One article counts the treatment of low-risk myelodysplastic syndromes

Myelodysplastic syndrome (MDS) is a group of heterogeneous clonal hematopoietic stem cell diseases, the clinical feature of which is bone marrow failure leading to cytopenia and other related complications
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In the past few years, researchers have learned more about clonal hematopoietic disorders from the uncertain potential of clonal hematopoiesis (CHIP), the unexplained clonal hematopoietic disease (CCUS) to MDS
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Accurate diagnosis of MDS requires comprehensive analysis from clinical, pathological, molecular and other aspects.
For pathological manifestations of abnormal morphology, an experienced blood pathologist is required to make judgments
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The next step is disease risk assessment, and the treatment plan and treatment goals are adjusted accordingly according to different disease risks
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The revised International Prognostic Scoring System (R-IPSS) (based on cytopenia and its degree, percentage of blasts, and cytogenetics) is the most commonly used tool for assessing disease risk.
R-IPSS can be used to assess somatic mutations and patient pooling.
Comprehensive evaluation of symptoms and other factors, more than half of MDS patients are ultimately classified as low-risk MDS (LR-MDS)
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In the past, the goal of LR-MDS treatment was to reduce cytopenias to prevent complications and improve the quality of life of patients.
Although treatment of cytopenias may indirectly affect survival, so far, there is no clear evidence that treatment of LR-MDS improves overall survival
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In most LR-MDS, the treatment goal is to improve anemia.
Erythropoiesis stimulant (ESA) is a good treatment option to control anemia.
In addition, it can be based on red blood cell (RBC) transfusion load and endogenous serum erythropoiesis.
It predicts the treatment response of ESA, and sufficient ESA treatment dose and duration are required to evaluate the response
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For patients with del(5q) MDS, lenalidomide is the preferred treatment method, with a higher incidence of RBC transfusion independence (TI)
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The reduction of blood cells in the early stage of treatment is predictable and controllable
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A recent data indicates that the use of low-dose lenalidomide earlier and for a certain period of time is associated with a longer transfusion-dependent time and a higher cytogenetic response rate
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It should be noted that the prognosis of patients with del(5q) after failure of lenalidomide treatment is very poor
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For patients with LR-MDS with ring sideroblast (RS) subtype, Luspatercept is approved for the treatment of anemia, which is the first approved drug for MDS in ten years
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Luspatercept is a pioneering red blood cell maturation agent (EMA) that can regulate the maturation of late red blood cells
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Luspatercept has a high incidence of RBC-TI, especially for patients with low blood transfusion burden
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Related research is exploring the efficacy of this new type of drug (compared with or combined with ESA) in the early treatment of LR-MDS patients
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For young non-del (5q) and non-RS LR-MDS patients, early in the course of the disease, bone marrow cells are insufficient, anti-thymocyte globulin (ATG) and cyclosporine immunosuppressive therapy (IST) can produce a long-lasting response
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For those patients who are not suitable for IST treatment, if isolated anemia and adequate platelet and neutrophil counts, lenalidomide can also be a treatment option
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Demethylating agents (HMA) (azacitidine and decitabine) should be used in patients with higher-risk disease characteristics, late course of disease, or double/pancytopenia, especially those who are not suitable for IST treatment Patient
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A shorter treatment "3-day plan" is being explored, which may be a new treatment option for patients with LR-MDS, and research on oral HMA treatment for LR-MDS is also ongoing
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In addition, several new drugs for the treatment of LR-MDS are undergoing clinical trials.
Metlestat, a telomerase inhibitor, has shown encouraging activity in phase II clinical trials and is currently being studied in phase III randomized clinical trials.

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Reference source: Rami Komrokji.
2021 SOHO.
EXABS-148-MDS.
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