How we manage adults with myelodysplastic syndrome

Area of Science:

• Hematology
• Oncology
• Clinical Medicine

Background:

• Prognosis in Myelodysplastic Syndromes (MDS) is stratified using conventional scores like IPSS into lower-risk (LR-MDS) and higher-risk (HR-MDS).
• Treatment strategies differ significantly based on risk stratification, targeting cytopenias in LR-MDS and disease progression in HR-MDS.

Purpose of the Study:

• To review current treatment approaches for Myelodysplastic Syndromes based on risk stratification.
• To highlight the efficacy of specific therapies for different MDS subtypes and risk groups.
• To discuss the role of emerging treatments and allogeneic stem cell transplantation.

Main Methods:

• Literature review of prognostic scoring systems and treatment guidelines for MDS.
• Analysis of treatment outcomes for anemia in LR-MDS, including erythropoietic stimulating factors and targeted therapies.
• Evaluation of treatment options for HR-MDS, including hypomethylating agents, intensive chemotherapy, and allogeneic stem cell transplantation.

Main Results:

• Erythropoietic stimulating factors are first-line for anemia in LR-MDS without del(5q); lenalidomide is highly effective for LR-MDS with del(5q).
• Allogeneic stem cell transplantation is the only potentially curative option for HR-MDS but is rarely applicable.
• Hypomethylating agents (HMA) can improve survival in HR-MDS, particularly in patients with excess blasts, and are often used prior to transplantation.

Conclusions:

• Treatment for MDS is risk-stratified, with distinct goals and therapeutic options for LR-MDS and HR-MDS.
• While established treatments exist, novel agents like venetoclax and IDH inhibitors are under investigation for specific MDS mutations.
• Allogeneic stem cell transplantation remains a key curative strategy for HR-MDS, often preceded by chemotherapy or HMAs.