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Endpoints for clinical trials and revised assessment in neuro-oncology

Nicholas Butowski1, Susan M Chang

  • 1Division of Neuro- Oncology, Department of Neurological Surgery, University of California San Francisco, San Francisco, California 94143-0350, USA. butowski@neurosurg.ucsf.edu

Current Opinion in Neurology
|September 26, 2012
PubMed
Abstract

Insights

Evaluating new brain tumor treatments requires effective clinical trial endpoints. Current imaging endpoints are limited, necessitating a focus on patient function, quality of life, and cognition for improved trial design and outcomes.

Area of Science:

  • Neuro-oncology
  • Clinical trial design
  • Biostatistics

Background:

  • Advances in brain tumor survival and treatment development have increased clinical trials.
  • Evaluating new therapeutics requires robust clinical trial endpoints.
  • Existing endpoints face challenges in accurately assessing treatment efficacy.

Purpose of the Study:

  • To review the challenges and advancements in clinical trial endpoints for brain tumors.
  • To highlight the limitations of current imaging-based endpoints.
  • To emphasize the growing importance of patient-centered outcomes.

Main Methods:

  • Review of recent literature on brain tumor clinical trial endpoints.
  • Analysis of the impact of pseudoprogression and pseudoresponse on imaging endpoints.
  • Discussion of novel endpoints including imaging, function, quality of life, and cognition.

Main Results:

  • Imaging endpoints like radiographic response and progression-free survival are confounded by pseudoprogression/pseudoresponse.
  • Overall survival interpretation is complicated by salvage therapies (e.g., bevacizumab).
  • Novel imaging, patient function, quality of life (QOL), and cognition are increasingly used as endpoints.

Conclusions:

  • Understanding endpoint benefits and limitations is key to improving clinical trial design.
  • Validated endpoints for patient function, QOL, and cognition are valuable secondary endpoints.
  • Improved endpoints will lead to more accurate evaluation of novel brain tumor therapeutics.

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