A pilot study investigated
topotecan (
Hycamtin, GlaxoSmithKline, Philadelphia, PA), a
topoisomerase I inhibitor, in treating uterine serous
carcinoma, a typically unresponsive aggressive
tumor. Fifteen patients were surgically staged, then treated with
topotecan (1.5 mg/m2, Days 1-5 every 21 days) as first-line
therapy (n = 12) or secondary to
platinum failure (n = 3). Patients received
topotecan through six courses,
disease progression, or unacceptable toxicity. Grade 3/4 hematologic toxicity prompted dose adjustments. Thirteen patients exhibited no gross evidence of residual disease postoperatively. At
topotecan initiation, one patient had 5-cm and one had < 1-cm residual disease. Seventy-eight courses (median, six) were administered; 12 (80%) patients completed the specified protocol. Common serious toxicities included grade 3
neutropenia (33%),
anemia (13%), and
thrombocytopenia (13%). Eight patients received
erythropoietin and/or
granulocyte colony-stimulating factor. Median follow-up for 14 evaluable patients was 26 months (range, 13-40). Of 11 evaluable first-line
topotecan patients, nine were alive at follow-up; five were disease-free. Of three second-line
topotecan patients, two died and one was alive with disease 31 months post-treatment. One patient with measurable disease achieved a complete and one a partial response as assessed by computed tomography scan. Median progression-free survival was 25 months; median survival has not been reached at 26 months. Although
topotecan's antitumor activity cannot yet be quantified, disease-free interval and survival outcomes compare favorably with other
therapies in uterine serous
carcinoma. Further evaluation of
topotecan in this population is warranted.