Best Practice & Research Clinical Haematology最新文献_第4页

Progress and challenges in the acute leukemia field 急性白血病领域的进展与挑战

IF 2.1 4区医学

Best Practice & Research Clinical Haematology Pub Date : 2023-11-07 DOI: 10.1016/j.beha.2023.101524

Daniel A. Pollyea

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The role of randomized controlled trials, registries, observational databases in evaluating new interventions 随机对照试验、登记、观察性数据库在评估新干预措施中的作用

IF 2.1 4区医学

Best Practice & Research Clinical Haematology Pub Date : 2023-10-31 DOI: 10.1016/j.beha.2023.101523

Robert Peter Gale , Mei-Jie Zhang , Hillard M. Lazarus

{"title":"The role of randomized controlled trials, registries, observational databases in evaluating new interventions","authors":"Robert Peter Gale , Mei-Jie Zhang , Hillard M. Lazarus","doi":"10.1016/j.beha.2023.101523","DOIUrl":"https://doi.org/10.1016/j.beha.2023.101523","url":null,"abstract":"<div><p>Approaches to comparing safety and efficacy of interventions include analyzing data from randomized controlled trials (RCTs), registries and observational databases (ODBs). RCTs are regarded as the <em>gold standard</em> but data from such trials are sometimes unavailable because a disease is uncommon, because the intervention is uncommon, because of structural limitations or because randomization cannot be done for practical or (seemingly) ethical reasons. There are many examples of an unproved intervention being so widely-believed to be effective that clinical trialists and potential subjects decline randomization. Often, when a RCT is finally done the intervention is proved ineffective or even harmful. These situations are termed <em>medical reversals</em> and are not uncommon [1,2]. There is also the dilemma of when seemingly similar RCTs report discordant conclisions</p><p>Data from high-quality registries, especially ODBs can be used when data from RCTs are unavailable but also have limitations. Biases and confounding co-variates may be unknown, difficult or impossible to identify and/or difficult to adjust for adequately. However, ODBs sometimes have large numbers of diverse subjects and often give answers more useful to clinicians than RCTs. Side-by-side comparisons suggest analyses from high-quality ODBs often give similar conclusions from high quality RCTs. Meta-analyses combining data from RCTs, registries and ODBs are sometimes appropriate. We suggest increased use of registries and ODBs to compare efficacy of interventions.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 4","pages":"Article 101523"},"PeriodicalIF":2.1,"publicationDate":"2023-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1521692623000841/pdfft?md5=a796816b4a1c3b921a354e3bc9ade39d&pid=1-s2.0-S1521692623000841-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"92046023","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

The international cooperative Gaucher group (ICCG) Gaucher registry 国际合作戈歇组织(ICCG)戈歇注册

IF 2.1 4区医学

Best Practice & Research Clinical Haematology Pub Date : 2023-10-31 DOI: 10.1016/j.beha.2023.101522

Neal J. Weinreb

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The Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry 澳大利亚再生障碍性贫血和其他骨髓衰竭综合征登记处

IF 2.1 4区医学

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

{"title":"The Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry","authors":"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","doi":"10.1016/j.beha.2023.101516","DOIUrl":"https://doi.org/10.1016/j.beha.2023.101516","url":null,"abstract":"<div><p>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.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 4","pages":"Article 101516"},"PeriodicalIF":2.1,"publicationDate":"2023-10-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1521692623000774/pdfft?md5=43482c7a15f293fa7591c9b94fa27199&pid=1-s2.0-S1521692623000774-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"91987013","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

WHO, what, when, where, and why: New classification systems for acute myeloid leukemia and their impact on clinical practice WHO, What, When, Where, and Why:急性髓性白血病的新分类系统及其对临床实践的影响

IF 2.1 4区医学

Best Practice & Research Clinical Haematology Pub Date : 2023-10-20 DOI: 10.1016/j.beha.2023.101518

Frederick R. Appelbaum

引用次数: 0

The best GVHD prophylaxis: Or at least progress towards finding it 最好的GVHD预防方法:或者至少朝着找到它的方向前进

IF 2.1 4区医学

Best Practice & Research Clinical Haematology Pub Date : 2023-10-20 DOI: 10.1016/j.beha.2023.101520

Daniel Weisdorf , Najla El Jurdi , Shernan G. Holtan

引用次数: 0

Mechanisms of resistance to hypomethylating agents and BCL-2 inhibitors 对低甲基化药物和BCL-2抑制剂的耐药性机制

IF 2.1 4区医学

Best Practice & Research Clinical Haematology Pub Date : 2023-10-20 DOI: 10.1016/j.beha.2023.101521

Sudhamsh Reddy Desai , Samarpana Chakraborty , Aditi Shastri

引用次数: 0

When to use which molecular prognostic scoring system in the management of patients with MDS? 在MDS患者的管理中，何时使用哪种分子预后评分系统?

IF 2.1 4区医学

Best Practice & Research Clinical Haematology Pub Date : 2023-10-17 DOI: 10.1016/j.beha.2023.101517

Tariq Kewan , Jan Philipp Bewersdorf , Carmelo Gurnari , Zhuoer Xie , Maximilian Stahl , Amer M. Zeidan

{"title":"When to use which molecular prognostic scoring system in the management of patients with MDS?","authors":"Tariq Kewan , Jan Philipp Bewersdorf , Carmelo Gurnari , Zhuoer Xie , Maximilian Stahl , Amer M. Zeidan","doi":"10.1016/j.beha.2023.101517","DOIUrl":"https://doi.org/10.1016/j.beha.2023.101517","url":null,"abstract":"<div><p>Myelodysplastic syndromes/neoplasms (MDS) are a heterogeneous group of hematopoietic cancers characterized by recurrent molecular alterations driving the disease pathogenesis with a variable propensity for progression to acute myeloid leukemia<span><span><span> (AML). Clinical decision making for MDS relies on appropriate risk stratification at diagnosis, with higher-risk patients requiring more intensive therapy. The conventional clinical prognostic systems including the </span>International Prognostic Scoring System (IPSS) and its revised version (IPSS-R) have dominated the risk stratification of MDS from 1997 until 2022. Concurrently, the use of next-generation sequencing has revolutionized the field by revealing multiple recurrent </span>genetic mutations<span>, which correlate with phenotype and prognosis. Significant efforts have been made to formally incorporate molecular data into prognostic tools to improve proper risk identification and personalize treatment strategies. In this review, we will critically compare the available molecular scoring systems for MDS focusing on areas of progress and potential limitations that can be improved in subsequent revisions of these tools.</span></span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 4","pages":"Article 101517"},"PeriodicalIF":2.1,"publicationDate":"2023-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"91987017","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Asparaginase dosing for obese patients with acute lymphoblastic leukemia and factors that contribute to outcomes 急性淋巴细胞白血病肥胖患者的天冬酰胺酶剂量和影响结果的因素

IF 2.1 4区医学

Best Practice & Research Clinical Haematology Pub Date : 2023-10-14 DOI: 10.1016/j.beha.2023.101519

Ryan D. Cassaday

引用次数: 0

Cytogenomics of B-cell non-Hodgkin lymphomas: The “old” meets the “new” b细胞非霍奇金淋巴瘤的细胞基因组学:“老”与“新”的相遇

IF 2.1 4区医学

Best Practice & Research Clinical Haematology Pub Date : 2023-10-10 DOI: 10.1016/j.beha.2023.101513

Marta Grau , Cristina López , José Ignacio Martín-Subero , Sílvia Beà

{"title":"Cytogenomics of B-cell non-Hodgkin lymphomas: The “old” meets the “new”","authors":"Marta Grau , Cristina López , José Ignacio Martín-Subero , Sílvia Beà","doi":"10.1016/j.beha.2023.101513","DOIUrl":"https://doi.org/10.1016/j.beha.2023.101513","url":null,"abstract":"<div><p>For the routine diagnosis of haematological neoplasms an integrative approach is used considering the morphology, and the immunophenotypic, and molecular features of the tumor sample, along with clinical information. The identification and characterization of recurrent chromosomal aberrations mainly detected by conventional and molecular cytogenetics in the tumor cells has a major impact on the classification of lymphoid neoplasms. Some of the B-cell non-Hodgkin lymphomas are characterized by particular chromosomal aberrations, highlighting the relevance of conventional and molecular cytogenetic studies in their diagnosis and prognosis. In the current genomics era, next generation sequencing provides relevant information as the mutational profiles of haematological malignancies, improving their classification and also the clinical management of the patients. In addition, other new technologies have emerged recently, such as the optical genome mapping, which can overcome some of the limitations of conventional and molecular cytogenetics and may become more widely used in the cytogenetic laboratories in the upcoming years. Moreover, epigenetic alterations may complement genetic changes for a deeper understanding of the pathogenesis underlying B-cell neoplasms and a more precise risk-based patient stratification. Overall, here we describe the current state of the genomic data integrating chromosomal rearrangements, copy number alterations, and somatic variants, as well as a succinct overview of epigenomic changes, which altogether constitute a comprehensive diagnostic approach in B-cell non-Hodgkin lymphomas.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 4","pages":"Article 101513"},"PeriodicalIF":2.1,"publicationDate":"2023-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49715976","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0