Progression-free rate as the principal end-point for phase II trials in soft-tissue sarcomas

Eur J Cancer. 2002 Mar;38(4):543-9. doi: 10.1016/s0959-8049(01)00398-7.

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.

Publication types

  • Meta-Analysis
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Antineoplastic Agents / therapeutic use*
  • Clinical Trials, Phase II as Topic / statistics & numerical data*
  • Disease-Free Survival
  • Female
  • Humans
  • Male
  • Middle Aged
  • Reference Values
  • Sarcoma / drug therapy*

Substances

  • Antineoplastic Agents