title: Outcome of poor response paediatric AML using early SCT.
abstract: BACKGROUND
  Children with poor response acute myeloid leukaemia (AML) generally have a very poor outcome. Allogeneic stem cell transplantation (SCT) is often recommended for these children but the benefit is unclear. The aim of this study was to investigate survival for poor response AML patients treated with SCT.
  
  
  METHODS
  Treatment was given according to the NOPHO-AML 2004 protocol. All patients received AIET (Cytarabine, Idarubicin, Etoposide, Thioguanine) and AM (Cytarabine, Mitoxantrone) as induction. We included poor response defined as > 15% blasts on day 15 after AIET (n = 17) or > 5% blasts after AM (n = 14, refractory disease). Poor response patients received intensively timed induction and proceeded to SCT when a donor was available.
  
  
  RESULTS
  Thirty-one of 267 evaluable patients (12%) had a poor response. SCT was performed in 25; using matched unrelated donors in 13, matched sibling donors in 6, cord blood donor in 4, and haploidentical donor in two. The median follow-up for the 31 poor responding patients was 2.6 years (range 0.4 - 8.1 years) and 3-year probability of survival 70% (95% CI 59-77%).
  
  
  CONCLUSIONS
  The poor responders in the NOPHO-AML 2004 protocol had a favourable prognosis treated with time-intensive induction followed by SCT.
authors.affiliation: Department of Paediatrics, The University Hospital Rigshospitalet, Copenhagen, Denmark.
authors.forename: Neval E
authors.initials: NE
authors.lastname: Wareham
authors.forename: Carsten
authors.initials: C
authors.lastname: Heilmann
authors.forename: Jonas
authors.initials: J
authors.lastname: Abrahamsson
authors.forename: Erik
authors.initials: E
authors.lastname: Forestier
authors.forename: Britt
authors.initials: B
authors.lastname: Gustafsson
authors.forename: Shau-Yin
authors.initials: SY
authors.lastname: Ha
authors.forename: Jesper
authors.initials: J
authors.lastname: Heldrup
authors.forename: Kirsi
authors.initials: K
authors.lastname: Jahnukainen
authors.forename: Ólafur G
authors.initials: ÓG
authors.lastname: Jónsson
authors.forename: Birgitte
authors.initials: B
authors.lastname: Lausen
authors.forename: Josefine
authors.initials: J
authors.lastname: Palle
authors.forename: Bernward
authors.initials: B
authors.lastname: Zeller
authors.forename: Henrik
authors.initials: H
authors.lastname: Hasle
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D003561:Cytarabine / Q000494:pharmacology / Q000627:therapeutic use*; D018572:Disease-Free Survival; D061345:Early Medical Intervention; D005047:Etoposide / Q000494:pharmacology / Q000627:therapeutic use*; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D015255:Idarubicin / Q000494:pharmacology / Q000627:therapeutic use*; D060828:Induction Chemotherapy; D007223:Infant; D007231:Infant, Newborn; D015470:Leukemia, Myeloid, Acute / Q000276:immunology / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D008297:Male; D008942:Mitoxantrone / Q000494:pharmacology / Q000627:therapeutic use*; D019653:Myeloablative Agonists / Q000494:pharmacology / Q000627:therapeutic use*; D013866:Thioguanine / Q000494:pharmacology / Q000627:therapeutic use*; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D019653:Myeloablative Agonists; D003561:Cytarabine; D005047:Etoposide; D008942:Mitoxantrone; D013866:Thioguanine; D015255:Idarubicin