A Quality Assessment of the ARM-Net Registry Design and Data Collection

Hageman, Isabel C.; Steeg, H. J. J. van der; Jenetzky, Ekkehart; Trajanovska, Misel; King, Sebastian K.; Blaauw, Ivo de; Rooij, Iris A. L. M. van; Aminoff, Dalia; Amerstorfer, Eva E.; Till, Holger; Bagolan, Pietro; Iacobelli, Barbara Daniela; Çavuşoğlu, Hakan; Onur, Özen; Deluggi, Stefan; Ludwiczek, Johanna; Divarcı, Emre; Fanjul, M; Fascetti-Leon, Francesco; Vázquez, Araceli García; Giné, C; Gorter, Ramon R.; Jong, Justin de; Goseman, Jan; Lacher, Martin; Grano, Caterina; Grasshoff‐Derr, Sabine; Haanen, Michel; Leva, Ernesto; Morandi, Anna; Lisi, Gabriele; Makedonsky, I. A.; Marcelis, Carlo; Midrio, Paola; Miserez, Marc; Mohideen, Mazeena; PiniPrato, Alessio; Reck-Burneo, Carlos A.; Reutter, Heiko; Rohleder, Stephan; Samuk, Inbal; Schmiedeke, Eberhard; Schwarzer, Nicole; Sloots, Pim; Stenström, Pernilla; Verhaak, Chris M.; Vilanova-Sánchez, Alejandra; Volk, Patrick; Witvliet, M.

doi:10.1016/j.jpedsurg.2023.02.049

Cited by 6 publications

(7 citation statements)

References 20 publications

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“…Sustainability also necessitates a long-term comprehensive financial plan including future-oriented exit strategies and succession planning [ 39 , 47 , 50 ]. A registry’s utility, effectiveness, efficiency, and agility are also important to ensure its long-term sustainability [ 27 , 55 ].…”

Section: Resultsmentioning

confidence: 99%

“…A data dictionary provides clear instructions for data entry and analysis by defining all data elements and their purpose as well as the coding values including permissible values, representation class, data type, and format [ 17 , 20 ]. Complete alignment between the variables described in the data dictionary and those captured by the registry’s interface is expected [ 55 ].…”

Section: Resultsmentioning

confidence: 99%

“…Data quality and assurance plans which include data validation (e.g., medical, clinical, and record audit) [ 61 ] and a review of RDR-generated studies ensure compliance with RDR-based studies’ protocols and ethical and regulatory requirements [ 12 , 25 , 32 , 69 ]. Data quality and assurance processes necessitate routine data quality checks and data cleaning to ensure the enrolment of eligible patients, data completeness, validity, and coherence while mitigating record duplication and errors [ 26 , 31 , 35 , 45 , 55 ]. Data audits can be performed by internal registry staff or an external service provider, or some combination [ 37 ].…”

Section: Resultsmentioning

confidence: 99%

“… Sustainability • Access to multiple funding sources to facilitate long-term sustainability [ 17 , 18 , 25 , 35 , 44 , 45 , 49 ]. • Long-term comprehensive financial plan [ 25 – 27 , 29 , 30 , 36 , 39 , 47 , 50 , 52 , 54 , 55 ]. Ethics, Legal, Privacy • Compliance with the relevant (international, national, or local) ethical, legal, and privacy regulations [ 23 , 25 , 32 , 33 , 35 , 36 , 39 , 42 , 49 , 50 ].…”

Section: Discussionmentioning

confidence: 99%

“… Data dictionary • Data dictionary provides clear instructions for data entry [ 17 , 20 , 25 ]. • Complete alignment between the data dictionary and the data being collected [ 55 ]. Data collection • Data collection procedure and standardized data collection forms [ 18 , 35 , 51 ].…”

Section: Discussionmentioning

confidence: 99%

See 4 more Smart Citations

Scoping review of the recommendations and guidance for improving the quality of rare disease registries

Tarride,

Okoh,

Aryal

et al. 2024

Orphanet J Rare Dis

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Background Rare disease registries (RDRs) are valuable tools for improving clinical care and advancing research. However, they often vary qualitatively, structurally, and operationally in ways that can determine their potential utility as a source of evidence to support decision-making regarding the approval and funding of new treatments for rare diseases. Objectives The goal of this research project was to review the literature on rare disease registries and identify best practices to improve the quality of RDRs. Methods In this scoping review, we searched MEDLINE and EMBASE as well as the websites of regulatory bodies and health technology assessment agencies from 2010 to April 2023 for literature offering guidance or recommendations to ensure, improve, or maintain quality RDRs. Results The search yielded 1,175 unique references, of which 64 met the inclusion criteria. The characteristics of RDRs deemed to be relevant to their quality align with three main domains and several sub-domains considered to be best practices for quality RDRs: (1) governance (registry purpose and description; governance structure; stakeholder engagement; sustainability; ethics/legal/privacy; data governance; documentation; and training and support); (2) data (standardized disease classification; common data elements; data dictionary; data collection; data quality and assurance; and data analysis and reporting); and (3) information technology (IT) infrastructure (physical and virtual infrastructure; and software infrastructure guided by FAIR principles (Findability; Accessibility; Interoperability; and Reusability). Conclusions Although RDRs face numerous challenges due to their small and dispersed populations, RDRs can generate quality data to support healthcare decision-making through the use of standards and principles on strong governance, quality data practices, and IT infrastructure.

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

See 3 more Smart Citations

Scoping review of the recommendations and guidance for improving the quality of rare disease registries

Tarride,

Okoh,

Aryal

et al. 2024

Orphanet J Rare Dis

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Congenital Heart Defects in Patients with Anorectal Malformations: A Retrospective Cohort Study of 281 Patients

de Beaufort,

Mackay,

Stevens

et al. 2024

Pediatr Cardiol

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In patients born with anorectal malformations (ARM), additional congenital heart defects (CHD) can occur. We aimed to provide an overview on disease and treatment details of CHD identified in patients born with ARM, from a unique large cohort of a very rare disease. We performed a retrospective single-center cohort study between January 2000 and July 2023. All consecutive patients with ARM were included. Outcomes were the number of patients with CHD, and screening percentage and percentage of patients diagnosed with CHD over 3 time periods (2000–2006, 2007–2014, 2015–2023). We used uni- and multi-variable logistic regression analyses to search for associations between CHD present and baseline characteristics. In total, 281 patients were included. Some 241 (85.8%) underwent echocardiography, of whom 80 (33.2%) had CHD. Screening percentage with echocardiography increased (74.1% vs. 85.7% vs. 95.9%, p < 0.001) and percentage of patients diagnosed with CHD remained similar over time (30.2% vs. 34.5% vs. 34.0%, p = 0.836). Atrial and ventricular septal defects (n = 36, n = 29), and persistent left superior vena cava (n = 17) were most identified. The presence of VACTERL-association or a genetic syndrome was independently associated with the presence of CHD. CHD were present in 33% of patients with ARM that underwent echocardiography. Over time, the number of CHD identified through screening remained similar. Patients with the presence of VACTERL-association or a genetic syndrome had a higher risk of having CHD. Therefore, acknowledging the potential presence of CHD in patients with ARM remains important.

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Transitional Care for Patients with Congenital Colorectal Diseases: An EUPSA Network Office, ERNICA, and eUROGEN Joint Venture

de Beaufort,

Aminoff,

de Blaauw

et al. 2023

Journal of Pediatric Surgery

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A Quality Assessment of the ARM-Net Registry Design and Data Collection

Cited by 6 publications

References 20 publications

Scoping review of the recommendations and guidance for improving the quality of rare disease registries

Scoping review of the recommendations and guidance for improving the quality of rare disease registries

Congenital Heart Defects in Patients with Anorectal Malformations: A Retrospective Cohort Study of 281 Patients

Transitional Care for Patients with Congenital Colorectal Diseases: An EUPSA Network Office, ERNICA, and eUROGEN Joint Venture

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