Chapter 2 - Clinical Presentation, Differential Diagnosis and Response Assessment of Gliomas

Accurate response measurements are needed to ensure the continuation of effective therapy, and modify or discontinue an ineffective treatment in non-responding patients.

For clinical trials enrolling brain tumour patients, overall survival or survival at specified time points are usually considered as the most reliable endpoints. However, these endpoints may be prone to the confounding effects of salvage therapy.

Quality of life endpoints, i.e. PROM (patient-related outcome measures) and PREM (patient-related experience measures), including neurological and cognitive symptoms, are of great value and should be used in clinical brain tumour studies.

Response rate and progression-free survival using MRI/CT for evaluating tumour burden are valuable endpoints when attempting to isolate the relative efficacy of a given treatment, and to increase the knowledge of the nature of follow-up on study.

Currently the most widely used system to assess first-line treatment response in glioma is the Response Assessment in Neuro-Oncology (RANO) criteria.

These surrogate markers present limitations, including the potential for variability, the potential for false-positive signals and the discordance in radiographic interpretation between radiologists.

An improved understanding of tumour biology and novel imaging techniques, e.g. PET and functional MRI, have opened new windows for the evaluation of tumours, even at the molecular level.

The introduction of targeted therapies and immunotherapies directly affecting contrast enhancement have also highlighted the need for new endpoints.

Response assessments (clinically and MRI/CT) are in general conducted every second or third month for patients during the initial 1–2 years after primary treatment, thereafter every six months or at the discretion of patient and physician.

Revision Questions

  1. What is the gold standard when evaluating the efficacy of a given treatment?
  2. What is the value of using surrogate markers in evaluating treatment?
  3. How often should a response assessment be done?

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Last update: 18 September 2017