PROs with Durvalumab plus GemCis in Advanced BTCs: The TOPAZ-1 Study

September 2022, Vol 3, No 3

Results of the randomized, double-blind, global, phase 3 TOPAZ-1 trial demonstrated that the first-line chemoimmunotherapy regimen of the PD-L1 inhibitor durvalumab plus gemcitabine/cisplatin (GemCis; median follow-up, 16.8 months) significantly improved overall survival (OS) versus placebo plus GemCis (median follow-up, 15.9 months) in patients with advanced biliary tract cancers (BTCs).1 A preplanned secondary objective of the TOPAZ-1 trial was to assess patient-reported outcomes (PROs) for the 2 treatment arms, and results of this analysis were presented at the 2022 ASCO annual meeting.

In the TOPAZ-1 trial, 685 patients with BTC were randomly assigned 1:1 to receive durvalumab (n = 341; 1500 mg) or placebo (n = 344) plus GemCis (gemcitabine 1000 mg/m2; cisplatin 25 mg/m2) for up to 8 cycles, followed by durvalumab or placebo alone until disease progression or unacceptable toxicity. PROs were assessed with the European Organisation for Research and Treatment of Cancer 30-item Quality of Life questionnaire (EORTC QLQ-C30) and the BTC-specific questionnaire consisting of a 21-item module (EORTC QLQ-BIL21). Time to deterioration (TTD) was the primary assessment of PROs, defined as the time from randomization to the date of the first prespecified, clinically meaningful deterioration in a PRO that is confirmed at a subsequent visit. The PRO analysis set included all patients from the full analysis set who completed a questionnaire.

For both the EORTC QLQ-C30 and QLQ-BIL21 PRO questionnaires, completion rates were high at baseline (>81%) and remained high (>70% for the majority of time points over 28 cycles) for both treatment groups. Baseline scores were comparable between treatment groups for EORTC QLQ-C30 and QLQ-BIL21 scales.

Across all visits, no clinically meaningful detriment in quality of life (QOL) was seen, including functional domains and symptom scores, with durvalumab versus placebo as assessed by adjusted mean change from baseline for EORTC QLQ-C30 and QLQ-BIL21 scores. Adjusted mean change from baseline in Global Health Status/QOL was higher with durvalumab, indicating a trend toward improved QOL with durvalumab versus placebo.

Median TTD of Global Health Status/QOL as assessed by EORTC QLQ-C30 was numerically longer with durvalumab than placebo (7.4 months vs 6.7 months; hazard ratio, 0.87; P = .279), with separation between the 2 curves occurring at approximately 7 months and favoring durvalumab.

Based on these results, the authors concluded that the addition of durvalumab to GemCis improved OS with no detriment in QOL, supporting durvalumab plus GemCis as a new first-line treatment option for patients with advanced BTCs.


  1. Oh D-Y, He AR, Qin S, et al. Durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer. NEJM Evid. 2022;1(8). Published online June 1, 2022.

Source: Burris HA, Okusaka T, Vogel A, et al. Patient-reported outcomes for the phase 3 TOPAZ-1 study of durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer. Abstract 4070.

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