Seminars in hematology最新文献

Intestinal microbiome and myelodysplastic syndromes: Current state of knowledge and perspectives for future. 肠道微生物群与骨髓增生异常综合征：知识现状与未来展望。

IF 5 3区医学

Seminars in hematology Pub Date : 2024-12-01 Epub Date: 2024-10-28 DOI: 10.1053/j.seminhematol.2024.10.006

Marin Simunic, Kathy McGraw, Steven Z Pavletic, Armin Rashidi

引用次数: 0

Beyond HMAs: Novel Targets and Therapeutic Approaches. 超越 HMAs：新的靶点和治疗方法。

IF 5 3区医学

Seminars in hematology Pub Date : 2024-12-01 Epub Date: 2024-08-30 DOI: 10.1053/j.seminhematol.2024.08.001

Ted M Getz, Jan P Bewersdorf, Tariq Kewan, Jessica M Stempel, Aram Bidikian, Rory M Shallis, Maximilian Stahl, Amer M Zeidan

{"title":"Beyond HMAs: Novel Targets and Therapeutic Approaches.","authors":"Ted M Getz, Jan P Bewersdorf, Tariq Kewan, Jessica M Stempel, Aram Bidikian, Rory M Shallis, Maximilian Stahl, Amer M Zeidan","doi":"10.1053/j.seminhematol.2024.08.001","DOIUrl":"10.1053/j.seminhematol.2024.08.001","url":null,"abstract":"Myelodysplastic syndromes/neoplasms (MDS) constitute a heterogeneous group of clonal hematopoietic disorders with extremely variable clinical features and outcomes. Management of MDS is largely based on risk stratification of patients into either lower-risk or higher-risk categories using the International Prognostic Scoring System-Revised and, more recently, on the Molecular International Prognostic Scoring System. Lower-risk MDS is often managed with the goal of ameliorating cytopenias and improving quality of life, while higher-risk MDS is treated with therapies aimed at extending survival and delaying progression to acute myeloid leukemia (AML). Therapeutic strategies in lower-risk MDS patients may consist of erythropoiesis stimulating agents, luspatercept, and lenalidomide for selected patients. Furthermore, imetelstat has recently been added to the FDA-approved therapeutic armamentarium for lower-risk MDS. In higher-risk MDS, monotherapy with hypomethylating agents continues to be the standard of care. While several novel hypomethylating agent combinations have and are being studied in large randomized phase 3 clinical trials, including the combination of azacitidine and venetoclax, no combination to date have improved overall survival to azacitidine monotherapy. Moreover, biomarker-directed therapies as well as immonotherapeutic approaches are currently being evaluated in early phase trials. Despite recent advancements, the lack of therapeutic agents, particularly after the failure of first line therapy in higher risk MDS, continues to be a major hurdle in the management of MDS. In this review, we discuss the current treatment landscape of MDS and provide an overview of novel agents currently in clinical development that have the potential to alter our current treatment paradigms.","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":" ","pages":"358-369"},"PeriodicalIF":5.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142401199","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

Infections and antimicrobial prophylaxis in patients with myelodysplastic syndromes. 骨髓增生异常综合征患者的感染和抗菌药预防。

IF 5 3区医学

Seminars in hematology Pub Date : 2024-12-01 Epub Date: 2024-08-03 DOI: 10.1053/j.seminhematol.2024.07.004

Mary M Czech, Eduard Schulz, Alain Mina, Juan Gea-Banacloche

{"title":"Infections and antimicrobial prophylaxis in patients with myelodysplastic syndromes.","authors":"Mary M Czech, Eduard Schulz, Alain Mina, Juan Gea-Banacloche","doi":"10.1053/j.seminhematol.2024.07.004","DOIUrl":"10.1053/j.seminhematol.2024.07.004","url":null,"abstract":"Infectious complications are an important cause of morbidity and mortality in patients with myelodysplastic syndromes (MDS). Preventing infections could significantly improve both survival and quality of life. Unfortunately, both infections and antimicrobial prophylaxis in patients with MDS are incompletely assessed due to the heterogeneity of disorders included in each publication, changing definitions over time, and lack of standardized prophylaxis practices. Despite these limitations, some basic statements can be made. Infections in MDS are associated with neutropenia. Patients with lower-risk (LR) MDS tend to have fewer infections compared to patients with higher-risk (HR) MDS, which may be related to the different prevalence of neutropenia in the 2 groups. Pneumonia is the most common infection, and bacteria are the most common pathogens. Invasive fungal infections (IFI) are uncommon. Reactivation of latent viruses are rare. With the limited data available, we agree that antibacterial prophylaxis can be considered in patients with HR-MDS during severe neutropenia and early cycles of therapy when infections are most likely to occur. Given the low prevalence of IFI and viral reactivation, antimicrobial prophylaxis for these pathogens is less likely to be advantageous for most patients, although antifungal prophylaxis with activity against mold is commonly used in patients with persistent, profound neutropenia. Ultimately, improved data collection regarding infections and antimicrobial prophylaxis is needed to improve care for patients with MDS.","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":" ","pages":"348-357"},"PeriodicalIF":5.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11646186/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142093712","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

Inflammation in myelodysplastic syndrome pathogenesis. 骨髓增生异常综合征发病机制中的炎症。

IF 5 3区医学

Seminars in hematology Pub Date : 2024-12-01 Epub Date: 2024-09-21 DOI: 10.1053/j.seminhematol.2024.09.005

Juan Jose Rodriguez-Sevilla, Simona Colla

引用次数: 0

A line in shifting sand: Can we define and target TP53 mutated MDS? 沙中之线：我们能否定义并锁定 TP53 突变的 MDS？

IF 5 3区医学

Seminars in hematology Pub Date : 2024-12-01 Epub Date: 2024-11-06 DOI: 10.1053/j.seminhematol.2024.10.009

Sarah Skuli, Andrew Matthews, Martin Carroll, Catherine Lai

{"title":"A line in shifting sand: Can we define and target TP53 mutated MDS?","authors":"Sarah Skuli, Andrew Matthews, Martin Carroll, Catherine Lai","doi":"10.1053/j.seminhematol.2024.10.009","DOIUrl":"10.1053/j.seminhematol.2024.10.009","url":null,"abstract":"Mutations in the tumor suppressor protein, TP53, lead to dismal outcomes in myeloid malignancies, including myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). Recent pathological reclassifications have integrated TP53 mutated MDS and AML under a unified category of TP53 mutated myeloid neoplasms, which allows for more flexibility in treatment approaches. Therapeutic strategies have predominantly mirrored those for AML, with allogeneic stem cell transplantation emerging as critical for long-term disease control. The question remains whether there are physiological distinctions within TP53 mutated myeloid neoplasms that will significantly impact prognosis and therapeutic considerations. This review explores the unique aspects of classically defined \"TP53 mutated MDS\", focusing on its distinct biological characteristics and outcomes. Our current understanding is that TP53 mutated MDS and AML are globally quite similar, but as a group have unique features compared to TP53 wildtype (WT) disease. Optimizing immunotherapy and targeting vulnerabilities due to co-mutations and/or chromosome abnormalities should be the focus of future research.","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":" ","pages":"449-456"},"PeriodicalIF":5.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142627554","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

Genetic predisposition to myelodysplastic syndrome: Genetic counseling and transplant implications. 骨髓增生异常综合征的遗传倾向：遗传咨询和移植影响。

IF 5 3区医学

Seminars in hematology Pub Date : 2024-12-01 Epub Date: 2024-09-22 DOI: 10.1053/j.seminhematol.2024.09.003

Yi Liu, Kathleen Calzone, Lisa J McReynolds

{"title":"Genetic predisposition to myelodysplastic syndrome: Genetic counseling and transplant implications.","authors":"Yi Liu, Kathleen Calzone, Lisa J McReynolds","doi":"10.1053/j.seminhematol.2024.09.003","DOIUrl":"10.1053/j.seminhematol.2024.09.003","url":null,"abstract":"The development of myelodysplastic syndromes (MDS) is influenced by various genetic predispositions. Several important genes contribute to disease susceptibility. This paper explores common genetic predisposition genes in MDS, including DDX41, CEBPA, and SAMD9/SAMD9L, which are linked to hereditary conditions presenting diagnostic and clinical challenges. It delves into hereditary conditions that affect platelet production and count, such as RUNX1, ETV6, and ANKRD26, detailing their clinical features and how they contribute to an increased risk of MDS. The discussion extends to additional genetic syndromes like GATA2 deficiency, telomere biology disorders, Fanconi anemia, and Li-Fraumeni syndrome, along with new findings on genes like ERG that offer new insights into disease etiology. The importance of genetic counseling in MDS is underscored, outlining its goals, methods for evaluating family history, risk assessment, and the ethical considerations involved. Furthermore, the role of hematopoietic cell transplantation in managing MDS, particularly in patients with germline syndromes, is reviewed, emphasizing the need for optimal donor selection and personalized treatment approaches. This comprehensive overview illustrates the critical role of genetic factors in MDS and highlights the need for continued research and tailored clinical practices to improve patient outcomes.","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":" ","pages":"370-378"},"PeriodicalIF":5.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142507134","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

A Call to Arms: Overcoming challenges in Myelodysplastic Syndromes therapy advances. 武器的召唤：克服骨髓增生异常综合症治疗进展中的挑战。

IF 5 3区医学

Seminars in hematology Pub Date : 2024-12-01 DOI: 10.1053/j.seminhematol.2024.11.005

Kathy L McGraw, Peter D Aplan, Steven Z Pavletic

引用次数: 0

Allogeneic hematopoietic cell transplantation in elderly patients with myelodysplastic syndromes: Considerations and challenges. 骨髓增生异常综合征老年患者的异基因造血细胞移植：考虑因素和挑战。

IF 5 3区医学

Seminars in hematology Pub Date : 2024-12-01 Epub Date: 2024-10-17 DOI: 10.1053/j.seminhematol.2024.10.004

Eduard Schulz, Steven Z Pavletic, Alain Mina

引用次数: 0

Management of Myelodysplastic Syndrome in Pregnant Patients: Treatment Approaches and Considerations. 妊娠患者骨髓增生异常综合征的管理：治疗方法和注意事项。

IF 5 3区医学

Seminars in hematology Pub Date : 2024-12-01 Epub Date: 2024-11-20 DOI: 10.1053/j.seminhematol.2024.11.001

Erin McDonald, David M Swoboda

引用次数: 0

Cellular and immunotherapies for myelodysplastic syndromes. 骨髓增生异常综合征的细胞和免疫疗法。

IF 5 3区医学

Seminars in hematology Pub Date : 2024-12-01 Epub Date: 2024-09-21 DOI: 10.1053/j.seminhematol.2024.09.006

Ryan J Stubbins, Hannah Cherniawsky, Aly Karsan

{"title":"Cellular and immunotherapies for myelodysplastic syndromes.","authors":"Ryan J Stubbins, Hannah Cherniawsky, Aly Karsan","doi":"10.1053/j.seminhematol.2024.09.006","DOIUrl":"10.1053/j.seminhematol.2024.09.006","url":null,"abstract":"In this review article, we outline the current landscape of immune and cell therapy-based approaches for patients with myelodysplastic syndromes (MDS). Given the well characterized graft-versus-leukemia (GVL) effect observed with allogeneic hematopoietic cell transplantation, and the known immune escape mechanisms observed in MDS cells, significant interest exists in developing immune-based approaches to treat MDS. These attempts have included antibody-based drugs that block immune escape molecules, such as inhibitors of the PD-1/PD-L1 and TIM-3/galectin-9 axes that mediate interactions between MDS cells and T-lymphocytes, as well as antibodies that block the CD47/SIRPα interaction, which mediates macrophage phagocytosis. Unfortunately, these approaches have been largely unsuccessful. There is significant potential for T-cell engaging therapies and chimeric antigen receptor T (CAR-T) cells, but there are also several limitations to these approaches that are unique to MDS. However, many of these limitations may be overcome by the next generation of cellular therapies, including those with engineered T-cell receptors or natural killer (NK)-cell based platforms. Regardless of the approach, all these immune cells are subject to the complex bone marrow microenvironment in MDS, which harbours a variable and heterogeneous mix of pro-inflammatory cytokines and immunosuppressive elements. Understanding this interaction will be paramount to ensuring the success of immune and cellular therapies in MDS.","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":" ","pages":"397-408"},"PeriodicalIF":5.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142473899","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