title: End points and statistical considerations in immuno-oncology trials: impact on multiple myeloma.
abstract: Unlike conventional cancer treatment, immuno-oncology therapies are commonly associated with delayed clinical benefit and durable responses, as seen with immuno-oncology therapies for multiple myeloma (MM). Therefore, a longer-term approach to immuno-oncology data assessment is required. Appropriate study designs, end points and statistical methods are essential for evaluating immuno-oncology therapies to assess treatment outcomes, and may better accommodate immuno-oncology clinical trial data. In addition to conventional end points including median progression-free survival (PFS) and overall survival (OS), end points such as hazard ratios for PFS and OS over time, PFS and OS landmark analyses beyond the median, and immune-response end points might provide better indications of the efficacy of immuno-oncology therapies. Long-term data with these agents will allow better prediction of outcomes in MM.
authors.affiliation: Cancer Research And Biostatistics (CRAB), Seattle, WA, USA.
authors.forename: Antje
authors.initials: A
authors.lastname: Hoering
authors.affiliation: Samuel Oschin Cancer Center, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
authors.forename: Brian
authors.initials: B
authors.lastname: Durie
authors.affiliation: Cancer Research And Biostatistics (CRAB), Seattle, WA, USA.
authors.forename: Hongwei
authors.initials: H
authors.lastname: Wang
authors.affiliation: Cancer Research And Biostatistics (CRAB), Seattle, WA, USA.
authors.forename: John
authors.initials: J
authors.lastname: Crowley
mesh_terms: D002986:Clinical Trials as Topic; D018572:Disease-Free Survival; D023381:Endpoint Determination; D006801:Humans; D007167:Immunotherapy; D008495:Medical Oncology / Q000639:trends; D009101:Multiple Myeloma / Q000453:epidemiology / Q000276:immunology* / Q000628:therapy*; D016896:Treatment Outcome
keywords: immuno-oncology trials; multiple myeloma; study end points