{"entity":{"id":"paper-libretto-531-nejm-2023","kind":"paper","name":"LIBRETTO-531: selpercatinib versus cabozantinib or vandetanib in advanced RET-mutant medullary thyroid cancer","aka":[],"tldr":"The selective RET inhibitor selpercatinib cut the risk of progression by more than 70 percent compared with the older multikinase drugs cabozantinib and vandetanib in RET-mutant medullary thyroid cancer, with fewer side effects.","summary":"Phase 3 trial of 291 patients with progressive advanced RET-mutant medullary thyroid cancer randomised 2:1 to selpercatinib or physician's choice of cabozantinib or vandetanib.\n\nProgression-free survival at 12 months was 86.8 versus 65.7 percent (hazard ratio 0.28), response 69.4 versus 38.8 percent, and treatment-failure-free survival also favoured selpercatinib; grade 3 or higher adverse events were 52.8 versus 76.3 percent.","asOf":"2026-09-17","links":[{"label":"N Engl J Med 2023","url":"https://doi.org/10.1056/NEJMoa2309719"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/37870969/"}],"tags":[],"related":[],"cancers":["medullary-thyroid-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":["selpercatinib"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["libretto-531"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["nejm"],"dependsOn":[],"notes":[],"journal":"New England Journal of Medicine","year":2023,"doi":"10.1056/NEJMoa2309719","pmid":"37870969","authors":"Hadoux J, Elisei R, Brose MS, et al.","paperType":"rct","findings":["Twelve-month progression-free survival 86.8 percent vs 65.7 percent; hazard ratio 0.28.","Objective response 69.4 percent vs 38.8 percent."],"whatItMeans":"Selpercatinib is the first-line standard for advanced RET-mutant medullary thyroid cancer; RET testing at diagnosis is essential.","caveats":["Overall survival data immature.","Open-label design."],"changedPractice":true,"participants":291},"route":"/key-papers/paper-libretto-531-nejm-2023/","neighbours":{"cancer":[{"id":"medullary-thyroid-cancer","kind":"cancer","name":"Medullary thyroid cancer","route":"/cancers/medullary-thyroid-cancer/"}],"drug":[{"id":"selpercatinib","kind":"drug","name":"Selpercatinib","route":"/drugs/selpercatinib/"}],"trial":[{"id":"libretto-531","kind":"trial","name":"LIBRETTO-531","route":"/trials/libretto-531/"}],"journal":[{"id":"nejm","kind":"journal","name":"New England Journal of Medicine","route":"/journals/nejm/"}]}}