Background: Recurrent cutaneous breast cancer is difficult to manage, with
surgery, radiotherapy and systemic therapy all having their limitations. Mi
ltefosine is a topical cytostatic agent which may provide an alternative ap
proach in its treatment. Patients and methods: Patients with previously tre
ated progressive cutaneous lesions from breast cancer were treated with mil
tefosine on a named-patient compassionate supply basis. Miltefosine was app
lied topically to the skin at a dose of 2 drops/10 cm(2) skin area. Results
: Twenty-five patients were treated, most of whom had been heavily pre-trea
ted. Treatment was continued for a median of 14 weeks (range 2-164). In 7 p
atients grade I skin toxicities were observed, and in 4 patients grade 3 lo
cal toxicities necessitated dose adjustments. A response was seen in 9 pati
ents (1 complete response, 2 partial responses, 6 minor responses) giving a
total response rate of 36%, with stable disease in 11 patients (44%) and p
rogressive disease in 5 (20%). Those lesions which were superficial or <2 c
m in diameter were most likely to respond. Conclusions: Miltefosine, either
used alone or in conjunction with other therapies for distant metastases,
is an effective and tolerable local treatment for cutaneous breast cancer.