Consensus on the criteria needed for creating a rare-disease patient registry. A Delphi study

Cavero‐Carbonell, Clara; Gras-Colomer, Elena; Guaita-Calatrava, Rosana; López-Briones, Carmen González; Amorós, Rubén; Abaitua, I.; Paz, Manuel Posada de la; Zurriaga, Óscar

doi:10.1093/pubmed/fdv099

Cited by 12 publications

(14 citation statements)

References 26 publications

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“…Rare disease registries are helpful repositories of information for health-care planning, implementation, and follow-up investigation in specific populations, but patients must be aware of their existence. 16 Innovative online methods for reaching patients with rare CHD have recently proven effective. For example, in 2014, Schumacher et al 17 successfully utilised social media to recruit a large international patient cohort with two rare complications of CHD: Fontanassociated protein-losing enteropathy and plastic bronchitis.…”

Section: Discussionmentioning

confidence: 99%

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Feasibility of a healthcare system-based tetralogy of Fallot patient registry

et al. 2017

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Feasibility of a healthcare system-based tetralogy of Fallot patient registry

et al. 2017

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“…Participants are guided to reach a form of consensus by anonymously communicating their opinions, considering the opinions of others and evaluating where their opinions align (Diamond et al, 2014; Fitch et al, 2001; Sinha et al, 2011). The e‐Delphi method is well suited to research in rare conditions because the guide for best practice will come from a relatively small group of experts (Cavero‐Carbonell et al, 2015; Linertova et al, 2012), therefore, large general surveys may be inappropriate.…”

Section: Methodsmentioning

confidence: 99%

What do I need to know? Essential educational concepts for complex regional pain syndrome

Moore

Braithwaite

Stanton

et al. 2022

European Journal of Pain

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Background Complex Regional Pain Syndrome (CRPS) is a rare but disabling pain condition. Accurate and timely education about CRPS is key to promote optimal clinical outcomes, but it is unclear what the content of that education should be. We aimed to determine the content that both people with CRPS and expert health care professionals (HCPs) reported as important. Methods An international three‐round e‐Delphi was conducted, recruiting adults diagnosed with CRPS and HCPs. In Round 1, participants were asked to list the most important information people with CRPS should know regarding the condition. Data were organized into concepts and allocated to themes. In Rounds 2 and 3, participants rated each concept on a 9‐point Likert Scale, categorized as ‘not important’ (0–3), ‘important’ (4–6) and ‘very important’ (7–9). A concept attained consensus when ≥75% agreement was reached within a category. Results Sixty‐two participants (HCPs: n = 7; CRPS: n = 55) proposed 193 concepts in Round 1, resulting in 22 themes. Fifteen additional concepts were identified in Round 2, resulting in a total of 208 concepts. From that list, 48 concepts that emphasized understanding and evidence‐based management of the disorder, the importance of self‐management strategies, pacing and movement, reached joint consensus as ‘very important’. One concept: ‘Advise that movement does not help’ reached joint consensus as ‘not important’. Conclusion Forty‐eight concepts were jointly considered ‘very important’ for future CRPS‐related educational content. Future research to better understand group differences and to canvas a broader HCP group is warranted. Significance This e‐Delphi study identified the 48 core concepts that those with the lived experience of CRPS, and advanced practitioner health care professionals jointly rated as ‘very important’ to include in fundamental and accessible educational material.

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“…En el ámbito de la salud suele distinguirse entre registro de pacientes y registro de enfermedades, aunque este es un asunto que va más allá de la diferencia semántica (Bellgard et al, 2013;Cavero-Carbonell et al, 2015).…”

Section: El Concepto De Registrounclassified

Registros y biobancos de enfermedades raras. Una oportunidad para avanzar

Zurriaga

Martínez

Corrochano

et al. 2018

Arbor

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Los registros y los biobancos de enfermedades raras se han convertido durante los últimos años en instrumentos clave para la investigación de este tipo de enfermedades. En el presente artículo se hace una revisión del concepto de registro y de la utilidad de los mismos en las enfermedades raras, se muestra un panorama de los registros de enfermedades raras en el ámbito internacional y nacional y se exponen los retos y las oportunidades que presentan los registros. Se presentan asimismo los biobancos como estructuras creadas con el fin de gestionar muestras biológicas para su uso en investigación. Se abordan los aspectos éticos, legales y sociales relacionados con la actividad de los biobancos y los aspectos técnicos asociados con la armonización, estandarización y control de calidad de las muestras y de la información relacionada y se ofrece un panorama general de los biobancos, recalcando su importancia en el ámbito de las enfermedades raras y la necesidad de colaboración con los registros de enfermedades raras.

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Consensus on the criteria needed for creating a rare-disease patient registry. A Delphi study

Abstract: The consensus method used made it possible to define the characteristics of a PR based on expert opinion within a rare-disease framework. This study may serve as a guide for helping other researchers plan and build a rare-disease PR.

Cited by 12 publications

References 26 publications

Feasibility of a healthcare system-based tetralogy of Fallot patient registry

Feasibility of a healthcare system-based tetralogy of Fallot patient registry

What do I need to know? Essential educational concepts for complex regional pain syndrome

Registros y biobancos de enfermedades raras. Una oportunidad para avanzar

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