title: Allogeneic bone marrow transplant improves outcome for juvenile myelomonocytic leukaemia.
abstract: OBJECTIVE
  To correlate clinical presentation and therapeutic outcomes in children with a diagnosis of juvenile myelomonocytic leukaemia.
  
  
  METHODS
  The medical records of 14 children who fulfilled the International Juvenile Myelomonocytic Leukaemia Working Group Criteria for a diagnosis of juvenile myelomonocytic leukaemia (JMML) presenting to a single institution were reviewed, and their clinical status at September 2000 was documented.
  
  
  RESULTS
  The most common presenting features were hepatosplenomegaly and lymphadenopathy. Fifty per cent of cases presented in the first year of life. Nine of 14 patients initially received chemotherapy otherwise used in the treatment of acute myeloid or lymphoblastic leukaemia with no apparent benefit. All six patients who received conditioning therapy with chemotherapy alone, followed by allogeneic bone marrow transplant (BMT), are in complete remission at a median follow-up duration of 12 months (range 5-91 months). Five of six patients surviving post-allogeneic BMT received marrow from an unrelated donor. Only one of seven patients who did not receive BMT survived long-term.
  
  
  CONCLUSIONS
  Children with a diagnosis of JMML should be treated with allogeneic BMT as soon as a suitable donor is found. The role of anti-leukaemic therapy in this disease, prior to BMT, requires further investigation in the context of a multicentre clinical trial.
authors.affiliation: Centre for Children's Cancer and Blood Disorders, Sydney Children's Hospital, High Street, Randwick, New South Wales, Australia.
authors.forename: A M
authors.initials: AM
authors.lastname: Maguire
authors.forename: M R
authors.initials: MR
authors.lastname: Vowels
authors.forename: S
authors.initials: S
authors.lastname: Russell
authors.forename: R J
authors.initials: RJ
authors.lastname: Cohn
authors.forename: C
authors.initials: C
authors.lastname: Cole
authors.forename: B
authors.initials: B
authors.lastname: Saxon
authors.forename: F
authors.initials: F
authors.lastname: Alvaro
authors.forename: G M
authors.initials: GM
authors.lastname: Marshall
mesh_terms: D000970:Antineoplastic Agents / Q000008:administration & dosage; D016026:Bone Marrow Transplantation; D005260:Female; D006776:Hospitals, Pediatric; D006801:Humans; D007223:Infant; D015479:Leukemia, Myelomonocytic, Acute / Q000188:drug therapy / Q000628:therapy*; D008297:Male; D009517:New South Wales; D012074:Remission Induction; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents