title: Do our current clinical trial designs help to guide clinical practice?
abstract: There is a markedly increasing contrast between the current refinement of nosologic classification of sarcoma integrating molecular typing with a growing number of subtypes and the usually standardized approach proposed in clinical practice guidelines for both local and systemic treatment. Although gastrointestinal stromal tumor (GIST), dermatofibrosarcoma protuberans, and a few other subtypes now have specific therapeutic strategies, the majority of sarcomas are still lumped together in clinical trials investigating novel therapeutic options. These trials may not provide sufficient information to guide routine clinical practice. Proof-of-concept trials exploring a targeted agent in a selected molecular subtype, randomized phase II trials, and trials focusing on histologic subtypes-all integrating translational research and aiming to identify the mechanisms of sensitivity and resistance to the treatment-are needed. This paper discusses the limitations of previous clinical trial designs to guide clinical practice in this complex context.
authors.affiliation: From the Department of Medicine, Centre Léon Bérard, Lyon, France; Unité INSERM 1052, Centre Léon Bérard, Lyon, France; Université Claude Bernard Lyon, Lyon, France.
authors.forename: Jean-Yves
authors.initials: JY
authors.lastname: Blay
authors.forename: Olfa
authors.initials: O
authors.lastname: Derbel
authors.forename: Isabelle
authors.initials: I
authors.lastname: Ray-Coquard
mesh_terms: D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D014408:Biomarkers, Tumor / Q000235:genetics / Q000378:metabolism; D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D017326:Clinical Trials, Phase III as Topic / Q000379:methods*; D023381:Endpoint Determination; D019317:Evidence-Based Medicine / Q000379:methods*; D006801:Humans; D025202:Molecular Diagnostic Techniques; D018579:Patient Selection; D057285:Precision Medicine; D011237:Predictive Value of Tests; D012107:Research Design; D012509:Sarcoma / Q000145:classification / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism / Q000401:mortality / Q000473:pathology; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D014408:Biomarkers, Tumor