Safety and efficacy of a multicenter study using intraarterial chemotherapy in conjunction with osmotic opening of the blood-brain barrier for the treatment of patients with malignant brain tumors

BACKGROUND. The aim of this study was to determine the safety and efficacy of intraarterial chemotherapy with osmotic opening of the blood-brain barri er (BBB) for the treatment of malignant brain tumors when administered acro ss multiple centers. METHODS. Patients with primary central nervous system lymphoma (PCNSL), pri mitive neuroectodermal tumor (PNET), germ cell tumor, cancer metastasis to the brain, or low or high grade glioma were eligible. Prior to entry, magne tic resonance imaging or computed tomography brain scan, medical history, n eurologic status, and Karnofsky performance status were reviewed at the coo rdinating center. Standardized anesthesia and intraarterial catheterization guidelines were followed by a multidisciplinary ream at each center. Betwe en March 1994 and November 1997, 5 universities treated 221 adult patients with intraarterial chemotherapy with or without osmotic opening of the BBB (2464 procedures). RESULTS. Of evaluable patients with PCNSL, 40 of 53 (75%) achieved complete response (CR). All evaluable patients with PNET (n = 17), metastatic disea se (n = 12), or germ cell tumor (n = 4) achieved stable disease (SD) or bet ter. Of 57 evaluable patients with glioblastoma multiforme, 45 (79%) achiev ed SD or better. Asymptomatic subintimal tear occurred in 11 of 221 patient s (5%), pulmonary embolism in 6 of 221 (2.7%), and renal toxicity in 4 of 2 21 (1.8%). One patient with extensive glioma expired within 48 hours after treatment. CONCLUSIONS. Using standard guidelines and protocols, intraarterial chemoth erapy with or without osmotic opening of the BBB is feasible across multipl e centers with a low incidence of carherer-related complications. Tn patien ts with chemotherapy-sensitive tumors, such as PCNSL, PNET, germ cell tumor , and cancer metastasis to the central nervous system, enhanced delivery re sults in a high degree of tumor response, with an efficacy profile that is reproducible across multiple centers. (C) 2000 American Cancer Society.