We performed a phase II study with mitoxantrone in patients with carcinoid tumours, islet cell tumours and medullary carcinomas of the thyroid. Thirty-five eligible patients received mitoxantrone 12 mg m(-2) i.v. every 3 weeks. Among 18 previously untreated patients, three responded (17%, 95% CI=4-41%); no responses were achieved in 17 previously treated patients. Of the 21 patients who had carcinoid tumours, 11 were previously untreated and two achieved a response (18%, 95% CI=2-52%). Overall response rate was 9% (95% CI=2-23%). At a median follow-up of 43 months, median overall survival was 16 months. The median survival of 21 patients with a normal alkaline phosphatase was 29 months and 9 months for 14 patients with elevated serum levels (P = 0.005). A similar observation was noticed for gamma-glutamyltransferase (P = 0.007). We concluded that mitoxantrone is not active in APUD tumours. Elevated alkaline phosphatase and gamma-glutamyltransferase are associated with a poor prognosis.
Neijt, JP. ; Lacave, AJ. ; Splinter, TAW. ; Taal, BG. ; Veenhof, CHN. ; et. al. Mitoxantrone in Metastatic Apudomas - a Phase-ii Study of the Eortc Gastrointestinal Cancer Cooperative Group. In: The British journal of cancer, Vol. 71, no. 1, p. 106-108 (1995)