Outcomes of pediatric patients with therapy-related myeloid neoplasms.


Journal

Bone marrow transplantation
ISSN: 1476-5365
Titre abrégé: Bone Marrow Transplant
Pays: England
ID NLM: 8702459

Informations de publication

Date de publication:
12 2021
Historique:
received: 19 05 2021
accepted: 20 08 2021
revised: 06 08 2021
pubmed: 5 9 2021
medline: 11 3 2022
entrez: 4 9 2021
Statut: ppublish

Résumé

Long-term outcomes after allogeneic hematopoietic cell transplantation (HCT) for therapy-related myeloid neoplasms (tMNs) are dismal. There are few multicenter studies defining prognostic factors in pediatric patients with tMNs. We have accumulated the largest cohort of pediatric patients who have undergone HCT for a tMN to perform a multivariate analysis defining factors predictive of long-term survival. Sixty-eight percent of the 401 patients underwent HCT using a myeloablative conditioning (MAC) regimen, but there were no statistically significant differences in the overall survival (OS), event-free survival (EFS), or cumulative incidence of relapse and non-relapse mortality based on the conditioning intensity. Among the recipients of MAC regimens, 38.4% of deaths were from treatment-related causes, especially acute graft versus host disease (GVHD) and end-organ failure, as compared to only 20.9% of deaths in the reduced-intensity conditioning (RIC) cohort. Exposure to total body irradiation (TBI) during conditioning and experiencing grade III/IV acute GVHD was associated with worse OS. In addition, a diagnosis of therapy-related myelodysplastic syndrome and having a structurally complex karyotype at tMN diagnosis were associated with worse EFS. Reduced-toxicity (but not reduced-intensity) regimens might help to decrease relapse while limiting mortality associated with TBI-based HCT conditioning in pediatric patients with tMNs.

Identifiants

pubmed: 34480120
doi: 10.1038/s41409-021-01448-x
pii: 10.1038/s41409-021-01448-x
pmc: PMC9260859
mid: NIHMS1820140
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

2997-3007

Subventions

Organisme : NCI NIH HHS
ID : K12 CA090625
Pays : United States
Organisme : NCI NIH HHS
ID : P30 CA008748
Pays : United States

Informations de copyright

© 2021. The Author(s), under exclusive licence to Springer Nature Limited.

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Auteurs

Akshay Sharma (A)

Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN, USA. akshay.sharma@stjude.org.

Sujuan Huang (S)

Biostatistics, St. Jude Children's Research Hospital, Memphis, TN, USA.

Ying Li (Y)

Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN, USA.

Russell J Brooke (RJ)

Biostatistics, St. Jude Children's Research Hospital, Memphis, TN, USA.

Ibrahim Ahmed (I)

Pediatric Hematology, Oncology and BMT, Children's Mercy Hospital Kansas City, Kansas City, MO, USA.

Heather B Allewelt (HB)

Pediatric Hematology Oncology, AdventHealth for Children, Orlando, FL, USA.

Persis Amrolia (P)

Department of Bone Marrow Transplant, Great Ormond St Children's Hospital, London, UK.

Alice Bertaina (A)

Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford School of Medicine, Stanford, CA, USA.

Neel S Bhatt (NS)

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.

Marc B Bierings (MB)

Stem cell transplantation, Princess Maxima Centre for Pediatric Oncology, Utrecht, Netherlands.

Joshua Bies (J)

Pediatrics, University of North Carolina, Chapel Hill, NC, USA.

Claire Brisset (C)

Hemato-immunology Department, Robert Debré Hospital, GHU APHP Nord-Université de Paris, Paris, France.

Jennifer E Brondon (JE)

Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC, USA.

Ann Dahlberg (A)

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.

Jean-Hugues Dalle (JH)

Hemato-immunology Department, Robert Debré Hospital, GHU APHP Nord-Université de Paris, Paris, France.

Hesham Eissa (H)

Blood and Marrow Transplant and Cellular Therapeutics, Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora, CO, USA.

Mony Fahd (M)

Hemato-immunology Department, Robert Debré Hospital, GHU APHP Nord-Université de Paris, Paris, France.

Adam Gassas (A)

Department of Haematology and Oncology, Royal Hospital for Children, Bristol, UK.

Nicholas J Gloude (NJ)

Pediatrics, University of California San Diego, Rady Children's Hospital San Diego, San Diego, CA, USA.

W Scott Goebel (WS)

Pediatrics, Riley Hospital for Children at IU Health, Indiana University School of Medicine, Indianapolis, IN, USA.

Erika S Goeckerman (ES)

Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC, USA.

Katherine Harris (K)

Blood and Marrow Transplantation, Children's National Hospital, Washington, DC, USA.

Richard Ho (R)

Pediatric Hematology, Oncology and BMT, Vanderbilt University Medical Center, Nashville, TN, USA.

Michelle P Hudspeth (MP)

Pediatric Hematology and Oncology, Medical University of South Carolina, Charleston, SC, USA.

Jeffrey S Huo (JS)

Pediatric Cellular Therapies, Cancer and Blood Disorders, Atrium Health Levine Children's Hospital, Charlotte, NC, USA.

David Jacobsohn (D)

Blood and Marrow Transplantation, Children's National Hospital, Washington, DC, USA.

Kimberly A Kasow (KA)

Pediatrics, University of North Carolina, Chapel Hill, NC, USA.

Emmanuel Katsanis (E)

Department of Pediatrics, University of Arizona, Tucson, AZ, USA.

Saara Kaviany (S)

Pediatric Hematology, Oncology and BMT, Vanderbilt University Medical Center, Nashville, TN, USA.

Amy K Keating (AK)

Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO, USA.

Nancy A Kernan (NA)

Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York City, NY, USA.

Yiouli P Ktena (YP)

Pediatric Oncology, Sidney Kimmel Cancer Center, Johns Hopkins University, Baltimore, MD, USA.

Colette R Lauhan (CR)

Pediatrics, University of California San Diego, Rady Children's Hospital San Diego, San Diego, CA, USA.

Gerardo López-Hernandez (G)

Bone Marrow Transplant and Cell therapy Department, National Institute of Pediatrics, Ciudad de Mexico, Mexico.

Paul L Martin (PL)

Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC, USA.

Kasiani C Myers (KC)

Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.

Swati Naik (S)

Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.

Alberto Olaya-Vargas (A)

Bone Marrow Transplant and Cell therapy Department, National Institute of Pediatrics, Ciudad de Mexico, Mexico.

Toshihiro Onishi (T)

Center for Cell and Gene Therapy, Texas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.

Mohamed Radhi (M)

Pediatric Hematology, Oncology and BMT, Children's Mercy Hospital Kansas City, Kansas City, MO, USA.

Shanti Ramachandran (S)

Oncology, Haematology, Blood and Marrow Transplantation, Child and Adolescent Health Services, Perth Children's Hospital, Nedlands, WA, Australia.

Kristie Ramos (K)

Department of Pediatrics, University of Arizona, Tucson, AZ, USA.

Hemalatha G Rangarajan (HG)

Hematology, Oncology, Blood and Marrow Transplant, Nationwide Children's Hospital, Columbus, OH, USA.

Philip A Roehrs (PA)

Pediatric Cellular Therapies, Cancer and Blood Disorders, Atrium Health Levine Children's Hospital, Charlotte, NC, USA.

Megan E Sampson (ME)

Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.

Peter J Shaw (PJ)

Children's Hospital at Westmead, Westmead, NSW, Australia.

Jodi L Skiles (JL)

Pediatrics, Riley Hospital for Children at IU Health, Indiana University School of Medicine, Indianapolis, IN, USA.

Katherine Somers (K)

Hematology, Oncology, Blood and Marrow Transplant, Nationwide Children's Hospital, Columbus, OH, USA.

Heather J Symons (HJ)

Pediatric Oncology, Sidney Kimmel Cancer Center, Johns Hopkins University, Baltimore, MD, USA.

Marie de Tersant (M)

Hemato-immunology Department, Robert Debré Hospital, GHU APHP Nord-Université de Paris, Paris, France.

Allison N Uber (AN)

Pediatric Hematology and Oncology, Medical University of South Carolina, Charleston, SC, USA.

Birgitta Versluys (B)

Stem cell transplantation, Princess Maxima Centre for Pediatric Oncology, Utrecht, Netherlands.

Cheng Cheng (C)

Biostatistics, St. Jude Children's Research Hospital, Memphis, TN, USA.

Brandon M Triplett (BM)

Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN, USA.

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