: Division of Bone Marrow Transplant, Seattle Cancer Care Alliance, Seattle, Washington.
authors.forename: Baldeep M
authors.initials: BM
authors.lastname: Wirk
authors.affiliation: Division of Hematology and Oncology, Center for Cell and Gene Therapy, Baylor College of Medicine Center for Cell and Gene Therapy, Houston, Texas.
authors.forename: Rammurti T
authors.initials: RT
authors.lastname: Kamble
authors.affiliation: Hematology Research Centre, Division of Experimental Medicine, Department of Medicine, Imperial College London, London, UK.
authors.forename: Robert P
authors.initials: RP
authors.lastname: Gale
authors.affiliation: Division of Hematology/Oncology, University of North Carolina, Chapel Hill, North Carolina.
authors.forename: William A
authors.initials: WA
authors.lastname: Wood
authors.affiliation: Department of Hematology/Oncology, All Children's Hospital, St. Petersburg, Florida.
authors.forename: Gregory
authors.initials: G
authors.lastname: Hale
authors.affiliation: Cancer Research Foundation of New York, Bronx, New York.
authors.forename: Peter H
authors.initials: PH
authors.lastname: Wiernik
authors.affiliation: Department of Blood and Marrow Transplantation, Mayo Clinic, Rochester, Minnesota.
authors.forename: Shahrukh K
authors.initials: SK
authors.lastname: Hashmi
authors.affiliation: Pediatric Bone Marrow Transplant, University Hospitals Bristol NHS Trust, Bristol, UK.
authors.forename: David
authors.initials: D
authors.lastname: Marks
authors.affiliation: Division of Hematology, Oncology, and Transplantation, University of Minneapolis, Minneapolis, Minnesota.
authors.forename: Celalettin
authors.initials: C
authors.lastname: Ustun
authors.affiliation: Section of Hematology/Oncology, Department of Internal Medicine, Louisiana State University Health Shreveport, Shreveport, Louisiana.
authors.forename: Reinhold
authors.initials: R
authors.lastname: Munker
authors.affiliation: Division of Hematology/Oncology, Department of Medicine, Vanderbilt University Medical Center, Nashville, Tennessee.
authors.forename: Bipin N
authors.initials: BN
authors.lastname: Savani
authors.affiliation: Division of Hematologic Malignancies and Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
authors.forename: Edwin
authors.initials: E
authors.lastname: Alyea
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Uday
authors.initials: U
authors.lastname: Popat
authors.affiliation: Blood and Marrow Transplant Program, Department of Hematology and Medical Oncology, Cleveland Clinic Taussig Cancer Institute, Cleveland, Ohio.
authors.forename: Ronald
authors.initials: R
authors.lastname: Sobecks
authors.affiliation: Blood and Marrow Transplant Program, Department of Hematology and Medical Oncology, Cleveland Clinic Taussig Cancer Institute, Cleveland, Ohio.
authors.forename: Matt
authors.initials: M
authors.lastname: Kalaycio
authors.affiliation: Adult Blood and Marrow Stem Cell Transplant Program, Knight Cancer Institute, Oregon Health and Science University, Portland, Oregon.
authors.forename: Richard
authors.initials: R
authors.lastname: Maziarz
authors.affiliation: Department of Pediatrics-Hematology, Oncology and Stem Cell Transplantation, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.
authors.forename: Nobuko
authors.initials: N
authors.lastname: Hijiya
authors.affiliation: CIBMTR (Center for International Blood and Marrow Transplant Research), Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Wael
authors.initials: W
authors.lastname: Saber
mesh_terms: D000328:Adult; D000367:Age Factors; D016026:Bone Marrow Transplantation / Q000592:standards; D002648:Child; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods / Q000592:standards*; D006801:Humans; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000628:therapy*; D008297:Male; D009017:Morbidity; D011379:Prognosis; D012189:Retrospective Studies; D035781:Siblings; D016019:Survival Analysis; D014019:Tissue Donors; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
keywords: CML; Pediatrics