title: Outcome of children less than three years old at diagnosis with non-metastatic medulloblastoma treated with chemotherapy on the "Head Start" I and II protocols.
abstract: OBJECTIVE
  To determine the survival of infants and young children with non-metastatic medulloblastoma using intensive myeloablative chemotherapy and autologous hematopoietic progenitor cell rescue (AuHCR).
  
  
  METHODS
  Twenty-one children less than 3 years old at diagnosis with non-metastatic medulloblastoma were enrolled on two identical serial studies, "Head Start" I and "Head Start" II. After surgery, patients received five cycles of induction chemotherapy consisting of vincristine, cisplatin, cyclophosphamide and etoposide. Following induction, all patients underwent myeloablative chemotherapy using carboplatin, thiotepa and etoposide with AuHCR. Irradiation was used only at relapse.
  
  
  RESULTS
  The 5-year event-free (EFS) and overall survival (OS) rates (+/-SE) for all patients, patients with gross total resection, and patients with residual tumor were 52 +/- 11% and 70 +/- 10%, 64 +/- 13% and 79 +/- 11%, and 29 +/- 17% and 57 +/- 19%, respectively. The 5-year EFS and OS ( +/- SE) for patients with desmoplastic and classical medulloblastoma were 67 +/- 16% and 78 +/- 14%, and 42 +/- 14 and 67 +/- 14%, respectively. There were four treatment related deaths. The majority of survivors (71%) avoided irradiation completely. Mean intellectual functioning and quality of life (QoL) for children surviving without irradiation was within average range for a majority of survivors tested.
  
  
  CONCLUSIONS
  This strategy of brief intensive chemotherapy for young children with non-metastatic medulloblastoma eliminated the need for craniospinal irradiation 52% of the patients, and may preserve QoL and intellectual functioning. The excellent survival rates are somewhat dampened by high toxic mortality.
authors.affiliation: Childrens Hospital Los Angeles, Los Angeles, California, USA. gdhall@chla.usc.edu
authors.forename: Girish
authors.initials: G
authors.lastname: Dhall
authors.forename: Howard
authors.initials: H
authors.lastname: Grodman
authors.forename: Lingyun
authors.initials: L
authors.lastname: Ji
authors.forename: Stephen
authors.initials: S
authors.lastname: Sands
authors.forename: Sharon
authors.initials: S
authors.lastname: Gardner
authors.forename: Ira J
authors.initials: IJ
authors.lastname: Dunkel
authors.forename: Geoffrey B
authors.initials: GB
authors.lastname: McCowage
authors.forename: Blanca
authors.initials: B
authors.lastname: Diez
authors.forename: Jeffrey C
authors.initials: JC
authors.lastname: Allen
authors.forename: Anjali
authors.initials: A
authors.lastname: Gopalan
authors.forename: Albert S
authors.initials: AS
authors.lastname: Cornelius
authors.forename: Amanda
authors.initials: A
authors.lastname: Termuhlen
authors.forename: Minnie
authors.initials: M
authors.lastname: Abromowitch
authors.forename: Richard
authors.initials: R
authors.lastname: Sposto
authors.forename: Jonathan L
authors.initials: JL
authors.lastname: Finlay
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002528:Cerebellar Neoplasms / Q000188:drug therapy* / Q000401:mortality / Q000601:surgery; D002652:Child Behavior; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007223:Infant; D007361:Intelligence Tests; D008297:Male; D008527:Medulloblastoma / Q000188:drug therapy* / Q000401:mortality / Q000601:surgery; D009483:Neuropsychological Tests; D011788:Quality of Life; D015996:Survival Rate