Outcomes of Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults with Chronic Myeloid Leukemia: A CIBMTR Cohort Analysis

Sonali Chaudhury, Rodney Sparapani, Zhen Huan Hu, Taiga Nishihori, Hisham Abdel-Azim, Adriana Malone, Richard Olsson, Mehdi Hamadani, Andrew Daly, Ulrike Bacher, Baldeep M. Wirk, Rammurti T. Kamble, Robert P. Gale, William A. Wood, Gregory Hale, Peter H. Wiernik, Shahrukh K. Hashmi, David Marks, Celalettin Ustun, Reinhold MunkerBipin N. Savani, Edwin Alyea, Uday Popat, Ronald Sobecks, Matt Kalaycio, Richard Maziarz, Nobuko Hijiya, Wael Saber

Research output: Contribution to journalArticle

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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.

Original languageEnglish (US)
Pages (from-to)1056-1064
Number of pages9
JournalBiology of Blood and Marrow Transplantation
Volume22
Issue number6
DOIs
StatePublished - Jun 1 2016

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Cell Transplantation
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Young Adult
Cohort Studies
Protein-Tyrosine Kinases
Morbidity
Graft vs Host Disease
Life Expectancy
Tissue Donors
Pediatrics
Transplants
Incidence

Keywords

  • CML
  • Pediatrics

Cite this

Outcomes of Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults with Chronic Myeloid Leukemia : A CIBMTR Cohort Analysis. / Chaudhury, Sonali; Sparapani, Rodney; Hu, Zhen Huan; Nishihori, Taiga; Abdel-Azim, Hisham; Malone, Adriana; Olsson, Richard; Hamadani, Mehdi; Daly, Andrew; Bacher, Ulrike; Wirk, Baldeep M.; Kamble, Rammurti T.; Gale, Robert P.; Wood, William A.; Hale, Gregory; Wiernik, Peter H.; Hashmi, Shahrukh K.; Marks, David; Ustun, Celalettin; Munker, Reinhold; Savani, Bipin N.; Alyea, Edwin; Popat, Uday; Sobecks, Ronald; Kalaycio, Matt; Maziarz, Richard; Hijiya, Nobuko; Saber, Wael.

In: Biology of Blood and Marrow Transplantation, Vol. 22, No. 6, 01.06.2016, p. 1056-1064.

Research output: Contribution to journalArticle

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 2016, 'Outcomes of Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults with Chronic Myeloid Leukemia: A CIBMTR Cohort Analysis', Biology of Blood and Marrow Transplantation, vol. 22, no. 6, pp. 1056-1064. https://doi.org/10.1016/j.bbmt.2016.02.015
Chaudhury, Sonali ; Sparapani, Rodney ; Hu, Zhen Huan ; Nishihori, Taiga ; Abdel-Azim, Hisham ; Malone, Adriana ; Olsson, Richard ; Hamadani, Mehdi ; Daly, Andrew ; Bacher, Ulrike ; Wirk, Baldeep M. ; Kamble, Rammurti T. ; Gale, Robert P. ; Wood, William A. ; Hale, Gregory ; Wiernik, Peter H. ; Hashmi, Shahrukh K. ; Marks, David ; Ustun, Celalettin ; Munker, Reinhold ; Savani, Bipin N. ; Alyea, Edwin ; Popat, Uday ; Sobecks, Ronald ; Kalaycio, Matt ; Maziarz, Richard ; Hijiya, Nobuko ; Saber, Wael. / Outcomes of Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults with Chronic Myeloid Leukemia : A CIBMTR Cohort Analysis. In: Biology of Blood and Marrow Transplantation. 2016 ; Vol. 22, No. 6. pp. 1056-1064.
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T1 - Outcomes of Allogeneic Hematopoietic Cell Transplantation in Children and Young Adults with Chronic Myeloid Leukemia

T2 - A CIBMTR Cohort Analysis

AU - Chaudhury, Sonali

AU - Sparapani, Rodney

AU - Hu, Zhen Huan

AU - Nishihori, Taiga

AU - Abdel-Azim, Hisham

AU - Malone, Adriana

AU - Olsson, Richard

AU - Hamadani, Mehdi

AU - Daly, Andrew

AU - Bacher, Ulrike

AU - Wirk, Baldeep M.

AU - Kamble, Rammurti T.

AU - Gale, Robert P.

AU - Wood, William A.

AU - Hale, Gregory

AU - Wiernik, Peter H.

AU - Hashmi, Shahrukh K.

AU - Marks, David

AU - Ustun, Celalettin

AU - Munker, Reinhold

AU - Savani, Bipin N.

AU - Alyea, Edwin

AU - Popat, Uday

AU - Sobecks, Ronald

AU - Kalaycio, Matt

AU - Maziarz, Richard

AU - Hijiya, Nobuko

AU - Saber, Wael

PY - 2016/6/1

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N2 - 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.

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