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 Munker; Bipin N. Savani; Edwin Alyea; Uday Popat; Ronald Sobecks; Matt Kalaycio; Richard Maziarz; Nobuko Hijiya; Wael Saber

doi:10.1016/j.bbmt.2016.02.015

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 journal › Article › peer-review

25 Scopus 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.

Original language	English (US)
Pages (from-to)	1056-1064
Number of pages	9
Journal	Biology of Blood and Marrow Transplantation
Volume	22
Issue number	6
DOIs	https://doi.org/10.1016/j.bbmt.2016.02.015
State	Published - Jun 1 2016

Keywords

CML
Pediatrics

ASJC Scopus subject areas

Hematology
Transplantation

Access to Document

10.1016/j.bbmt.2016.02.015

Cite this

Chaudhury, S., Sparapani, R., Hu, Z. H., Nishihori, T., Abdel-Azim, H., Malone, A., Olsson, R., Hamadani, M., Daly, A., Bacher, U., Wirk, B. M., Kamble, R. T., Gale, R. P., Wood, W. A., Hale, G., Wiernik, P. H., Hashmi, S. K., Marks, D., Ustun, C., ... 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, 22(6), 1056-1064. https://doi.org/10.1016/j.bbmt.2016.02.015

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

@article{ee6b978e449641a8a75cdd6bf90a05f3,

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.",

keywords = "CML, Pediatrics",

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

note = "Publisher Copyright: {\textcopyright} 2016 The American Society for Blood and Marrow Transplantation.",

year = "2016",

month = jun,

day = "1",

doi = "10.1016/j.bbmt.2016.02.015",

language = "English (US)",

volume = "22",

pages = "1056--1064",

journal = "Biology of Blood and Marrow Transplantation",

issn = "1083-8791",

publisher = "Elsevier Inc.",

number = "6",

}

TY - JOUR

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

Y1 - 2016/6/1

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.

AB - 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.

KW - CML

KW - Pediatrics

UR - http://www.scopus.com/inward/record.url?scp=84963595294&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84963595294&partnerID=8YFLogxK

U2 - 10.1016/j.bbmt.2016.02.015

DO - 10.1016/j.bbmt.2016.02.015

M3 - Article

C2 - 26964698

AN - SCOPUS:84963595294

SN - 1083-8791

VL - 22

SP - 1056

EP - 1064

JO - Biology of Blood and Marrow Transplantation

JF - Biology of Blood and Marrow Transplantation

IS - 6

ER -

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

Abstract

Keywords

ASJC Scopus subject areas

Access to Document

Other files and links

Fingerprint

Cite this