Results Summary
PCORI funded the Pilot Projects to explore how to conduct and use patient-centered outcomes research in ways that can better serve patients and the healthcare community. Learn more.
Background
When patients have swollen or irritated blood vessels, doctors call it vasculitis. Vasculitis occurs when the body’s immune system attacks blood vessels by mistake. One type of vasculitis is called ANCA-associated vasculitis, or AAV. It is named after the part of the immune system that is not working right. Even with treatment, vasculitis can last for a long time.
It’s important for doctors to learn about the overall health and well-being of patients living with a condition like vasculitis. The Patient-Reported Outcomes Measurement Information System (PROMIS®) provides surveys doctors can use to ask patients about what they are able to do in daily life and how they feel.
Project Purpose
The researchers wanted to find out what matters most to people living with AAV. They also wanted to create a survey that could help doctors to better understand how to treat patients with AAV.
This study had three main goals:
- To see if surveys from PROMIS include questions about the things that matter most to patients with AAV
- To create a new method for measuring how AAV affects patients’ daily lives
- To match AAV symptoms to symptoms listed in the International Classification of Functioning, Disability and Health (ICF). The ICF is a detailed list of ways that the human body functions and how people experience life with their illnesses. It is often used to help researchers think about how to measure health and the impact of illness. The ICF can be used by doctors to choose measures and surveys that help them understand their patients better.
Methods
The researchers did interviews and held discussion groups. They also created a survey and looked at patient health information. The study took place at clinics in the United States, Canada, and the United Kingdom.
The researchers gave the PROMIS surveys to patients with AAV to see if the survey could accurately measure patients’ health outcomes and identify if AAV was still causing symptoms.
The researchers interviewed more than 30 patients with AAV individually and in groups to learn what matters most to them about AAV and to identify what health outcomes they thought were important. They also talked with a committee of experts on AAV about what they thought mattered to patients.
The researchers created a new draft survey, called the AAV-PRO, based on what they heard from the patients and input from the experts. The survey had 35 questions about how AAV affects patients’ lives. Three hundred patients with AAV filled out the AAV-PRO two times, three months apart. The researchers then looked at the patients’ survey answers to see if the survey was accurately measuring patients’ experience with AAV.
Finally, the researches asked an international group of experts who treat AAV to identify the parts of the ICF that matched what patients had said was most important to them.
Findings
The researchers reported that they made progress in
- Learning how patients with AAV experience their illness and what health outcomes matter to them
- Finding out if the PROMIS surveys can help doctors know whether a patient is still experiencing symptoms
- Developing the AAV-PRO, a new survey that is specifically for patients with the condition
- Finding out what parts of the ICF match up with patients’ experiences with AAV
The researchers believe that it will be possible to use the PROMIS approach to develop a patient-reported outcomes survey for vasculitis.
The researchers found it helpful to include patients in the research process. The researchers learned how to work together with patients and felt it made the study better.
Limitations
The patients who took part in this study spoke English. The results might be different for patients who don’t speak English.
Conclusions
PROMIS surveys worked well for patients with AAV. The new survey, AAV-PRO, was designed to capture information that is specific to AAV. It is ready to be tested. The ICF included many of the things that patients with AAV say matter to them. The ICF can be used to help doctors or researchers figure out how to better measure the impact of AAV on patients.
Sharing the Results
The researchers will provide the study results to the public through websites and newsletters. They will send the results to patients and clinicians in the VCRC Patient Contact Registry, the Vasculitis Patient-Powered Research Network, the Vasculitis Foundation, and Vasculitis United Kingdom. They will present the study to researchers and patients at conferences. The researchers will submit articles about the project to medical journals.
Professional Abstract
PCORI funded the Pilot Projects to explore how to conduct and use patient-centered outcomes research in ways that can better serve patients and the healthcare community. Learn more.
Background
The overarching goal of the Patient-Reported Outcomes for Vasculitis project was to more effectively incorporate patients’ perspectives into the assessment of disease activity and burden among patients with antineutrophil cytoplasmic antibodies (ANCA)-associated vasculitis (AAV) and develop valid and precise patient-reported outcomes for use in clinical research.
Project Purpose
- Objective 1: Evaluate the feasibility and construct validity of instruments within the Patient-Reported Outcome Measurement Information System (PROMIS) to record important components of the disease experience among patients with AAV.
- Objective 2: Create a disease-specific, patient-based measure of disease burden associated with AAV.
- Objective 3: Analyze physician-based and patient-reported outcomes in AAV within the framework of the International Classification of Function (ICF).
These three interrelated objectives all address the need to capture patient experiences with vasculitis for use as outcomes in clinical research and practice and to better understand the scope of health and disease among patients with vasculitis. The project has included direct engagement and involvement of patient research partners on the steering committee. The final results/products may benefit patients and physicians as clinical research is conducted in vasculitis and lead to a broader understanding of the extent of the impact of vasculitis on patients.
Study Design
This project included qualitative work, prospective cross-sectional and longitudinal data collection, and a Delphi exercise.
Participants, Interventions, Settings, and Outcomes
Participants were patients with vasculitis, settings were academic medical centers, and outcome measures were PROMIS and AAV-PRO.
Data Sources
Data were from >30 individual interviews conducted in the US, Canada, and the UK and from several focus groups.
Researchers collected data on PROMIS through the Vasculitis Clinical Research Consortium Longitudinal Studies cohort using both the PROMIS Computer Adaptive Test instruments and the PROMIS short forms. Researchers also collected data through PROMIS short forms in several international randomized clinical trials on vasculitis.
Data Analysis
To assess whether PROMIS measures discriminated between active disease and remission in this population, a mixed linear model was constructed with a random intercept introduced for each study subject.
Researchers created a draft AAV patient-reported outcome questionnaire—AAV-PRO—based on all of the accumulated qualitative data and analysis and feedback from the steering committee. More than 300 patients in the US and UK completed the 35 candidate questionnaire items on the AAV-PRO, in addition to information about their disease, at baseline and three months (included transition item charting change). In the US, a test-retest exercise was completed three to five days following baseline. Exploratory factor analysis and Rasch analysis defined the underlying scale (domain) structure. The following properties were determined for each domain: convergent validity, using Pearson correlations between domain scores and the three-level EuroQol five dimensions questionnaire; known groups validity, using t tests to compare mean scores for different disease states; test-retest reliability, analyzing intraclass correlation coefficients, with respondents reporting “no change;” and longitudinal construct validity, analyzing mean change scores and effect sizes in relation to transition item responses at three months.
Researchers completed the analyses for the ICF project. This project led to new initiatives to help best capture the patient experience with vasculitis. An international group of clinicians with expertise in the clinical care of patients with vasculitis participated in a three-round Delphi exercise to identify relevant domains.
Findings
Completing the work of the interrelated set of studies conducted within the overall Patient-Reported Outcomes for Vasculitis project led to substantial progress in the following areas:
- Understanding patients’ perspectives of illness in AAV, including about the impact of (1) the disease itself and (2) commonly prescribed treatments.
- Development, validation, and application of patient-centered outcome assessment tools to quantitatively capture the domains of illness identified as important by patients and often not addressed by currently used physician-based measures. Specifically,
- PROMIS to measure patient experiences in vasculitis using well-validated “generic” (nondisease-specific) measures in several domains
- Development of a new disease-specific patient-reported outcome measure—AAV-PRO—which collects and quantitates the patient experience with AAV
- Analysis of the coverage of currently used outcome measures in relation to the spectrum of health, based on the structure of the World Health Organization’s ICF.
- Incorporating patients directly in all aspects of the research process. This approach was a learning process for both the academic investigators and the patients and led to outstanding partnerships, fully patient-centered research, higher quality work, and excellent working systems.
The outcome measures tested/developed will affect the field of vasculitis through their application in clinical trials. Ongoing work on validation and incorporation into clinical trials will lead to substantially better capture of patients’ perspectives of vasculitis as a core measure in research.
Limitations
This work was limited to patients who spoke English and may not be fully applicable to patients whose primary language is not English.
Conclusions
- Through the Patient-Reported Outcomes for Vasculitis project, researchers developed methods for collecting data from patients with vasculitis that describe patients’ own assessment of their disease and progress.
- Validity and feasibility of PROMIS for vasculitis were acceptable. These measures have the potential to enhance clinical research, including clinical trials in this set of complex diseases.
- AAV-PRO captures disease-specific elements of the patient experience in AAV and will be tested in the context of clinical trials.
The study of the ICF in ANCA–associated vasculitis has highlighted the spectrum of outcome measures in this disease and will aid in attaining comprehensive disease assessment.