Pub Date : 2023-07-01DOI: 10.1097/PPO.0000000000000671
Michael M Allevato, Joshua D Smith, Michael J Brenner, Steven B Chinn
Abstract: The global incidence of human papillomavirus-positive (HPV+) head and neck squamous cell carcinoma (HNSCC) has surged in recent decades, with HPV+ HNSCC accounting for >70% of oropharynx cancers in the United States. Its incidence in men has surpassed that of HPV+ cervical cancer in women, and reliable assays are needed for early detection and to monitor response to therapy. Human papillomavirus-positive OPSCC has a more favorable response to therapy and prognosis than HPV-negative (HPV-) HNSCC, motivating regimens to deintensify curative surgery or chemoradiotherapy protocols. A barrier to deintensifying and personalizing therapy is lack of reliable predictive biomarkers. Furthermore, HPV- HNSCC survival rates are static without reliable surveillance biomarkers available. The emergence of circulating plasma-based biomarkers reflecting the tumor-immune microenvironment heralds a new era in HNSCC diagnosis and therapy. We review evidence on tumor-derived extracellular vesicles (exosomes) as biomarkers for diagnosis, prognostication, and treatment in HPV+ and HPV- HNSCC.
{"title":"Tumor-Derived Exosomes and the Role of Liquid Biopsy in Human Papillomavirus Oropharyngeal Squamous Cell Carcinoma.","authors":"Michael M Allevato, Joshua D Smith, Michael J Brenner, Steven B Chinn","doi":"10.1097/PPO.0000000000000671","DOIUrl":"10.1097/PPO.0000000000000671","url":null,"abstract":"<p><strong>Abstract: </strong>The global incidence of human papillomavirus-positive (HPV+) head and neck squamous cell carcinoma (HNSCC) has surged in recent decades, with HPV+ HNSCC accounting for >70% of oropharynx cancers in the United States. Its incidence in men has surpassed that of HPV+ cervical cancer in women, and reliable assays are needed for early detection and to monitor response to therapy. Human papillomavirus-positive OPSCC has a more favorable response to therapy and prognosis than HPV-negative (HPV-) HNSCC, motivating regimens to deintensify curative surgery or chemoradiotherapy protocols. A barrier to deintensifying and personalizing therapy is lack of reliable predictive biomarkers. Furthermore, HPV- HNSCC survival rates are static without reliable surveillance biomarkers available. The emergence of circulating plasma-based biomarkers reflecting the tumor-immune microenvironment heralds a new era in HNSCC diagnosis and therapy. We review evidence on tumor-derived extracellular vesicles (exosomes) as biomarkers for diagnosis, prognostication, and treatment in HPV+ and HPV- HNSCC.</p>","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":"29 4","pages":"230-237"},"PeriodicalIF":2.2,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10372688/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9884051","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-07-01DOI: 10.1097/PPO.0000000000000670
Molly E Heft Neal, Heather M Walline, Catherine T Haring
Abstract: Circulating tumor DNA (ctDNA) has become an area of intense study in many solid malignancies including head and neck cancer. This is of particular interest for human papillomavirus-mediated oropharyngeal squamous cell carcinoma as this cohort of patients has excellent survival and is undergoing current clinical trials aimed at treatment de-escalation. Recent studies have demonstrated the prognostic implications of pretreatment ctDNA and the utility of monitoring ctDNA during and posttreatment; however, there is a need for a more critical understanding of ctDNA as it is beginning to be incorporated into clinical trials. This review discusses the current state of ctDNA in oropharynx cancer focusing on ctDNA kinetics and minimal residual disease detection and ends with a discussion of future applications.
{"title":"Circulating Tumor DNA in Human Papillomavirus-Mediated Oropharynx Cancer: Leveraging Early Data to Inform Future Directions.","authors":"Molly E Heft Neal, Heather M Walline, Catherine T Haring","doi":"10.1097/PPO.0000000000000670","DOIUrl":"10.1097/PPO.0000000000000670","url":null,"abstract":"<p><strong>Abstract: </strong>Circulating tumor DNA (ctDNA) has become an area of intense study in many solid malignancies including head and neck cancer. This is of particular interest for human papillomavirus-mediated oropharyngeal squamous cell carcinoma as this cohort of patients has excellent survival and is undergoing current clinical trials aimed at treatment de-escalation. Recent studies have demonstrated the prognostic implications of pretreatment ctDNA and the utility of monitoring ctDNA during and posttreatment; however, there is a need for a more critical understanding of ctDNA as it is beginning to be incorporated into clinical trials. This review discusses the current state of ctDNA in oropharynx cancer focusing on ctDNA kinetics and minimal residual disease detection and ends with a discussion of future applications.</p>","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":"29 4","pages":"215-219"},"PeriodicalIF":2.2,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9845066","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}
Pub Date : 2023-05-01DOI: 10.1097/PPO.0000000000000658
Jan Philipp Bewersdorf, Zhuoer Xie, Amer M Zeidan
Abstract: Myelodysplastic syndromes/neoplasms (MDSs) constitute a heterogeneous group of clonal disorders that are clinically characterized by dysplastic changes in multiple hematopoietic lineages, cytopenias, and a variable risk of progression to acute myeloid leukemia. Patients with MDS are classified as either lower- or higher-risk based on risk stratification tools such as the International Prognostic Scoring System and its revised version, which continue to be the basis for prognosis and treatment selection. Although anemic patients with lower-risk MDS are currently treated with an erythropoiesis-stimulating agent, luspatercept, and transfusions, the telomerase inhibitor imetelstat and the hypoxia-inducible factor α inhibitor roxadustat have shown encouraging early results and are now in phase III clinical trials. For higher-risk MDS patients, hypomethylating agent monotherapy continues to be the standard of care. However, with various novel hypomethylating agent-based combination therapies in advanced clinical testing and an increased emphasis on individualized biomarker-driven treatment decisions, the standard therapy paradigms might change in the future.
{"title":"Novel Approaches and Future Directions in Myelodysplastic Syndrome Treatment.","authors":"Jan Philipp Bewersdorf, Zhuoer Xie, Amer M Zeidan","doi":"10.1097/PPO.0000000000000658","DOIUrl":"https://doi.org/10.1097/PPO.0000000000000658","url":null,"abstract":"<p><strong>Abstract: </strong>Myelodysplastic syndromes/neoplasms (MDSs) constitute a heterogeneous group of clonal disorders that are clinically characterized by dysplastic changes in multiple hematopoietic lineages, cytopenias, and a variable risk of progression to acute myeloid leukemia. Patients with MDS are classified as either lower- or higher-risk based on risk stratification tools such as the International Prognostic Scoring System and its revised version, which continue to be the basis for prognosis and treatment selection. Although anemic patients with lower-risk MDS are currently treated with an erythropoiesis-stimulating agent, luspatercept, and transfusions, the telomerase inhibitor imetelstat and the hypoxia-inducible factor α inhibitor roxadustat have shown encouraging early results and are now in phase III clinical trials. For higher-risk MDS patients, hypomethylating agent monotherapy continues to be the standard of care. However, with various novel hypomethylating agent-based combination therapies in advanced clinical testing and an increased emphasis on individualized biomarker-driven treatment decisions, the standard therapy paradigms might change in the future.</p>","PeriodicalId":9655,"journal":{"name":"Cancer journal","volume":"29 3","pages":"195-202"},"PeriodicalIF":2.2,"publicationDate":"2023-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9498193","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}
Pub Date : 2023-05-01DOI: 10.1097/PPO.0000000000000659
Mina L Xu, Robert P Hasserjian
Abstract: 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.
{"title":"Updates in Classification of Myelodysplastic Syndrome.","authors":"Mina L Xu, 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":"29 3","pages":"122-129"},"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}
Pub Date : 2023-05-01DOI: 10.1097/PPO.0000000000000665
Lara K Rotter, Shai Shimony, Kelly Ling, Evan Chen, Rory M Shallis, Amer M Zeidan, Maximilian Stahl
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.
{"title":"Epidemiology and Pathogenesis of Myelodysplastic Syndrome.","authors":"Lara K Rotter, Shai Shimony, Kelly Ling, Evan Chen, Rory M Shallis, Amer M Zeidan, 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":"29 3","pages":"111-121"},"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}
Pub Date : 2023-05-01DOI: 10.1097/PPO.0000000000000656
Zhuoer Xie, Evan C Chen, Lourdes M Mendez, Rami Komrokji, Amer M Zeidan
Abstract: 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.
{"title":"Spectrum From Clonal Hematopoiesis to Myelodysplastic Neoplasm/Syndromes and Other Myeloid Neoplasms.","authors":"Zhuoer Xie, Evan C Chen, Lourdes M Mendez, Rami Komrokji, 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":"29 3","pages":"130-137"},"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}
Pub Date : 2023-05-01DOI: 10.1097/PPO.0000000000000661
Jessica M Stempel, Nikolai A Podoltsev, Talib Dosani
Abstract: 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.
{"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":"29 3","pages":"168-178"},"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}
Pub Date : 2023-05-01DOI: 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, 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":"29 3","pages":"109-110"},"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}
Pub Date : 2023-05-01DOI: 10.1097/PPO.0000000000000660
Georgina Gener-Ricos, Yoheved S Gerstein, Danielle Hammond, Courtney D DiNardo
Abstract: 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.
{"title":"Germline Predisposition to Myelodysplastic Syndromes.","authors":"Georgina Gener-Ricos, Yoheved S Gerstein, Danielle Hammond, 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":"29 3","pages":"143-151"},"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}
Pub Date : 2023-05-01DOI: 10.1097/PPO.0000000000000657
Yasmin Abaza, Anand Ashwin Patel
Abstract: 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.
{"title":"Novel Therapies in Myelodysplastic Syndrome: Where Do Venetoclax and Isocitrate Dehydrogenase Inhibitors Fit in?","authors":"Yasmin Abaza, 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":"29 3","pages":"188-194"},"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}