Open AccessOutcomes of pediatric patients with therapy-related myeloid neoplasms
Author(s) -
Akshay Sharma,
Sujuan Huang,
Ying Li,
Russell J. Brooke,
Ibrahim Ahmed,
Heather Allewelt,
Persis Amrolia,
Alice Bertaina,
Neel S. Bhatt,
Marc Bierings,
Joshua J. Bies,
Claire Brisset,
Jennifer Brondon,
Ann Dahlberg,
Jean Hugues Dalle,
Hesham Eissa,
Mony Fahd,
Adam Gassas,
Nicholas J. Gloude,
W. Scott Goebel,
Erika S. Goeckerman,
Katherine E. Harris,
Richard Ho,
Michelle Hudspeth,
Jeffrey S. Huo,
David A. Jacobsohn,
Kimberly A. Kasow,
Emmanuel Katsanis,
Saara Kaviany,
Amy K. Keating,
Nancy A. Kernan,
Yiouli P. Ktena,
Colette Lauhan,
Gerardo LópezHernández,
Paul Martin,
Kasiani C. Myers,
Swati Naik,
Alberto OlayaVargas,
Toshihiro Onishi,
Mohamed Radhi,
Shanti Ramachandran,
Kristie N. Ramos,
Hemalatha G. Rangarajan,
Philip Roehrs,
Megan Sampson,
Peter J. Shaw,
Jodi Skiles,
Katherine M. Somers,
Heather J. Symons,
Marie de Tersant,
Allison Uber,
Birgitta Versluys,
Cheng Cheng,
Brandon M. Triplett
Publication year - 2021
Publication title -
bone marrow transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.609
H-Index - 127
eISSN - 1476-5365
pISSN - 0268-3369
DOI - 10.1038/s41409-021-01448-x
Subject(s) - medicine , total body irradiation , cohort , cumulative incidence , myelodysplastic syndromes , hematopoietic stem cell transplantation , transplantation , oncology , myeloid , incidence (geometry) , regimen , chemotherapy , bone marrow , cyclophosphamide , physics , optics
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.