The Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry
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(Published version)
Date
2023
Authors
Fox, L.C.
McQuilten, Z.K.
Firkin, F.
Fox, V.
Badoux, X.
Bajel, A.
Barbaro, P.
Cole-Sinclair, M.F.
Forsyth, C.
Gibson, J.
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Journal article
Citation
Best Practice and Research: Clinical Haematology, 2023; 36(4):101516-1-101516-10
Statement of Responsibility
Lucy C. Fox, Zoe K. McQuilten, Frank Firkin, Vanessa Fox, Xavier Badoux, Ashish Bajel, Pasquale Barbaro, Merrole F. Cole-Sinclair, Cecily Forsyth, John Gibson, Devendra K. Hiwase, Anna Johnston, Anthony Mills Fernando Roncolato, Robyn Sutherland, Jeff Szer, Stephen B. Ting, Shahla Vilcassim, Lauren Young, Neil A. Waters, Erica M. Wood, on behalf of the Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry (AAR) Investigators
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Abstract
The bone marrow failure syndromes (BMFS) are a diverse group of acquired and inherited diseases which may manifest in cytopenias, haematological malignancy and/or syndromic multisystem disease. Patients with BMFS frequently experience poor outcomes, and improved treatment strategies are needed. Collation of clinical characteristics and patient outcomes in a national disease-specific registry represents a powerful tool to identify areas of need and support clinical and research collaboration. Novel treatment strategies such as gene therapy, particularly in rare diseases, will depend on the ability to identify eligible patients alongside the molecular genetic features of their disease that may be amenable to novel therapy. The Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry (AAR) aims to improve outcomes for all paediatric and adult patients with BMFS in Australia by describing the demographics, treatments (including supportive care) and outcomes, and serving as a resource for research and practice improvement.
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© 2023 The Authors. Published by Elsevier Ltd. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).