Emily Menendez

GI Oncology Now

A final analysis of the phase 3 GAIN trial <a href="https://meetings.asco.org/abstracts-presentations/245459" target="_blank" rel="noopener">presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting</a> has provided new insight into the efficacy of neoadjuvant/perioperative gemcitabine with cisplatin for patients with biliary tract cancer (BTC).
Although radical surgical resection serves as the only potentially curative treatment for patients with BTC and incidental gallbladder cancer, the 5-year overall survival (OS) rate of this procedure is only 20% to 40%, and data regarding pure adjuvant chemotherapy in BTCs are inconsistent.
The GAIN trial began as a result of prior encouraging data on neoadjuvant/perioperative treatment concepts in other diseases. The trial included 68 patients with localized or locally advanced resectable nonmetastatic BTC who were randomized to receive either neoadjuvant/perioperative systemic chemotherapy consisting of gemcitabine plus cisplatin for three cycles before and after surgery followed by radical surgery in arm A or to undergo direct surgery followed by adjuvant treatment in arm B.
The primary end point of the study was OS, and secondary end points included progression-free survival/event-free survival (PFS/EFS), R0 resection rate, toxicity, perioperative morbidity, mortality, and quality of life. Because of a slow enrollment rate, recruitment for the trial was stopped after 68 patients.
The intention-to-treat population included 32 patients in arm A and 30 patients in arm B. Adjuvant treatment was completed in 43.8% of patients in arm A. Of the patients in arm B, 23.3% received adjuvant treatment.
With a median follow-up of 11.8 months, neoadjuvant treatment was found to improve OS (median OS: 27.8 in arm A vs 14.6 months in arm B; hazard ratio, 0.46 [95% CI, 0.22-0.96]; P=0.04) and R0 resection rate (62.5% vs 33.3%), and a similar effect was seen in EFS.
Postoperative morbidity rates were similar in both arms: 33.3% in arm A versus 32% in arm B. The 30- and 90-day mortality rates were lower for arm A than arm B (30-day: 4.2% vs 24%; 90-day: 4.2% vs 28%, respectively). No new safety/toxicity signals were observed. At least one treatment-related adverse event (TRAE) occurred in 12 patients (38.7%) at grade 3 and in one patient (3.2%) at grade 4. No fatal TRAEs were observed.
The use of neoadjuvant/perioperative gemcitabine with cisplatin can boost OS and R0 resection rates in patients with BTC versus direct surgery and can almost double median OS and decrease mortality rates.

A final analysis of the phase 3 GAIN trial <a href="https://meetings.asco.org/abstracts-presentations/245459" target="_blank" rel="noopener">presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting</a> has provided new insight into the efficacy of neoadjuvant/perioperative gemcitabine with cisplatin for patients with biliary tract cancer (BTC).Although radical surgical resection serves as the only potentially curative treatment for patients with BTC and incidental gallbladder cancer, the 5-year overall survival (OS) rate of this procedure is only 20% to 40%, and data regarding pure adjuvant chemotherapy in BTCs are inconsistent.The GAIN trial began as a result of prior encouraging data on neoadjuvant/perioperative treatment concepts in other diseases. The trial included 68 patients with localized or locally advanced resectable nonmetastatic BTC who were randomized to receive either neoadjuvant/perioperative systemic chemotherapy consisting of gemcitabine plus cisplatin for three cycles before and after surgery followed by radical surgery in arm A or to undergo direct surgery followed by adjuvant treatment in arm B.The primary end point of the study was OS, and secondary end points included progression-free survival/event-free survival (PFS/EFS), R0 resection rate, toxicity, perioperative morbidity, mortality, and quality of life. Because of a slow enrollment rate, recruitment for the trial was stopped after 68 patients.The intention-to-treat population included 32 patients in arm A and 30 patients in arm B. Adjuvant treatment was completed in 43.8% of patients in arm A. Of the patients in arm B, 23.3% received adjuvant treatment.With a median follow-up of 11.8 months, neoadjuvant treatment was found to improve OS (median OS: 27.8 in arm A vs 14.6 months in arm B; hazard ratio, 0.46 [95% CI, 0.22-0.96]; P=0.04) and R0 resection rate (62.5% vs 33.3%), and a similar effect was seen in EFS.Postoperative morbidity rates were similar in both arms: 33.3% in arm A versus 32% in arm B. The 30- and 90-day mortality rates were lower for arm A than arm B (30-day: 4.2% vs 24%; 90-day: 4.2% vs 28%, respectively). No new safety/toxicity signals were observed. At least one treatment-related adverse event (TRAE) occurred in 12 patients (38.7%) at grade 3 and in one patient (3.2%) at grade 4. No fatal TRAEs were observed.The use of neoadjuvant/perioperative gemcitabine with cisplatin can boost OS and R0 resection rates in patients with BTC versus direct surgery and can almost double median OS and decrease mortality rates.

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The use of neoadjuvant/perioperative gemcitabine with cisplatin can boost OS and R0 resection rates in patients with BTC.