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Setting a research agenda for vascular Ehlers-Danlos syndrome using a patient and stakeholder engagement model.

Published on Feb 27, 2020in Journal of Vascular Surgery3.243
· DOI :10.1016/J.JVS.2019.12.043
Liz Sage1
Estimated H-index: 1
,
Melissa Russo (Brown University)+ 5 AuthorsSherene Shalhub13
Estimated H-index: 13
Abstract
Abstract Objective Vascular Ehlers-Danlos syndrome (vEDS) is a rare, syndromic, heritable condition with life-threatening complications that include aortic and arterial aneurysms, dissection, and rupture. This study describes the formation of the vEDS Research Collaborative and methods used for stakeholder engagement. Methods The vEDS Research Collaborative was established with an engagement award from the Patient-Centered Outcomes Research Institute to create a framework for a patient-researcher partnership. Between October 1, 2017, and September 30, 2018, the Collaborative used the Patient-Centered Outcomes Research Institute Engagement Rubric to conduct stakeholder engagement to develop a patient-centered research agenda. A modified Delphi technique was used to develop and to refine research topics and questions, gathering input from all stakeholders during three rounds of feedback. Results Four topic areas were deemed important: mental health and quality of life issues, creating a care team, a holistic approach to vEDS management (medical and surgical), and pregnancy management. An online survey to rank a list of 12 research questions in these topic areas in order of importance was disseminated. The questions were ranked in order of importance through an online survey (N = 197 responses). The survey showed a high degree of alignment in the top priorities among stakeholders. There was a high degree of interest in pragmatic clinical trials evaluating medical management options and health-related quality of life outcomes. Conclusions The vEDS Research Collaborative has built a sustainable, coalition model of patient and stakeholder engagement, supported by the vEDS community, to identify a patient-centered, prioritized list of research questions. In articulating a shared vision for the future of vEDS research, the Collaborative has laid the groundwork for developing research protocols aligned with the highest priority questions for the individuals affected by this serious condition that can be translated into future clinical trials.
  • References (24)
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References24
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#1Sherene Shalhub (UW: University of Washington)H-Index: 13
#2Peter H. Byers (UW: University of Washington)H-Index: 70
Last. Karen Woo (UCLA: University of California, Los Angeles)H-Index: 10
view all 24 authors...
Abstract Objective Vascular Ehlers-Danlos syndrome (vEDS) is a rare disorder and 1 of 13 types of EDS. The syndrome results in aortic and arterial aneurysms and dissections at a young age. Diagnosis is confirmed with molecular testing via skin biopsy or genetic testing for COL3A1 pathogenic variants. We describe a multi-institutional experience in the diagnosis of vEDS from 2000 to 2015. Methods This is a multi-institutional cross-sectional retrospective study of individuals with vEDS. The insti...
Source
#1Sherene Shalhub (UW: University of Washington)H-Index: 13
#2Liz Sage (UW: University of Washington)H-Index: 1
view all 9 authors...
Abstract Objective Patient centered research requires active engagement of patients. The Vascular Ehlers-Danlos Syndrome (vEDS) research collaborative was established to ascertain patient-centered vEDS research priorities and to engage affected individuals as research partners. Evaluation of access to information and interest in research among individuals with vEDS was the first step undertaken as part of this work. Methods A 28 question survey was created to evaluate four domains of interest: D...
Source
#1Sherene Shalhub (UW: University of Washington)H-Index: 13
#2Peter H. Byers (UW: University of Washington)H-Index: 70
Last. Karen Woo (UCLA: University of California, Los Angeles)H-Index: 10
view all 24 authors...
Abstract Objective Vascular Ehlers-Danlos syndrome (vEDS) is a rare connective tissue disorder owing to pathogenic variants in COL3A1 that lead to impaired type III collagen production. We aim to describe the contemporary multi-institutional experience of aortic and arterial pathology in individuals with vEDS, to evaluate disease patterns and refine management recommendations. Methods This cross-sectional, retrospective study of individuals with genetically confirmed vEDS was conducted between 2...
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#1Sherene Shalhub (UW: University of Washington)H-Index: 13
#2Enid Neptune (Johns Hopkins University)H-Index: 17
Last. Dianna M. Milewicz (University of Texas Health Science Center at Houston)H-Index: 58
view all 7 authors...
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#1De Backer J (Ghent University Hospital)H-Index: 41
#2T De Backer (Ghent University Hospital)H-Index: 21
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#1Michael Frank (French Institute of Health and Medical Research)H-Index: 13
#2Salma Adham (Paris IV: Paris-Sorbonne University)H-Index: 1
Last. Xavier JeunemaitreH-Index: 69
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Abstract Background Vascular Ehlers-Danlos syndrome (vEDS) is a rare genetic connective tissue disorder secondary to pathogenic variants within the COL3A1 gene, resulting in exceptional arterial and organ fragility and premature death. The only published clinical trial to date demonstrated the benefit of celiprolol on arterial morbimortality. Objectives The authors herein describe the outcomes of a large cohort of vEDS patients followed ≤17 years in a single national referral center. Methods All...
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#1James K. Stoller (Cleveland Clinic)H-Index: 51
Rare diseases pose particular challenges to patients who are affected, to the clinicians who care for them, and to the investigators who study their conditions. Although individually uncommon, rare diseases are common in the aggregate, with approximately 7,000 described rare diseases affecting 25 to 30 million US adults. Challenges posed to affected individuals and their families largely regard being diagnosed, receiving optimal care, and affording disease-specific medications. Challenges facing...
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#1Naveen Pemmaraju (University of Texas MD Anderson Cancer Center)H-Index: 29
#2Audun UtengenH-Index: 6
Last. Michael A. ThompsonH-Index: 33
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Purpose of review The use of social media has now become a standard means of communication for many individuals worldwide. The use of one specific form of social media, Twitter, has increased among healthcare providers, both as a means of information gathering and as a conduit for original content creation. Recently, major efforts by users have been put forward to help streamline the unprecedented amount of information that can be found on Twitter. These efforts have led to the creation of disea...
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#1Christopher-Paul Milne (Tufts Center for the Study of Drug Development)H-Index: 11
#2Wendi Ni (Tufts Center for the Study of Drug Development)H-Index: 1
Abstract Social media has transformed how people interact with one another through the Internet, and it has the potential to do the same for orphan drug development. Currently, social media influences the orphan drug development process in the following three ways: assisting the study of orphan diseases, increasing the awareness of orphan disease, and playing a vital role in clinical trials. However, there are some caveats to the utilization of social media, such as the need to protect patient p...
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#1Susan Sheridan (Patient-Centered Outcomes Research Institute)H-Index: 1
#2Suzanne Schrandt (Patient-Centered Outcomes Research Institute)H-Index: 3
Last. Kathryn Paez (AIR: American Institutes for Research)H-Index: 6
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