Patient-Reported Outcomes in a Phase III Study of Everolimus Versus Placebo in Patients with Metastatic Carcinoma of the Kidney That Has Progressed on Vascular Endothelial Growth Factor Receptor Tyrosine Kinase Inhibitor Therapy

摘要: Purpose. A phase III, randomized, double-blind, placebo-controlled trial was conducted in patients with metastatic renal cell carcinoma. The focus of this paper is to evaluate the patient-reported outcomes. Methods.Patients were randomly assigned (2:1) receive oral everolimus 10 mg once daily or placebo. Functional Assessment Cancer Therapy Kidney Symptom Index—Disease-Related Symptoms (FKSIDRS) and European Organization for Research Treatment (EORTC) QLQ-C30 administeredbeforerandomizationandonday1ofeachcycle. FKSI-DRS EORTC Physical Functioning Global Quality Life scores primary endpoints examined. Longitudinal models used compare treatment arms. Sensitivity analyses explore impact missing data assumptions. Results.LongitudinaltrendsforFKSI-DRSscoresdid not differ by arm. Taking nonignorable into account, there significant differences betweentreatmentarmsinthetrendovertimeforphysical functioning global quality life, arm exhibiting greater decreases. All three these measures health-related life significantly related progression-free survival. Conclusions. There no evidence a difference between placebo longitudinal patterns disease-related symptoms, little