Background: Though the pathology of optic pathway tumor is mostly pilocytic astrocytoma, the benign tumor behaves like malignant tumor because total resection is not feasible. Adjuvant chemotherapy might be a reasonable strategy for management of these low grade tumors which keep growing. We evaluate the responsiveness of optic pathway tumor to cisplatin-based chemotherapy. Methods: Patients with unresectable and progressive optic pathway tumors received conventional chemotherapy including cisplatin, etoposide, and vinblastine were enrolled in this study from 1992 to 2007. Patients treated with radiotherapy previously were excluded. Brain MRI was performed every 3 months to evaluate the objective response to chemotherapy. Results: There are seven girls and nine boys enrolled in this study. The median age at diagnosis was 30 months old (range from 3 months to 11 years old). The median follow-up duration was 81.5 months (range from 24 months to 14.5 years). The pathology showed pilocytic astrocytomas in 11 patients, astrocytoma in one patient, and anaplastic astrocytomas in two patients. The 6-month progression-free survival (PFS) is 100%, 12-month PFS is 81.3%, 3-year PFS is 71.4% and 5-year PFS is 55.5% respectively. The toxicity of the cisplatin-based chemotherapy showed mild bone marrow suppression in 13 patients (81.3%), infection in nine patients (56.3%), gastrointestinal discomfort in seven patients (43.8%), renal insufficiency in two patient (12.5%), cerebral salt wasting syndrome with hyponatremia in one patient (6.25%) and high pitch hearing loss in two patients (12.5%). Conclusion: Cisplatin-based chemotherapy is an effective regimen for control of progressive optic pathway tumors.
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