Daily Oral Miltefosine (Hexadecyl-Phosphocholine) in Patients with Advanced Non-Small Cell Lung Cancer. A Phase II Study

Abstract

A clinical phase II trial with an oral formulation of miltefosine (MIL) was performed in patients with non-small cell lung cancer (NSCLC). Eligibility criteria for this phase II study included histological proof of NSCLC, measurable locally advanced or metastatic disease without previous radiotherapy of the indicator lesions, no chemotherapy for the diagnosis of cancer prior to entry into the study, WHO performance status of grade < 2, and informed consent. The capsulated drug was given twice daily at a single dose of 50 mg for the first week with the option of increasing the daily dose to 150 mg (50 mg×3) in the second week if tolerability was good. The minimum intended treatment duration was 9 weeks. Twenty-six patients have been entered into the study and 25 are evaluable for toxicity (WHO criteria). Twenty-one patients are evaluable for toxicity and response (WHO criteria). There was 1 PR and 3 patients had a NC of their disease with a duration exceeding twice the period of time for which progression was documented prior to entry into this study (criteria of the AIO phase I/II-study group). Progressive disease was observed in 17 patients with 3 early deaths. Severe side effects were seen in the gastrointestinal tract as abdominal pain, loss of appetite, nausea and vomiting (up to grade 4, WHO) or diarrhea (up to grade 2, WHO). In conclusion, oral MIL given daily showed no major therapeutic activity in patients with NSCLC.