Vol 2, No 2 (2011)
Review paper
Published online: 2011-07-15
The relations between therapy results and accepted evaluation criteria of progression free survival and event free survival — analysis on the basis of tyrosine kinase inhibitors use in patients with chronic myeloid leukemia
Hematologia 2011;2(2):99-104.
Abstract
From many years a lot of attempts were undertaken to consolidate the criteria of evaluation of
results of randomized clinical trials (RCT). Acceptation in 1996 and 2001 of precised standards
of treatment efficacy evaluation (CONSORT, Consolidated Standards of Reporting Trials)
significantly improved quality of reported RCT later on. Also Food and Drug Administration
(FDA) data published in 2003 improved quality of reports by underlining the significance of
overall survival (OS) and quality of life in study evaluating the efficacy of new drugs in cancer
patients. FDA also suggested to introduce the surrogates of OS in patients with low probability
of long term survival, between others progression free survival (PFS) or event free survival
(EFS). Despite this, there is still a lot of discrepancies in terms of accepted therapy efficacy
evaluation criteria in recently published clinical trials. It is especially evident in case of RCT
evaluating the efficacy of tyrosine kinase inhibitors in patients with chronic myeloid leukemia.
The discrepancies are related to the differences in defined criteria of PFS, EFS, and drug
intolerance. It makes difficult to compare different drugs evaluated in RCT and may influence
decision making process when therapy should be change.
Hematologia 2011; 2, 2: 99–104
Hematologia 2011; 2, 2: 99–104
Keywords: randomized clinical trialsPFSEFSOSchronic myeloid leukemiatyrosine kinase inhibitors
