Abstract

Nine trials of the antitumor chemotherapeutic agent, melphalan (L-phenylalanine mustard; Alkeran) were carried out in eight patients with the malignant carcinoid syndrome. In six trials, this drug was given on a low-dose, daily basis. In three of these trials, there was definite evidence of antitumor effect as determined by measurable decreases in liver size, which began within two to four weeks and lasted for between 6 and 12 months. In three trials, melphalan was given at a high intermittent dose, and two of the three patients in this group had a significant decrease in liver size. There were inconsistent changes in urinary 5-hydroxyindoleacetic acid excretion, but exacerbation of carcinoid symptoms after initiation of antitumor chemotherapy was the rule. Results of trials with other drugs in patients with malignant carcinoid tumors were also compared to results with melphalan.

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