Faculty Collaboration Database

Outcomes of Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults with Chronic Myeloid Leukemia: A CIBMTR Cohort Analysis. Biol Blood Marrow Transplant 2016 Jun;22(6):1056-1064

Date

03/12/2016

Pubmed ID

26964698

Pubmed Central ID

PMC4877686

DOI

10.1016/j.bbmt.2016.02.015

Scopus ID

2-s2.0-84963595294 (requires institutional sign-in at Scopus site)   25 Citations

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.

Author List

Chaudhury S, Sparapani R, Hu ZH, Nishihori T, Abdel-Azim H, Malone A, Olsson R, Hamadani M, Daly A, Bacher U, Wirk BM, Kamble RT, Gale RP, Wood WA, Hale G, Wiernik PH, Hashmi SK, Marks D, Ustun C, Munker R, Savani BN, Alyea E, Popat U, Sobecks R, Kalaycio M, Maziarz R, Hijiya N, Saber W

Authors

Mehdi H. Hamadani MD Professor in the Medicine department at Medical College of Wisconsin
Wael Saber MD, MS Professor in the Medicine department at Medical College of Wisconsin
Rodney Sparapani PhD Associate Professor in the Institute for Health and Equity department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adult
Age Factors
Bone Marrow Transplantation
Child
Graft vs Host Disease
Hematopoietic Stem Cell Transplantation
Humans
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Male
Morbidity
Prognosis
Retrospective Studies
Siblings
Survival Analysis
Tissue Donors
Transplantation, Homologous
Treatment Outcome
Young Adult