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
dx.doi.org/10.1007/BF01211805, hdl.handle.net/1765/65501
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