The Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry

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dc.contributor.authorFox, L.C.
dc.contributor.authorMcQuilten, Z.K.
dc.contributor.authorFirkin, F.
dc.contributor.authorFox, V.
dc.contributor.authorBadoux, X.
dc.contributor.authorBajel, A.
dc.contributor.authorBarbaro, P.
dc.contributor.authorCole-Sinclair, M.F.
dc.contributor.authorForsyth, C.
dc.contributor.authorGibson, J.
dc.contributor.authorHiwase, D.K.
dc.contributor.authorJohnston, A.
dc.contributor.authorMills, A.
dc.contributor.authorRoncolato, F.
dc.contributor.authorSutherland, R.
dc.contributor.authorSzer, J.
dc.contributor.authorTing, S.B.
dc.contributor.authorVilcassim, S.
dc.contributor.authorYoung, L.
dc.contributor.authorWaters, N.A.
dc.contributor.authoret al.
dc.date.issued2023
dc.description.abstractThe 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.
dc.description.statementofresponsibilityLucy 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
dc.identifier.citationBest Practice and Research: Clinical Haematology, 2023; 36(4):101516-1-101516-10
dc.identifier.doi10.1016/j.beha.2023.101516
dc.identifier.issn1521-6926
dc.identifier.issn1532-1924
dc.identifier.orcidHiwase, D.K. [0000-0002-6666-3056]
dc.identifier.urihttps://hdl.handle.net/2440/143150
dc.language.isoen
dc.publisherElsevier BV
dc.relation.granthttp://purl.org/au-research/grants/nhmrc/1194811
dc.relation.granthttp://purl.org/au-research/grants/nhmrc/1177784
dc.rights© 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/).
dc.source.urihttp://dx.doi.org/10.1016/j.beha.2023.101516
dc.subjectAplastic anaemia; Inherited bone marrow failure; Inherited predisposition to haematological; malignancy; Rare disease registry
dc.subject.meshHumans
dc.subject.meshAnemia, Aplastic
dc.subject.meshBone Marrow Diseases
dc.subject.meshSyndrome
dc.subject.meshRegistries
dc.subject.meshAdult
dc.subject.meshChild
dc.subject.meshAustralia
dc.subject.meshBone Marrow Failure Disorders
dc.titleThe Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry
dc.typeJournal article
pubs.publication-statusPublished

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