title: Survival Impact of Early Post-Transplant Toxicities in Pediatric and Adolescent Patients Undergoing Allogeneic Hematopoietic Cell Transplantation for Malignant and Nonmalignant Diseases: Recognizing Risks and Optimizing Outcomes.
abstract: In pediatric and adolescent patients undergoing allogeneic hematopoietic cell transplantation, treatment-related toxicities remain a clinical challenge. A paucity of data investigates the risks for and survival impact of treatment-related toxicities in this population. Here the authors assess the relative toxicity of myeloablative, reduced-toxicity, and reduced-intensity conditioning regimens; identify patient-related predictors of post-transplant toxicities; and investigate the impact of early post-transplant toxicities on transplant-related mortality (TRM). In this retrospective study, 164 patients (aged 1 to 22 years) underwent allogeneic stem cell transplantation after busulfan-based conditioning for malignant and nonmalignant diseases between 2000 and 2014. The number of grades III to IV toxicities between days 0 and +30 was calculated for each patient. TRM was calculated to 2 years. Median patient age was 9 years, and median number of toxicities was 3 (range, 0 to 17). The 100-person day incidence of post-transplant toxicities in myeloablative conditioning was not different from the incidence in reduced-toxicity conditioning (13.88 versus 13.59, P = .812). Reduced intensity was less toxic than both myeloablative and reduced toxicity (13.75 versus 8.41, P < .001). Age ≥ 12 years (.276 with SE = .138, P = .045) and unrelated donor transplant (.318 with SE = 0.113, P = .005) were risk factors for ≥3 toxicities. Having ≥3 toxicities or a performance score < 90 conferred higher risk of TRM (P = .021). In pediatric and adolescent patients undergoing hematopoietic cell transplantation, reduced-toxicity conditioning was not significantly less toxic than myeloablative conditioning. Additionally, the number of post-transplant toxicities correlated with the risk of mortality. Further investigations to confirm our findings are warranted.
authors.affiliation: Department of Pediatrics, Columbia University Medical Center, New York, New York.
authors.forename: Naima
authors.initials: N
authors.lastname: Al Mulla
authors.affiliation: Department of Pediatrics, Columbia University Medical Center, New York, New York.
authors.forename: Justine M
authors.initials: JM
authors.lastname: Kahn
authors.affiliation: Department of Pediatrics, Columbia University Medical Center, New York, New York; Department of Biostatistics, Mailman School of Public Health, Columbia University, New York, New York.
authors.forename: Zhezhen
authors.initials: Z
authors.lastname: Jin
authors.affiliation: Department of Pediatrics, Columbia University Medical Center, New York, New York.
authors.forename: Mahvish
authors.initials: M
authors.lastname: Qureshi
authors.affiliation: Department of Pediatrics, Columbia University Medical Center, New York, New York.
authors.forename: Esra
authors.initials: E
authors.lastname: Karamehmet
authors.affiliation: Columbia University, New York, New York.
authors.forename: Grace
authors.initials: G
authors.lastname: Yoon-Jeong Kim
authors.affiliation: Department of Pediatrics, Columbia University Medical Center, New York, New York.
authors.forename: Anya L
authors.initials: AL
authors.lastname: Levinson
authors.affiliation: Department of Pediatrics, Columbia University Medical Center, New York, New York.
authors.forename: Monica
authors.initials: M
authors.lastname: Bhatia
authors.affiliation: Department of Pediatrics, Columbia University Medical Center, New York, New York.
authors.forename: James H
authors.initials: JH
authors.lastname: Garvin
authors.affiliation: Department of Pediatrics, Columbia University Medical Center, New York, New York.
authors.forename: Diane
authors.initials: D
authors.lastname: George
authors.affiliation: Department of Pediatrics, Columbia University Medical Center, New York, New York.
authors.forename: Andrew L
authors.initials: AL
authors.lastname: Kung
authors.affiliation: Department of Pediatrics, Columbia University Medical Center, New York, New York. Electronic address: Ps2087@cumc.columbia.edu.
authors.forename: Prakash
authors.initials: P
authors.lastname: Satwani
mesh_terms: D000293:Adolescent; D000328:Adult; D002066:Busulfan / Q000008:administration & dosage / Q000633:toxicity; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects* / Q000401:mortality; D006801:Humans; D007223:Infant; D008297:Male; D019653:Myeloablative Agonists / Q000633:toxicity; D012189:Retrospective Studies; D018570:Risk Assessment / Q000379:methods*; D012307:Risk Factors; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000009:adverse effects* / Q000401:mortality; D014184:Transplantation, Homologous / Q000009:adverse effects / Q000401:mortality; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D019653:Myeloablative Agonists; D002066:Busulfan
keywords: Adolescent; Allogeneic; Morbidity; Mortality; Pediatric; Predictors