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.
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.
Dhall
Girish
G
Childrens Hospital Los Angeles, Los Angeles, California, USA. gdhall@chla.usc.edu
Grodman
Howard
H
Ji
Lingyun
L
Sands
Stephen
S
Gardner
Sharon
S
Dunkel
Ira J
IJ
McCowage
Geoffrey B
GB
Diez
Blanca
B
Allen
Jeffrey C
JC
Gopalan
Anjali
A
Cornelius
Albert S
AS
Termuhlen
Amanda
A
Abromowitch
Minnie
M
Sposto
Richard
R
Finlay
Jonathan L
JL
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