Sharma, A;
Huang, S;
Li, Y;
Brooke, RJ;
Ahmed, I;
Allewelt, HB;
Amrolia, P;
... Triplett, BM; + view all
(2021)
Outcomes of pediatric patients with therapy-related myeloid neoplasms.
Bone Marrow Transplantation
, 56
pp. 2997-3007.
10.1038/s41409-021-01448-x.
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Abstract
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.
| Type: | Article |
|---|---|
| Title: | Outcomes of pediatric patients with therapy-related myeloid neoplasms |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1038/s41409-021-01448-x |
| Publisher version: | https://doi.org/10.1038/s41409-021-01448-x |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. |
| Keywords: | Science & Technology, Life Sciences & Biomedicine, Biophysics, Oncology, Hematology, Immunology, Transplantation, STEM-CELL TRANSPLANTATION, MYELODYSPLASTIC SYNDROME, MALIGNANT NEOPLASMS, RISK-FACTORS, LEUKEMIA, AML, EVOLUTION, CANCER, INDEX |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10140391 |
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