Objectives: Glioblastoma multiforme (GBM) care is surgery followed by radiotherapy (RT) plus temozolomide (TMZ) chemotherapy. At the recurrence no standard chemotherapy is actually available. Fotemustine is a third-generation nitrosourea with high brain permeability coefficient. The aim of the present study was to evaluate
efficacy and toxicity of fotemustine as rescue therapy in patients with ... [Show full abstract] histologically confirmed GBM, recurring or progressing, after loco-regional treatment and first-line TMZ chemotherapy. Patients: 23 pts (median age 54,2 years (range 28-72,5); median KPS 80) were considered eligible. After surgery, 14/23 pts were recurred or progressed after RT plus concomitant and adjuvant TMZ first-line chemotherapy, 9/23 pts after RT plus
adjuvant TMZ. The primary end-point was progression-free survival at 6 months. Treatment consisted of fotemustine induction infusion (100mg/m2 days 1,8 and 15) with five weeks rest. If pts responded or were stabilized at magnetic resonance imaging control (MRI), fotemustine was continued at the same dose every three weeks until the progression. Results: The median time to progression (TTP) was 4 months; the median survival was 6 months for all pts. The progression-free survival (PFS) 6-12 months was 31.8% and 9% respectively; overall survival (OS) 6-12 months was 59% and 13% respectively. PFS-6 for 14/23 pts with RT plus concomitant and adjuvant TMZ was 28.57%; median time to progression was 17 weeks. Three partial responses (PR) (13.6%) and nine stable diseases (SD) (40.9%) were obtained. Main toxicity was haematologic (leucopenia and, above all, thrombocytopenia). Conclusions: Fotemustine seems active and no-cross resistant in pts with GBM recurrent or progressive after TMZ-based chemotherapy.