{"title":"Treatment of high-risk myelodysplastic syndromes.","authors":"Nicolaus Kröger","doi":"10.3324/haematol.2023.284946","DOIUrl":null,"url":null,"abstract":"<p><p>Myelodysplastic syndrome (MDS) is considered to be a heterogeneous myeloid malignancy with a common origin in the hematopoietic stem cell compartment and is generally divided into lower- and higher-risk forms. While the treatment goals for lower-risk MDS are to decrease transfusion requirements and transformation into acute leukemia, the major aims for higher-risk MDS are to prolong survival and ultimately cure the patient. Although novel agents such as luspatercept and imetelstat have recently been approved as new treatment options for lower-risk MDS, hypomethylating agents currently remain the only approved non-transplant option for higher-risk MDS and are the standard of care for patients not eligible for allogeneic hematopoietic stem cell transplantation (HSCT). Combinations with other drugs as first-line treatment have to date not proven more efficacious than monotherapy in higher-risk MDS, and outcome after the failure of treatment with hypomethylating agents is poor. The only potential cure and standard of care for eligible patients is HSCT and even though the number of transplanted - especially older - MDS patients has increased over time due to better management and greater donor availability, the majority of MDS patients will not be eligible for this curative approach. Current challenges include decreasing the relapse risk, the main cause of HSCT failure. This review summarizes current knowledge on the options of transplant and non-transplant treatment approaches for these patients and demonstrate the unmet clinical need for more effective therapies.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"339-349"},"PeriodicalIF":8.2000,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11788630/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Haematologica","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.3324/haematol.2023.284946","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Myelodysplastic syndrome (MDS) is considered to be a heterogeneous myeloid malignancy with a common origin in the hematopoietic stem cell compartment and is generally divided into lower- and higher-risk forms. While the treatment goals for lower-risk MDS are to decrease transfusion requirements and transformation into acute leukemia, the major aims for higher-risk MDS are to prolong survival and ultimately cure the patient. Although novel agents such as luspatercept and imetelstat have recently been approved as new treatment options for lower-risk MDS, hypomethylating agents currently remain the only approved non-transplant option for higher-risk MDS and are the standard of care for patients not eligible for allogeneic hematopoietic stem cell transplantation (HSCT). Combinations with other drugs as first-line treatment have to date not proven more efficacious than monotherapy in higher-risk MDS, and outcome after the failure of treatment with hypomethylating agents is poor. The only potential cure and standard of care for eligible patients is HSCT and even though the number of transplanted - especially older - MDS patients has increased over time due to better management and greater donor availability, the majority of MDS patients will not be eligible for this curative approach. Current challenges include decreasing the relapse risk, the main cause of HSCT failure. This review summarizes current knowledge on the options of transplant and non-transplant treatment approaches for these patients and demonstrate the unmet clinical need for more effective therapies.
期刊介绍:
Haematologica is a journal that publishes articles within the broad field of hematology. It reports on novel findings in basic, clinical, and translational research.
Scope:
The scope of the journal includes reporting novel research results that:
Have a significant impact on understanding normal hematology or the development of hematological diseases.
Are likely to bring important changes to the diagnosis or treatment of hematological diseases.