EGPA Patient and Doctor Differences in Disease Reporting Noted
People with eosinophilic granulomatosis with polyangiitis (EGPA) are more likely to report a greater range of symptoms than their doctors, a study that drew on patient- and physician-reported databases found, while showing similarities on other points, like relapse frequency.
Given the importance of the patient perspective in both research and the clinic, this study “comparing patient-reported and physician-reported characteristics and outcomes in patients with EGPA should encourage rethinking and refinement of how patients are recruited and how their data are collected for complex diseases,” the researchers wrote.
Specifically, a “common set” of disease-specific symptoms and outcomes, and the use of similar definitions or wording for clarity are needed across registries to be able to compare their data with confidence.
The study, “Self-reported data and physician-reported data in patients with eosinophilic granulomatosis with polyangiitis: comparative analysis,” was published in the Interactive Journal of Medical Research.
EGPA is the rarest form of anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV), which comprises a group of autoimmune disorders characterized by blood vessel damage and inflammation. EGPA, in particular, is considered a complex and multisystem disease that can have a wide array of manifestations involving several organs.
Researchers in Canada and the U.S. wanted to compare how often EGPA symptoms, treatments, and outcomes are reported by adult patients as opposed to doctors.
“Patient-based registries can help advance research on rare diseases,” they wrote.
But research into EGPA, particularly, is less extensive “compared to several other forms of vasculitis,” the team added. “A better understanding of the utility of patient-based research registries could help advance research on this rare disease.”
Patient or caregiver responses came from the Vasculitis Patient-Powered Research Network (VPPRN), an online and self-reported registry for health-related information in vasculitis. The 195 EGPA patients registered in the VPPRN were adults living in Canada or the U.S., and had an established EGPA diagnosis.
Physicians entered findings on patients for the Vasculitis Clinical Research Consortium (VCRC) database, and for this study included information covering 354 EGPA patients, ages 18 or older, enrolled in either the VCRC longitudinal study (NCT00315380) or the One-time DNA study (NCT01241305) between 2013–19.
More women were in the VPPRN study than the VCRC database (69.2% vs. 59%), and adults in this patient-reporting group were on average 2.7 years younger at diagnosis than the database group (47.3 vs. 50 years). The proportion of patients who tested positive for ANCAs, the self-reactive antibodies that cause vasculitis, was higher among those in the VPPRN than the VCRC (48.9% vs. 38.9%).
Both patients and doctors reported a wide array of symptoms. But patients reported higher frequencies of almost all symptoms than did doctors, including fever (55.4% vs. 17.5%), weight loss (55.6% vs. 29.9%), numbness or tingling (87.6% vs. 60.5%), problems with the nose or sinuses (92.2% vs. 82.5), joint pain or swelling (67.1% vs. 39.5%), and kidney issues (22.4% vs. 10.2%) or coughing up blood (14.4% vs. 5.9%).
The frequency of reporting asthma and heart issues like inflammation were similar between these groups, while there was lesser reporting of lung issues apart from asthma by patients than doctors (72.4% vs 83.6%).
Relapses, or a worsening of EGPA symptoms, were noted at similar frequencies among both the patient-reported VPPRN and physician-reported VCRC (32.3% vs. 35.7%).
A similar frequency in the reported use of glucocorticoids, cyclophosphamide, or Nucala (mepolizumab) as EGPA treatments was also evident. But EGPA reporting by patients showed more greater use of rituximab than did the physician database (24.1% vs. 10.5%).
“Both patients and physicians reported a spectrum of outcomes and relative frequencies of manifestations, relapse rates, and medication use that are quite consistent with what is expected for this heterogeneous multisystem disease,” the researchers wrote. “However, some interesting differences in how this disease was reported were also observed between the 2 cohorts.”
How the disease manifests may account — at least partly — for some of these differences, they noted. For instance, a higher proportion of patients in the VPPRN were positive for ANCAs, and more might have required treatment with rituximab for that reason.
Likewise, “misinterpretation or the use of different definitions of disease manifestations,” like that of asthma as opposed to “lung manifestations” meant to capture non-asthma symptoms, “may also have contributed to some differences.”
Patients might also “over-report” their symptoms, noting some not necessarily due to EGPA.
However, including the patient perspective in disease work is a “clear mandate,” the researchers concluded.
“These results support the use of patient-reported data in EGPA but also the need for careful consideration of these 2 types of registries, how patient- and physician-reported data are collected, as well as disease-specific definitions for the study of EGPA,” they wrote.
“Establishing a common set of disease-specific items and outcomes in EGPA and using similar definitions or wording is advised when seeking to combine data from both patients and physicians.”
About the Author
