SUPERIORITY OF PCNU OVER AZQ IN THE TREATMENT OF PRIMARY BRAIN-TUMORS- RESULTS OF A PROSPECTIVE RANDOMIZED TRIAL-(81-20) BY THE BRAIN-TUMOR STUDY-GROUP

Purpose. A two-arm randomized clinical trial was performed to determin e the efficacy of PCNU and AZQ in the treatment of de novo or recurren t primary brain tumors. An additional objective was to gather informat ion on the administration and toxicity of these compounds, supplementi ng that obtained previously in phase I/II studies. Methods. During 198 2 and 1983 the Brain Tumor Study Group randomized 152 adult patients w ith primary brain tumors to receive PCNU 75-100 mg/m(2) intravenously (IV) every 8 weeks or AZQ 15 mg/m(2) IV once a week for 4 weeks, every 6-8 weeks. All patients who had not received 'full dose' radiotherapy before randomization received it concurrently with the first course o f protocol chemotherapy. The data were analyzed for the total randomiz ed population (RP), and for 130 patients in the valid study group (VSG ) formed by excluding 22 patients for whom the histologic diagnosis wa s not documented by central review. Results. Median survival times wer e 11.0 months for the PCNU group and 8.4 months for the AZQ group. The difference in survival curves was statistically significant for the R P (p = 0.01) and the VSG (p = 0.02). Life-table analysis of the VSG sh owed estimated 2-year survivals of 34% for PCNU and 11% for AZQ. The a dvantage of PCNU remained significant (p = 0.006) after adjustment for histopathologic category, age, initial performance status, and interv al from initial reported surgery. Myelosuppression was the principal t oxicity in both groups. Conclusion. PCNU conferred a significant survi val advantage to patients with newly diagnosed or recurrent primary br ain tumors compared to AZQ.