Personalized Prognostic Risk Score for Long-Term Survival for Children with Acute Leukemia after Allogeneic Transplantation

Menachem Bitan; Kwang Woo Ahn; Heather R. Millard; Michael A. Pulsipher; Hisham Abdel-Azim; Jeffery J. Auletta; Valerie Brown; Ka Wah Chan; Miguel Angel Diaz; Andrew Dietz; Marta González Vincent; Gregory Guilcher; Gregory A. Hale; Robert J. Hayashi; Amy Keating; Parinda Mehta; Kasiani Myers; Kristin Page; Tim Prestidge; Nirali N. Shah; Angela R. Smith; Ann Woolfrey; Elizabeth Thiel; Stella M. Davies; Mary Eapen

doi:10.1016/j.bbmt.2017.05.011

Personalized Prognostic Risk Score for Long-Term Survival for Children with Acute Leukemia after Allogeneic Transplantation

Menachem Bitan, Kwang Woo Ahn, Heather R. Millard, Michael A. Pulsipher, Hisham Abdel-Azim, Jeffery J. Auletta, Valerie Brown, Ka Wah Chan, Miguel Angel Diaz, Andrew Dietz, Marta González Vincent, Gregory Guilcher, Gregory A. Hale, Robert J. Hayashi, Amy Keating, Parinda Mehta, Kasiani Myers, Kristin Page, Tim Prestidge, Nirali N. ShahAngela R. Smith, Ann Woolfrey, Elizabeth Thiel, Stella M. Davies, Mary Eapen

Pediatric Blood & Marrow Transplant & Cellular Therapy

Research output: Contribution to journal › Article › peer-review

12 Scopus citations

Abstract

We studied leukemia-free (LFS) and overall survival (OS) in children with acute myeloid (AML, n = 790) and acute lymphoblastic leukemia (ALL, n = 1096) who underwent transplantation between 2000 and 2010 and who survived for at least 1 year in remission after related or unrelated donor transplantation. Analysis of patient-, disease-, and transplantation characteristics and acute and chronic graft-versus-host disease (GVHD) was performed to identify factors with adverse effects on LFS and OS. These data were used to develop risk scores for survival. We did not identify any prognostic factors beyond 4 years after transplantation for AML and beyond 3 years for ALL. Risk score for survival for AML includes age, disease status at transplantation, cytogenetic risk group, and chronic GVHD. For ALL, the risk score includes age at transplantation and chronic GVHD. The 10-year probabilities of OS for AML with good (score 0, 1, or 2), intermediate (score 3), and poor risk (score 4, 5, 6, or 7) were 94%, 87%, and 68%, respectively. The 10-year probabilities of OS for ALL were 89% and 80% for good (score 0 or 1) and poor risk (score 2), respectively. Identifying children at risk for late mortality with early intervention may mitigate some excess late mortality.

Original language	English (US)
Pages (from-to)	1523-1530
Number of pages	8
Journal	Biology of Blood and Marrow Transplantation
Volume	23
Issue number	9
DOIs	https://doi.org/10.1016/j.bbmt.2017.05.011
State	Published - Sep 2017

Bibliographical note

Funding Information:
Financial disclosure: The CIBMTR is supported primarily by Public Health Service Grant/Cooperative Agreement 5U24-CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID); a grant/cooperative agreement 5U10HL069294 from NHLBI and NCI; a contract HHSH250201200016C with Health Resources and Services Administration (HRSA/DHHS); grants N00014-15-1-0848 and N00014-16-1-2020 from the Office of Naval Research. Other support to the CIBMTR include: Actinium Pharmaceuticals, Inc.; Alexion; Amgen, Inc.; anonymous donation to the Medical College of Wisconsin; Astellas Pharma US; AstraZeneca; Atara Biotherapeutics, Inc.; Be the Match Foundation; Bluebird Bio, Inc.; Bristol Myers Squibb Oncology; Celgene Corporation; Cellular Dynamics International, Inc.; Cerus Corporation; Chimerix, Inc.; Fred Hutchinson Cancer Research Center; Gamida Cell Ltd.; Genentech, Inc.; Genzyme Corporation; Gilead Sciences, Inc.; Health Research, Inc. Roswell Park Cancer Institute; HistoGenetics, Inc.; Incyte Corporation; Janssen Scientific Affairs, LLC; Jazz Pharmaceuticals, Inc.; Jeff Gordon Children's Foundation; Medac, GmbH; MedImmune; The Medical College of Wisconsin; Merck & Co, Inc.; Mesoblast; MesoScale Diagnostics, Inc.; Miltenyi Biotec, Inc.; National Marrow Donor Program; Neovii Biotech NA, Inc.; Novartis Pharmaceuticals Corporation; Onyx Pharmaceuticals; Optum Healthcare Solutions, Inc.; Otsuka America Pharmaceutical, Inc.; Otsuka Pharmaceutical Co, Ltd. – Japan; PCORI; Perkin Elmer, Inc.; Pfizer, Inc.; Sanofi US; Seattle Genetics; Spectrum Pharmaceuticals, Inc.; St. Baldrick's Foundation; Sunesis Pharmaceuticals, Inc.; Swedish Orphan Biovitrum, Inc.; Takeda Oncology; Telomere Diagnostics, Inc.; University of Minnesota; and Wellpoint, Inc.

Publisher Copyright:
© 2017 The American Society for Blood and Marrow Transplantation

Keywords

Acute lymphoblastic leukemia
Acute myeloid leukemia
Risk score
Survival
Transplantation

Publisher link

10.1016/j.bbmt.2017.05.011

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5683075

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Bitan, M., Ahn, K. W., Millard, H. R., Pulsipher, M. A., Abdel-Azim, H., Auletta, J. J., Brown, V., Chan, K. W., Diaz, M. A., Dietz, A., Vincent, M. G., Guilcher, G., Hale, G. A., Hayashi, R. J., Keating, A., Mehta, P., Myers, K., Page, K., Prestidge, T., ... Eapen, M. (2017). Personalized Prognostic Risk Score for Long-Term Survival for Children with Acute Leukemia after Allogeneic Transplantation. Biology of Blood and Marrow Transplantation, 23(9), 1523-1530. https://doi.org/10.1016/j.bbmt.2017.05.011

Bitan, M, Ahn, KW, Millard, HR, Pulsipher, MA, Abdel-Azim, H, Auletta, JJ, Brown, V, Chan, KW, Diaz, MA, Dietz, A, Vincent, MG, Guilcher, G, Hale, GA, Hayashi, RJ, Keating, A, Mehta, P, Myers, K, Page, K, Prestidge, T, Shah, NN, Smith, AR, Woolfrey, A, Thiel, E, Davies, SM & Eapen, M 2017, 'Personalized Prognostic Risk Score for Long-Term Survival for Children with Acute Leukemia after Allogeneic Transplantation', Biology of Blood and Marrow Transplantation, vol. 23, no. 9, pp. 1523-1530. https://doi.org/10.1016/j.bbmt.2017.05.011

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title = "Personalized Prognostic Risk Score for Long-Term Survival for Children with Acute Leukemia after Allogeneic Transplantation",

abstract = "We studied leukemia-free (LFS) and overall survival (OS) in children with acute myeloid (AML, n = 790) and acute lymphoblastic leukemia (ALL, n = 1096) who underwent transplantation between 2000 and 2010 and who survived for at least 1 year in remission after related or unrelated donor transplantation. Analysis of patient-, disease-, and transplantation characteristics and acute and chronic graft-versus-host disease (GVHD) was performed to identify factors with adverse effects on LFS and OS. These data were used to develop risk scores for survival. We did not identify any prognostic factors beyond 4 years after transplantation for AML and beyond 3 years for ALL. Risk score for survival for AML includes age, disease status at transplantation, cytogenetic risk group, and chronic GVHD. For ALL, the risk score includes age at transplantation and chronic GVHD. The 10-year probabilities of OS for AML with good (score 0, 1, or 2), intermediate (score 3), and poor risk (score 4, 5, 6, or 7) were 94%, 87%, and 68%, respectively. The 10-year probabilities of OS for ALL were 89% and 80% for good (score 0 or 1) and poor risk (score 2), respectively. Identifying children at risk for late mortality with early intervention may mitigate some excess late mortality.",

keywords = "Acute lymphoblastic leukemia, Acute myeloid leukemia, Risk score, Survival, Transplantation",

author = "Menachem Bitan and Ahn, {Kwang Woo} and Millard, {Heather R.} and Pulsipher, {Michael A.} and Hisham Abdel-Azim and Auletta, {Jeffery J.} and Valerie Brown and Chan, {Ka Wah} and Diaz, {Miguel Angel} and Andrew Dietz and Vincent, {Marta Gonz{\'a}lez} and Gregory Guilcher and Hale, {Gregory A.} and Hayashi, {Robert J.} and Amy Keating and Parinda Mehta and Kasiani Myers and Kristin Page and Tim Prestidge and Shah, {Nirali N.} and Smith, {Angela R.} and Ann Woolfrey and Elizabeth Thiel and Davies, {Stella M.} and Mary Eapen",

note = "Funding Information: Financial disclosure: The CIBMTR is supported primarily by Public Health Service Grant/Cooperative Agreement 5U24-CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID); a grant/cooperative agreement 5U10HL069294 from NHLBI and NCI; a contract HHSH250201200016C with Health Resources and Services Administration (HRSA/DHHS); grants N00014-15-1-0848 and N00014-16-1-2020 from the Office of Naval Research. Other support to the CIBMTR include: Actinium Pharmaceuticals, Inc.; Alexion; Amgen, Inc.; anonymous donation to the Medical College of Wisconsin; Astellas Pharma US; AstraZeneca; Atara Biotherapeutics, Inc.; Be the Match Foundation; Bluebird Bio, Inc.; Bristol Myers Squibb Oncology; Celgene Corporation; Cellular Dynamics International, Inc.; Cerus Corporation; Chimerix, Inc.; Fred Hutchinson Cancer Research Center; Gamida Cell Ltd.; Genentech, Inc.; Genzyme Corporation; Gilead Sciences, Inc.; Health Research, Inc. Roswell Park Cancer Institute; HistoGenetics, Inc.; Incyte Corporation; Janssen Scientific Affairs, LLC; Jazz Pharmaceuticals, Inc.; Jeff Gordon Children's Foundation; Medac, GmbH; MedImmune; The Medical College of Wisconsin; Merck & Co, Inc.; Mesoblast; MesoScale Diagnostics, Inc.; Miltenyi Biotec, Inc.; National Marrow Donor Program; Neovii Biotech NA, Inc.; Novartis Pharmaceuticals Corporation; Onyx Pharmaceuticals; Optum Healthcare Solutions, Inc.; Otsuka America Pharmaceutical, Inc.; Otsuka Pharmaceutical Co, Ltd. – Japan; PCORI; Perkin Elmer, Inc.; Pfizer, Inc.; Sanofi US; Seattle Genetics; Spectrum Pharmaceuticals, Inc.; St. Baldrick's Foundation; Sunesis Pharmaceuticals, Inc.; Swedish Orphan Biovitrum, Inc.; Takeda Oncology; Telomere Diagnostics, Inc.; University of Minnesota; and Wellpoint, Inc. Publisher Copyright: {\textcopyright} 2017 The American Society for Blood and Marrow Transplantation",

year = "2017",

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doi = "10.1016/j.bbmt.2017.05.011",

language = "English (US)",

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T1 - Personalized Prognostic Risk Score for Long-Term Survival for Children with Acute Leukemia after Allogeneic Transplantation

AU - Bitan, Menachem

AU - Ahn, Kwang Woo

AU - Millard, Heather R.

AU - Pulsipher, Michael A.

AU - Abdel-Azim, Hisham

AU - Auletta, Jeffery J.

AU - Brown, Valerie

AU - Chan, Ka Wah

AU - Diaz, Miguel Angel

AU - Dietz, Andrew

AU - Vincent, Marta González

AU - Guilcher, Gregory

AU - Hale, Gregory A.

AU - Hayashi, Robert J.

AU - Keating, Amy

AU - Mehta, Parinda

AU - Myers, Kasiani

AU - Page, Kristin

AU - Prestidge, Tim

AU - Shah, Nirali N.

AU - Smith, Angela R.

AU - Woolfrey, Ann

AU - Thiel, Elizabeth

AU - Davies, Stella M.

AU - Eapen, Mary

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PY - 2017/9

Y1 - 2017/9

N2 - We studied leukemia-free (LFS) and overall survival (OS) in children with acute myeloid (AML, n = 790) and acute lymphoblastic leukemia (ALL, n = 1096) who underwent transplantation between 2000 and 2010 and who survived for at least 1 year in remission after related or unrelated donor transplantation. Analysis of patient-, disease-, and transplantation characteristics and acute and chronic graft-versus-host disease (GVHD) was performed to identify factors with adverse effects on LFS and OS. These data were used to develop risk scores for survival. We did not identify any prognostic factors beyond 4 years after transplantation for AML and beyond 3 years for ALL. Risk score for survival for AML includes age, disease status at transplantation, cytogenetic risk group, and chronic GVHD. For ALL, the risk score includes age at transplantation and chronic GVHD. The 10-year probabilities of OS for AML with good (score 0, 1, or 2), intermediate (score 3), and poor risk (score 4, 5, 6, or 7) were 94%, 87%, and 68%, respectively. The 10-year probabilities of OS for ALL were 89% and 80% for good (score 0 or 1) and poor risk (score 2), respectively. Identifying children at risk for late mortality with early intervention may mitigate some excess late mortality.

AB - We studied leukemia-free (LFS) and overall survival (OS) in children with acute myeloid (AML, n = 790) and acute lymphoblastic leukemia (ALL, n = 1096) who underwent transplantation between 2000 and 2010 and who survived for at least 1 year in remission after related or unrelated donor transplantation. Analysis of patient-, disease-, and transplantation characteristics and acute and chronic graft-versus-host disease (GVHD) was performed to identify factors with adverse effects on LFS and OS. These data were used to develop risk scores for survival. We did not identify any prognostic factors beyond 4 years after transplantation for AML and beyond 3 years for ALL. Risk score for survival for AML includes age, disease status at transplantation, cytogenetic risk group, and chronic GVHD. For ALL, the risk score includes age at transplantation and chronic GVHD. The 10-year probabilities of OS for AML with good (score 0, 1, or 2), intermediate (score 3), and poor risk (score 4, 5, 6, or 7) were 94%, 87%, and 68%, respectively. The 10-year probabilities of OS for ALL were 89% and 80% for good (score 0 or 1) and poor risk (score 2), respectively. Identifying children at risk for late mortality with early intervention may mitigate some excess late mortality.

KW - Acute lymphoblastic leukemia

KW - Acute myeloid leukemia

KW - Risk score

KW - Survival

KW - Transplantation

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Personalized Prognostic Risk Score for Long-Term Survival for Children with Acute Leukemia after Allogeneic Transplantation

Abstract

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