Exploring Reproductive Experiences With Women Enrolled in the International Vasculitis Pregnancy Registry.

OBJECTIVE: This study explored the reproductive journeys of women with vasculitis, including their conversations with healthcare providers, disease activity, medication changes, and delivery experiences. METHODS: Interviews were conducted with women registered in the Vasculitis Pregnancy Registry (VPREG), an online patient-reported registry of pregnant women with vasculitis. A team of physicians, patients, and qualitative researchers developed a qualitative interview guide. Participant responses were evaluated using thematic analysis. RESULTS: Eighteen patients with vasculitis who had experienced pregnancy were interviewed (10 antineutrophil cytoplasmic antibody-associated vasculitis, 4 Takayasu arteritis, 2 Behçet disease, 1 IgA vasculitis, 1 relapsing polychondritis). Thematic analysis revealed common experiences in the decision-making process during pregnancy planning, including accessing information from multiple sources, communicating with medical professionals, and changing treatment for vasculitis. Women sought information about vasculitis and pregnancy from various sources, including social media; however, opinions from their physicians and family members were most influential. Patients were more likely than providers to initiate conversations regarding family planning. Balancing differing opinions from subspecialists was challenging as many patients recalled acting as a liaison between multiple physicians during pregnancy. The need for self-advocacy was a common experience among patients. Most women had pregnancies that resulted in live births with delivery at term. CONCLUSION: When making decisions about pregnancy, women of reproductive age with vasculitis used multiple resources. Patients consistently valued their medical provider's opinion over alternative sources of information. To ensure comprehensive medical care, half of women relied on self-advocacy to coordinate communication among subspecialists. Most women had pregnancies that resulted in live births with delivery at term.

Reproductive Outcomes for Women with Vasculitis.

OBJECTIVE: There are limited data on the reproductive health of women with vasculitis. This study utilized a prospective, international vasculitis pregnancy registry to survey women during and after pregnancy. METHODS: The Vasculitis Pregnancy Registry (VPREG) is imbedded within the Vasculitis Patient-Powered Research Network (VPPRN), an international online research infrastructure. Any pregnant woman with a diagnosis of vasculitis can self-enroll. After enrollment, women are invited to complete online surveys at study entry, once per trimester, and postpartum. Descriptive statistics are reported here. RESULTS: Between 2015-2022, 147 women with 149 pregnancies enrolled in VPREG from 16 countries. Data on 78 pregnancies with known outcomes were included in this analysis. During pregnancy, women on average experienced low levels of pain related to vasculitis (scale 0-10, median 2 (IQR 1-5)) and preserved feelings of wellness (scale 0-10, median 3 (IQR 1-5)). Thirty-six percent of women reported their vasculitis was active during pregnancy. Of the 14 women requiring hospitalization during pregnancy outside of delivery, four cited active vasculitis as the indication. Most women (54/73, 74%) were prescribed medications for vasculitis during pregnancy. Seventy-six (97%) pregnancies resulted in live births with 64% delivering vaginally and 21% experiencing a preterm delivery. CONCLUSION: These results demonstrate that most women with vasculitis can experience pregnancies that result in live births delivered at term. During pregnancy, a minority of women reported flares of vasculitis or the need for hospitalization due to vasculitis. These data are useful to rheumatologists and patients to inform and facilitate discussions about reproductive health and vasculitis.

Plain Language Summary of principles for improving the care of people with eosinophil-associated diseases.

WHAT IS THIS SUMMARY ABOUT?: Eosinophil-associated diseases (EADs) are a group of conditions in which eosinophils (a type of white blood cell) are thought to play a key role in the disease and how it develops. Some EADs are common, such as atopic dermatitis (also called eczema) and a subtype of asthma called eosinophilic asthma, while others are rare, such as hypereosinophilic syndrome (a condition in which a person has a very high number of eosinophils in both the blood and one or more organs). People with EADs face many problems related to their conditions. Symptoms such as severe abdominal pain, itch, or shortness of breath impact both the patient as well as their friends and family. Patients with EADs also experience delays to diagnosis and treatment as well as financial barriers. Healthcare professionals sometimes fail to recognize the complex set of symptoms that characterize an EAD, and this may cause delays in reaching a correct diagnosis. As a result, it may take longer for a patient to get the best care and the most effective treatments, which may contribute to poor health. The goal of this charter is to describe the key elements of good quality care, which all people with EADs deserve, as well as to present an action plan to improve health and overall well-being for people with EADs. Proposed use of this patient charter: The principles described in this charter (a written guide to achieve an outcome) show the core elements of quality care that people with EADs must receive. They also describe clear steps to reduce the burden on patients and their caregivers and to improve patient health outcomes. We urge healthcare professionals, hospitals, and policymakers around the world to adopt these principles quickly. By doing this, people with EADs will be more likely to receive an accurate and timely diagnosis and have access to quality care and treatment in the right setting.

Improving Care in Eosinophil-Associated Diseases: A Charter.

Eosinophil-associated diseases (EADs) are a range of heterogeneous conditions in which eosinophils are believed to play a critical pathological role. EADs include common illnesses such as eosinophilic asthma and chronic rhinosinusitis and rare conditions such as hypereosinophilic syndromes (HES) and eosinophilic gastrointestinal disorders (EGIDs). EADs are associated with substantial burdens for the patient, including chronic, debilitating symptoms, increased financial burden, decreased health-related quality of life, and the need for repeated visits to multiple different healthcare professionals (HCPs), emergency departments, and/or hospitals. Poor EAD recognition by HCPs often contributes to delayed diagnoses, which further delays patient access to appropriate care and effective treatments, contributing to poor health outcomes. The objective of this charter is to outline key patient rights and expectations with respect to the management of their condition(s) and to set forth an ambitious action plan to improve health outcomes for patients with EADs: (1) people with EADs, their caretakers, HCPs, and the public must have greater awareness and education about EADs; (2) people with EADs must receive a timely, accurate diagnosis; (3) all people with EADs must have access to an appropriate multidisciplinary team, when necessary; and (4) people with EADs must have access to safe and effective treatment options without unnecessary regulatory delays. The principles described in this charter demonstrate the core elements of quality care that people with EADs must receive, and they represent clear steps by which to reduce patient and caregiver burden and improve patient outcomes. We urge HCPs, healthcare systems, and policymakers worldwide to swiftly adopt these principles to ensure patients with EADs have an accurate diagnosis in a timely manner and access to high-level care and treatment in an appropriate setting.

Diagnostic delays in vasculitis and factors associated with time to diagnosis.

BACKGROUND: Patients with vasculitis, a set of rare diseases, encounter delays in obtaining an accurate diagnosis which can lead to substantial morbidity and increased mortality. This study sought to describe the diagnostic journey of patients with vasculitis and identify factors associated with time to diagnosis. METHODS: Patients with vasculitis enrolled in an online registry completed a two-stage study: Stage 1: survey of open-ended questions about patients' diagnostic journeys and perceived factors associated with rapid or delayed diagnosis; Stage 2: survey with specific questions based on data from Stage 1 and additional investigator-identified factors. RESULTS: 375 patients with vasculitis participated in Stage 1; 456 patients participated in Stage 2. 85% of patients were seen by a healthcare provider within 3 months of the onset of symptoms. The median time to diagnosis of vasculitis was 7 months. 313/456 (73%) of patients were misdiagnosed initially. 40% of diagnoses were made in a hospital setting; 2% of diagnoses were made at a specialized vasculitis center. 60% of patients had at least 1 visit to an emergency room prior to diagnosis. Unemployment, time to travel to a medical center > 1 h, initial misdiagnosis, and delays in seeing a specialist were all associated with longer times to diagnosis. 373/456 (82%) of patients reported that a delayed diagnosis had negative consequences on their health. CONCLUSION: Patients with vasculitis encounter substantial delays in achieving an accurate diagnosis and these delays are associated with negative health consequences. Both patient-related factors and healthcare-related factors are associated with diagnostic delays.

Effects of the COVID-19 Pandemic on Patients Living With Vasculitis.

OBJECTIVE: This study aimed to analyze the concerns and health-related behaviors in patients with vasculitis during the early phase of the coronavirus disease 2019 (COVID-19) pandemic in North America. METHODS: Patients with vasculitis in North America were invited to complete an online survey through the Vasculitis Patient-Powered Research Network in collaboration with the Vasculitis Foundation and the Relapsing Polychondritis Foundation. Questions focused on concerns and behaviors related to doctors' visits, tests, medication, and telehealth use. Factors affecting their concern and health-related behaviors were determined. RESULTS: Data from 662 patients were included: 90% of patients were White, 78% were women, 83% expressed moderate or high levels of concern about COVID-19, and 87% reported that their vasculitis moderately or extremely affected their level of concern. Older age, female sex, lung disease, and immunosuppression were associated with greater concern. Doctors' visits, laboratory tests, and other tests were avoided by 66%, 46%, and 40% of patients, respectively. Younger age, urban location, higher income, higher concern levels, and prednisone use (>10 mg/day) were associated with greater likelihood of avoiding visits or tests. Ten percent of patients on immunosuppressive therapy stopped their medication. Twenty-nine percent patients on rituximab avoided an infusion. Forty-four percent of patients had telehealth visits; more visits were reported for younger patients, for patients on glucocorticoids, and in Canada versus the United States. CONCLUSION: During the COVID-19 pandemic, patients with vasculitis have high levels of concern and exhibit potentially harmful health-related behaviors. Health care use varies across different demographic groups and geographic regions. Specific strategies are warranted to facilitate engagement of these patients with the health care system during the pandemic.

ANCA-associated vasculitis.

The anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitides (AAVs) are a group of disorders involving severe, systemic, small-vessel vasculitis and are characterized by the development of autoantibodies to the neutrophil proteins leukocyte proteinase 3 (PR3-ANCA) or myeloperoxidase (MPO-ANCA). The three AAV subgroups, namely granulomatosis with polyangiitis (GPA), microscopic polyangiitis and eosinophilic GPA (EGPA), are defined according to clinical features. However, genetic and other clinical findings suggest that these clinical syndromes may be better classified as PR3-positive AAV (PR3-AAV), MPO-positive AAV (MPO-AAV) and, for EGPA, by the presence or absence of ANCA (ANCA+ or ANCA-, respectively). Although any tissue can be involved in AAV, the upper and lower respiratory tract and kidneys are most commonly and severely affected. AAVs have a complex and unique pathogenesis, with evidence for a loss of tolerance to neutrophil proteins, which leads to ANCA-mediated neutrophil activation, recruitment and injury, with effector T cells also involved. Without therapy, prognosis is poor but treatments, typically immunosuppressants, have improved survival, albeit with considerable morbidity from glucocorticoids and other immunosuppressive medications. Current challenges include improving the measures of disease activity and risk of relapse, uncertainty about optimal therapy duration and a need for targeted therapies with fewer adverse effects. Meeting these challenges requires a more detailed knowledge of the fundamental biology of AAV as well as cooperative international research and clinical trials with meaningful input from patients.

Patient involvement in medical research: what patients and physicians learn from each other.

BACKGROUND: There is increasing interest in actively involving patients in the process of medical research to help ensure research is relevant and important to both researchers and people affected by the disease under study. This project examined the recently formed Vasculitis Patient-Powered Research Network (VPPRN), a rare disease research network, to better understand what investigators and patients learned from working on research teams together. METHODS: Qualitative interviews were conducted by phone with patients, physician/PhD-investigators, and study managers/staff who participated in the network. The question guiding the interviews and observational analysis was: "What have investigators and patients learned about working together while working on VPPRN teams?" Interview transcripts were analyzed in combination with observations from multiple in-person and telephone meetings. RESULTS: Transcripts and notes were reviewed and coded from 22 interviews conducted among 13 patient-partners, 5 study managers/staff, and 4 MD or PhD-investigators, and 6 in-person and 42 telephone/web-conference meetings. Patient-partners and investigators characterized their working relationships with one another, what they learned from their collaborations, and provided recommendations for future teams of patient-partners and investigators. Major themes included the great benefits of communicating about activities, being open to listening to each group member, and the importance of setting reasonable expectations. CONCLUSIONS: Direct engagement in research design and development by patient-partners and co-learning between investigators and patient-partners can result in a positive and productive working relationship for all members of a medical research team. This bi-directional engagement directly benefits and impacts research design, participant recruitment to studies, and study subject retention.

Experience With Direct-to-Patient Recruitment for Enrollment Into a Clinical Trial in a Rare Disease: A Web-Based Study.

BACKGROUND: The target sample size for clinical trials often necessitates a multicenter (center of excellence, CoE) approach with associated added complexity, cost, and regulatory requirements. Alternative recruitment strategies need to be tested against this standard model. OBJECTIVES: The aim of our study was to test whether a Web-based direct recruitment approach (patient-centric, PC) using social marketing strategies provides a viable option to the CoE recruitment method. METHODS: PC recruitment and Web-based informed consent was compared with CoE recruitment for a randomized controlled trial (RCT) of continuing versus stopping low-dose prednisone for maintenance of remission of patients with granulomatosis with polyangiitis (GPA). RESULTS: The PC approach was not as successful as the CoE approach. Enrollment of those confirmed eligible by their physician was 10 of 13 (77%) and 49 of 51 (96%) in the PC and CoE arms, respectively (P=.05). The two approaches were not significantly different in terms of eligibility with 34% of potential participants in the CoE found to be ineligible as compared with 22% in the PC arm (P=.11) nor in provider acceptance, 22% versus 26% (P=.78). There was no difference in the understanding of the trial as reflected in the knowledge surveys of individuals in the PC and CoE arms. CONCLUSIONS: PC recruitment was substantially less successful than that achieved by the CoE approach. However, the PC approach was good at confirming eligibility and was as acceptable to providers and as understandable to patients as the CoE approach. The PC approach should be evaluated in other clinical settings to get a better sense of its potential.