title: Safety of hematopoietic stem cell transplantation in children less than three years of age.
abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) is a standard treatment for a variety of hematologic conditions. However, very young children may experience different complications of HSCT compared to older patients. The authors retrospectively analyzed the results of 51 transplants performed on children less than 3 years of age between June 1987 and October 2005. Donors were matched-related (n = 21), partially mismatched related (n = 3), and unrelated (n = 27). The majority of patients had one or more grade III organ toxicities, but all nonrelapse deaths were attributable to infection. Perineal dermatitis was found in a large number (73%) of recipients of cyclophosphamide-based conditioning regimens. The 1-year transplant-related mortality (TRM) was 14%, but significantly declined in the more modern period. Grades II-IV acute graft-versus-host-disease (GvHD) was seen in 22% of patients, while chronic extensive GvHD developed in only 7% of patients. Relapse was seen in 40% of transplants performed for a malignant condition, most commonly in those patients not in remission at time of HSCT. The 5-year event-free survival (EFS) and overall survival (OS) were 53 and 64%, respectively. Recipients of fractionated total body irradiation (fTBI) were more likely to have at least one long-term sequelae than patients who received chemotherapy-based regimens (p = .014). These data demonstrate that HSCT can be performed safely in very young children, especially as supportive-care techniques improve. Cyclophosphamide-related perineal dermatitis is a unique complication in very young children. Finally, the incidence of acute and chronic GvHD in this population is low.
authors.affiliation: Division of Pediatric Stem Cell Transplantation, Stanford University School of Medicine, Stanford, CA, USA. dvorakc@peds.ucsf.edu
authors.forename: Christopher C
authors.initials: CC
authors.lastname: Dvorak
authors.forename: Nicola Bobey
authors.initials: NB
authors.lastname: Wright
authors.forename: Wendy B
authors.initials: WB
authors.lastname: Wong
authors.forename: Karen M
authors.initials: KM
authors.lastname: Kristovich
authors.forename: Edward W
authors.initials: EW
authors.lastname: Matthews
authors.forename: Kenneth I
authors.initials: KI
authors.lastname: Weinberg
authors.forename: Michael D
authors.initials: MD
authors.lastname: Amylon
authors.forename: Rajni
authors.initials: R
authors.lastname: Agarwal
mesh_terms: D002675:Child, Preschool; D005260:Female; D006084:Graft Rejection / Q000276:immunology; D006086:Graft vs Host Disease / Q000209:etiology / Q000276:immunology / Q000401:mortality*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects* / Q000401:mortality / Q000706:statistics & numerical data; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007239:Infections / Q000209:etiology / Q000401:mortality*; D053208:Kaplan-Meier Estimate; D008297:Male; D012189:Retrospective Studies; D019172:Transplantation Conditioning; D014916:Whole-Body Irradiation