Glioma treatment goals traditionally have focused on tumor shrinkage or prolonging survival, but it’s time for those endpoints to be supplemented by clinical outcomes that are meaningful to the patient, according to a recently published report from a neuro-oncology working group.
The group, which includes representatives of previous oncology working groups, the Food and Drug Administration, and observers from the European Medicines Agency, has established a core set of symptoms and functional points that they say could be used in clinical trials and clinical care for patients with high-grade gliomas.
“Patients want to live longer, but they also want to continue to function as well as possible for as long as possible,” said, of the National Cancer Institute (NCI), and coauthors in a report that sums up the work to date of the Fast Track COA Group.
That work, while specific to gliomas, echoes results from broader initiatives that seek to standardize patient-reported outcomes in oncology trials, Dr. Armstrong and coauthors wrote. The report was published in the Lancet Oncology.
The core set of symptom constructs and functional issues identified by the work group are represented already in patient-reported outcome measures, according to the authors.
The symptoms worth measuring fall into five categories, including pain, difficulty communicating, perceived cognition, seizures, and symptomatic adverse events. The functional issues were divided into two categories, physical functioning, including weakness or walking, and role functioning, which they defined as the ability to work or participate in social or leisure activities.
Some of those outcomes can be challenging or cumbersome to track, Dr. Armstrong and coauthors said.
Pain has “many dimensions“ and is important to track, the group wrote. Likewise, patients’ concerns related to language function also are important, but are very “noisy“ as a variable and can be specific to tumor location.
Collecting data on seizure frequency and severity is important yet complicated, because of the variability in seizures and considerable difference between focal and generalized seizures. Assessment of cognitive functioning can be lengthy and burdensome to patients.
Adverse events of relevance will vary, depending on the drug used, its mechanism of action, and available data, though some allowance needs to be made for the possibility of “overlap“ with disease-related symptoms, the report said.
Physical functioning, including walking and weakness, should be evaluated. It also would be useful to distinguish the duration of time that patients have deficits in physical functioning in the later stages of their disease progression, authors said.
Role and social functioning should be assessed in most patients with high-grade gliomas, who will have symptoms and deficits that prevent returning to a job. “Patients might spend a substantial portion of their lives feeling ill, unable to do usual activities, or meet occupational, social, financial, and family obligations,” said Dr. Armstrong and coauthors in the report.
The scales and tools used to measure symptoms and functional concerns need to be those that best fit a particular clinical trial or clinical practice scenario. Several instruments that would be appropriate are discussed in the report, including the NCI Patient-Reported Outcome of the Common Toxicity Criteria Adverse Events (NCI PRO-CTCAE) and symptom and function scales or items in the Patient-Reported Outcomes Measurement System (PROMIS).
, such as time to recurrence or survival.
“Strategies for introducing these constructs to clinical trial cooperative groups and sponsors will be necessary,” they concluded.
Dr. Armstrong reported employment as a senior investigator and deputy chief of the neuro-oncology branch of the Center for Cancer Research at the NCI. His coauthors reported disclosures related to several companies and interests, including AbbVie, AstraZeneca, Bristol-Myers Squibb, Genentech, Merck, Taiho, and Tocagen.
SOURCE: Armstrong TS et al. .