EMJ Hematology最新文献_第8页

Management of Adult Patients with Myelodysplastic Syndromes 成年骨髓增生异常综合征患者的管理

EMJ Hematology Pub Date : 2017-08-01 DOI: 10.7892/BORIS.110110

N. Bonadies

{"title":"Management of Adult Patients with Myelodysplastic Syndromes","authors":"N. Bonadies","doi":"10.7892/BORIS.110110","DOIUrl":"https://doi.org/10.7892/BORIS.110110","url":null,"abstract":"The myelodysplastic syndromes (MDS) form a heterogeneous group of clonal disorders with an increasing incidence in the elderly population and an emerging impact on healthcare resources. MDS are caused by gene mutations affecting the haematopoietic stem cells, leading to ineffective haematopoiesis, characterised by dysplasia and cytopenia, and a propensity to evolve towards secondary acute myeloid leukaemia (AML). Accurate diagnosis and risk assessment are essential for the correct treatment allocation. In lower-risk MDS patients, median survival reaches 3–8 years and mortality is mainly caused by cytopenia (cardiovascular events, infections, and bleeding). Therefore, the treatment for these patients should be focussed on reduction of disease-related complications, disease progression, and improvement of quality of life. In contrast, in higher-risk MDS patients, median survival ranges from 1–3 years and death from transformation to AML exceeds non-leukaemic mortality. Treatment should be aimed to delay progression to AML and improve overall survival. Allogeneic haematopoietic stem cell transplant remains the only curative option for higher-risk MDS patients. However, only a minority of patients are eligible for such intensive treatment. Consequently, most patients are managed with supportive care and palliative treatment, including growth factors, immune-modulators, and hypomethylating agents. Since elderly patients with chronic cytopenia are frequently seen in general practice, awareness of the wide spectrum of presentations of MDS and potential courses of lower and higher-risk diseases are important for primary healthcare physicians.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":"57 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2017-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"122062110","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

The Pivotal Role of Proteasome Inhibition in Myeloma Treatment 蛋白酶体抑制在骨髓瘤治疗中的关键作用

EMJ Hematology Pub Date : 2017-07-28 DOI: 10.33590/emjhematol/10314234

Mia Cahill

引用次数: 0

Advances in the Treatment of Non-Hodgkin’s Lymphoma: Exploring New Frontiers 非霍奇金淋巴瘤的治疗进展:探索新领域

EMJ Hematology Pub Date : 2016-07-28 DOI: 10.33590/emjhematol/10312986

T. Ibbotson

引用次数: 0

Thrombocytopenia Caused By Inherited Haematopoietic Transcription Factor Mutation: Clinical Phenotypes and Diagnostic Considerations 由遗传性造血转录因子突变引起的血小板减少症:临床表型和诊断考虑

EMJ Hematology Pub Date : 2016-07-28 DOI: 10.33590/emjhematol/10314585

D. Rabbolini, C. Ward, W. Stevenson

{"title":"Thrombocytopenia Caused By Inherited Haematopoietic Transcription Factor Mutation: Clinical Phenotypes and Diagnostic Considerations","authors":"D. Rabbolini, C. Ward, W. Stevenson","doi":"10.33590/emjhematol/10314585","DOIUrl":"https://doi.org/10.33590/emjhematol/10314585","url":null,"abstract":"Inherited thrombocytopenias comprise a heterogeneous group of blood disorders with abnormalities in genes related to glycoproteins and adhesion molecules, signalling pathways, cytoskeletal components, granule formation, and transcription factor complexes. Recent improvements in sequencing technology have increased the number of transcription factor mutations that have been implicated as causative for these platelet disorders. Mutations in RUNX1, GATA1, GFI1B, FLI1, and ETV6 share common features, including a variable bleeding history often associated with abnormal but non-specific changes in platelet morphology and platelet function testing. The phenotype of the underlying platelet disorder is often variable despite mutations in the same transcription factor, suggesting that the site of mutation and the protein domain that is perturbed is an important determinant of the clinical syndrome. Importantly, some of these transcription factor mutations are associated with other physical abnormalities, including an increased risk of acute leukaemia as well as solid organ malignancies. Genetic diagnosis of these disorders allows rational medical management to prevent bleeding, as well as providing an opportunity for family screening in order to reduce disease burden.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":"33 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2016-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"122720713","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 1

Minimisation of Bleeding Risks Due to Direct Oral Anticoagulants 直接口服抗凝剂引起的出血风险最小化

EMJ Hematology Pub Date : 2016-07-28 DOI: 10.33590/emjhematol/10312079

O. Vornicu, A. Larock, J. Douxfils, F. Mullier, Virginie Dubois, Jean-Michel DognÃ©, M. Gourdin, S. Lessire, A. Dincq

引用次数: 0

Management of Children With Sickle Cell Disease in Europe: Current Situation and Future Perspectives 欧洲镰状细胞病儿童的管理:现状和未来展望

EMJ Hematology Pub Date : 2016-07-28 DOI: 10.33590/emjhematol/10310534

R. Colombatti, L. Sainati

引用次数: 6

The Relative Contributions of Germline Variation, Epimutation, and Somatic Mutation to Paediatric Leukaemia Predisposition 种系变异、上皮化和体细胞突变对儿童白血病易感性的相对贡献

EMJ Hematology Pub Date : 2016-07-28 DOI: 10.33590/emjhematol/10312282

T. Druley

{"title":"The Relative Contributions of Germline Variation, Epimutation, and Somatic Mutation to Paediatric Leukaemia Predisposition","authors":"T. Druley","doi":"10.33590/emjhematol/10312282","DOIUrl":"https://doi.org/10.33590/emjhematol/10312282","url":null,"abstract":"The next-generation sequencing era has repeatedly demonstrated that the amount of acquired somatic mutations in paediatric cancers can rarely account for the total incidence of any cancer subtype. In addition, many cancer-related mutations can be found in healthy individuals. These findings strongly suggest that additional genetic or epigenetic variation is required for malignant transformation, particularly in children who have significantly less environmental exposure and resulting genetic damage. Current studies now suggest that 3–33% of paediatric cancer patients have a predisposition to cancer. These germline genetic or epigenetic changes are frequently found in molecular mechanisms regulating normal human development which have long informed our understanding of developmental biology. Blockade of development is a mechanism of transformation consistent with the higher number of immature cancer cell types in paediatric patients. Thus, while nearly every cancer is a combination of germline variation and somatic mutation, the relative contribution to tumourigenesis in paediatrics is weighted toward germline changes. This review will explore how paediatric predisposition to leukaemia is influenced by germline genetic and epigenetic variability of variable penetrance. Improved understanding of these critical developmental mechanisms will lead to improved surveillance and perhaps guide a new class of therapeutics aimed at promoting normal differentiation rather than widespread cytotoxicity.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":"482 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2016-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"133481754","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Biosimilars: Shaping the Future of Haematology 生物仿制药:塑造血液学的未来

EMJ Hematology Pub Date : 2016-07-28 DOI: 10.33590/emjhematol/10310413

Blair R. Hesp

{"title":"Biosimilars: Shaping the Future of Haematology","authors":"Blair R. Hesp","doi":"10.33590/emjhematol/10310413","DOIUrl":"https://doi.org/10.33590/emjhematol/10310413","url":null,"abstract":"Prof Robin Foà opened the symposium by highlighting how improving healthcare and an ageing population are increasing the burden on healthcare resources and creating challenges in maintaining the high level of healthcare provision that many people expect. Dr Armando López-Guillermo discussed the role of biosimilars in maintaining sustainable and affordable healthcare systems and the need to balance this against ensuring that biosimilars offer comparable efficacy and safety compared with their reference products. Dr Martin Schiestl outlined the differences in approval processes for biosimilars compared with novel biological therapies and generic versions of small-molecule drugs, and how this ensures similarity between biosimilars and their reference products. Prof Steffen Thirstrup reviewed the processes that European Union regulatory authorities undertake when deciding whether it is appropriate to extrapolate indications for biosimilars beyond a single approved indication. The meeting objectives were to discuss the role of biosimilars in meeting healthcare needs and to review what regulatory assessments biosimilars undergo prior to receiving marketing approval, and how additional extrapolated indications can be scientifically justified.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":"49 12","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2016-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"114060035","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 1

Current Management of Adult Acute Lymphoblastic Leukaemia: Emerging Insights and Outstanding Questions 成人急性淋巴细胞白血病的当前管理:新兴的见解和悬而未决的问题

EMJ Hematology Pub Date : 2016-07-28 DOI: 10.33590/emjhematol/10312198

X. Thomas, C. Le Jeune

{"title":"Current Management of Adult Acute Lymphoblastic Leukaemia: Emerging Insights and Outstanding Questions","authors":"X. Thomas, C. Le Jeune","doi":"10.33590/emjhematol/10312198","DOIUrl":"https://doi.org/10.33590/emjhematol/10312198","url":null,"abstract":"Less than 50% of patients with adult acute lymphoblastic leukaemia (ALL) experience long-term survival and for those adults >60 years old, long-term survival rates are only 10%. However, significant advances have been reported over the last decade. Both the efficacy of chemotherapy and the safety of transplants have improved. Improved outcomes have been seen in younger adults treated with paediatric-inspired chemotherapy regimens. Minimal residual disease has been identified as an independent predictor of relapse risk and is currently widely used for risk-adapted treatment. Newly developed targeted therapies have been developed to improve treatment outcomes. Tyrosine kinase inhibitors (TKI) have become an integral part of front-line therapy for Philadelphia (Ph) chromosome positive ALL. Ph-positive ALL serves as the first example of truly targeted treatment, although the choice of the most effective TKI is not yet settled. The last few years have also seen a surge in immune therapies for B cell lineage ALL. The success of the anti-CD20 monoclonal antibody rituximab provided proof-of-principle for exploiting the immune system therapeutically. Novel immune therapies recruit (bispecific T cell engager) or modify (chimeric antigen receptor T cells) the patient’s own T cells to fight leukaemic cells. These new approaches led us to predict that ALL therapy might be based heavily on non-chemotherapeutic approaches in the near future. The role of allogeneic stem cell transplantation is also increasingly called into question. Herein, we review the background and development of these distinct treatments, and assess the current clinical knowledge of their efficacy and safety.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":"1 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2016-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"125798324","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 2

Navigating the Changing Multiple Myeloma Treatment Landscape 导航不断变化的多发性骨髓瘤治疗前景

EMJ Hematology Pub Date : 2016-07-26 DOI: 10.33590/emjhematol/10312371

Mia Cahill

引用次数: 0