title: Successful rescue of early graft failure in pediatric patients using T-cell-depleted haploidentical hematopoietic SCT.
abstract: Graft failure (GF) is a significant complication after allogeneic hematopoietic stem cell transplantation (HCT) and is associated with a high mortality rate. We performed re-transplantation using haploidentical-related donors to rescue children with early GF. Between 2008 and 2013, 10 patients received re-transplantation from haploidentical family donors. The median age at HCT was 13.5 years and the median time between transplantations was 52.5 days. Conditioning regimen with fludarabine and CY was used in seven patients, and TBI was added in three patients. All 10 patients received T-cell-depleted grafts using CD3 or CD3/CD19 MoAb. The median numbers of CD34(+) and CD3(+) cells were 5.52 × 10(6)/kg and 1.08 × 10(6)/kg, respectively. For GVHD prophylaxis, mycophenolate mofetil (MMF) and tacrolimus or MMF and CYA were used. All 10 patients achieved a sustained neutrophil engraftment and maintained a complete donor chimerism at the time of analysis (median 23 months, range 6-62 months). Nine of 10 patients were alive, and one patient with moyamoya disease with AML died of encephalopathy 7 months post transplant. This study suggests that fludarabine- and CY-based conditioning with T-cell-depleted haploidentical HCT is a feasible option to rescue pediatric patients with primary GF.
authors.affiliation: Department of Pediatrics, Haeundae Paik Hospital, Inje University College of Medicine, Busan, Korea.
authors.forename: J A
authors.initials: JA
authors.lastname: Park
authors.affiliation: Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center Children's Hospital, Seoul, Korea.
authors.forename: K N
authors.initials: KN
authors.lastname: Koh
authors.affiliation: Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center Children's Hospital, Seoul, Korea.
authors.forename: E S
authors.initials: ES
authors.lastname: Choi
authors.affiliation: Department of Laboratory Medicine, University of Ulsan College of Medicine, Asan Medical Center Children's Hospital, Seoul, Korea.
authors.forename: S
authors.initials: S
authors.lastname: Jang
authors.affiliation: Department of Laboratory Medicine, University of Ulsan College of Medicine, Asan Medical Center Children's Hospital, Seoul, Korea.
authors.forename: S W
authors.initials: SW
authors.lastname: Kwon
authors.affiliation: Department of Laboratory Medicine, University of Ulsan College of Medicine, Asan Medical Center Children's Hospital, Seoul, Korea.
authors.forename: C-J
authors.initials: CJ
authors.lastname: Park
authors.affiliation: Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center Children's Hospital, Seoul, Korea.
authors.forename: J J
authors.initials: JJ
authors.lastname: Seo
authors.affiliation: Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center Children's Hospital, Seoul, Korea.
authors.forename: H J
authors.initials: HJ
authors.lastname: Im
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival / Q000276:immunology*; D006086:Graft vs Host Disease / Q000517:prevention & control*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D007223:Infant; D008212:Lymphocyte Depletion / Q000009:adverse effects*; D008297:Male; D012189:Retrospective Studies; D019172:Transplantation Conditioning / Q000009:adverse effects*; D014184:Transplantation, Homologous; D055815:Young Adult