التفاصيل البيبلوغرافية
| العنوان: |
The Metreleptin Effectiveness and Safety Registry (MEASuRE): concept, design and challenges |
| المؤلفون: |
Haymond, Morey W, Araújo-Vilar, David, Balser, John, Lewis, James H, Louzado, Ruth, Musso, Carla, von Schnurbein, Julia, Wabitsch, Martin |
| المساهمون: |
Amryt Pharmaceuticals Inc. |
| المصدر: |
Orphanet Journal of Rare Diseases ; volume 18, issue 1 ; ISSN 1750-1172 |
| بيانات النشر: |
Springer Science and Business Media LLC |
| سنة النشر: |
2023 |
| مصطلحات موضوعية: |
Pharmacology (medical), Genetics (clinical), General Medicine |
| الوصف: |
Background Metreleptin, a recombinant analog of human leptin, is an approved therapy, adjunct to diet, to treat the metabolic complications of leptin deficiency in patients with lipodystrophy – a group of rare diseases characterized by a paucity of adipose tissue. MEASuRE (Metreleptin Effectiveness And Safety Registry) is a post-authorization, voluntary registry that gathers long-term safety and effectiveness data on metreleptin. Here, we present the aims and evolution of MEASuRE. Methods MEASuRE was established to collect data from patients receiving commercially supplied metreleptin in the United States (US) and European Union (EU). MEASuRE aims to determine the incidence and severity of safety events and describe the clinical characteristics and therapeutic outcomes in the metreleptin-treated population. A key feature of MEASuRE is that it accumulates data from different sources to meet post-authorization objectives. US data are received directly from treating physicians via a contract research organization-mediated electronic data capture system. In the EU, data are received via the European Registry of Lipodystrophies managed by the European Consortium of Lipodystrophies (ECLip), a platform established by researchers and physicians to advance the knowledge of lipodystrophy. MEASuRE complies with applicable regulatory requirements governing privacy, and the storage, management, and access of data. Results Leveraging processes, infrastructure, and data from the ECLip registry presented several challenges that were addressed during MEASuRE’s development, including the expansion of the ECLip registry to accommodate MEASuRE-specific data elements, extensive data matching processes to ensure data consistency regardless of source, and rigorous data validation following the amalgamation of global data. Through the support of ECLip, MEASuRE is now a fully operational registry with the capacity for gathering and integrating standardized US- and EU-derived data. As of 31st October 2022, 15 US and four EU ... |
| نوع الوثيقة: |
article in journal/newspaper |
| اللغة: |
English |
| DOI: |
10.1186/s13023-023-02714-5 |
| DOI: |
10.1186/s13023-023-02714-5.pdf |
| DOI: |
10.1186/s13023-023-02714-5/fulltext.html |
| الإتاحة: |
https://doi.org/10.1186/s13023-023-02714-5Test |
| حقوق: |
https://creativecommons.org/licenses/by/4.0Test ; https://creativecommons.org/licenses/by/4.0Test |
| رقم الانضمام: |
edsbas.CEE0C9CF |
| قاعدة البيانات: |
BASE |
