TREATMENT OF DIENCEPHALIC SYNDROME WITH CHEMOTHERAPY - GROWTH, TUMOR RESPONSE, AND LONG-TERM CONTROL

BACKGROUND. The diencephalic syndrome (DS), which is manifested by pro gressive emaciation and failure to thrive in an apparently alert, chee rful infant, usually is due to a low grade hypothalamic glioma. Treatm ent with aggressive surgery and/or radiotherapy is variably successful in controlling disease and may result in severe neurologic sequelae. Chemotherapy recently has been shown to be effective in patients with low grade gliomas of childhood, but it is used infrequently in those w ith DS. METHODS, The authors evaluated the efficacy of a regimen of ca rboplatin and vincristine on improving weight, causing tumor shrinkage , and delaying the need for alternative therapies in seven children (a ges 9-20 months; median age, 11 months) with DS. Five patients weighed less than the 5th percentile for their age at the start of the study, one weighed within the 10th percentile, and one weighed within the 25 th percentile. RESULTS. At follow-up (range, 6-54 months; median, 28 m onths), the patients' weights had increased by 66-95% (median, 80%). O n magnetic resonance imaging, four patients had a >50% reduction in tu mor mass, one had a 25-50% reduction, and two had stable disease. In t hose patients with radiographic response to treatment, weight gain was accomplished with oral feedings in four of five patients, whereas tho se with stable disease required nasogastric, nasojejunal, or gastrosto my tube supplementation to maintain weight. Disease progression occurr ed at a median of 24 months after initiation of chemotherapy, and two patients remained free of progressive disease at last follow-up. Five patients were alive a median of 59 months from diagnosis. The need for radiation or other therapies was delayed in six of seven children. Th erapy was tolerated without significant toxicities. CONCLUSION. The au thors conclude that treatment of DS with a carboplatin and vincristine regimen results in demonstrable weight gain, may result in tumor shri nkage, and in some cases, significantly delays the need for alternativ e therapies. (C) 1998 American Cancer Society.