Randomized, double-blind, placebo-controlled, multicenter trial of 6% miltefosine solution, a topical chemotherapy in cutaneous metastases from breast cancer

Purpose: To compare 6% miltefosine solution (Miltex, Asta Medica, Frankfurt , Germany), a new topical cytostatic drug, with placebo as palliative treat ment for cutaneous metastases from breast cancer. Patients and Methods. In a double-blind, placebo-controlled, multicenter ph ase III study, a total of 52 patients with inoperable progressive skin. les ions from histologically or cytologically confirmed breast cancer, not mana geable by radiotherapy or systemic treatment, with superficial or flat skin lesions (estimated depth of invasion less than or equal to 1 cm) were rand omized to receive either 6% miltefosine solution or placebo. The solution w as applied at the dose of 2 drops/10 cm(2). once daily during the first wee k and twice daily thereafter until treatment failure. Results. Treatment groups were well balanced for patient characteristics at study entry except for a small difference in age. Time to treatment failur e (TTF), the primary parameter of this study, showed miltefosine solution t o be significantly superior to placebo (P = .007); the median TTF in the mi ltefosine, solution group was nearly three times longer than that in the pl acebo group (56 days v 21 days). The rate of response based on intention to treat patients was 33.3% for miltefosine solution compared with 3.70% for placebo (P = .006). Cutaneous reactions were seen mainly in the miltefosine group, with the type and frequency similar to those observed in previous s tudies. Conclusion: 6% Miltefosine solution is confirmed as an effective palliative treatment option for cutaneous metastases from breast cancer. Skin reactio ns, when present, are well tolerated and only occasionally require cessatio n of treatment. (C) 2001 by American Society of Clinical Oncology.