DAILY ORAL MILTEFOSINE (HEXADECYLPHOSPHOCHOLINE) IN PATIENTS WITH ADVANCED BREAST-CANCER - A PHASE-II STUDY

Background: A clinical phase II study with an oral formulation of Miltefosine (INN of hexadecylphosphocholine) was performed in patients with locally advanced or metastatic breast cancer. Material and Methods: Eligibility criteria included histological proof of breast cancer, measurable disease without previous radiotherapy of the indicator lesions, pretreatment with chemotherapy, WHO performance status of grade less-than-or-equal-to 2, and informed consent. The 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 in the second week if tolerability was good. The minimum treatment duration was 9 weeks. Results: 23 patients have been entered into the study and 17 were evaluable for toxicity. 11 patients were evaluable for response. There was no objective tumor response. 4 out of 11 patients had reached a no-change status. Progressive disease was seen in 7 patients with 2 early deaths. Gastrointestinal side effects like nausea and vomiting (15/17) and loss of appetite (15/17) reached a maximum of WHO grade 4, diarrhea (4/17) was at the most of WHO grade 3. Conclusion: Miltefosine given daily by oral route showed no therapeutic activity in patients with advanced metastatic breast cancer.