When does myelodysplastic syndrome (MDS) require treatment?

From the Guidelines

Treatment for myelodysplastic syndrome (MDS) is required when patients develop significant cytopenias, such as hemoglobin levels below 10 g/dL, neutrophil counts under 1,800/μL, or platelet counts below 100,000/μL, causing symptoms like fatigue, frequent infections, or bleeding problems, as indicated by the revised International Prognostic Scoring System (IPSS-R) risk classification 1.

Key Factors for Treatment Initiation

The decision to initiate treatment in MDS patients is based on several factors, including:

• Symptom severity
• Blood count abnormalities
• Risk classification using the IPSS or IPSS-R
• Transfusion dependence
• Bone marrow blast percentage
• Genetic mutations like TP53 or complex cytogenetic abnormalities

Treatment Options

Treatment options for MDS range from:

• Supportive care (transfusions, growth factors) for lower-risk disease
• Hypomethylating agents (azacitidine, decitabine) for higher-risk disease
• Stem cell transplantation for higher-risk disease As recommended by the European Society for Medical Oncology (ESMO) clinical practice guidelines for diagnosis, treatment, and follow-up of MDS 1.

Monitoring and Disease Progression

Regular monitoring through complete blood counts and periodic bone marrow examinations is essential to track disease progression and determine the appropriate timing for treatment initiation. The IPSS-R risk classification is crucial in determining treatment timing, with higher-risk patients typically requiring immediate intervention 1.

Specific Treatment Considerations

For IPSS-R intermediate-risk MDS patients, treatment decisions are based on factors like age, comorbidities, and somatic mutations. Patients with a chromosomal abnormality in addition to del(5q) may have similar outcomes to those with isolated del(5q), except for certain additional abnormalities 1.

Adverse Events and Monitoring

Close monitoring of blood counts is required during treatment, particularly for grade 3 or 4 neutropaenia and thrombocytopaenia, which are common adverse events of lenalidomide (LEN) treatment 1.

Treatment Algorithm

The treatment algorithm for lower-risk MDS is outlined in Figure 3 of the ESMO guidelines, which includes options like antithymocyte globulin (ATG), erythropoietin (EPO), granulocyte colony-stimulating factor (G-CSF), and thrombopoietin receptor agonist (TPO-RA) 1. The choice of treatment should be individualized based on patient-specific factors, including symptom severity, blood count abnormalities, and risk classification, with the goal of improving quality of life and reducing morbidity and mortality 1.

From the FDA Drug Label

Patients with myelodysplastic syndromes (MDS) require treatment if they have certain criteria, including: required packed RBC transfusions; had platelet counts <50.0 x 10^9/L; required platelet transfusions; or were neutropenic (ANC <1.0 x 10^9/L) with infections requiring treatment with antibiotics.

The decision to treat myelodysplastic syndrome (MDS) depends on the presence of specific criteria, such as:

• Need for packed RBC transfusions
• Platelet counts <50.0 x 10^9/L
• Need for platelet transfusions
• Neutropenia (ANC <1.0 x 10^9/L) with infections requiring antibiotic treatment Treatment with azacitidine or decitabine may be considered for patients who meet these criteria 2, 3.

From the Research

Treatment Requirements for Myelodysplastic Syndrome (MDS)

Myelodysplastic syndrome (MDS) requires treatment based on various factors, including the risk level of the disease, symptoms, and individual patient needs. The following points outline the treatment requirements for MDS:

• Treatment is typically required for patients with higher-risk MDS, as well as those with lower-risk MDS who are symptomatic or have a high risk of progression to acute myeloid leukemia (AML) 4, 5.
• The Revised International Prognosis Scoring System (IPSS-R) is used to divide patients into low- and high-risk groups, which helps guide treatment decisions 4.
• For lower-risk patients, treatment goals include improving cytopenia, quality of life, and overall survival, while for higher-risk patients, the goal is to prolong survival 5, 6.
• Treatment options for MDS include:
  • Supportive care, such as red blood cell transfusions and iron chelation 6, 7.
  • Erythropoiesis-stimulating agents (ESAs) for anemic patients 5, 7.
  • Lenalidomide for patients with del(5q) MDS 4, 6.
  • Hypomethylating agents, such as 5-azacytidine, for higher-risk patients 4, 5.
  • Allogeneic stem cell transplantation for eligible patients, particularly those with higher-risk disease 4, 5.
• New therapeutic options are being developed, including thrombopoietin analogues, TGF-beta family ligand trapping drugs, and telomerase inhibitors 6.
• Treatment decisions should be individualized based on patient-specific factors, including risk level, symptoms, and comorbidities 5, 8.