Results Summary
What was the research about?
In autoimmune and inflammatory illnesses, the immune system attacks healthy cells by mistake. Examples are rheumatoid arthritis, ankylosing spondylitis, and vasculitis. Treatments include two types of medicines that limit inflammation and reduce damage to joints and tissues. Questions remain about how safe and effective these medicines are.
In this study, the research team wanted to see if they could use PCORnet® data to look at the safety and effectiveness of these medicines. PCORnet is a research network. It merges health records, prescription, and health insurance data from health systems, including hospitals and clinics. The team used the data to compare rates of infection among patients with different autoimmune illnesses taking the two types of medicines. They looked at rates of shingles and hospital stays due to infection.
What were the results?
The research team had problems getting consistent and complete data. For example, prescription data were missing for patients in some health systems.
Among patients with different autoimmune illnesses, the research team found that patients taking the two types of medicines had similar rates of shingles and hospital stays due to infection. But the rates varied among patients with different autoimmune illnesses. For example, among patients with illness related to blood vessels and bones:
- Patients with vasculitis had the highest rate of shingles. Patients with ankylosing spondylitis had the lowest rate.
- Patients with vasculitis had the highest rate of hospital stays due to infection. Patients with ankylosing spondylitis had the lowest rate.
What did the research team do?
The research team used PCORnet data. The data were from patients taking medicines to treat autoimmune illnesses between 2016 and 2020.
Patients with autoimmune illnesses gave input throughout the study.
What were the limits of the study?
Many patients in the study didn’t have prescription or refill data recorded. Results may have differed with complete data.
Future research could look at ways to reduce missing prescription data.
How can people use the results?
Researchers can consider these results when using data from research networks. Doctors and patients can use these results when considering medicines for autoimmune illnesses.
ArthritisPower, IBD Partners, and Vasculitis PPRN formerly were Network Partners in PCORnet®, the National Patient-Centered Clinical Research Network. PCORnet® has been developed with funding from the Patient-Centered Outcomes Research Institute (PCORI®). |
Professional Abstract
Objective
To evaluate the feasibility of using clinical data from research networks to compare the effect of biologic and non-biologic medications on incidence of infections requiring hospitalizations and on incidence of herpes zoster associated with treatments among patients with autoimmune diseases
Study Design
Design Element | Description |
---|---|
Design | Retrospective cohort study |
Population | Electronic health record data from 204,247 patients with autoimmune disease, including rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, psoriasis, ulcerative colitis, Crohn’s disease, ankylosing spondylitis, and vasculitis |
Interventions/ Comparators |
|
Outcomes | Incidence of infections associated with treatment resulting in hospitalization; incidence of herpes zoster associated with treatment |
Timeframe | Up to 4-year follow-up for study outcomes |
This retrospective cohort study examined the feasibility of using clinical data obtained from PCORnet® to compare the effect of biologic and non-biologic medications among patients with autoimmune disease.
Researchers used PCORnet data for people treated for autoimmune diseases, including rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, psoriasis, ulcerative colitis, Crohn’s disease, ankylosing spondylitis, and vasculitis. The study included data from 204,247 patients with an autoimmune diagnosis participating in three PCORnet Clinical Research Networks. Clinical Research Networks consist of two or more healthcare systems, including hospitals, integrated delivery systems, and federally qualified health centers. Researchers identified patients with autoimmune diseases receiving biologic and non-biologic medications between 2016 and 2020.
Researchers then examined the comparative risks of infections requiring hospitalization and the incidence of herpes zoster associated with treatments using biologic and non-biologic medications. They compared outcomes by disease cohort, such as patients with inflammatory bowel disease.
Patients with autoimmune diseases gave input on the study design and outcomes.
Results
Researchers had problems getting consistent and complete data from the health systems.
Across disease cohorts, patients treated with biologic and non-biologic medications had similar rates of hospitalization due to infection and similar rates of herpes zoster. Infection rates varied across disease cohorts. For example, researchers found that, among patients with musculoskeletal autoimmune diseases treated with biologic and non-biologic medications:
- Rates of hospitalization due to infection were highest among patients with vasculitis (9.7–11.5 events per 100 person years) and lowest among patients with ankylosing spondylitis (0.55–0.9 events per 100 person years).
- Herpes zoster rates were highest among patients with vasculitis (1.24–2.94 events per 100 person years) and lowest among patients with ankylosing spondylitis (0.21–0.33 events per 100 person years).
Among patients with inflammatory bowel disease treated with biologic and non-biologic medications:
- Rates of hospitalization due to infection were highest among patients with ulcerative colitis (6.1–7.2 events per 100 person years).
- Herpes zoster rates were similar among patients with ulcerative colitis and Crohn’s disease (0.41–0.5 events and 0.33–0.47 events per 100 patient years, respectively).
Limitations
Most prescription refill information was missing. Also, immunomodulatory prescription data were not uniformly available for patients with autoimmune diseases and varied between 14% and 68% across health systems. Results may have differed with more data.
Conclusions and Relevance
In this study, clinical data from PCORnet allowed researchers to study some elements of the comparative risks of biologic and non-biologic medications on rates of hospitalization due to infection and on rates of herpes zoster. Researchers found similar rates among patients with autoimmune diseases treated with biologic and non-biologic medications.
Future Research Needs
Future research could examine ways to mitigate issues with missing data related to biologic and non-biologic medications.
ArthritisPower, IBD Partners, and Vasculitis PPRN formerly were Network Partners in PCORnet®, the National Patient-Centered Clinical Research Network. PCORnet® has been developed with funding from the Patient-Centered Outcomes Research Institute (PCORI®). |
Final Research Report
View this project's final research report.
Journal Citations
Results of This Project
Related Journal Citations
Peer-Review Summary
Peer review of PCORI-funded research helps make sure the report presents complete, balanced, and useful information about the research. It also assesses how the project addressed PCORI’s Methodology Standards. During peer review, experts read a draft report of the research and provide comments about the report. These experts may include a scientist focused on the research topic, a specialist in research methods, a patient or caregiver, and a healthcare professional. These reviewers cannot have conflicts of interest with the study.
The peer reviewers point out where the draft report may need revision. For example, they may suggest ways to improve descriptions of the conduct of the study or to clarify the connection between results and conclusions. Sometimes, awardees revise their draft reports twice or more to address all of the reviewers’ comments.
Peer reviewers commented and the researchers made changes or provided responses. Those comments and responses included the following:
The reviewers noted that the researchers overstated their conclusions in saying that the initiation of biologic agents led to significant, clinically important improvements because there was no comparison group to indicate that the improvements were the result of the biologics. The researchers revised their conclusions to remove this language.
The reviewers thanked the researchers for providing the important account of difficulties they had completing the study. They requested more information about the challenges the researchers faced in obtaining refill information for medications overall and specifically for injectables. The researchers added information about their experience to the discussion and in particular when discussing future research possibilities.
The reviewers encouraged the researchers to discuss the particular difficulties they experienced in gathering data elements collected outside of the PCORnet common data model as this information would be helpful for future research. The researchers revised the report to specify which data came from the PCORnet databases and which data came from outside, or “sidecar,” sources.
The reviewers noted that the researchers used a relatively small sample of patients whose data could be linked to Medicare claims that was likely to be very different from the overall sample of patients collected from all sites in the clinical research networks. The researchers agreed and revised their interpretation of results to take into account the differences between the two patient groups.