Outcomes of pediatric patients with therapy-related myeloid neoplasms

Akshay Sharma, Sujuan Huang, Ying Li, Russell J. Brooke, Ibrahim Ahmed, Heather B. Allewelt, Persis Amrolia, Alice Bertaina, Neel S. Bhatt, Marc B. Bierings, Joshua Bies, Claire Brisset, Jennifer E. Brondon, Ann Dahlberg, Jean Hugues Dalle, Hesham Eissa, Mony Fahd, Adam Gassas, Nicholas J. Gloude, W. Scott GoebelErika S. Goeckerman, Katherine Harris, Richard Ho, Michelle P. Hudspeth, Jeffrey S. Huo, David Jacobsohn, Kimberly A. Kasow, Emmanuel Katsanis, Saara Kaviany, Amy K. Keating, Nancy A. Kernan, Yiouli P. Ktena, Colette R. Lauhan, Gerardo López-Hernandez, Paul L. Martin, Kasiani C. Myers, Swati Naik, Alberto Olaya-Vargas, Toshihiro Onishi, Mohamed Radhi, Shanti Ramachandran, Kristie Ramos, Hemalatha G. Rangarajan, Philip A. Roehrs, Megan E. Sampson, Peter J. Shaw, Jodi L. Skiles, Katherine Somers, Heather J. Symons, Marie de Tersant, Allison N. Uber, Birgitta Versluys, Cheng Cheng, Brandon M. Triplett

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish (US)
Pages (from-to)2997-3007
Number of pages11
JournalBone marrow transplantation
Issue number12
StatePublished - Dec 2021

ASJC Scopus subject areas

  • Transplantation
  • Hematology


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