25 June 2019 : Original article
Risk Factors for Transplant Outcomes in Children and Adolescents with Non-Malignant Diseases Following Allogeneic Hematopoietic Stem Cell TransplantationAgnieszka Zaucha-Prażmo1ABCDEF*, Elżbieta Sadurska2BDE, Anna Pieczonka3BDE, Jolanta Goździk4BD, Robert Dębski5BD, Katarzyna Drabko1BDE, Joanna Zawitkowska1DE, Monika Lejman1BDF, Jacek Wachowiak3DE, Jan Styczyński5ADE, Jerzy R. Kowalczyk1ADE
Ann Transplant 2019; 24:374-382
BACKGROUND: The objective of this study was the analysis of transplant outcomes and survival in children treated with allogeneic hematopoietic cell transplantation (alloHCT) for non-malignant disorders, with a focus on risk factor analysis of transplant-related mortality (TRM).
MATERIAL AND METHODS: The treatment outcome was analyzed retrospectively in 10 consecutive years in 4 pediatric transplant centers in Poland. To compare the outcomes, patient data were analyzed according to the diagnosis, age at transplant, donor type, stem cell source, conditioning regimens, transplanted CD34+ cells dose, and pediatric TRM score.
RESULTS: From 183 analyzed patients, 27 (14.8%) died, all of them due to transplant-related complications. TRM occurred more frequently in matched unrelated donor (MUD) transplant recipients vs. matched sibling donor (MSD) transplant recipients (p=0.02); in peripheral blood (PB) recipients vs. bone marrow (BM) recipients (p=0.004); and in patients receiving >5×10⁶/kg CD34+ cells (p<0.0001). OS differed significantly according to underlying disease comparing to other diagnoses. Lower survival was found in patients transplanted from MUD (p=0.02). OS was higher in patients receiving BM (p=0.001) and in those receiving ≤5×10⁶/kg CD34+ cells (p<0.001). Multivariate analysis showed lower probability of TRM in BM recipients (p=0.04). The probability of TRM was higher in SCID patients (p=0.02) and in patients receiving >5×10⁶/kg CD34+ cells (p=0.0001).
CONCLUSIONS: Underlying disease, stem cell source, and CD34+ dose higher than 5×10⁶/kg were the most important risk factors for TRM, and they all affected OS.
Keywords: Adolescent, Child, Risk Factors, Anemia, Aplastic, Child, Preschool, Congenital Bone Marrow Failure Syndromes, Graft vs Host Disease, Granulomatous Disease, Chronic, primary immunodeficiency diseases, Prognosis, Retrospective Studies, Survival Rate, Transplantation Conditioning, Treatment Outcome
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