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.
{"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":null,"pages":null},"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}
Pub Date : 2017-07-28DOI: 10.33590/emjhematol/10314234
Mia Cahill
The main objectives of this symposium were to explore new insights into the biology of multiple myeloma (MM) in the context of new treatment options, discuss the clinical evidence supporting continuous therapy (CT) as a means of enhancing autologous stem cell transplant (ASCT) outcomes, and explore the modern treatment options for patients with relapsed/refractory MM (RRMM), including proteasome inhibitors (PI). Prof Nikhil C. Munshi introduced the latest research on the biology of MM and its possible translation to the clinic and treatment decisions. Prof Pieter Sonneveld then discussed the current clinical knowledge and evidence for the relative roles of ASCT and CT in treating MM in the context of three clinical questions, with expert panel perspectives on each question. Prof Meletios Dimopoulos closed the symposium with an in-depth look at treatment options for RRMM and the results of the TOURMALINE-MM1 trial. Clinical case studies added relevance to these key learnings and demonstrated the importance of a holistic approach to treatment.
本次研讨会的主要目的是在新的治疗方案背景下探索多发性骨髓瘤(MM)生物学的新见解，讨论支持持续治疗(CT)作为增强自体干细胞移植(ASCT)结果的手段的临床证据，并探讨复发/难治性MM (RRMM)患者的现代治疗方案，包括蛋白酶体抑制剂(PI)。Nikhil C. Munshi教授介绍了MM生物学的最新研究及其可能转化为临床和治疗决策的可能性。Pieter Sonneveld教授随后讨论了目前的临床知识和证据，在三个临床问题的背景下，ASCT和CT在治疗MM中的相对作用，每个问题都有专家小组的观点。Meletios Dimopoulos教授在研讨会结束时深入探讨了RRMM的治疗方案和TOURMALINE-MM1试验的结果。临床病例研究增加了这些关键知识的相关性，并证明了整体治疗方法的重要性。
{"title":"The Pivotal Role of Proteasome Inhibition in Myeloma Treatment","authors":"Mia Cahill","doi":"10.33590/emjhematol/10314234","DOIUrl":"https://doi.org/10.33590/emjhematol/10314234","url":null,"abstract":"The main objectives of this symposium were to explore new insights into the biology of multiple myeloma (MM) in the context of new treatment options, discuss the clinical evidence supporting continuous therapy (CT) as a means of enhancing autologous stem cell transplant (ASCT) outcomes, and explore the modern treatment options for patients with relapsed/refractory MM (RRMM), including proteasome inhibitors (PI). Prof Nikhil C. Munshi introduced the latest research on the biology of MM and its possible translation to the clinic and treatment decisions. Prof Pieter Sonneveld then discussed the current clinical knowledge and evidence for the relative roles of ASCT and CT in treating MM in the context of three clinical questions, with expert panel perspectives on each question. Prof Meletios Dimopoulos closed the symposium with an in-depth look at treatment options for RRMM and the results of the TOURMALINE-MM1 trial. Clinical case studies added relevance to these key learnings and demonstrated the importance of a holistic approach to treatment.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2017-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"133980044","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}
Pub Date : 2016-07-28DOI: 10.33590/emjhematol/10312986
T. Ibbotson
A recent symposium at the European Hematology Association (EHA) congress, chaired by Prof Eva Kimby, explored the changing paradigms in the treatment of non-Hodgkin’s lymphoma (NHL) and the potential impact of new approaches to diagnosis and treatment. Prof Kimby opened the symposium by discussing the recent therapeutic advances in the treatment of follicular lymphoma (FL). Prof Georg Lenz then spoke about the clinical implications of diffuse large B cell lymphoma (DLBCL) diagnosis and the manner in which disease subtyping can foster effective use of targeted therapies. Prof Catherine Thieblemont presented on post-induction treatment in DLBCL, and the importance of effective treatment options to limit the number of patients who fail first-line therapy. Prof Pier Luigi Zinzani then concluded the symposium by presenting data on the new immuno-oncology treatments being evaluated in patients with relapsed or refractory NHL.
最近在欧洲血液学协会(EHA)大会上，Eva Kimby教授主持了一次研讨会，探讨了非霍奇金淋巴瘤(NHL)治疗模式的变化以及新诊断和治疗方法的潜在影响。Kimby教授首先讨论了滤泡性淋巴瘤(滤泡性淋巴瘤)治疗的最新进展。Georg Lenz教授随后谈到了弥漫性大B细胞淋巴瘤(DLBCL)诊断的临床意义，以及疾病亚型可以促进有效使用靶向治疗的方式。Catherine Thieblemont教授介绍了DLBCL的诱导后治疗，以及有效的治疗方案对于限制一线治疗失败的患者数量的重要性。Pier Luigi Zinzani教授随后介绍了在复发或难治性NHL患者中正在评估的新的免疫肿瘤学治疗方法的数据，从而结束了研讨会。
{"title":"Advances in the Treatment of Non-Hodgkin’s Lymphoma: Exploring New Frontiers","authors":"T. Ibbotson","doi":"10.33590/emjhematol/10312986","DOIUrl":"https://doi.org/10.33590/emjhematol/10312986","url":null,"abstract":"A recent symposium at the European Hematology Association (EHA) congress, chaired by Prof Eva Kimby, explored the changing paradigms in the treatment of non-Hodgkin’s lymphoma (NHL) and the potential impact of new approaches to diagnosis and treatment. Prof Kimby opened the symposium by discussing the recent therapeutic advances in the treatment of follicular lymphoma (FL). Prof Georg Lenz then spoke about the clinical implications of diffuse large B cell lymphoma (DLBCL) diagnosis and the manner in which disease subtyping can foster effective use of targeted therapies. Prof Catherine Thieblemont presented on post-induction treatment in DLBCL, and the importance of effective treatment options to limit the number of patients who fail first-line therapy. Prof Pier Luigi Zinzani then concluded the symposium by presenting data on the new immuno-oncology treatments being evaluated in patients with relapsed or refractory NHL.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2016-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"115152801","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}
Pub Date : 2016-07-28DOI: 10.33590/emjhematol/10314585
D. Rabbolini, C. Ward, W. Stevenson
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.
{"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":null,"pages":null},"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}
Direct oral anticoagulants (DOAC) are used in several indications for the prevention and treatment of thrombotic events. As highlighted by data from clinical trials and case studies, all DOAC carry the risk of bleeding despite careful selection and patient management. Previous publications have demonstrated the limited knowledge of many physicians concerning the indications for, and correct management of, these anticoagulants. Health institutions should develop risk minimisation strategies and educational materials to prevent major adverse events related to DOAC administration. Major bleeding events are reported in clinical practice and specific antidotes are emerging from Phase III trials. Some antidotes are licensed but their high cost might limit routine use. We therefore illustrate approaches and tools that can help physicians prescribe DOAC appropriately. We focus on screening for modifiable bleeding risk factors and adapting doses according to the individual benefit-risk profile. We also provide recommendations on managing a missed dose, switching, bridging, and resumption.
Pub Date : 2016-07-28DOI: 10.33590/emjhematol/10310534
R. Colombatti, L. Sainati
Sickle cell disease (SCD) is the most common haemoglobinopathy worldwide and its frequency has steadily increased in Europe in the past decades. SCD is a complex multisystem disorder characterised by chronic haemolytic anaemia, vaso-occlusive crisis, and vasculopathy. Clinical manifestations can be very different, ranging from mild haemolysis to life-threatening acute clinical complications and chronic disabilities. This review will explore service delivery across Europe to children with SCD, reporting on the available minimum standards of care and future perspectives.
{"title":"Management of Children With Sickle Cell Disease in Europe: Current Situation and Future Perspectives","authors":"R. Colombatti, L. Sainati","doi":"10.33590/emjhematol/10310534","DOIUrl":"https://doi.org/10.33590/emjhematol/10310534","url":null,"abstract":"Sickle cell disease (SCD) is the most common haemoglobinopathy worldwide and its frequency has steadily increased in Europe in the past decades. SCD is a complex multisystem disorder characterised by chronic haemolytic anaemia, vaso-occlusive crisis, and vasculopathy. Clinical manifestations can be very different, ranging from mild haemolysis to life-threatening acute clinical complications and chronic disabilities. This review will explore service delivery across Europe to children with SCD, reporting on the available minimum standards of care and future perspectives.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2016-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"128253907","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}
Pub Date : 2016-07-28DOI: 10.33590/emjhematol/10312282
T. Druley
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.
{"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":null,"pages":null},"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}
Pub Date : 2016-07-28DOI: 10.33590/emjhematol/10310413
Blair R. Hesp
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.
Robin foom教授在研讨会开幕时强调，医疗保健的改善和人口老龄化如何增加了医疗资源的负担，并在维持许多人所期望的高水平医疗保健服务方面带来了挑战。Armando博士López-Guillermo讨论了生物仿制药在维持可持续和负担得起的医疗保健系统中的作用，以及平衡这与确保生物仿制药提供与其参考产品相当的功效和安全性的必要性。Martin Schiestl博士概述了与新型生物疗法和小分子药物的仿制药相比，生物仿制药的批准程序的差异，以及这如何确保生物仿制药与其参考产品之间的相似性。Steffen Thirstrup教授回顾了欧盟监管机构在决定是否适合将生物仿制药的适应症推断出单一已批准的适应症时所采取的程序。会议的目标是讨论生物仿制药在满足医疗保健需求方面的作用，审查生物仿制药在获得市场批准之前进行的监管评估，以及如何科学地证明其他推断适应症。
{"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":null,"pages":null},"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}
Pub Date : 2016-07-28DOI: 10.33590/emjhematol/10312198
X. Thomas, C. Le Jeune
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.
{"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":null,"pages":null},"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}
Pub Date : 2016-07-26DOI: 10.33590/emjhematol/10312371
Mia Cahill
The treatment landscape for patients with multiple myeloma (MM) is constantly evolving. Over the past decade, the introduction of novel agents including proteasome inhibitors (PI) and immunomodulatory agents has led to notable changes in therapeutic strategy and significant improvements in survival. Understanding this landscape and what this means in terms of translating clinical trials to everyday practice is essential. Prof Paul Richardson opened the symposia with an introduction to currently available agents and recent developments in MM, and highlighted the importance of how we think about current studies. Prof Roman Hájek explored clonal evolution, how it can be prevented in the context of relapsed disease, and the evidence from clinical trials supporting the use of combination therapy. Dr Antonio Palumbo addressed the concept of continuous therapy in MM and where the field is at present. Prof Shaji Kumar described the early phase development of ixazomib. Prof Paul Richardson presented the results from the TOURMALINE-MM1 trial.
{"title":"Navigating the Changing Multiple Myeloma Treatment Landscape","authors":"Mia Cahill","doi":"10.33590/emjhematol/10312371","DOIUrl":"https://doi.org/10.33590/emjhematol/10312371","url":null,"abstract":"The treatment landscape for patients with multiple myeloma (MM) is constantly evolving. Over the past decade, the introduction of novel agents including proteasome inhibitors (PI) and immunomodulatory agents has led to notable changes in therapeutic strategy and significant improvements in survival. Understanding this landscape and what this means in terms of translating clinical trials to everyday practice is essential.\u0000\u0000Prof Paul Richardson opened the symposia with an introduction to currently available agents and recent developments in MM, and highlighted the importance of how we think about current studies. Prof Roman Hájek explored clonal evolution, how it can be prevented in the context of relapsed disease, and the evidence from clinical trials supporting the use of combination therapy. Dr Antonio Palumbo addressed the concept of continuous therapy in MM and where the field is at present. Prof Shaji Kumar described the early phase development of ixazomib. Prof Paul Richardson presented the results from the TOURMALINE-MM1 trial.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2016-07-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"114916158","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}
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