title: Salvage allogeneic hematopoietic SCT for primary graft failure in children.
abstract: Primary graft failure (pGF) is associated with considerable morbidity and mortality. Salvage hematopoietic SCT (HSCT) can rescue pGF patients; however, the optimal preconditioning regimen and stem cell source are yet to be determined, particularly in children. In this study, we retrospectively analyzed 102 pediatric patients who received salvage allogeneic HSCT for pGF. Salvage HSCT from matched or one-Ag-mismatched related donors (rMM01) provided superior OS compared with that from two- or three-Ags-mismatched related donors (rMM23) or cord blood transplantation (CBT). CBT showed a trend toward a slightly lower engraftment rate and late engraftment achievement compared with rMM23; however, the OS rate was similar between the two groups (47.6±7.7% for rMM23 and 45.7±8.6% for CBT, at 1 year after salvage HSCT). Multivariate analysis showed that preconditioning regimens with fludarabine or irradiation were associated with a higher engraftment rate and those with alkylating agents were associated with better OS. In conclusion, our results showed that rMM01 was the most suitable donor for salvage HSCT for pediatric pGF, and that CBT was an equally important option compared with rMM23 for patients without rMM01.
authors.affiliation: Department of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan. katom-tky@umin.ac.jp
authors.forename: M
authors.initials: M
authors.lastname: Kato
authors.forename: K
authors.initials: K
authors.lastname: Matsumoto
authors.forename: R
authors.initials: R
authors.lastname: Suzuki
authors.forename: H
authors.initials: H
authors.lastname: Yabe
authors.forename: M
authors.initials: M
authors.lastname: Inoue
authors.forename: H
authors.initials: H
authors.lastname: Kigasawa
authors.forename: J
authors.initials: J
authors.lastname: Inagaki
authors.forename: K
authors.initials: K
authors.lastname: Koh
authors.forename: Y
authors.initials: Y
authors.lastname: Hashii
authors.forename: H
authors.initials: H
authors.lastname: Tauchi
authors.forename: A
authors.initials: A
authors.lastname: Suminoe
authors.forename: A
authors.initials: A
authors.lastname: Kikuta
authors.forename: H
authors.initials: H
authors.lastname: Sakamaki
authors.forename: K
authors.initials: K
authors.lastname: Kawa
authors.forename: K
authors.initials: K
authors.lastname: Kato
authors.forename: T
authors.initials: T
authors.lastname: Fukuda
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D006084:Graft Rejection / Q000601:surgery*; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects* / Q000379:methods*; D006801:Humans; D007223:Infant; D011379:Prognosis; D012307:Risk Factors; D016879:Salvage Therapy / Q000379:methods*; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D016896:Treatment Outcome