Georgetown University


Grant Number: U01AR057971-01 and 3U01AR057971-02S1
Project Title: Validation of PROMIS in Diverse Cancer Populations and Supplement study: Estimating Cancer Specific Norms
PI Name, Title and Contact Information:
  1. Arnold Potosky, PhD, MHS, Principal Investigator Director of Health Services Research at Georgetown University, Lombardi Comprehensive Cancer Center, Cancer Control Program, Professor of Oncology
  2. Carol Moinpour, PhD, Principal Investigator Fred Hutchinson Cancer Research Center (FHCRC), Public Health Sciences Division, Cancer Prevention Program/Southwest Oncology Group Statistical Center, Affiliate Associate Professor, Health Services
Other Key Personnel: Nigel Bush, PhD, Investigator
Vivien Chen, MD, PhD, co-principal investigator
Rosemary Cress, DrPH, Principal Investigator
Kim Davis, PhD Investigator
Sally Glaser, PhD, co-principal investigator
Kevin Henry, PhD, Principal Investigator
James Hwang, MD Clinician
Theresa Keegan, PhD, Principal Investigator
Subha Madhaven, PhD, Data Coordinator
Jeanne Mandelblatt, MD, , MPH, Investigator
Mildred Ramirez, PhD, Investigator
Vanessa Sheppard, PhD, Investigator
Jeanne Teresi, PhD, Principal Investigator
Richard Jones, PhD, Investigator
Judy Wang, PhD, Investigator
Xiaocheng Wu, MD, MPH, Principal Investigator
Roxanne Jensen, PhD, Investigator
Web Site:  

Abstract:

Patient-reported outcomes (PROs) have gained increasing recognition as legitimate biomedical endpoints and in 2004 NIH launched the PROMIS Initiative to improve outcome measurement by building and validating item banks. If the "promise" of using patient–reported outcomes to monitor and improve health is to be realized, it is essential to ensure that PROMIS tools are appropriate and psychometrically equivalent for diverse populations cared for outside of specialized health care settings. This is especially important given the substantial health disparities across race/ethnic, age and social class groups. However, there has been limited research establishing equivalence of measures across diverse groups.

To fill this gap, we propose a three–pronged approach to evaluate PROMIS item bank measures in a population-based sample of White, Hispanic, Black and Asian adult cancer patients. We focus on cancer since it is a leading chronic disease and one where there are important disparities in experience and outcomes across the disease trajectory. We have selected seven cancers (female breast, uterine and cervical cancer, prostate cancer, and male and female colorectal cancer, non–Hodgkin’s Lymphoma and non–small cell lung cancer) to ensure a wide age range and symptoms. We use IRT–based methods and construct validation to assess the use of multiple items for the PROMIS domains of pain, depression, anxiety, sleep disturbance, fatigue, social function, and physical function.

In our first phase, we will conduct a qualitative study, including translating PROMIS items (selected items from 7 domains) into Chinese, translating the full instrument (non-PROMIS items) into Chinese and Spanish and conducting cognitive debriefing in a small sample, similar to our target population in the population-based study, to obtain linguistic validations and identify areas requiring revisions in order to improve standardization of survey items across respondent groups.

In our second phase, we will conduct a population-based study using four cancer registries (from California (2), New Jersey, Louisiana) to randomly recruit approximately 5, 000 patients diagnosed in the previous six to nine months. We will interview at least 1,000 patients from each race/ethnic group divided equally among two age groups (ages 21–64, and ages 65-84). Participants will be surveyed using a self–administered, mailed survey at initial recruitment and again six months later. A telephone interview will be attempted for subjects who do not respond to the mailed survey, or for subjects expressing a preference to complete the survey via telephone. Our large-scale, population-based recruitment and survey administration provide the opportunity to collect additional data that will enable the estimation of norm scores for cancer patients, with a specific focus on establishing norms by cancer type, severity, age, and gender. These cancer-specific norms, will be able to be used in future cancer clinical trials and studies allowing for comparisons within both cancer populations and the general U.S. population.

Our third phase is an exploratory clinical study (n=200) to examine whether the PROMIS fatigue measures generate sufficiently precise estimates of change over time during the first six months of chemotherapy to help guide care management.

Our study will provide a comprehensive set of empirical data on PROMIS measurement performance across sub–populations, indicating likely causes of any observed differences. Our approach leverages investments in, and extends the scope of, the PROMIS Initiative by developing measures that can be used to evaluate the impact of interventions, inform practice, and monitor population health disparities.

We will accomplish these goals by pursuing four specific aims:

AIM 1: Use item–response theory to evaluate the measurement properties of PROMIS items across age and race/ethnic groups from a population-based sample of cancer patients.

AIM 2: Evaluate the ability of PROMIS measures to detect differences in population-based patient outcomes across age, race-ethnicity, and cancer sub-groups defined by type, stage/severity, comorbidity, treatments, and disease phase (construct validity).

AIM 3: Evaluate the responsiveness of measures to detect clinically meaningful changes in selected health–related quality of life domains.

AIM 4: To estimate cancer-specific population norms by patient age, severity, and other clinically important characteristics.

Patient Population(s):

In our population-based study we will survey approximately 5, 000 cancer patients diagnosed in the previous six to nine months, residing in three different states covered by four centralized population-based cancer registries. We will draw a stratified random, population-based sample of patients to yield an average of 600–650 patients in each of eight sub–groups, defined by four race-ethnic groups and two age groups: Black, Latino, Asian and White, ages 21-64 and 65-84. Participants will be surveyed using a self-administered, mailed survey six to nine months after initial diagnosis to evaluate whether there is measurement equivalence between groups as well as the ability of the measures to distinguish between groups of patients in the broader population. We will re–interview patients six months later (12–15 months post–diagnosis) to evaluate the responsiveness of measures to change as patients transition from the active treatment period to post-active treatment. In the analyses we will focus on age and race/ethnicity, but will also examine the role of many other characteristics such as gender, socioeconomic status, acculturation, access to care, health literacy, and comorbidity. We will collect clinical information from medical records in a random 40% sample of surveyed cases to assess the correlation of PROMIS measures with clinical status and events (e.g., stage at diagnosis, comorbidity, treatment, acute complications, and active treatment versus disease–free survivor).

We will also conduct an exploratory clinical study to assess the responsiveness of items during the early, active treatment phase of care, since this time period cannot be captured from population-based registries as a result of reporting delays. Two groups of 100 cancer patients ages 21 and older receiving chemotherapy will be enrolled: women with early stage (I, II, or III) breast cancer and men and women with advanced stage colorectal cancer (Stages III and IV). Patients (n=200) in this phase will be recruited from treatment centers and will complete computer adaptive test assessments monthly for six months, starting prior to therapy.

Disease Groups and Domains

We focus on cancer since it is a leading chronic disease and one where there are important disparities in experience and outcomes across the disease trajectory. We have selected seven cancers (female breast, uterine, cervical, prostate, and male and female colorectal, non–Hodgkin’s Lymphoma and non–small cell lung cancer) to ensure a wide age range of adults with varying treatment experiences and potential symptoms. We focus on the domains of pain, anxiety, sleep disturbance, depression, fatigue, social function, and physical function. We selected these domains because of their importance in overall health-related quality of life (HRQOL) and impact on the ability to participate in the work force, the potential for differential expression across groups differing in characteristics such as age, race/ethnicity and socioeconomic status, and their broad applicability to other chronic diseases.

For questions concerning Georgetown’s role in the PROMIS network, please contact Arnold Potosky, PhD, by e-mail at alp49@georgetown.edu. For questions concerning University of Washington/FHCRC’s role in the PROMIS network, please contact Carol Moinpour, PhD, by e-mail at cmoinpou@fhcrc.org For more information about the PROMIS network, go to http://www.nihpromis.org.