title: T-cell-replete haploidentical stem cell transplantation is highly efficacious for relapsed and refractory childhood acute leukaemia.
abstract: BACKGROUND
  Despite improvements in first-line therapies, the outcomes of relapsed or refractory childhood acute leukaemia that has not achieved complete remission after relapse, has relapsed after stem cell transplantation (SCT), has primary induction failure and has relapsed with a very unfavourable cytogenetic risk profile, are dismal.
  
  
  OBJECTIVE
  We evaluated the feasibility and efficacy of T-cell-replete haploidentical peripheral blood stem cell transplantation (haplo-SCT) with low-dose anti-human thymocyte immunoglobulin (ATG), tacrolimus, methotrexate and prednisolone (PSL) in 14 paediatric patients with high-risk childhood acute leukaemia.
  
  
  RESULTS
  All patients achieved complete engraftment. The median time to reaching an absolute neutrophil count of more than 0.5 × 10(9) L(-1) was 14 days. Acute graft-vs-host disease (aGVHD) of grades II-IV and III-IV developed in 10 (71%) and 2 (14%) patients, respectively. Treatment-related mortality and relapse occurred in one (7%) patient and six (43%) patients, respectively. Eleven patients were alive and seven of them were disease-free with a median follow-up of 36 months (range: 30-159 months). The probability of event-free survival after 2 years was 50%.
  
  
  CONCLUSIONS
  These findings indicate that T-cell-replete haplo-SCT, with low-dose ATG and PSL, provides sustained remission with an acceptable risk of GVHD in paediatric patients with advanced haematologic malignancies.
authors.affiliation: Department of Pediatric Oncology.
authors.forename: S
authors.initials: S
authors.lastname: Kobayashi
authors.forename: M
authors.initials: M
authors.lastname: Ito
authors.forename: H
authors.initials: H
authors.lastname: Sano
authors.forename: K
authors.initials: K
authors.lastname: Mochizuki
authors.forename: M
authors.initials: M
authors.lastname: Akaihata
authors.forename: T
authors.initials: T
authors.lastname: Waragai
authors.forename: Y
authors.initials: Y
authors.lastname: Ohara
authors.forename: M
authors.initials: M
authors.lastname: Hosoya
authors.forename: H
authors.initials: H
authors.lastname: Ohto
authors.forename: A
authors.initials: A
authors.lastname: Kikuta
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D064591:Allografts; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000097:blood / Q000401:mortality / Q000628:therapy; D006801:Humans; D007223:Infant; D007938:Leukemia / Q000097:blood / Q000401:mortality / Q000628:therapy*; D007958:Leukocyte Count; D017710:Lymphocyte Transfusion; D008297:Male; D012008:Recurrence; D033581:Stem Cell Transplantation; D015996:Survival Rate; D013601:T-Lymphocytes / Q000637:transplantation*
keywords: HLA-haploidentical stem cell transplantation; T-cell-replete haploidentical stem cell transplantation; children; graft-vs-leukaemia effect; refractory leukaemia