Treatment of Low-Risk MDS According to NCCN Recommendations Version 1.2023

Low-Risk MDS

Low-Risk MDS (MDS-LR) is defined based on the IPSS-R (Revised International Prognostic Scoring System) score value in the very low, low, and intermediate ranges, i.e., ≤ 3.5. Treatment is recommended for patients with clinically significant cytopenia. Supportive care is an integral part of therapy.

Treatment of Patients with MDS-LR and Clinically Relevant Thrombocytopenia or Neutropenia

In the case of clinically relevant thrombocytopenia or neutropenia, azacitidine is preferred according to the recommendations, or the patient can be enrolled in a clinical trial. Other recommended modalities are intravenous decitabine or the combination of oral decitabine + cedazuridine. Immunosuppressive therapy with the possible addition of eltrombopag can be beneficial under certain circumstances. If the disease progresses or there is no response to treatment, hypomethylating agents, clinical trial participation, and allogeneic hematopoietic cell transplantation in selected patients can be considered.

Treatment of Patients with MDS-LR and Symptomatic Anemia

The recommended approach for patients with symptomatic anemia is shown in the following diagram.

Fig.  NCCN Recommendations (version 1. 2023) for the treatment of Low-Risk MDS with symptomatic anemia (all recommendations are class 2A)

Approach with Serum Erythropoietin Levels > 500 mU/ml

If serum erythropoietin levels exceed 500 mU/ml and the probability of response to immunosuppressive therapy is sufficiently high, the preferred recommendation is antithymocyte globulin (ATG) + cyclosporine A + eltrombopag, or ATG + cyclosporine A can be administered; another recommended option is ATG + eltrombopag, or under certain circumstances, ATG alone can be used (recommendation class 3). For patients without response or with intolerance to this treatment, it is recommended to proceed as with a low probability of response to immunotherapy.

In the case of serum erythropoietin levels > 500 mU/ml and low probability of response to immunosuppression, azacitidine is the preferred choice, or enrolling in a clinical trial is suitable. Other options are decitabine or oral decitabine and cedazuridine. Lenalidomide can be considered under certain circumstances. If there is no treatment response after 6 cycles of azacitidine or 4 cycles of decitabine or oral decitabine and cedazuridine, or if the patient is intolerant to this treatment, enrolling in a clinical study is recommended, or allogeneic hematopoietic stem cell transplantation can be considered for selected patients.

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Source: Myelodysplastic Syndromes. NCCN Clinical Practice Guidelines in Oncology. Version 1.2023. Available at: www.nccn.org/guidelines/guidelines-detail?category=1&id=1446