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Correlation between efficacy endpoints in patients with advanced biliary tract cancer treated by systemic second-line therapies: Analysis of aggregated data from a systematic literature review - 24/11/22

Doi : 10.1016/j.clinre.2022.102010 
Cindy Neuzillet a, , David Malka b, Astrid Lièvre c, Inderpreet Singh Khurana d, Dewi Vernerey e
a Institut Curie, Versailles Saint-Quentin University - Paris Saclay University, Saint-Cloud, France 
b Institut Mutualiste Montsouris, Paris-Saclay University, Paris, France 
c Department of Gastroenterology, Rennes University Hospital, University of Rennes 1, INSERM Unité 1242, Rennes, France 
d BresMed Health Solutions Pvt. Ltd., India 
e Methodology and Quality of Life Unit in Oncology, University Hospital of Besançon, Besançon, France, Bourgogne Franche-Comté University, INSERM, EFS BFC, UMR1098, Interactions Hôte-Greffon-Tumeur/Ingénierie Cellulaire et Génique, Besançon, France 

Corresponding author: Cindy NEUZILLET, GI Oncology, Medical Oncology Department, Curie Institute, Versailles Saint-Quentin University - Paris Saclay University, 35 rue Dailly, 92210, Saint-Cloud, FranceGI OncologyMedical Oncology DepartmentCurie InstituteVersailles Saint-Quentin University - Paris Saclay University35 rue DaillySaint-Cloud92210France

Highlights

New effective drugs for biliary tract cancer (BTC) are emerging from genomic analyses.
Genomic-based therapies are directed toward small subsets of patients.
We found a strong correlation between overall response rate (ORR) and overall survival (OS).
A 10%-ORR gain in second line translated into a 4.7-month benefit in OS.
This should be considered when designing new clinical trials for pretreated advanced BTC.

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Abstract

Background

Overall response rate (ORR) and progression-free survival (PFS) are commonly used as endpoints for phase II trials. However, the ultimate goal is to bring survival benefit for the patients. We aimed to assess the correlation between ORR, median PFS and overall survival (OS) using aggregated data from a systematic review of second-line systemic therapies in advanced biliary tract cancer (BTC) patients.

Methods

Clinical trials were identified using Medline database. Studies only enrolling patients with gallbladder cancer were not included. Searches were last updated on April 2020. Eligible studies reported OS, PFS and/or ORR data for BTC patients receiving second-line systemic chemotherapy. Pearson weighted correlation was estimated between OS and ORR and between median OS and PFS.

Results

Seventeen studies (N = 912 patients) were selected. There was a strong correlation between median OS/ORR in the overall analysis (r = 0.85; P < 0.0001), both for trials with chemotherapy (r = 0.90; P=0.0152) and targeted therapy (r = 0.84; P = 0.0006). In contrast, the correlation between median OS/PFS, albeit significant in the overall analysis (r = 0.80; P < 0.0001), remained significant only for targeted therapies in the sensitivity analysis (r = 0.83; P = 0.0009).

Conclusions

ORR seems to be a more interesting intermediate endpoint in BTC in second line for both chemotherapy and targeted therapies, while PFS may be relevant only for targeted therapy trials. Further well-designed studies for surrogacy evaluation should be performed to confirm this observation.

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Keywords : Chemotherapy, Cholangiocarcinoma, Endpoint, Correlation, Targeted therapy

Abbreviations : ORR, PFS, OS, BTC, iCCA, eCCA, FGFR, IDH, HRQoL, CR, RECIST, CI, PS


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Vol 46 - N° 10

Article 102010- décembre 2022 Retour au numéro
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