The Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry

IF 2.2 4区 医学 Q3 HEMATOLOGY Best Practice & Research Clinical Haematology Pub Date : 2023-10-23 DOI:10.1016/j.beha.2023.101516
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
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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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澳大利亚再生障碍性贫血和其他骨髓衰竭综合征登记处
骨髓衰竭综合征(BMFS)是一组不同的获得性和遗传性疾病,可能表现为细胞减少、血液恶性肿瘤和/或综合征性多系统疾病。BMFS患者往往预后不佳,需要改进治疗策略。在国家疾病特异性登记中整理临床特征和患者结果是确定需要领域并支持临床和研究合作的有力工具。新的治疗策略,如基因治疗,特别是在罕见疾病中,将取决于识别符合条件的患者及其疾病的分子遗传特征的能力,这些特征可能适用于新的治疗方法。澳大利亚再生障碍性贫血和其他骨髓衰竭综合征登记处(AAR)旨在通过描述人口统计学、治疗(包括支持性护理)和结果,并作为研究和实践改进的资源,改善澳大利亚所有患有BMFS的儿科和成人患者的结局。
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来源期刊
CiteScore
4.20
自引率
0.00%
发文量
42
审稿时长
35 days
期刊介绍: Best Practice & Research Clinical Haematology publishes review articles integrating the results from the latest original research articles into practical, evidence-based review articles. These articles seek to address the key clinical issues of diagnosis, treatment and patient management. Each issue follows a problem-orientated approach which focuses on the key questions to be addressed, clearly defining what is known and not known, covering the spectrum of clinical and laboratory haematological practice and research. Although most reviews are invited, the Editor welcomes suggestions from potential authors.
期刊最新文献
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