title: Haploidentical HCT using an αβ T-cell-depleted graft with targeted αβ(+) cells by add-back after a reduced intensity preparative regimen containing low-dose TBI.
abstract: Between 2012 and 2015, 42 pediatric patients underwent haploidentical hematopoietic cell transplantation using an αβ(+) T-cell-depleted graft with targeted αβ cells at 1-5 × 10(5)/kg by add-back; 31 had hematologic malignancy (HM), 8 had non-malignant disease (NM) and 3 had solid tumors. All patients received uniform reduced-intensity conditioning with fludarabine, cyclophosphamide, rabbit anti-thymocyte globulin and low-dose TBI. All 42 patients achieved neutrophil engraftment at a median of 10 days. The cumulative incidences (CIs) of ⩾grade II and ⩾grade III acute GvHD were 31±7.1% (SE) and 12±5.0%, respectively, and 1-year CI of chronic GvHD was 15±5.8%. One patient died of CMV pneumonia, leading to transplant-related mortality (TRM) of 2.6±2.5%. Sixteen patients relapsed and 11 died of disease. At a median follow-up of 19 months (range, 5-43 months), the estimated 2-year event-free survival for NM and HM were 88±11.7 and 50±10.1%, respectively. Our study demonstrated that haploidentical hematopoietic cell transplantation after ex vivo depletion of αβ(+) T cells with targeted dose noticeably reduced the graft failure rate and TRM in pediatric patients and could be applied to patients lacking a suitable related or unrelated donor.
authors.affiliation: Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center, Seoul, Korea.
authors.forename: H J
authors.initials: HJ
authors.lastname: Im
authors.affiliation: Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center, Seoul, Korea.
authors.forename: K N
authors.initials: KN
authors.lastname: Koh
authors.affiliation: Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center, Seoul, Korea.
authors.forename: J K
authors.initials: JK
authors.lastname: Suh
authors.affiliation: Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center, Seoul, Korea.
authors.forename: S W
authors.initials: SW
authors.lastname: Lee
authors.affiliation: Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center, 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, 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, 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, Seoul, Korea.
authors.forename: C-J
authors.initials: CJ
authors.lastname: Park
authors.affiliation: Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center, Seoul, Korea.
authors.forename: J J
authors.initials: JJ
authors.lastname: Seo
mesh_terms: D000293:Adolescent; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000473:pathology; D019337:Hematologic Neoplasms / Q000628:therapy*; D006801:Humans; D007223:Infant; D008212:Lymphocyte Depletion / Q000379:methods*; D008297:Male; D009369:Neoplasms / Q000628:therapy; D016693:Receptors, Antigen, T-Cell, alpha-beta; D016019:Survival Analysis; D013601:T-Lymphocytes / Q000276:immunology / Q000637:transplantation; D019172:Transplantation Conditioning / Q000379:methods*; D000075442:Transplantation, Haploidentical / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D016896:Treatment Outcome; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives; D014916:Whole-Body Irradiation / Q000379:methods; D055815:Young Adult
chemical_list: D000961:Antilymphocyte Serum; D016693:Receptors, Antigen, T-Cell, alpha-beta; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine