The ether lipid miltefosine (hexadecylphosphocholine) was orally given to patients with various tumours in a dose-finding study. All patients initially received a daily total dose of 100 mg, which in the absence of side-effects was increased to 150 mg and further to 200 mg. A total of 54 patients were entered and were evaluable for gastrointestinal toxicity. Nausea and vomiting were found to be dose-limiting; 22% of patients ultimately tolerated a dose of 100 mg, 59% tolerated a dose of 150 mg and 19% tolerated a dose of 200 mg. In addition 30% of patients developed renal dysfunction, which was thought to be related to the drug. No other toxities were observed. For further phase II studies it is recommended that one starts with a dose of 150 mg daily, divided over three administrations.

Dose finding, Hexadecylphosphocholine, Miltefosine, Solid tumours,
Journal of Cancer Research and Clinical Oncology
Department of Medical Oncology

Verweij, J, Planting, A.S.Th, van der Burg, M.E.L, & Stoter, G. (1992). A dose-finding study of miltefosine (hexadecylphosphocholine) in patients with metastatic solid tumours. Journal of Cancer Research and Clinical Oncology, 118(8), 606–608. doi:10.1007/BF01211805