Chemotherapy drug maintains quality of life despite side effects

Patients with advanced neuroendocrine tumors (NETs) reported similar health-related quality of life (HRQOL) during treatment with a recently approved chemotherapy drug as compared to patients receiving a placebo treatment, according to a clinical trial published in Lancet Oncology.

These findings, in conjunction with previous research showing delayed disease progression, suggest the drug, called everolimus, may be able to preserve quality of life even in light of chemotherapy's often-toxic side effects. The findings support the usefulness of HRQOL as an endpoint in clinical trials studying NETs, in a secondary analysis of their findings, according to the study.

This study was co-authored by David Cella, PhD, chair of Medical Social Sciences, the Ralph Seal Paffenbarger Professor and director of the Center for Patient-Centered Outcomes at the Institute for Public Health and Medicine.

NETs develop from specialized cells that receive inputs from nerve cells and release hormones into the blood, linking the nervous and endocrine systems.

According to the study, while prognosis varies widely depending on tumor location, treatment goals are largely consistent: control of tumor growth and preservation of health-related quality of life (HRQOL).

In this double-blind, placebo-controlled trial performed at multiple centers, investigators collected data from over 300 patients with advanced NETs of lung or gastrointestinal origin, who had been randomly assigned to receive everolimus or placebo treatments. HRQOL was assessed using the Functional Assessment of Cancer Therapy-General (FACT-G) questionnaire throughout the 15 month duration of the study.

The investigators observed that patients in the treatment group maintained similar FACT-G scores when compared with the placebo group, despite experiencing the toxic effects of chemotherapy. Patients in the treatment group had improved emotional well-being scores, pointing to the optimism engendered by a successful treatment.

"Toxic effects seem to be counterbalanced by longer progression-free survival and potentially fewer disease-related symptoms with everolimus than with placebo," according to the study.

The authors also found the connection between disease progression and HRQOL is supported by the observed decline in FACT-G score if a patient's cancer got worse.

The observed relationship between quality of life and disease progression also supports the use of HRQOL as an endpoint in clinical trials, according to the authors. With an active research community around NETs and no definitive treatment, HRQOL is a meaningful and patient-relevant endpoint that avoids the long tail of overall survival, a measure than can take years to collect, according to the study.