Document Detail

Assessing oncologic benefit in clinical trials of immunotherapy agents.
MedLine Citation:
PMID:  20237004     Owner:  NLM     Status:  MEDLINE    
BACKGROUND: USA Food and Drug Administration approval for cancer therapy requires demonstration of patient benefit as a marker of clinical efficacy. Prolonged survival is the gold standard for demonstration of efficacy, but other end points such as antitumor response, progression-free survival, quality of life, or surrogate end points may be used.
DESIGN: This study was developed based on discussion during a roundtable meeting of experts in the field of immunotherapy.
RESULTS: In most clinical trials involving cytotoxic agents, response end points use RECIST based on the premise that 'effective' therapy causes tumor destruction, target lesion shrinkage, and prevention of new lesions. However, RECIST may not be appropriate in trials of immunotherapy. Like other targeted agents, immunotherapies may mediate cytostatic rather than direct cytotoxic effects, and these may be difficult to quantify with RECIST. Furthermore, significant time may elapse before clinical effects are quantifiable because of complex response pathways. Effective immunotherapy may even mediate transient lesion growth secondary to immune cell infiltration.
CONCLUSIONS: RECIST may not be an optimal indicator of clinical benefit in immunotherapy trials. This article discusses alternative clinical trial designs and end points that may be more relevant for immunotherapy trials and may offer more effective prediction of survival in pivotal phase III studies.
R K Hales; J Banchereau; A Ribas; A A Tarhini; J S Weber; B A Fox; C G Drake
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Publication Detail:
Type:  Journal Article; Research Support, Non-U.S. Gov't; Review     Date:  2010-03-17
Journal Detail:
Title:  Annals of oncology : official journal of the European Society for Medical Oncology / ESMO     Volume:  21     ISSN:  1569-8041     ISO Abbreviation:  Ann. Oncol.     Publication Date:  2010 Oct 
Date Detail:
Created Date:  2010-09-23     Completed Date:  2011-01-25     Revised Date:  2014-07-30    
Medline Journal Info:
Nlm Unique ID:  9007735     Medline TA:  Ann Oncol     Country:  England    
Other Details:
Languages:  eng     Pagination:  1944-51     Citation Subset:  IM    
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MeSH Terms
Antineoplastic Agents / therapeutic use*
Clinical Trials as Topic
Neoplasms / therapy*
Reg. No./Substance:
0/Antineoplastic Agents

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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