Abstract

The authors describe a large experience using short courses of irinotecan for palliative therapy in patients with neuroblastoma (NB). Quality of life was a major issue in choosing this regimen for patients whose disease was resistant to standard anti-NB therapies. A retrospective review was conducted of all patients who were followed by the Department of Pediatrics at Memorial Sloan-Kettering Cancer Center and treated for resistant NB with irinotecan at 50 mg/m2 per day for 5 days as a 1-hour intravenous infusion. Treatment was outpatient, and there was a minimum 2-week rest period between courses. Granulocyte colony-stimulating factor was used to keep the absolute neutrophil count >500-1000/mL. Forty-four patients had been treated aggressively and/or extensively before they received one or more five-day courses of irinotecan. Emetogenic, diarrheal, and myelosuppressive effects were readily managed. Hospitalizations were limited to three patients with bacteremia. Twenty-three patients had a change in therapy, although they did not have progressive disease (PD) after receiving 1 (n=10), 2 (n=3), 3 (n=1), 4 (n=6), 7 (n=1 patient), 22 (n=1 patient), or 24 (n=1) courses. The most common reasons for changing treatment were to intensify retrieval therapy or to pursue immunotherapy. Of those 23 patients, 15 patients had stable disease, 7 were not evaluable for response because of concurrent radiotherapy, and 1 patient had a major response. Twenty-one patients had PD after 1 (n=3, 2 (n=9), 4 (n=2), 5 (n=1), 6 (n=3), 7 (n=1), 9 (n=1), and 11 (n=1) courses. In heavily treated patients, the regimen studied was well tolerated, allowed patients to continue most normal life activities, and produced anti-NB effects. Its modest toxicity supported use with other antitumor agents.

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