An assessment of the benefit-risk balance of FOLFIRINOX in metastatic pancreatic adenocarcinoma
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Julien Péron1,2, Pascal Roy1, Thierry Conroy3, Françoise Desseigne4, Marc Ychou5, Sophie Gourgou-Bourgade5, Trevor Stanbury6, Laurent Roche1, Brice Ozenne1, Marc Buyse7
1CNRS, UMR 5558, Laboratoire de Biométrie et Biologie Evolutive, Equipe Biostatistique-Santé, Service de Biostatistiques, Centre Hospitalier Lyon-Sud, Hospices Civils de Lyon, 69495 Pierre-Bénite, France
2Medical Oncology Department, Centre Hospitalier Lyon-Sud, Institut de Cancérologie des Hospices Civils de Lyon-IC-HCL, 69495 Pierre-Bénite, France
3Institut de Cancérologie de Lorraine, Alexis Vautrin Center, 54500 Vandœuvre-lès-Nancy, France
4Medical Oncology Department, Centre Léon Bérard, 69373 Lyon, France
5Institut Régional du Cancer Montpellier, Val d’Aurelle, 34298 Montpellier, France
6R&D UNICANCER, 75013 Paris, France
7International Drug Development Institute (IDDI), San Francisco, CA 94109, USA
Julien Péron, email: email@example.com
Keywords: multi-criteria analysis, pancreatic cancer, benefit-risk assessment, randomized controlled trial, statistics as topic
Received: July 28, 2016 Accepted: September 29, 2016 Published: October 19, 2016
Background: We sought to assess the benefit-risk balance of FOLFIRINOX versus gemcitabine in patients with metastatic pancreatic adenocarcinoma.
Methods: We used generalized pairwise comparisons. This statistical method permits the simultaneous analysis of several prioritized outcome measures. The first priority outcome was survival time (OS). Differences in OS that exceeded two months were considered clinically relevant. The second priority outcome was toxicity. The overall treatment effect was quantified using the net chance of a better outcome, which can be interpreted as the net probability for a random patient treated in the FOLFIRINOX group to have a better overall outcome than a random patient in the gemcitabine group.
Results: In this trial 342 patients received either FOLFIRINOX or gemcitabine. The net chance of a better outcome favored strongly and significantly the FOLFIRINOX group (24.7; P<.001), suggesting a favorable benefit-risk balance of FOLFIRINOX versus gemcitabine. The positive benefit-risk balance of FOLFIRINOX was observed throughout all sensitivity analyses.
Conclusions: Generalized pairwise comparisons are useful to perform a quantitative assessment of the benefit-risk balance of new treatments. It provides a clinically intuitive way of comparing patients with respect to all important efficacy and toxicity outcomes. Overall the benefit-risk balance of FOLFIRINOX was strongly positive.
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