Progression-free survival in children with optic pathway tumors: dependence on age and the quality of the response to chemotherapy--results of the first French prospective study for the French Society of Pediatric Oncology.
OBJECTIVE
To evaluate a strategy aimed at avoiding radiotherapy during first-line treatment of children with progressive optic pathway tumors (OPT), by exclusively administering multiagent chemotherapy during 16 months.


METHODS
Between 1990 and 1998, 85 children with progressive OPT were enrolled onto this multicenter nationwide trial. Chemotherapy alternating procarbazine plus carboplatin, etoposide plus cisplatin, and vincristine plus cyclophosphamide was given every 3 weeks. At the time of relapse or progression, second-line chemotherapy was authorized before recourse to radiotherapy.


RESULTS
Objective response rate (partial response [PR] + complete response [CR]) to chemotherapy was 42%. Five-year progression-free survival (PFS) and overall survival rates were 34% and 89%, respectively. The 5-year radiotherapy-free survival rate was 61%. In the multivariate analysis of the 85 patients that entered onto the study, factors associated with the risk of disease progression were age younger than 1 year at diagnosis (P =.047) and absence of neurofibromatosis type 1 (P =.035). In the multivariate analysis of the 74 patients that remained on study after the first cycle of chemotherapy, factors associated with the risk of disease progression were age younger than 1 year at diagnosis (P =.0053) and no objective response to chemotherapy (P =.0029). Three-year PFS was 44% in infants < or = 1 year versus 66% in children older than 1 year. Three-year PFS was 53% in the absence of an objective response to chemotherapy versus 68% after a PR or CR.


CONCLUSIONS
A significant proportion of children with OPT can avoid radiotherapy after prolonged chemotherapy. Deferring irradiation with chemotherapy protocols did not compromise overall survival of the entire population or visual function.
Laithier
Véronique
V
Department of Pediatric and Adolescent Medicine, Insitut Gustave Roussy, 39, rue Camille Desmoulins, 94805 Villejuif, France.
Grill
Jacques
J
Le Deley
Marie-Cécile
MC
Ruchoux
Marie-Madeleine
MM
Couanet
Dominique
D
Doz
François
F
Pichon
Fabienne
F
Rubie
Hervé
H
Frappaz
Didier
D
Vannier
Jean-Paul
JP
Babin-Boilletot
Annie
A
Sariban
Eric
E
Chastagner
Pascal
P
Zerah
Michel
M
Raquin
Marie-Anne
MA
Hartmann
Olivier
O
Kalifa
Chantal
C
D000367:Age Factors; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D016009:Chi-Square Distribution; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018572:Disease-Free Survival; D005163:Factor Analysis, Statistical; D005260:Female; D005602:France; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D019574:Optic Nerve Neoplasms / Q000188:drug therapy*; D011446:Prospective Studies; D016019:Survival Analysis