title: Progression-free rate as the principal end-point for phase II trials in soft-tissue sarcomas.
abstract: We have estimated progression-free rates (PFR) for various groups of soft-tissue sarcoma patients from our clinical trials database, to provide reference values for conducting phase II studies with PFR as the principal end-point. In 146 pretreated patients receiving an active agent, the PFR estimates were 39 and 14% at 3 and 6 months; with inactive regimens (234 patients), those estimates were 21 and 8% respectively. In 1154-non-pretreated patients, PFR estimates varied from 77% (synovial sarcoma) to 57% (malignant fibrous histiocytoma (MFH)) at 3 months, and from 56% (synovial sarcoma) to 38% (MFH) at 6 months. In 61 leiomyosarcomas from gastrointestinal origin, the corresponding figures were 44 and 30%, respectively. Consequently, for first-line therapy, a 6-month PFR of > or = 30-56% (depending on histology) can be considered as a reference value to suggest drug activity; for second-line therapy, a 3-month PFR of > or = 40% would suggest a drug activity, and < or = 20% would suggest inactivity.
authors.affiliation: EORTC Data Center, Av Mounier 83/11, B1200, Brussels, Belgium. mvg@eortc.be
authors.forename: M
authors.initials: M
authors.lastname: Van Glabbeke
authors.forename: J
authors.initials: J
authors.lastname: Verweij
authors.forename: I
authors.initials: I
authors.lastname: Judson
authors.forename: O S
authors.initials: OS
authors.lastname: Nielsen
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D017322:Clinical Trials, Phase II as Topic / Q000706:statistics & numerical data*; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D012016:Reference Values; D012509:Sarcoma / Q000188:drug therapy*
chemical_list: D000970:Antineoplastic Agents