BACKGROUNDCurrently, the survival of patients age > 70 years with glioblastoma multiforme (GBM) ranges from 4 months to 6 months, although radiotherapy and/or chemotherapy may prolong survival in certain subgroups. Temozolomide is an oral chemotherapeutic agent with efficacy against malignant gliomas and a favorable safety profile. This open‐label, single‐center, Phase II study was designed to evaluate the efficacy and safety of temozolomide as first‐line chemotherapy and exclusive treatment in elderly patients with newly diagnosed GBM.METHODSChemotherapy‐naïve patients (age > 70 years) were treated with temozolomide at a dose of 150–200 mg/m2 per day for 5 consecutive days of a 28‐day cycle until they developed disease progression. No radiation therapy was administered. The primary endpoint was median overall survival (OS); secondary endpoints included progression‐free survival (PFS) and toxicity.RESULTSThirty‐two patients (median age, 75 years; median Karnofsky performance status, 70) experienced a median OS of 6.4 months and a median PFS of 5.0 months. Of 29 patients who were assessed for response, 9 patients (31%) achieved a partial response, 12 patients (41%) maintained stable disease, and 8 patients (28%) developed progressive disease. Adverse events primarily were mild, with NCI CTC Grade 3–4 thrombocytopenia and neutropenia reported to occur in 6% and 9% of patients, respectively. No neurotoxicity was observed. Treatment delays and dose reductions occurred in 13% and 14% of cycles, respectively.CONCLUSIONSTemozolomide represents a safe, easily administered, and effective therapeutic approach for elderly patients with newly diagnosed GBM. Cancer 2004. © 2004 American Cancer Society.