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Primary Chemotherapy for Intracranial Nongerminomatous Germ Cell Tumors: Results of the Second International CNS Germ Cell Study Group Protocol

From the Oncology Unit, The Children's Hospital at Westmead and the Discipline of Paediatrics and Child Health at the University of Sydney, Sydney, Australia; Adult and Pediatric Neuro-Oncology Program, The New York University Cancer Institute and Departments of Pediatrics and Pathology, Memorial Sloan-Kettering Cancer Center, New York, NY; and Gutierrez Children's Hospital, Buenos Aires, Argentina

Address reprint requests to Stewart J. Kellie, MB, BS, Oncology Unit, The Children's Hospital at Westmead, Locked Bag 4001, Westmead, NSW 2145, Australia; e-mail: stewartk{at}chw.edu.au

PURPOSE: The optimum therapy for intracranial nongerminomatous germ cell tumors (NGGCT) remains controversial. The primary objective of this study was to determine whether intensive cisplatin and cyclophosphamide-based combination chemotherapy was effective in patients with intracranial NGGCT.

PATIENTS AND METHODS: Twenty patients were enrolled, aged 5 to 41 years (median, 13 years). Initial therapy included two courses of Regimen A (cisplatin, etoposide, cyclophosphamide, and bleomycin). Patients achieving a complete remission (CR) then received two courses of Regimen B (carboplatin, etoposide, and bleomycin). Those in CR after four courses of treatment received one additional course of Regimen A and Regimen B, while those not in CR after four treatment courses underwent second-look surgery and/or irradiation.

RESULTS: Sixteen of 17 patients assessable for response after two courses of treatment achieved a CR or partial response (CR + partial response, 0.94; 95% CI, 0.73 to 1.0). With a median follow-up of 6.3 years, 14 of 20 patients are alive without disease; eight patients were without relapse or progression, of whom three received local irradiation in first complete remission in violation of protocol, and six patients were in durable second or third complete remission after further chemotherapy and/or irradiation. The 5-year overall survival and event-free survival were 0.75 (95% CI, 0.56 to 0.94) and 0.36 (95% CI, 0.13 to 0.59), respectively.

CONCLUSION: Intensive chemotherapy was effective in one-third of patients in this study. Salvage therapy, including irradiation, was feasible in patients with recurrent disease.

Authors' disclosures of potential conflicts of interest are found at the end of this article.

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