Trifluridine/tipiracil polypill shows modest clinical activity against refractory ESCC

The fixed-dose combination of trifluridine/tipiracil may be feasible for use in the treatment of patients with refractory esophageal squamous cell carcinoma (ESCC), with the drug showing modest clinical activity in the phase II ECTAS study.

ECTAS enrolled 42 patients across seven hospitals in Japan. All patients had unresectable advanced/recurrent esophageal cancer that was refractory or intolerant to 5-fluorouracil, platinum-containing regimens, and taxanes. All tumours were squamous cell carcinomas.

The primary endpoint was progression-free survival (PFS) rate at 3 months. The secondary endpoints were the 6-month PFS rate, PFS, overall survival, response rate, disease control rate, and toxicity.

The PFS rates with trifluridine/tipiracil were 15.4 percent (90 percent confidence interval [CI], 7.4–26.0) at 3 months and 7.7 percent (90 percent CI, 2.6–16.6) at 6 months. The median PFS was 1.3 months (95 percent CI, 1.0–1.8), while the median overall survival was 4.5 months (95 percent CI, 3.6–5.7).

The response rate was 0 percent, while the disease control rate was 23.8 percent (95 percent CI, 13.5–38.5).

In terms of safety, the major grade 3–4 toxicities included neutropoenia (47.6 percent), leukocytopenia (35.7 percent), and anaemia (21.4 percent). There were no reports of treatment-related deaths. Exploratory subgroup analyses showed better PFS in the subgroup without distant metastasis at diagnosis.