المؤلفون: Springer, Jason Michael, Kermani, Tanaz A, Sreih, Antoine, Shaw, Dianne G, Young, Kalen, Burroughs, Cristina M, Merkel, Peter A

المصدر: Journal of Medical Internet Research, Vol 22, Iss 7, p e17231 (2020)

مصطلحات موضوعية: Computer applications to medicine. Medical informatics, R858-859.7, Public aspects of medicine, RA1-1270

الوصف: BackgroundUtilizing the traditional centers of excellence approach to conduct clinical trials involving rare diseases remains challenging. Patient-based registries have been shown to be both feasible and valid in several other diseases. ObjectiveThis report outlines the clinical characteristics of a large internet registry cohort of participants with a self-reported diagnosis of granulomatosis with polyangiitis or microscopic polyangiitis. MethodsPatients with a self-reported diagnosis of granulomatosis with polyangiitis or microscopic polyangiitis in an internet-based prospective longitudinal cohort (from the Vasculitis Patient-Powered Research Network) were included. Data on symptoms, diagnostic testing, and treatment were collected using standardized questionnaires. ResultsThe study compared patients with granulomatosis with polyangiitis (n=762) and patients with microscopic polyangiitis (n=164). Of the cohort, 97.7% (904/925) reported the diagnosis had been confirmed by a physician. Compared to microscopic polyangiitis, patients with granulomatosis with polyangiitis reported significantly more ear, nose, and throat manifestations (granulomatosis with polyangiitis: 641/723, 88.7%; microscopic polyangiitis: 89/164, 54.3%; z=10.42, P

وصف الملف: electronic resource

العلاقة: https://www.jmir.org/2020/7/e17231Test; https://doaj.org/toc/1438-8871Test

الوصول الحر: https://doaj.org/article/e7bccbae24a841bcbb49e2d2a49e1ed4Test

المؤلفون: Haller, Michael J, Long, S Alice, Blanchfield, J Lori, Schatz, Desmond A, Skyler, Jay S, Krischer, Jeffrey P, Bundy, Brian N, Geyer, Susan M, Warnock, Megan V, Miller, Jessica L, Atkinson, Mark A, Becker, Dorothy J, Baidal, David A, DiMeglio, Linda A, Gitelman, Stephen E, Goland, Robin, Gottlieb, Peter A, Herold, Kevan C, Marks, Jennifer B, Moran, Antoinette, Rodriguez, Henry, Russell, William E, Wilson, Darrell M, Greenbaum, Carla J, Battaglia, Manuela, Becker, Dorothy, Bingley, Penelope, Bosi, Emanuele, Buckner, Jane, Clements, Mark, Colman, Peter G, DiMeglio, Linda, Evans-Molina, Carmella, Gottlieb, Peter, Herold, Kevan, Knip, Mikael, Lernmark, Ake, Moore, Wayne, Muir, Andrew, Palmer, Jerry, Peakman, Mark, Philipson, Louis, Raskin, Philip, Redondo, Maria, Russell, William, Sosenko, Jay M, Spain, Lisa, Wentworth, John, Wherrett, Diane, Winter, William, Ziegler, Anette, Anderson, Mark, Antinozzi, Peter, Insel, Richard, Kay, Thomas, Pugliese, Alberto, Roep, Bart, Toppari, Jorma, Leschek, Ellen, Bourcier, Katarzyna, Ridge, John, Rafkin, Lisa, Santiago, Irene, Bundy, Brian, Abbondondolo, Michael, Adams, Timothy, Asif, Ilma, Bjellquist, Jenna, Boonstra, Matthew, Burroughs, Cristina, Cleves, Mario, Cuthbertson, David, DeSalvatore, Meagan, Eberhard, Christopher, Fiske, Steve, Ford, Julie, Garmeson, Jennifer, Geyer, Susan, Hays, Brian

المصدر: Diabetes. 68(6)

مصطلحات موضوعية: Prevention, Diabetes, Clinical Trials and Supportive Activities, Clinical Research, 6.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, Metabolic and endocrine, Adolescent, Adult, Antilymphocyte Serum, C-Peptide, CD4-CD8 Ratio, Child, Diabetes Mellitus, Type 1, Double-Blind Method, Female, Flow Cytometry, Glycated Hemoglobin, Granulocyte Colony-Stimulating Factor, Humans, Immunologic Factors, Male, T-Lymphocytes, Regulatory, Young Adult, Type 1 Diabetes TrialNet ATG-GCSF Study Group, Medical and Health Sciences, Endocrinology & Metabolism

الوصف: A three-arm, randomized, double-masked, placebo-controlled phase 2b trial performed by the Type 1 Diabetes TrialNet Study Group previously demonstrated that low-dose anti-thymocyte globulin (ATG) (2.5 mg/kg) preserved β-cell function and reduced HbA1c for 1 year in new-onset type 1 diabetes. Subjects (N = 89) were randomized to 1) ATG and pegylated granulocyte colony-stimulating factor (GCSF), 2) ATG alone, or 3) placebo. Herein, we report 2-year area under the curve (AUC) C-peptide and HbA1c, prespecified secondary end points, and potential immunologic correlates. The 2-year mean mixed-meal tolerance test-stimulated AUC C-peptide, analyzed by ANCOVA adjusting for baseline C-peptide, age, and sex (n = 82) with significance defined as one-sided P < 0.025, was significantly higher in subjects treated with ATG versus placebo (P = 0.00005) but not ATG/GCSF versus placebo (P = 0.032). HbA1c was significantly reduced at 2 years in subjects treated with ATG (P = 0.011) and ATG/GCSF (P = 0.022) versus placebo. Flow cytometry analyses demonstrated reduced circulating CD4:CD8 ratio, increased regulatory T-cell:conventional CD4 T-cell ratios, and increased PD-1+CD4+ T cells following low-dose ATG and ATG/GCSF. Low-dose ATG partially preserved β-cell function and reduced HbA1c 2 years after therapy in new-onset type 1 diabetes. Future studies should determine whether low-dose ATG might prevent or delay the onset of type 1 diabetes.

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/9x62j278Test

المؤلفون: Sreih, Antoine G., Cronin, Keri, Shaw, Dianne G., Young, Kalen, Burroughs, Cristina, Kullman, Joyce, Machireddy, Kirthi, McAlear, Carol A., Merkel, Peter A.

المساهمون: Patient-Centered Outcomes Research Institute, National Center for Advancing Translational Science (NCATS) and the National Institute of Arthritis and Musculoskeletal and Skin Diseases

المصدر: Orphanet Journal of Rare Diseases ; volume 16, issue 1 ; ISSN 1750-1172

مصطلحات موضوعية: Pharmacology (medical), Genetics (clinical), General Medicine

الوصف: 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.

الإتاحة: https://doi.org/10.1186/s13023-021-01794-5Test

المؤلفون: Banerjee, Shubhasree, George, Michael, Young, Kalen, Venkatachalam, Shilpa, Gordon, Jennifer, Burroughs, Cristina, Curtis, David, Ferrada, Marcela, Gavigan, Kelly, Grayson, Peter C., Kullman, Joyce, Danila, Maria I., Curtis, Jeffrey R., Shaw, Dianne G., Nowell, W. Benjamin, Merkel, Peter A.

المساهمون: Vasculitis Patient‐Powered Research Network

الوصف: 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. 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. 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. 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. ; Lewis Katz School of Medicine ; Neuroscience and Neurovirology

وصف الملف: 8 pages

العلاقة: Wiley; https://doi.org/10.1002/acr2.11204Test; ACR Open Rheumatology, Vol. 3, Issue 1; COVID-19 Research; Banerjee, S., George, M., Young, K., Venkatachalam, S., Gordon, J., Burroughs, C., Curtis, D., Ferrada, M., Gavigan, K., Grayson, P.C., Kullman, J., Danila, M.I., Curtis, J.R., Shaw, D.G., Benjamin Nowell, W., Merkel, P.A. and (2021), Effects of the COVID‐19 Pandemic on Patients Living With Vasculitis. ACR Open Rheumatology, 3: 17-24. https://doi.org/10.1002/acr2.11204Test; http://hdl.handle.net/20.500.12613/6173Test

الإتاحة: https://doi.org/20.500.12613/6173Test
https://doi.org/10.1002/acr2.11204Test
https://hdl.handle.net/20.500.12613/6173Test

المؤلفون: Applequist, Janelle, Burroughs, Cristina, Ramirez, Artemio, Merkel, Peter A., Rothenberg, Marc E., Trapnell, Bruce, Desnick, Robert J., Sahin, Mustafa, Krischer, Jeffrey P.

المساهمون: National Heart, Lung, and Blood Institute, Rare Diseases Clinical Research Network Data Coordinating Center, National Center for Advancing Translational Sciences & National Institute of Diabetes and Digestive Kidney Diseases, National Center for Advancing Translational Sciences & National Heart, Lung, and Blood Institute, National Center for Advancing Translational Sciences, National Institute of Neurological Disorders, and National Institute of Mental Health, National Center for Advancing Translational Sciences & National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Center for Advancing Translational Sciences & National Institute of Allergy and Infectious Diseases

المصدر: BMC Medical Research Methodology ; volume 20, issue 1 ; ISSN 1471-2288

مصطلحات موضوعية: Health Informatics, Epidemiology

الوصف: Background Participant recruitment for clinical research studies remains a significant challenge for researchers. Novel approaches to recruitment are necessary to ensure that populations are easier to reach. In the context of rare diseases, social media provides a unique opportunity for connecting with patient groups that have representatively lower diagnosis rates when compared with more common diseases or illness. We describe the implementation of designing a patient-centered approach to message design for the purposes of recruiting patients for clinical research studies for rare disease populations. Methods Using an iterative research approach, we analyzed our previous experience of using web-based direct-to-patient recruitment methods to compare these online strategies with traditional center of excellence recruitment strategies. After choosing six research studies for inclusion in the previous study, in-depth, online interviews ( n = 37) were conducted with patients represented in each disease category to develop and test recruitment message strategies for social media and a Web-based platform for patients to access study information and pre-screen. Finally, relationships were established with Patient Advocacy Groups representing each rare disease category to ensure further dissemination of recruitment materials via their own social media networks. Results Guided by social marketing theory, we created and tested various recruitment message designs. Three key message concepts preferred by patients emerged: (1) infographic; (2) positive emotional messages; and (3) educational information for sharing. A base study website was designed and created based on data from patient interviews. This website includes the option for potential participants to pre-screen and determine their eligibility for the study. Conclusions Study participants report wanting to be involved in the design and implementation of recruitment approaches for clinical research studies. The application of the aforementioned methods ...

الإتاحة: https://doi.org/10.1186/s12874-020-00926-yTest

المؤلفون: Chen, Xiaoxue, Agiro, Abiy, Nowell, W. Benjamin, Loud, Sara, McBurney, Robert, Young, Kalen, Sutphen, Rebecca, Bourquardez Clark, Elizabeth, Burroughs, Cristina M., Curtis, Jeffrey R., Sreih, Antoine G., Merkel, Peter A., Haynes, Kevin

المساهمون: Patient-Centered Outcomes Research Institute

المصدر: BMC Health Services Research ; volume 20, issue 1 ; ISSN 1472-6963

مصطلحات موضوعية: Health Policy

الوصف: Background Patient-powered research networks (PPRNs) have been employing and exploring different methods to engage patients in research activities specific to their conditions. One way to intensify patient engagement is to partner with payer stakeholders. The objective of this study was to evaluate the effectiveness of two common payer-initiated outreach methods (postal mail versus email) for inviting prospective candidates to participate in their initiatives. Methods This descriptive study linked members of a nationally-representative private insurance network to four disease-specific PPRN registries. Eligible members meeting diagnostic criteria who were not registered in any of the four PPRNs by 02/28/2018 were identified, and randomly assigned to either the mail or email group. They were contacted in two outreach efforts: first on 04/23/2018, and one follow-up on 05/23/2018. New registration rates by outreach method as of 8/31/2018 were determined by relinking. We compared registrants and non-registrants using bivariate analysis. Results A total of 14,571 patients were assigned to the mail group, and 14,574 to the email group. Invitations were successfully delivered to 13,834 (94.9%) mail group and 10,205 (70.0%) email group members. A small but significantly larger proportion of mail group members, ( n = 78; 0.54, 95% Confidence Interval [CI] {0.42–0.67%}) registered in PPRNs relative to the email group ( n = 24; 0.16, 95% CI {0.11–0.25%}), p < 0.001. Members who registered had more comorbidities, were more likely to be female, and had marginally greater medical utilization, especially emergency room visits, relative to non-registrants (52.0% vs. 42.5%, p = 0.05). Conclusion A health plan outreach to invite members to participate in PPRNs was modestly effective. Regular mail outperformed less costly email. Providing more value-add to participants may be a possible way to increase recruitment success.

الإتاحة: https://doi.org/10.1186/s12913-020-05325-zTest

المؤلفون: Young, Kalen, Kaminstein, Dana, Olivos, Ana, Burroughs, Cristina, Castillo-Lee, Celeste, Kullman, Joyce, McAlear, Carol, Shaw, Dianne G., Sreih, Antoine, Casey, George, Merkel, Peter A.

المساهمون: Patient-Centered Outcomes Research Institute

المصدر: Orphanet Journal of Rare Diseases ; volume 14, issue 1 ; ISSN 1750-1172

مصطلحات موضوعية: Pharmacology (medical), Genetics (clinical), General Medicine

الإتاحة: https://doi.org/10.1186/s13023-018-0969-1Test

المؤلفون: Agiro, Abiy, Chen, Xiaoxue, Eshete, Biruk, Sutphen, Rebecca, Bourquardez Clark, Elizabeth, Burroughs, Cristina M, Nowell, W Benjamin, Curtis, Jeffrey R, Loud, Sara, McBurney, Robert, Merkel, Peter A, Sreih, Antoine G, Young, Kalen, Haynes, Kevin

المساهمون: Patient-Centered Outcomes Research Institute, PCOR Institute, PCORI, iConquerMS

المصدر: Journal of the American Medical Informatics Association ; volume 26, issue 7, page 594-602 ; ISSN 1527-974X

مصطلحات موضوعية: Health Informatics

الوصف: Objective Patient-powered research networks (PPRNs) are a valuable source of patient-generated information. Diagnosis code-based algorithms developed by PPRNs can be used to query health plans’ claims data to identify patients for research opportunities. Our objective was to implement privacy-preserving record linkage processes between PPRN members’ and health plan enrollees’ data, compare linked and nonlinked members, and measure disease-specific confirmation rates for specific health conditions. Materials and Methods This descriptive study identified overlapping members from 4 PPRN registries and 14 health plans. Our methods for the anonymous linkage of overlapping members used secure Health Insurance Portability and Accountability Act–compliant, 1-way, cryptographic hash functions. Self-reported diagnoses by PPRN members were compared with claims-based computable phenotypes to calculate confirmation rates across varying durations of health plan coverage. Results Data for 21 616 PPRN members were hashed. Of these, 4487 (21%) members were linked, regardless of any expected overlap with the health plans. Linked members were more likely to be female and younger than nonlinked members were. Irrespective of duration of enrollment, the confirmation rates for the breast or ovarian cancer, rheumatoid or psoriatic arthritis or psoriasis, multiple sclerosis, or vasculitis PPRNs were 72%, 50%, 75%, and 67%, increasing to 91%, 67%, 93%, and 80%, respectively, for members with ≥5 years of continuous health plan enrollment. Conclusions This study demonstrated that PPRN membership and health plan data can be successfully linked using privacy-preserving record linkage methodology, and used to confirm self-reported diagnosis. Identifying and confirming self-reported diagnosis of members can expedite patient selection for research opportunities, shorten study recruitment timelines, and optimize costs.

الإتاحة: https://doi.org/10.1093/jamia/ocz012Test
http://academic.oup.com/jamia/article-pdf/26/7/594/34151456/ocz012.pdfTest

المؤلفون: Golenbiewski, Jon, Young, Kalen, Burroughs, Cristina, Kullman, Joyce, Merkel, Peter, Clowse, Megan

المصدر: Rheumatology ; volume 58, issue Supplement_2 ; ISSN 1462-0324 1462-0332

مصطلحات موضوعية: Pharmacology (medical), Rheumatology

الإتاحة: https://doi.org/10.1093/rheumatology/kez059.017Test
http://academic.oup.com/rheumatology/article-pdf/58/Supplement_2/kez059.017/28281311/kez059.017.pdfTest

المؤلفون: Sreih, Antoine G, Shaw, Dianne, Young, Kalen, Burroughs, Cristina, Kullman, Joyce, Machireddy, Kirthi, McAlear, Carol A, Casey, George, Merkel, Peter A

المصدر: Rheumatology ; volume 58, issue Supplement_2 ; ISSN 1462-0324 1462-0332

مصطلحات موضوعية: Pharmacology (medical), Rheumatology

الإتاحة: https://doi.org/10.1093/rheumatology/kez058.062Test
http://academic.oup.com/rheumatology/article-pdf/58/Supplement_2/kez058.062/28280883/kez058.062.pdfTest