PCORnet®, the National Patient-Centered Clinical Research Network, aims to improve the nation’s capacity to conduct health research by creating a large, highly representative network for conducting clinical outcomes research. In 2020, PCORI launched an initiative on Conducting Rare Disease Research using PCORnet to answer important questions about the treatment and management of rare diseases or conditions. The initiative funded this research project and others.
This research project is in progress. PCORI will post the research findings on this page within 90 days after the results are final.
What is the research about?
Neuroendocrine tumors, or NETs, are a rare type of cancer that can occur anywhere in the body. NETs often occur in the stomach, intestines, pancreas, or lungs. Several treatments are available for NETs. But questions remain about which treatments work best and the order in which to use them.
In this study, the research team is looking at:
- How often doctors use different treatments and how these treatments affect symptoms and patients’ quality of life
- What factors, such as the location of the tumor or patient traits, affect the choice of treatment and the patient-reported outcomes
- How combining treatments and the order in which doctors use treatments affect quality of life, symptoms, and survival
Who can this research help?
Results may help patients and doctors when considering treatments for NETs and the approach to treatment.
What is the research team doing?
The research team is enrolling more than 3,000 adults who are newly diagnosed with NETs at one of 14 PCORnet® sites across the United States. PCORnet is made up of networks of health systems that gather data from electronic health records, or EHRs, and transform them into a common format. Doctors and patients are deciding what NET treatments to use.
The research team is looking at how often doctors use different treatments and in what order they use them. Patients are answering surveys at the start of the study and 6, 12, and 18 months later. Surveys ask about quality of life, symptoms, and patient experience. The team is also reviewing EHRs for information about:
- Patient traits
- The NETs, such as their size and how many NETs a patient has
- Treatments and procedures
- Kidney and liver function
- If the NETs have spread
Patients with NETs, patient advocacy organizations focused on NETs, and doctors with expertise in treating NETs are helping to design and plan the study.
Research methods at a glance
|Design||Observational: cohort study|
|Population||3,010 patients ages 18 and older with a new diagnosis of a NET|
Primary: quality of life, kidney function, treatment sequencing
Secondary: overall survival; progression-free survival; NET symptoms; patient experience with cancer care; adverse toxicities including acute renal failure, liver failure, and receipt of dialysis; change in serum creatinine; health-related quality of life
|Timeframe||Up to 60-month follow-up for primary outcomes|