title: Allogeneic stem cell transplantation for refractory acute myeloid leukemia in pediatric patients: the UK experience.
abstract: We report outcomes for 44 children who underwent stem cell transplantation (SCT) for refractory AML in the UK between 2000 and 2012. Median age at SCT was 11.5 years. Twenty-three patients had primary refractory and 21 relapsed refractory AML. Refractory disease was confirmed by cytogenetics/molecular genetics in 24 cases. Median follow-up of the whole cohort is 6.8 years (2.1-14.9 years). Thirty patients (68%) achieved a CR following SCT. Transplant-related mortality at 1 year was 18%. Acute GVHD incidence was 52% (grade ⩾III 19%), chronic 7%. Relapse was the major cause of treatment failure and occurred in 32% of patients at a median of 61 days post SCT. Five-year overall survival and leukemia-free survival (LFS) were 43% (95% CI 31-61%). All patients with favorable cytogenetics (n=6) are alive in CR. Outcomes in patients with primary refractory disease were equivalent to those with relapsed refractory AML. Blast percentage ⩽30% in the BM pre-SCT, myeloablative conditioning and acute GVHD proved to be favorable prognostic features. We could stratify patients according to age ⩾10 years and >30% blasts in BM pre-SCT. Patients with none/one of these risk factors were highly salvageable (5 years LFS 53%) whereas those with both factors had a very poor prognosis (5 years LFS 10%). This may facilitate decision making on whether it is appropriate to consider transplant in such patients.
authors.affiliation: Bone Marrow Transplantation Department, Great Ormond Street Hospital, London, UK.
authors.forename: P
authors.initials: P
authors.lastname: O'Hare
authors.affiliation: Bone Marrow Transplantation Department, Great Ormond Street Hospital, London, UK.
authors.forename: G
authors.initials: G
authors.lastname: Lucchini
authors.affiliation: Pediatric Bone Marrow Transplant Department, Bristol Royal Children's Hospital, Bristol, UK.
authors.forename: M
authors.initials: M
authors.lastname: Cummins
authors.affiliation: Bone Marrow Transplantation Department, Great Ormond Street Hospital, London, UK.
authors.forename: P
authors.initials: P
authors.lastname: Veys
authors.affiliation: Hemato-Oncology Unit, Royal Marsden Hospital, London, UK.
authors.forename: M
authors.initials: M
authors.lastname: Potter
authors.affiliation: Department of Haematology, Birmingham Children's Hospital, Birmingham, UK.
authors.forename: S
authors.initials: S
authors.lastname: Lawson
authors.affiliation: Department of Paediatric Haematology, Sheffield Children's Hospital, Sheffield, UK.
authors.forename: A
authors.initials: A
authors.lastname: Vora
authors.affiliation: Royal Manchester Children's Hospital, Manchester, UK.
authors.forename: R
authors.initials: R
authors.lastname: Wynn
authors.affiliation: Hematology Department, Oxford University Hospital, Oxford, UK.
authors.forename: A
authors.initials: A
authors.lastname: Peniket
authors.affiliation: British Society for Blood and Marrow Transplantation Registry, Guy's Hospital, London, UK.
authors.forename: K
authors.initials: K
authors.lastname: Kirkland
authors.affiliation: British Society for Blood and Marrow Transplantation Registry, Guy's Hospital, London, UK.
authors.forename: R
authors.initials: R
authors.lastname: Pearce
authors.affiliation: British Society for Blood and Marrow Transplantation Registry, Guy's Hospital, London, UK.
authors.forename: J
authors.initials: J
authors.lastname: Perry
authors.affiliation: Bone Marrow Transplantation Department, Great Ormond Street Hospital, London, UK.
authors.forename: P J
authors.initials: PJ
authors.lastname: Amrolia
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D064591:Allografts; D002648:Child; D002675:Child, Preschool; D002869:Chromosome Aberrations; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000235:genetics / Q000401:mortality* / Q000473:pathology; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000235:genetics / Q000401:mortality* / Q000473:pathology / Q000628:therapy*; D008297:Male; D012008:Recurrence; D015996:Survival Rate; D006113:United Kingdom / Q000453:epidemiology