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Stable disease is not preferentially observed with targeted therapies and as currently defined has limited value in

Tatiana Vidaurre¹, Julia Wilkerson, Richard Simon

¹Medical Oncology Branch, National Cancer Institute, NIH, Bethesda, MD 20892, USA.

Cancer Journal (Sudbury, Mass.)

|October 15, 2009

Abstract:

The assertion that efficacy of "targeted therapies" (TAR) cannot be assessed by traditional response measures has become conventional wisdom often guiding trial design and interpretation. Because stable disease (SD) has been increasingly reported as a measure of activity even for "cytotoxic therapies" (CTX), we sought to compare the occurrence of SD in phase II trials of cytotoxic CTX and TAR. We catalogued response assessments in 143 phase II studies reported in 5 journals between October 2006 and March 2008. Eighty-five studies incorporated CTX and 58 administered TAR. Both groups had comparable distribution of histologies and similar progression free survival (PFS) (median 4.8/2.35 months) and OS (median 10.9/9.15 months). SD was defined in only 28.6% of studies (median 10 weeks). SD rates were nearly identical-mean/median 35.05/34.7% for CTX, and 32.3/31.05% for TAR-with similar distributions across histologies, suggesting SD may not reflect drug activity. There were no positive correlations between %SD and PFS or OS. The overall response rate (complete response + partial response) was higher with CTX therapies (mean/median, 28/25% versus 13.1/5.3%) and in both groups overall response rate demonstrated a strong correlation (P < 0.0001) with PFS and OS. As currently defined and measured SD is not a property of TAR but is as frequently found with CTX therapies and may not reflect antitumor activity. Responses are observed with "both classes" of therapy and should be sought as a measure of activity. Studies that use SD as an end point require an adequate control to distinguish antitumor activity from normal variability in time to progression.

Insights

Stable disease (SD) occurs similarly in trials of targeted therapies (TAR) and cytotoxic therapies (CTX), suggesting it may not reflect drug activity. Overall response rates, not SD, correlate with progression-free survival and overall survival.

Area of Science:

Oncology
Clinical Trial Design
Cancer Therapeutics

Background:

Conventional wisdom suggests targeted therapies (TAR) efficacy requires novel assessment methods beyond traditional response measures.
Stable disease (SD) is increasingly used to indicate activity for cytotoxic therapies (CTX), prompting a comparative analysis.

Purpose of the Study:

To compare the occurrence and significance of stable disease (SD) as a measure of activity in phase II clinical trials of cytotoxic therapies (CTX) versus targeted therapies (TAR).

Main Methods:

A systematic cataloguing of response assessments from 143 phase II studies published in 5 journals between October 2006 and March 2008.
Analysis included studies administering CTX (n=85) and TAR (n=58), comparing SD rates, progression-free survival (PFS), and overall survival (OS).

Main Results:

SD was defined in only 28.6% of studies and occurred at nearly identical rates for CTX (mean 35.05%) and TAR (mean 32.3%), irrespective of histology.
No positive correlations were found between the percentage of SD and PFS or OS.
Overall response rates (complete + partial response) were higher for CTX (mean 28%) than TAR (mean 13.1%) and strongly correlated with PFS and OS in both groups.

Conclusions:

Current definitions and measurements of stable disease (SD) do not appear to reflect antitumor activity uniquely for targeted therapies (TAR).
SD occurs with similar frequency in both cytotoxic therapies (CTX) and TAR, suggesting it may not be a reliable indicator of drug efficacy on its own.
Overall response rates demonstrate a stronger correlation with survival outcomes (PFS, OS) than SD, and should be prioritized as a measure of therapeutic activity. Studies using SD as an endpoint require robust controls to differentiate true activity from natural progression variability.

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