Hematology focuses on diseases of the blood, bone marrow, and lymphatic system. Learn about the diagnosis and treatment of anemia, leukemia, and lymphoma.
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The treatment landscape for Myelodysplastic Syndrome is highly stratified. Because the disease ranges from indolent to aggressive, the “one size fits all” approach does not work. At Liv Hospital, our treatment strategy is risk adapted. For lower risk patients, the priority is minimizing symptoms, reducing transfusion needs, and maximizing quality of life. For higher risk patients, the goal is to alter the natural history of the disease, delaying the transformation to leukemia and attempting a cure through transplantation whenever possible.
For patients with Very Low or Low risk MDS who have only mild anemia and no symptoms, immediate treatment may not be necessary.
Patients are monitored with regular blood counts (e.g., every 1 to 3 months) to check for disease progression.
Therapy is typically withheld until the blood counts drop to a level that causes symptoms or requires transfusions. This avoids exposing the patient to the side effects of drugs before they are needed.
This is the backbone of therapy for all MDS patients, regardless of risk.
Patients with severe anemia or thrombocytopenia rely on transfusions of red blood cells or platelets to maintain safe levels. While lifesaving, chronic transfusions carry risks of iron overload and antibody formation.
Erythropoietin (EPO) stimulating agents (ESAs) are injections that signal the bone marrow to produce more red blood cells. They are most effective in lower risk patients who have low natural EPO levels. Granulocyte colony stimulating factor (G CSF) may be used to boost white blood cells during severe infections.
Prophylactic antibiotics or antifungals may be prescribed for patients with very low neutrophil counts to prevent infection.
For the specific subgroup of patients with MDS associated with the deletion of chromosome 5q (del 5q), a drug called Lenalidomide is highly effective.
Lenalidomide works by modulating the immune system and directly targeting the clone of cells with the genetic defect.
It can eliminate the need for transfusions in the majority of these specific patients and can also suppress the abnormal genetic clone for a period of years.
For patients with higher risk MDS who are not candidates for immediate transplant, epigenetic therapy is the standard of care.
Drugs like Azacitidine and Decitabine are “hypomethylating agents.” In MDS, certain tumor suppressor genes are silenced by a chemical process called methylation. These drugs remove those chemical tags, re awakening the genes that control cell growth and maturation.
These drugs are given as injections or IV infusions for several days in a row, every 4 weeks. They are not a cure, but they can improve blood counts, delay the progression to leukemia, and improve survival. They act slowly, often taking 4 to 6 months to show maximum benefit.
A small subset of MDS patients (usually younger ones) have “hypocellular” MDS, which resembles aplastic anemia. In these cases, the body’s T cells are attacking the marrow.
Anti thymocyte globulin (ATG) and cyclosporine are drugs that suppress the immune system. By stopping the T cell attack, they allow the bone marrow to recover. This approach is specifically selected for patients with the HLA DR15 marker or a PNH clone.
For fit patients with higher risk MDS, stem cell transplantation (bone marrow transplant) is the only treatment capable of permanently eradicating the disease.
The patient receives high dose chemotherapy to destroy the diseased bone marrow. They then receive healthy stem cells from a matched donor (sibling or unrelated registry donor).
The new immune system from the donor recognizes the remaining MDS cells as foreign and attacks them. This immunological effect is crucial for the cure.
Transplant carries significant risks, including Graft vs Host Disease (GVHD) and infection. Therefore, it is generally reserved for patients with higher risk disease where the risk of the MDS outweighs the risk of the procedure.
In patients with very high blast counts (MDS EB 2) or those transforming to AML, intensive chemotherapy (like the “7+3” regimen used in leukemia) may be used to knock down the blast count before proceeding to a transplant.
Each unit of transfused blood contains iron that the body cannot excrete. After 20 to 30 units, iron builds up in the liver and heart.
Drugs like Deferasirox or Deferoxamine bind to the excess iron and help the body excrete it. This is important for patients with a reasonably long life expectancy who are transfusion dependent.
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The only curative treatment is an allogeneic stem cell transplant. However, other treatments can manage the disease for many years.
It varies. Some patients never need them, while others may need 1 or 2 units every week. The goal of treatment is to make you “transfusion independent.”
No, Erythropoietin (EPO) takes time. It usually takes 8 to 12 weeks of injections to see if the hemoglobin level will rise.
About 50% of patients respond to Azacitidine. It can normalize blood counts and improve survival, but it usually must be continued indefinitely to maintain the response.
Generally, no. With drugs like Azacitidine or Lenalidomide, stopping the drug usually causes the MDS to return. Treatment is usually continuous.
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