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

• Published in: European journal of cancer (1990) 2002-03, Vol.38 (4), p.543-549
• Main Authors: Van Glabbeke, M, Verweij, J, Judson, I, Nielsen, O.S
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Aged; Antineoplastic agents; Antineoplastic Agents - therapeutic use; Biological and medical sciences; Chemotherapy; Clinical Trials, Phase II as Topic - statistics & numerical data; Disease-Free Survival; End-point; Female; Humans; Male; Medical sciences; Middle Aged; Pharmacology. Drug treatments; Phase II; Progression-free rate; Reference Values; Response criteria; Sarcoma - drug therapy; Soft-tissue sarcoma
• ISSN: 0959-8049; 1879-0852
• DOI: 10.1016/S0959-8049(01)00398-7

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 ⩾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 ⩾40% would suggest a drug activity, and ⩽20% would suggest inactivity.