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Updates in Classification of Myelodysplastic Syndrome. 骨髓增生异常综合征分类的最新进展。
IF 2.2 4区 医学
Cancer journal Pub Date : 2023-05-01 DOI: 10.1097/PPO.0000000000000659
Mina L Xu, Robert P Hasserjian
{"title":"Updates in Classification of Myelodysplastic Syndrome.","authors":"Mina L Xu,&nbsp;Robert P Hasserjian","doi":"10.1097/PPO.0000000000000659","DOIUrl":"https://doi.org/10.1097/PPO.0000000000000659","url":null,"abstract":"<p><strong>Abstract: </strong>Myelodysplastic syndrome includes a broad range of myeloid neoplasms characterized by cytopenia and morphologic dysplasia. Recently, 2 new classification systems emerged to further define how these diseases are diagnosed and risk stratified. This review compares these models, provides detailed approaches, and reveals practical ways to move forward in clinical practice of myelodysplastic syndrome diagnosis.</p>","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9498186","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
Epidemiology and Pathogenesis of Myelodysplastic Syndrome. 骨髓增生异常综合征的流行病学和发病机制。
IF 2.2 4区 医学
Cancer journal Pub Date : 2023-05-01 DOI: 10.1097/PPO.0000000000000665
Lara K Rotter, Shai Shimony, Kelly Ling, Evan Chen, Rory M Shallis, Amer M Zeidan, Maximilian Stahl
{"title":"Epidemiology and Pathogenesis of Myelodysplastic Syndrome.","authors":"Lara K Rotter,&nbsp;Shai Shimony,&nbsp;Kelly Ling,&nbsp;Evan Chen,&nbsp;Rory M Shallis,&nbsp;Amer M Zeidan,&nbsp;Maximilian Stahl","doi":"10.1097/PPO.0000000000000665","DOIUrl":"https://doi.org/10.1097/PPO.0000000000000665","url":null,"abstract":"Abstract Myelodysplastic syndrome (MDS) is a clonal disorder characterized by ineffective hematopoiesis and variable cytopenias with a considerable risk of progression to acute myeloid leukemia. Epidemiological assessment of MDS remains challenging because of evolving classification systems, but the overall incidence in the United States is estimated to be approximately 4 per 100,000 and increases with age. The sequential accumulation of mutations drives disease evolution from asymptomatic clonal hematopoiesis (CH) to CH of indeterminate potential, clonal cytopenia of unknown significance, to frank MDS. The molecular heterogeneity seen in MDS is highly complex and includes mutations of genes involved in splicing machinery, epigenetic regulation, differentiation, and cell signaling. Recent advances in the understanding of the molecular landscape of MDS have led to the development of improved risk assessment tools and novel therapies. Therapies targeting the underlying pathophysiology will hopefully further expand the armamentarium of MDS therapeutics, bringing us closer to a more individualized therapeutic approach based on the unique molecular profile of each patient and eventually improving the outcomes of patients with MDS. We review the epidemiology of MDS and the newly described MDS precursor conditions CH, CH of indeterminate potential, and CCUS. We then discuss central aspects of MDS pathophysiology and outline specific strategies targeting hallmarks of MDS pathophysiology, including ongoing clinical trials examining the efficacy of these therapeutic modalities.","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9498187","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
Spectrum From Clonal Hematopoiesis to Myelodysplastic Neoplasm/Syndromes and Other Myeloid Neoplasms. 从克隆造血到骨髓增生异常肿瘤/综合征和其他髓系肿瘤的频谱。
IF 2.2 4区 医学
Cancer journal Pub Date : 2023-05-01 DOI: 10.1097/PPO.0000000000000656
Zhuoer Xie, Evan C Chen, Lourdes M Mendez, Rami Komrokji, Amer M Zeidan
{"title":"Spectrum From Clonal Hematopoiesis to Myelodysplastic Neoplasm/Syndromes and Other Myeloid Neoplasms.","authors":"Zhuoer Xie,&nbsp;Evan C Chen,&nbsp;Lourdes M Mendez,&nbsp;Rami Komrokji,&nbsp;Amer M Zeidan","doi":"10.1097/PPO.0000000000000656","DOIUrl":"https://doi.org/10.1097/PPO.0000000000000656","url":null,"abstract":"<p><strong>Abstract: </strong>Clonal hematopoiesis (CH) confers a high risk of aging-related diseases and hematologic malignancy. There are still significant knowledge gaps in identifying high-risk patients with CH and managing such patients. In this review, we focus on 3 areas: (1) the natural history of CH; (2) the risks of progression of CH, including CH of indeterminate potential, clonal cytopenia of undetermined significance, and therapy-related CH, to myeloid malignancy; and (3) the challenges and unmet needs of CH management and research.</p>","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9498188","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
Supportive Care for Patients With Myelodysplastic Syndromes. 骨髓增生异常综合征患者的支持治疗。
IF 2.2 4区 医学
Cancer journal Pub Date : 2023-05-01 DOI: 10.1097/PPO.0000000000000661
Jessica M Stempel, Nikolai A Podoltsev, Talib Dosani
{"title":"Supportive Care for Patients With Myelodysplastic Syndromes.","authors":"Jessica M Stempel, Nikolai A Podoltsev, Talib Dosani","doi":"10.1097/PPO.0000000000000661","DOIUrl":"10.1097/PPO.0000000000000661","url":null,"abstract":"<p><strong>Abstract: </strong>Myelodysplastic syndromes are a heterogeneous group of bone marrow disorders characterized by ineffective hematopoiesis, progressive cytopenias, and an innate capability of progressing to acute myeloid leukemia. The most common causes of morbidity and mortality are complications related to myelodysplastic syndromes rather than progression to acute myeloid leukemia. Although supportive care measures are applicable to all patients with myelodysplastic syndromes, they are especially essential in patients with lower-risk disease who have a better prognosis compared with their higher-risk counterparts and require longer-term monitoring of disease and treatment-related complications. In this review, we will address the most frequent complications and supportive care interventions used in patients with myelodysplastic syndromes, including transfusion support, management of iron overload, antimicrobial prophylaxis, important considerations in the era of COVID-19 (coronavirus infectious disease 2019), role of routine immunizations, and palliative care in the myelodysplastic syndrome population.</p>","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9500765","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
Myelodysplastic Syndromes/Neoplasms-Insights Into Pathogenesis Leading to Improved Classification, Risk Stratification, and Treatments. 骨髓增生异常综合征/肿瘤-导致改进分类，风险分层和治疗的发病机制的见解。
IF 2.2 4区 医学
Cancer journal Pub Date : 2023-05-01 DOI: 10.1097/PPO.0000000000000663
Michal G Rose, Amer M Zeidan
{"title":"Myelodysplastic Syndromes/Neoplasms-Insights Into Pathogenesis Leading to Improved Classification, Risk Stratification, and Treatments.","authors":"Michal G Rose,&nbsp;Amer M Zeidan","doi":"10.1097/PPO.0000000000000663","DOIUrl":"https://doi.org/10.1097/PPO.0000000000000663","url":null,"abstract":"","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9498185","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
Germline Predisposition to Myelodysplastic Syndromes. 骨髓增生异常综合征的种系易感性。
IF 2.2 4区 医学
Cancer journal Pub Date : 2023-05-01 DOI: 10.1097/PPO.0000000000000660
Georgina Gener-Ricos, Yoheved S Gerstein, Danielle Hammond, Courtney D DiNardo
{"title":"Germline Predisposition to Myelodysplastic Syndromes.","authors":"Georgina Gener-Ricos,&nbsp;Yoheved S Gerstein,&nbsp;Danielle Hammond,&nbsp;Courtney D DiNardo","doi":"10.1097/PPO.0000000000000660","DOIUrl":"https://doi.org/10.1097/PPO.0000000000000660","url":null,"abstract":"<p><strong>Abstract: </strong>While germline predisposition to myelodysplastic syndromes is well-established, knowledge has advanced rapidly resulting in more cases of inherited hematologic malignancies being identified. Understanding the biological features and main clinical manifestations of hereditary hematologic malignancies is essential to recognizing and referring patients with myelodysplastic syndrome, who may underlie inherited predisposition, for appropriate genetic evaluation. Importance lies in individualized genetic counseling along with informed treatment decisions, especially with regard to hematopoietic stem cell transplant-related donor selection. Future studies will improve comprehension of these disorders, enabling better management of affected patients and their families.</p>","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9498189","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
Novel Therapies in Myelodysplastic Syndrome: Where Do Venetoclax and Isocitrate Dehydrogenase Inhibitors Fit in? 骨髓增生异常综合征的新疗法:Venetoclax和异柠檬酸脱氢酶抑制剂适用于哪里?
IF 2.2 4区 医学
Cancer journal Pub Date : 2023-05-01 DOI: 10.1097/PPO.0000000000000657
Yasmin Abaza, Anand Ashwin Patel
{"title":"Novel Therapies in Myelodysplastic Syndrome: Where Do Venetoclax and Isocitrate Dehydrogenase Inhibitors Fit in?","authors":"Yasmin Abaza,&nbsp;Anand Ashwin Patel","doi":"10.1097/PPO.0000000000000657","DOIUrl":"https://doi.org/10.1097/PPO.0000000000000657","url":null,"abstract":"<p><strong>Abstract: </strong>Myelodysplastic syndromes (MDSs) are a heterogeneous group of clonal hematopoietic stem cell disorders with treatment approaches tailored to the presence of cytopenias, disease risk, and molecular mutation profile. In higher-risk MDSs, the standard of care are DNA methyltransferase inhibitors, otherwise referred to as hypomethylating agents (HMAs), with consideration for allogeneic hematopoietic stem cell transplantation in appropriate candidates. Given modest complete remission rates (15%-20%) with HMA monotherapy and median overall survival of approximately 18 months, there is much interest in the investigation of combination and targeted treatment approaches. Furthermore, there is no standard treatment approach in patients with progression of disease after HMA therapy. In this review, we aim to summarize the current evidence for the B-cell lymphoma-2 inhibitor, venetoclax, and a variety of isocitrate dehydrogenase inhibitors in the treatment of MDSs along with discussing their potential role in the treatment paradigm of this disease.</p>","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9500762","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
Evolution of Therapeutic Benefit Measurement Criteria in Myelodysplastic Syndromes/Neoplasms. 骨髓增生异常综合征/肿瘤治疗获益测量标准的演变。
IF 2.2 4区 医学
Cancer journal Pub Date : 2023-05-01 DOI: 10.1097/PPO.0000000000000666
Jessica M Stempel, Zhuoer Xie, Jan Philipp Bewersdorf, Maximilian Stahl, Amer M Zeidan
{"title":"Evolution of Therapeutic Benefit Measurement Criteria in Myelodysplastic Syndromes/Neoplasms.","authors":"Jessica M Stempel,&nbsp;Zhuoer Xie,&nbsp;Jan Philipp Bewersdorf,&nbsp;Maximilian Stahl,&nbsp;Amer M Zeidan","doi":"10.1097/PPO.0000000000000666","DOIUrl":"https://doi.org/10.1097/PPO.0000000000000666","url":null,"abstract":"<p><strong>Abstract: </strong>Myelodysplastic syndromes/neoplasms (MDS) are heterogeneous, clonal myeloid neoplasms characterized by ineffective hematopoiesis, progressive cytopenias, and an increased risk of progression to acute myeloid leukemia. The diversity in disease severity, morphology, and genetic landscape challenges not only novel drug development but also therapeutic response assessment. The MDS International Working Group (IWG) response criteria were first published in the year 2000 focusing on measures of blast burden reduction and hematologic recovery. Despite revision of the IWG criteria in 2006, correlation between IWG-defined responses and patient-focused outcomes, including long-term benefits, remains limited and has potentially contributed to failures of several phase III clinical trials. Several IWG 2006 criteria also lacked clear definitions leading to problems in practical applications and interobserver and intraobserver consistency of response reporting. Although the 2018 revision addressed lower-risk MDS, the most recent update in 2023 redefined responses for higher-risk MDS and has set out to provide clear definitions to enhance consistency while focusing on clinically meaningful outcomes and patient-centered responses. In this review, we analyze the evolution of the MDS response criteria, limitations, and areas of improvement.</p>","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9500761","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: 1
Updates in Risk Stratification in Myelodysplastic Syndromes. 骨髓增生异常综合征风险分层的最新进展。
IF 2.2 4区 医学
Cancer journal Pub Date : 2023-05-01 DOI: 10.1097/PPO.0000000000000654
Luis E Aguirre, David A Sallman, Richard Stone, Rami S Komrokji
{"title":"Updates in Risk Stratification in Myelodysplastic Syndromes.","authors":"Luis E Aguirre,&nbsp;David A Sallman,&nbsp;Richard Stone,&nbsp;Rami S Komrokji","doi":"10.1097/PPO.0000000000000654","DOIUrl":"https://doi.org/10.1097/PPO.0000000000000654","url":null,"abstract":"ABSTRACT Risk stratification plays an essential role in treatment planning in myelodysplastic syndromes. For decades, the International Prognostic Scoring System and its revised version have provided unified consensus for clinical trial enrollment and design. These models relied on laboratory and cytogenetic data to estimate prognosis and dictate treatment paradigms. Critical developments in DNA sequencing techniques in recent years, as well as our growing understanding of the clonal dynamics of myelodysplastic syndromes and the role that specific mutations have in shaping disease-specific phenotypes and treatment susceptibilities, have made it possible to identify molecular markers that carry critical diagnostic and therapeutic relevance and remained unaccounted for in the older models. The Molecular International Prognostic Scoring System is a novel risk stratification model that integrates clinical, cytogenetic, and molecular data to devise a more refined prognostic tool that builds on the accuracy of the traditional models.","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9498190","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
Hematopoietic Stem Cell Transplantation for Myelodysplastic Syndromes: The Current Landscape and Future Directions. 骨髓增生异常综合征的造血干细胞移植:现状和未来方向。
IF 2.2 4区 医学
Cancer journal Pub Date : 2023-05-01 DOI: 10.1097/PPO.0000000000000662
Jean Sabile, Steven Pavletic, Yazan Migdady
{"title":"Hematopoietic Stem Cell Transplantation for Myelodysplastic Syndromes: The Current Landscape and Future Directions.","authors":"Jean Sabile,&nbsp;Steven Pavletic,&nbsp;Yazan Migdady","doi":"10.1097/PPO.0000000000000662","DOIUrl":"https://doi.org/10.1097/PPO.0000000000000662","url":null,"abstract":"<p><strong>Abstract: </strong>Myelodysplastic syndromes (MDSs) are characterized by a clonal proliferation of hematopoietic stem cells with potential life-threatening cytopenia(s) and transformation to acute myeloid leukemia. Individualized risk stratification is evolving with new molecular models, such as the Molecular International Prognostic Scoring System, for better estimation of leukemic transformation and overall survival. The only potential cure for MDSs is allogeneic transplant, although it is underutilized in MDSs because of advanced patient age and multiple comorbidities. Optimization of transplant relies on improved identification of high-risk patients pretransplant, using targeted therapies leading to deeper molecular response, developing lower toxicity conditioning regimens, engineering better molecular tools for early detection and relapse monitoring, and adding maintenance treatment strategies for high-risk patients posttransplant. This review provides an overview of transplant in MDSs with updates, future directions, and role for novel therapies.</p>","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9500756","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
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