TOPAZ-1
Trial question
What is the role of durvalumab plus chemotherapy in patients with advanced biliary tract cancer?
Study design
Multi-center
Double blinded
RCT
Population
Characteristics of study participants
50.0% female
50.0% male
N = 685
685 patients (340 female, 345 male).
Inclusion criteria: patients with previously untreated unresectable or metastatic biliary tract cancer or with recurrent disease.
Key exclusion criteria: ampullary carcinoma; active or prior documented autoimmune or inflammatory disorders; known allergy or hypersensitivity to any study treatment.
Interventions
N=341 durvalumab (1,500 mg durvalumab plus gemcitabine and cisplatin).
N=344 placebo (matching placebo plus gemcitabine and cisplatin).
Primary outcome
Overall survival
41.9%
34.3%
41.9 %
31.4 %
20.9 %
10.5 %
0.0 %
Durvalumab
Placebo
Significant
increase ▲
NNT = 13
Significant increase in overall survival (41.9% vs. 34.3%; HR 1.22, 95% CI 1.03 to 1.52).
Secondary outcomes
Significant increase in progression-free survival (7.2 months vs. 5.7 months; HR 1.33, 95% CI 1.12 to 1.59).
Significant increase in objective response rate (26.7% vs. 18.7%; OR 1.6, 95% CI 1.11 to 2.31).
Safety outcomes
No significant difference in grade 3 or 4 adverse events.
Conclusion
In patients with previously untreated unresectable or metastatic biliary tract cancer or with recurrent disease, durvalumab was superior to placebo with respect to overall survival.
Reference
Do-Youn Oh, Aiwu Ruth He, Shukui Qin et al. Durvalumab plus Gemcitabine and Cisplatin in Advanced Biliary Tract Cancer. NEJM Evid. 2022 Aug;1(8):EVIDoa2200015.
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