Current Opinion in Hematology最新文献_第9页

VEXAS: where do we stand 2 years later? 维克斯:两年后我们的情况如何?

IF 3.2 3区医学

Current Opinion in Hematology Pub Date : 2023-03-01 DOI: 10.1097/MOH.0000000000000750

Pierre Sujobert, Maël Heiblig, Yvan Jamilloux

{"title":"VEXAS: where do we stand 2 years later?","authors":"Pierre Sujobert, Maël Heiblig, Yvan Jamilloux","doi":"10.1097/MOH.0000000000000750","DOIUrl":"https://doi.org/10.1097/MOH.0000000000000750","url":null,"abstract":"Purpose of review: Two years after the recognition of VEXAS (for Vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome, we propose an extensive review of the current understanding of VEXAS pathophysiology and therapeutic options.Recent findings: Among the nearly 150 articles published about VEXAS, some have provided determinant insights into VEXAS pathophysiology and treatment. Clinical data from retrospective series support the JAK inhibitor ruxolitinib as the most efficient strategy to control inflammation, and interesting results were also described with azacytidine. Allogeneic stem cell transplantation remains the only curative option, but should be proposed to carefully selected patients.Summary: Although waiting for more robust evidence from prospective clinical trials, therapeutic options emerge from retrospective studies. We propose a set of criteria that should be systematically reported to harmonize the evaluation of therapeutic outcomes. This will allow the collection of high-quality data and facilitate their subsequent meta-analysis with the overall aim of improving the management of VEXAS patients.","PeriodicalId":55196,"journal":{"name":"Current Opinion in Hematology","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10633099","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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: 3

Is it the time to integrate novel sequencing technologies into clinical practice? 现在是将新型测序技术融入临床实践的时候了吗？

IF 3.1 3区医学

Current Opinion in Hematology Pub Date : 2023-03-01 Epub Date: 2022-12-30 DOI: 10.1097/MOH.0000000000000754

Jennifer VanOudenhove, Stephanie Halene, Lourdes Mendez

{"title":"Is it the time to integrate novel sequencing technologies into clinical practice?","authors":"Jennifer VanOudenhove, Stephanie Halene, Lourdes Mendez","doi":"10.1097/MOH.0000000000000754","DOIUrl":"10.1097/MOH.0000000000000754","url":null,"abstract":"Purpose of review: The aim of this study was to provide insight into how novel next-generation sequencing (NGS) techniques are set to revolutionize clinical practice.Recent findings: Advances in sequencing technologies have focused on improved capture of mutations and reads and cellular resolution. Both short and long read DNA sequencing technology are being refined and combined in novel ways with other multiomic approaches to gain unprecedented biological insight into disease. Single-cell (sc)DNA-seq and integrated scDNA-seq with immunophenotyping provide granular information on disease composition such as clonal hierarchy, co-mutation status, zygosity, clonal diversity and genotype phenotype correlations. These and other techniques can identify rare cell populations providing the opportunity for increased sensitivity in measurable residual disease monitoring and precise characterization of residual clones permitting distinction of leukemic from pre/nonmalignant clones.Summary: Increasing genetics-based mechanistic insights and classification of myeloid diseases along with a decrease in the cost of high-throughput NGS mean novel sequencing technologies are closer to being a reality in standard clinical practice. These technologies are poised to improve diagnostics, our ability to monitor treatment response and minimal residual disease and allow the study of premalignant conditions such as clonal haematopoiesis.","PeriodicalId":55196,"journal":{"name":"Current Opinion in Hematology","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10266076","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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

The fifth edition of the World Health Organization Classification and the International Consensus Classification of myeloid neoplasms: evolving guidelines in the molecular era with practical implications. 世界卫生组织分类和髓系肿瘤国际共识分类第五版:分子时代具有实际意义的发展指南。

IF 3.2 3区医学

Current Opinion in Hematology Pub Date : 2023-03-01 DOI: 10.1097/MOH.0000000000000748

Gang Zheng, Peng Li, Xiaohui Zhang, Zenggang Pan

{"title":"The fifth edition of the World Health Organization Classification and the International Consensus Classification of myeloid neoplasms: evolving guidelines in the molecular era with practical implications.","authors":"Gang Zheng, Peng Li, Xiaohui Zhang, Zenggang Pan","doi":"10.1097/MOH.0000000000000748","DOIUrl":"https://doi.org/10.1097/MOH.0000000000000748","url":null,"abstract":"Purpose of review: There have been major advances in our understanding of molecular pathogenesis of myeloid neoplasms, which prompt the updates in the classification of myeloid neoplasms in the fifth edition of World Health Organization Classification (WHO-5) and the new International Consensus Classification (ICC). The purpose of this review is to provide an overview of these two classification systems for myeloid neoplasms.Recent findings: The definition, classification, and diagnostic criteria in many myeloid entities have been refined in WHO-5 and ICC with improved understanding of morphology and integration of new genetic findings. Particularly, molecular and cytogenetic studies have been increasingly incorporated into the classification, risk stratification, and selection of therapy of myeloid neoplasms. Overall, despite some revisions and discrepancies between WHO-5 and ICC, the major categories of myeloid neoplasms remain the same. Further validation studies are warranted to fine-tune and, ideally, integrate these two classifications.Summary: Integration of clinical information, laboratory parameters, morphologic features, and cytogenetic and molecular studies is essential for the classification of myeloid neoplasms, as recommended by both WHO-5 and ICC.","PeriodicalId":55196,"journal":{"name":"Current Opinion in Hematology","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10616030","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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: 2

Editorial: Molecular advances make the day in myeloid diseases. 社论：髓系疾病的分子研究取得进展

IF 3.2 3区医学

Current Opinion in Hematology Pub Date : 2023-03-01 DOI: 10.1097/MOH.0000000000000751

Stephanie Halene

引用次数: 0

Molecular landscape of myelodysplastic neoplasms in disease classification and prognostication. 骨髓增生异常肿瘤在疾病分类和预后中的分子景观。

IF 3.2 3区医学

Current Opinion in Hematology Pub Date : 2023-03-01 DOI: 10.1097/MOH.0000000000000752

Giulia Maggioni, Matteo G Della Porta

{"title":"Molecular landscape of myelodysplastic neoplasms in disease classification and prognostication.","authors":"Giulia Maggioni, Matteo G Della Porta","doi":"10.1097/MOH.0000000000000752","DOIUrl":"https://doi.org/10.1097/MOH.0000000000000752","url":null,"abstract":"Purpose of review: The aim of this review is to provide a complete perspective of the evidence that led to the three recent new landmarks of myelodysplastic syndromes (MDS) definition and prognostication: the WHO 2022 and International Consensus Classification (ICC) 2022 classification and the Molecular Intermational Prognostic Scoring System (IPSS-M) score.Recent findings: The molecular founding lesions of MDS are strongly linked with disease phenotype and prognosis, therefore the genetic assessment have become part of MDS classifications and prognostication.Summary: The WHO 2022 now recognizes the class 'MDS with defining genetic abnormalities'. It includes 'MDS with SF3B1 mutation', and 'MDS with biallelic TP53 inactivation'. The ICC 2022 further introduces the category 'MDS/acute myeloid leukemia (AML)' emphasizing the biological continuum existing between the diseases, with the aim to expand therapeutic possibilities for MDS patients with more than 10% of blasts; it further identifies 9 MDS-funding lesions, defying the 'MDS/AML with myelodysplasia-related gene mutations' class. In recent years, many efforts have been done in order to specify and weight the role of mutations in disease prognostication; the IPSS-M proposed in 2022 finally integrates the molecular profile of the disease with the clinical and cytogenetic data, providing a better prognostication at patient level compared to IPSS-R.","PeriodicalId":55196,"journal":{"name":"Current Opinion in Hematology","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10633098","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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: 1

Hepcidin mimetics in polycythemia vera: resolving the irony of iron deficiency and erythrocytosis. 多发性红细胞症中的拟肝素：解决缺铁与红细胞增多症的矛盾。

IF 3.2 3区医学

Current Opinion in Hematology Pub Date : 2023-03-01 Epub Date: 2022-12-29 DOI: 10.1097/MOH.0000000000000747

Shivani Handa, Yelena Ginzburg, Ronald Hoffman, Marina Kremyanskaya

{"title":"Hepcidin mimetics in polycythemia vera: resolving the irony of iron deficiency and erythrocytosis.","authors":"Shivani Handa, Yelena Ginzburg, Ronald Hoffman, Marina Kremyanskaya","doi":"10.1097/MOH.0000000000000747","DOIUrl":"10.1097/MOH.0000000000000747","url":null,"abstract":"Purpose of review: Development of hepcidin therapeutics has been a ground-breaking discovery in restoring iron homeostasis in several haematological disorders. The hepcidin mimetic, rusfertide, is in late-stage clinical development for treating polycythemia vera patients with a global phase 3 trial [NCT05210790] currently underway. Rusfertide serves as the first possible noncytoreductive therapeutic option to maintain haematocrit control and avoid phlebotomy in polycythemia vera patients. In this comprehensive review, we discuss the pathobiology of dysregulated iron metabolism in polycythemia vera, provide the rationale for targeting the hepcidin-ferroportin axis and elaborate on the preclinical and clinical trial evidence supporting the role of hepcidin mimetics in polycythemia vera.Recent findings: Recently, updated results from two phase 2 clinical trials [NCT04057040 & NCT04767802] of rusfertide (PTG300) demonstrate that the drug is highly effective in eliminating the need for therapeutic phlebotomies, normalizing haematological parameters, repleting iron stores and relieving constitutional symptoms in patients with polycythemia vera. In light of these findings, additional hepcidin mimetic agents are also being evaluated in polycythemia vera patients.Summary: Hepcidin agonists essentially serve as a 'chemical phlebotomy' and are poised to vastly improve the quality of life for phlebotomy requiring polycythemia vera patients.","PeriodicalId":55196,"journal":{"name":"Current Opinion in Hematology","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9908837/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10642874","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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

Editorial Introductions. 编辑介绍。

IF 3.2 3区医学

Current Opinion in Hematology Pub Date : 2023-03-01 DOI: 10.1097/MOH.0000000000000757

引用次数: 0

Novel immune directed therapies in myelodysplastic syndromes and acute myeloid leukemia. 骨髓增生异常综合征和急性髓性白血病的新型免疫导向疗法。

IF 3.1 3区医学

Current Opinion in Hematology Pub Date : 2023-03-01 Epub Date: 2022-12-23 DOI: 10.1097/MOH.0000000000000749

Andrew M Brunner

{"title":"Novel immune directed therapies in myelodysplastic syndromes and acute myeloid leukemia.","authors":"Andrew M Brunner","doi":"10.1097/MOH.0000000000000749","DOIUrl":"10.1097/MOH.0000000000000749","url":null,"abstract":"Purpose of review: Therapies that target the immune system are increasingly used across oncology, including in hematologic malignancies such as myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). While allogeneic transplant has been a key therapy in these cancers, new approaches that target the immune system are being explored including immune checkpoint therapies, antibody-drug conjugates, and cellular therapies.Recent findings: This review outlines updates in the preclinical rationale for immune directed therapies in MDS and AML, as well as recent clinical trials exploring these therapies. This manuscript summarizes the development of therapies targeting T cell immunoglobulin and mucin domain-containing protein 3 (TIM-3) and CD47, which are being evaluated in late phase studies in MDS and AML. It also reviews the landscape of other immune based therapies including antibody-drug conjugates, chimeric antigen receptor-T cells, bispecific antibodies, and tumor vaccines.Summary: The treatment landscape in MDS and AML is rapidly changing; with a goal of improving the quality and duration of responses, a number of immune based therapies are under investigation. This review outlines recent advances with these therapies as well as some of the challenges that remain to incorporate them into leukemia care.","PeriodicalId":55196,"journal":{"name":"Current Opinion in Hematology","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10017015/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10616031","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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

Old age: the crown of life, our play's last act. Question and answers on older patients undergoing allogeneic hematopoietic cell transplantation. 老年:人生的王冠，我们戏剧的最后一幕。老年患者异基因造血细胞移植的问题与解答。

IF 3.2 3区医学

Current Opinion in Hematology Pub Date : 2023-01-01 DOI: 10.1097/MOH.0000000000000743

Enrico Maffini, Moreno Festuccia, Margherita Ursi, Francesco Barbato, Michele Dicataldo, Marcello Roberto, Elena Campanini, Elisa Dan, Francesco De Felice, Serena De Matteis, Gianluca Storci, Massimiliano Bonafè, Mario Arpinati, Francesca Bonifazi

{"title":"Old age: the crown of life, our play's last act. Question and answers on older patients undergoing allogeneic hematopoietic cell transplantation.","authors":"Enrico Maffini, Moreno Festuccia, Margherita Ursi, Francesco Barbato, Michele Dicataldo, Marcello Roberto, Elena Campanini, Elisa Dan, Francesco De Felice, Serena De Matteis, Gianluca Storci, Massimiliano Bonafè, Mario Arpinati, Francesca Bonifazi","doi":"10.1097/MOH.0000000000000743","DOIUrl":"https://doi.org/10.1097/MOH.0000000000000743","url":null,"abstract":"Purpose of review: Several studies showed that age alone should not be used as an arbitrary parameter to exclude patients from allogeneic hematopoietic cell transplantation (HCT). The accessibility to allogeneic HCT programs for older patients with hematological diseases is growing up constantly. The Center for International Blood and Marrow Transplant Research has recently shown that over 30% of allogeneic HCT recipients are at least 60 years old and that nearly 4% are aged 70 or more. Historically, the use of allogeneic HCT among elderly patients has been limited by age restrictions, reflecting physicians' concerns regarding prohibitive transplant-related mortality and HCT-associated morbidity.Recent findings: The introduction of reduced intensity/toxicity conditioning regimens has allowed transplant Centers to carry out allogeneic HCT on patients previously considered not ideal candidates. The integration of specific risk scores could lead to better capture mental and physical frailties of older patients. Older adults less frequently have available medically fit siblings, able to donate, so, unrelated donors, familial haploidentical donors or umbilical cord blood grafts could potentially abrogate such a difficulty, allowing the curative potential of allogeneic HCT.Summary: The appropriate assessing of allogeneic HCT feasibility for elderly patients should be the resonate application of different clinical and biological principles.","PeriodicalId":55196,"journal":{"name":"Current Opinion in Hematology","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10777132","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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

N6-methyladenosine in hematological malignancies: a concise review. n6 -甲基腺苷在血液系统恶性肿瘤中的作用:简明综述。

IF 3.2 3区医学

Current Opinion in Hematology Pub Date : 2023-01-01 DOI: 10.1097/MOH.0000000000000741

Wan-Jie Wang, Ting-Ting Xu, Jing Bao

{"title":"N6-methyladenosine in hematological malignancies: a concise review.","authors":"Wan-Jie Wang, Ting-Ting Xu, Jing Bao","doi":"10.1097/MOH.0000000000000741","DOIUrl":"https://doi.org/10.1097/MOH.0000000000000741","url":null,"abstract":"Purpose of review: Hematological malignancies are a kind of systemic cancers mostly related to abnormal differentiation of blood stem cells. Because of the poor prognosis, chemotherapy resistance and common recurrence, new mechanisms and treatment therapies are looking forward to be discovered.Recent findings: Over the years, epigenetic abnormalities have been known to act a key part in occurrence and development of hematological tumors. In the internal modifications on long noncoding eukaryotic mRNA, there is a common type called N6-methyladenosine that can change the expression of target genes and participate in the translation, degradation and splicing of mRNA. M6A is related to a wealth of cancers, such as HNRNPA2B1's elevation in multiple myeloma, METTLE3's elevation in acute myeloid leukemia and lung cancer. Immune cells, playing a significant role in hematological cancers, can also be regulated by m6A.Summary: In the review, we summarized the recent progress on hematological malignancies associating with m6A and immune cells, which may offer a new road for the treatment of them.","PeriodicalId":55196,"journal":{"name":"Current Opinion in Hematology","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10833832","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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