Design and development of the Australian and New Zealand (ANZ) myeloma and related diseases registry
MetadataShow full item record
© 2016 The Author(s). Background: Plasma cell dyscrasias (PCD) are a spectrum of disorders resulting from the clonal expansion of plasma cells, ranging from the pre-malignant condition monoclonal gammopathy of undetermined significance (MGUS) to multiple myeloma (MM). MM generates a significant burden of disease on the community and it is predicted that it will increase in both incidence and prevalence owing to an ageing population and longer survival secondary to new therapeutic options. Robust and comprehensive clinical data are currently lacking but are required to define current diagnostic, investigational and management patterns in Australia and New Zealand (ANZ) for comparison to both local and international guidelines for standards of care. A clinical registry can provide this information and subsequently support development of strategies to address any differences, including providing a platform for clinical trials. The Myeloma and Related Diseases Registry (MRDR) was developed to monitor and explore variations in practices, processes and outcomes in ANZ and provide benchmark outcomes nationally and internationally for PCD. This paper describes the MRDR aims, development and implementation and discusses challenges encountered in the process. Methods: The MRDR was established in 2012 as an online database for a multi-centre collaboration across ANZ, collecting prospective data on patients with a diagnosis of MGUS, MM, solitary plasmacytoma or plasma cell leukaemia. Development of the MRDR required multi-disciplinary team participation, IT and biostatistical support as well as financial resources. Results: More than 1250 patients have been enrolled at 23 sites to date. Here we describe how database development, data entry and securing ethics approval have been major challenges for participating sites and the coordinating centre, and our approaches to resolving them. Now established, the MRDR will provide clinically relevant and credible monitoring, therapy and 'real world' outcome data, to support the conduction of high quality studies. In addition, the Myeloma 1000 sub-study is establishing a repository of paired peripheral blood specimens from registry patients to study mechanisms underlying disease progression. Conclusion: Establishment of the MRDR has been challenging, but it is a valuable investment that will provide a platform for coordinated national and international collaboration for clinical research in PCD in ANZ.
This open access article is distributed under the Creative Commons license https://creativecommons.org/licenses/by/4.0/
Showing items related by title, author, creator and subject.
Design of a framework for the deployment of collaborative independent rare disease-centric registries: Gaucher disease registry modelBellgard, M.; Napier, K.; Bittles, A.; Szer, J.; Fletcher, S.; Zeps, Nikolajs; Hunter, A.; Goldblatt, J. (2016)Orphan drug clinical trials often are adversely affected by a lack of high quality treatment efficacy data that can be reliably compared across large patient cohorts derived from multiple governmental and country ...
Wilson, P.; D'Agostino, R.; Bhatt, D.; Eagle, K.; Pencina, M.; Smith, S.; Alberts, M.; Dallongeville, J.; Goto, S.; Hirsch, A.; Liau, C.; Ohman, E.; Röther, J.; Reid, Christopher; Mas, J.; Steg, P. (2012)Background: Prediction models for cardiovascular events and cardiovascular death in patients with established cardiovascular disease are not generally available. Methods: Participants from the prospective REduction of ...
Kent, Peter; Kongsted, A.; Jensen, T.; Albert, H.; Schiøttz-Christensen, B.; Manniche, C. (2015)© 2015 Kent et al. Background: Large-scale clinical registries are increasingly recognized as important resources for quality assurance and research to inform clinical decision-making and health policy. We established a ...