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 Véronique Laithier, Jacques Grill, Marie-Cécile Le Deley, Marie-Madeleine Ruchoux, Dominique Couanet, François Doz, Fabienne Pichon, Hervé Rubie, Didier Frappaz, Jean-Paul Vannier, Annie Babin-Boilletot, Eric Sariban, Pascal Chastagner, Michel Zerah, Marie-Anne Raquin, Olivier Hartmann, Chantal Kalifa From the Departments of Pediatric and Adolescent Medicine, Public Health and Biostatistics, and Radiology, Institut Gustave Roussy, Villejuif; Department of Pediatric Oncology-Hematology, University Hospital, Besançon; Laboratory of Neuropathology, Roger Salengro Hospital; Department of Pediatrics, Oscar Lambret Center, Lille; Department of Pediatrics, Curie Institute; Department of Pediatric Neurosurgery, Necker Hospital, Paris; Department of Hematology-Oncology, Children’s Hospital, Toulouse; Department of Pediatrics, Léon Bérard Center, Lyon; Department of Pediatric Hematology-Oncology, University Hospital, Rouen; Department of Pediatric Hematology-Oncology, Hautepierre Hospital, Strasbourg; Department of Pediatric Hematology-Oncology, Children’s Hospital, Vandoeuvre, France; and Department of Pediatric Hematology-Oncology, Queen Fabiola Hospital, Bruxelles, Belgium. Address reprint requests to Jacques Grill, MD, Département de Cancérologie de l’Enfant et de l’Adolescent, Institut Gustave Roussy, 39, rue Camille Desmoulins, 94805 Villejuif, France; e-mail: grill@igr.fr. Purpose: 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. Patients and 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 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. Conclusion: 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. Supported by a grant from the Fondation de France and Fédération Nationale des Centres de Lutte Contre le Cancer to V.L. Journal of Clinical Oncology, Vol 21, Issue 24 (December), 2003: 4572-4578 Remember we are NOT Doctors and have NO medical training. This site is like an Encylopedia - there are many pages, many links on many topics. Support our work with any size DONATION - see left side of any page - for how to donate. You can help raise awareness of CAM.