Adrenal crisis and sick day episodes among CAH patients: preliminary report based on international CAH (I-CAH) registry

Karunasena, Nayananjani; Daniel, Eleni; Bryce, Jillian; Jiang, Jipu; Faisal, Ahmed S; Güran, Tülay; Mendonça, Berenice Bilharinho; Bachega, Tânia A.S.S.; Blankenstein, Oliver; Koehler, B; Neumann, Uta; Acerini, Carlo L.; Krone, Nils; Bonfig, Walter; Mohnike, Klaus; Elsedfy, Heba; Ross, Richard

doi:10.1530/endoabs.41.ep14

Cited by 2 publications

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“…The I-CAH longitudinal module can also be used to collect prospective data on the occurrence on adverse events in CAH, including sick day episodes and adrenal crises. A preliminary analysis of this data has demonstrated that patients with CAH experience frequent sick day episodes [23] and further studies are ongoing to determine factors which may be associated with the occurrence of adrenal insufficiency related adverse events. There are currently seven ongoing studies that are using the I-CAH Registry including industry supported-studies that are examining the feasibility of studies using novel therapeutic options in CAH.…”

Section: Past and Current Research Activities Of The I-cah Registrymentioning

confidence: 99%

The Role of International Databases in Understanding the Aetiology and Consequences of Differences/Disorders of Sex Development

Ali

Lucas‐Herald

Bryce

et al. 2019

IJMS

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The International Disorders of Sex Development (I-DSD) and International Congenital Adrenal Hyperplasia registry (I-CAH) Registries were originally developed over 10 years ago and have since supported several strands of research and led to approximately 20 peer-reviewed publications. In addition to acting as an indispensable tool for monitoring clinical and patient-centered outcomes for improving clinical practice, the registries can support a wide nature of primary and secondary research and can also act as a platform for pharmacovigilance, given their ability to collect real world patient data within a secure, ethics approved virtual research environment. The challenge for the future is to ensure that the research community continues to use the registries to improve our understanding of Disorders of Sex Development (DSD).

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Section: Past and Current Research Activities Of The I-cah Registrymentioning

confidence: 99%

The Role of International Databases in Understanding the Aetiology and Consequences of Differences/Disorders of Sex Development

Ali

Lucas‐Herald

Bryce

et al. 2019

IJMS

Self Cite

View full text Add to dashboard Cite

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An assessment of the quality of the I-DSD and the I-CAH registries - international registries for rare conditions affecting sex development

Kourime

Bryce

Jiang

et al. 2017

Orphanet J Rare Dis

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BackgroundWith the proliferation of rare disease registries, there is a need for registries to undergo an assessment of their quality against agreed standards to ensure their long-term sustainability and acceptability.This study was performed to evaluate the I-DSD and I-CAH Registries and identify their strengths and weaknesses.MethodsThe design and operational aspects of the registries were evaluated against published quality indicators. Additional criteria included the level of activity, international acceptability of the registries and their use for research.ResultsThe design of the I-DSD and I-CAH Registries provides them with the ability to perform multiple studies and meet the standards for data elements, data sources and eligibility criteria. The registries follow the standards for data security, governance, ethical and legal issues, sustainability and communication of activities. The data have a high degree of validity, consistency and accuracy and the completeness is maximal for specific conditions such as androgen insensitivity syndrome and congenital adrenal hyperplasia. In terms of research output, the external validity is strong but the wide variety of cases needs further review. The internal validity of data was condition specific and highest for conditions such as congenital adrenal hyperplasia. The shift of the registry from a European registry to an international registry and the creation of a discrete but linked CAH registry increased the number of users and stakeholders as well as the international acceptability of both registries.ConclusionsThe I-DSD and I-CAH registries comply with the standards set by expert organisations. Recent modifications in their operation have allowed the registries to increase their user acceptability.

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Adrenal crisis and sick day episodes among CAH patients: preliminary report based on international CAH (I-CAH) registry

Cited by 2 publications

References 0 publications

The Role of International Databases in Understanding the Aetiology and Consequences of Differences/Disorders of Sex Development

The Role of International Databases in Understanding the Aetiology and Consequences of Differences/Disorders of Sex Development

An assessment of the quality of the I-DSD and the I-CAH registries - international registries for rare conditions affecting sex development

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