Only pathologic complete response to neoadjuvant chemotherapy improves significantly the long term survival of patients with resectable esophageal squamous cell carcinoma - Final report of a randomized, controlled trial of preoperative chemotherapy versus surgery alone
E. Ancona et al., Only pathologic complete response to neoadjuvant chemotherapy improves significantly the long term survival of patients with resectable esophageal squamous cell carcinoma - Final report of a randomized, controlled trial of preoperative chemotherapy versus surgery alone, CANCER, 91(11), 2001, pp. 2165-2174
BACKGROUND. Surgery is the standard treatment for patients with resectable
esophageal carcinoma, but the long term prognosis of these patients is unsa
tisfactory. Some randomized trials of preoperative chemotherapy suggest tha
t the prognosis of patients who respond may be improved.
METHODS. This randomized, controlled trial compared patients with clinicall
y resectable esophageal epidermoid carcinoma who underwent surgery alone (A
rm A) with those who received preoperative chemotherapy (Arm B). Overall su
rvival and the prognostic impact of major response to chemotherapy were ana
lyzed. Forty-eight patients were enrolled in each arm. Chemotherapy consist
ed of two or three cycles of cisplatin (100 mg/m(2) on Day 1) and 5-fluorou
racil (1000 mg/m(2) per day continuous infusion on Days 1-5). In both study
arms, transthoracic esophagectomy plus two-field lymphadenectomy was perfo
rmed. The two groups were comparable in terms of patient characteristics.
RESULTS. Forty-seven patients were evaluable in each arm. The curative rese
ction rate was 74.4% (35 of 47 patients) in Arm A and 78.7% (37 of 47 patie
nts) in Arm B. Treatment-related mortality was 4.2% in both arms. The respo
nse rate to preoperative chemotherapy was 40% (19 of 47 patients), includin
g 6 patients (12.8%) who achieved a pathologic complete responses. Overall
survival was not improved significantly. The 19 patients in Arm B who respo
nded to chemotherapy and underwent curative resection had significantly bet
ter 3-year and 5-year survival rates (74% and 60%, respectively) compared w
ith both nonresponders (24% and 12%, respectively; P = 0.0002) and patients
in Arm A who underwent complete resection (46% and 26%, respectively; P =
0.01): Patients who achieved a pathologic complete response (P = 0.01), but
not those who achieved a partial response (P = 0.2), had significantly imp
roved survival.
CONCLUSIONS. Patients with resectable esophageal carcinoma who underwent pr
eoperative chemotherapy and obtained a pathologic complete response had a s
ignificantly improved long term survival. Major efforts should be undertake
n to identify patients before neoadjuvant treatments who are likely to resp
ond. Cancer 2001;91:2165-74, (C) 2001 American Cancer Society.