TREATMENT OF PATIENTS WITH PINEALOBLASTOMA WITH HIGH-DOSE CYCLOPHOSPHAMIDE

The outcome for patients with pineoblastoma has historically been very poor, with most patients dying of disseminated disease despite irradi ation. Furthermore, the low incidence of this tumor has hindered progr ess toward defining better treatment strategies. Here we report the ac tivity and toxicity of cyclophosphamide administered as a single agent at a dose schedule of 2 g/m(2)/day for 2 successive days at monthly i ntervals for a maximum of four courses. Eight patients were evaluated, six newly diagnosed and two recurrent. Amongst the six newly diagnose d patients, there were three patients demonstrating partial responses, and three had stable disease throughout the cyclophosphamide treatmen t period. All six patients are alive and disease free after further th erapy. One patient with recurrent disease demonstrated tumor progressi on on cyclophosphamide, and the other had stable disease throughout th e cyclophosphamide treatment period. Both patients subsequently died o f progressive disease. The major toxicity of high dose cyclophosphamid e was hematopoietic, with one patient requiring a dose reduction after three courses due to prolonged thrombocytopenia. One patient was also withdrawn from treatment with cyclophosphamide due to impaired pulmon ary function. This study demonstrates the activity of high dose cyclop hosphamide in the treatment of pineoblastoma and may serve as basis fo r the design of future studies of this tumor. (C) 1996 Wiley-Liss, Inc .