How can the patient's perspective of well-being be incorporated scientifically into cancer research? Little information detailing the relative merits of PRO measures exists, leaving clinical researchers guessing which measures to choose and when best to deploy them. The overall goal of this R01 investigation is to demonstrate that recently-developed, simple, brief assessments of patient-reported well-being and symptom distress are psychometrically compatible and practical prognostic biomarkers for cancer clinical research. Specifically, we will compare the NIH-funded Patient-Reported Outcome Measurement Information System (PROMIS) short forms with the PRO-based version of the Common Terminology Criteria for Adverse Events (CTCAE) or PRO-CTCAE, and simple, single-item symptom linear analogue self-assessment (LASA) measures. We will implement a multi-site master protocol that will accrue 1800 cancer patients over four years from the Mayo Clinic Cancer Center, the M.D. Anderson Cancer Center, and the Memorial-Sloan-Kettering Cancer Center. PRO data will be collected at three observation points per patient, roughly one month apart to test psychometric and prognostic properties at baseline and longitudinally. Patients will be followed for overall survival (OS), disease-free survival (DFS), and progression-free survival (PFS) with an average followup per patient of two years. We will test the effect of data collection mode (paper versus electronic). This research will provide a complete psychometric profile of the PROMIS measures relative to the new PRO-CTCAE measures and simple single-item numerical analogues in cancer patients and assess whether they could serve as practical prognostic biomarkers for cancer clinical research. Although the focus of this proposal is on cancer, this investigation is relevant to other conditions as well.
Three specific aims will be pursued:
Specific Aim 1 : Compare psychometric properties of PROMIS, PRO-CTCAE, and other simple, patient- reported measures to produce standardised PROs for patient well-being and safety in cancer clinical research. Extensive psychometric testing will be carried out to evaluate the validity, responsiveness, redundancy, and compatibility between the PROMIS and PRO-CTCAE efforts relative to simple, single item assessments.
Specific Aim 2 : Test the PROMIS and other simple, patient-reported outcome measures for prognostic capability for overall survival (OS), disease-free survival (DFS), and progression-free survival (PFS). The purpose of this aim is to estimate the independent prognostic ability of the PROMIS, PRO-CTCAE, and LASA measures for OS, DFS, and PFS across multiple disease sites and stages.
Specific Aim 3 : Develop and test new strategies for collecting PROMIS and other simple, patient- reported outcome measures in clinical settings. The purpose of this aim is to evaluate the relative merits of various data collection modes for the aforementioned PRO measures in a clinic setting. We will test patient receptivity and measurement error paper-based, computer-based, and interactive voice response (IVR) versions of the PRO measures.

Public Health Relevance

The purpose of this project is to compare three different questionnaires that are designed to evaluate patient well-being, especially for people with cancer. The project will also test different ways of collecting questionnaire data such as pencil-and-paper forms, computerized forms, and automated telephone surveys.

National Institute of Health (NIH)
National Cancer Institute (NCI)
Research Project (R01)
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Special Emphasis Panel (ZRG1-AARR-F (52))
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Mitchell, Sandra A
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Mayo Clinic, Rochester
United States
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Sloan, Jeff A; Halyard, Michele; El Naqa, Issam et al. (2016) Lessons From Large-Scale Collection of Patient-Reported Outcomes: Implications for Big Data Aggregation and Analytics. Int J Radiat Oncol Biol Phys 95:922-929
Bingener, Juliane; Sloan, Jeff A; Seisler, Drew K et al. (2015) PROMIS for Laparoscopy. J Gastrointest Surg 19:917-26
Eton, David T; Beebe, Timothy J; Hagen, Philip T et al. (2014) Harmonizing and consolidating the measurement of patient-reported information at health care institutions: a position statement of the Mayo Clinic. Patient Relat Outcome Meas 5:7-15
Piderman, Katherine M; Johnson, Mary E; Frost, Marlene H et al. (2014) Spiritual quality of life in advanced cancer patients receiving radiation therapy. Psychooncology 23:216-21
Sloan, Jeff A (2011) Metrics to assess quality of life after management of early-stage lung cancer. Cancer J 17:63-7