Sample size calculations for detecting disease-modifying osteoarthritis drug effects on the incidence of end-stage knee osteoarthritis in clinical trials: Data from the Osteoarthritis Initiative

摘要

Objective: We previously published data representing calculations for sample sizes assuming that the reduction of the incidence of knee joint replacement (KJR) would be an endpoint to prove efficacy of a disease modifying drug in osteoarthritis (DMOAD). The sample sizes required for such hypothetical studies appeared to be high, rendering those studies unrealistic in the clinical research setting for practical reasons. The purpose of this work is to calculate sample sizes for hypothetical trials for DMOAD efficacy using a proxy for reaching end-stage knee osteoarthritis (esKOA) as an endpoint.