Treatment given to relieve the symptoms of a disease and the treatment’s side effects is known as supportive care. The goal of supportive care is to improve the patient’s quality of life and to relieve discomfort as much as possible. Supportive care is an important part of MDS treatment.
Transfusions of red blood cells can help some patients by improving their blood cell counts or by relieving anemia symptoms, such as shortness of breath, dizziness, extreme fatigue and chest pain. Transfusion can help relieve symptoms for a short time but more transfusions may be needed over time. In MDS, 60 to 80 percent of patients have anemia at the time of diagnosis and up to 90 percent of patients will require one or more transfusions during the course of their illness.
Platelet transfusions may be used in patients with thrombocytopenia (low platelet counts), which can cause symptoms such as easy bruising or bleeding. Transfusions are typically required once a patient’s platelet count falls below 10,000/μL. Aminocaproic acid, an antifibrinolytic agent, is recommended for bleeding episodes that do not respond to platelet transfusion, and for cases of severe thrombocytopenia. This medication works by stopping blood clots from breaking down too quickly.
Iron Chelation Therapy
Iron is found in red blood cells. When a person receives a large number of red blood cell transfusions, too much iron can build up in the body. This is called iron overload and it can injure vital organs over time.
Iron chelation therapy uses drugs called “chelators,” which bind to excess iron and remove it from the body. This therapy may be appropriate for anemic patients who need frequent blood transfusions (more than 4 units of red blood cells over 8 weeks). The most common drugs used in this therapy include
- Deferasirox (Exjade®, Jadenu®)
- Deferoxamine mesylate (DFO; Desferal®)
For patients who need frequent red blood cell transfusions, it is recommended that doctors monitor serum ferritin (iron) levels and check often for signs of organ damage.
Blood Cell Growth Factors
Agents called “growth factors” promote blood cell production in the bone marrow. These agents are used to treat some patients whose blood cell counts show decreased numbers of cells.
Red Blood Cell Growth Factors
Erythropoietin (EPO) is a hormone created in the kidneys. It encourages red blood cell production in response to low oxygen levels in the body. A shortage of EPO can also cause anemia.
Erythropoiesis-stimulating agents (ESAs) are red blood cell growth factors that are pharmaceutical analogues of natural EPO. They are used for MDS patients who have anemia associated with low EPO levels. Treatment with ESAs may decrease transfusion needs and possibly improve survival.
- Epoetin alfa (Procrit®) and darbepoetin alfa (Aranesp®) are types of ESAs. They are given by an injection under the skin (subcutaneously [SC]). Darbepoetin alfa is a longer-acting form of EPO than epoetin alfa.
Most patients with MDS do not have low EPO levels, so administration of ESAs is not a useful treatment for their anemia. Nonetheless, all MDS patients should have their EPO levels checked.
Some MDS patients with low EPO levels may not benefit from treatment with ESAs alone; however, an ESA given along with G-CSF may increase their hemoglobin concentration (see below).
White Blood Cell Growth Factors
White blood cell growth factors are naturally produced by the body, and help increase the production of white blood cells. Synthetic versions of these substances may be used to treat patients with frequent infections due to neutropenia, but they are not known to help patients live longer. The two main types are
- Granulocyte colony-stimulating factor (G-CSF) which helps the body increase white blood cell production. Filgrastim (Neupogen®) and Pegfilgrastim (Neulasta®) are examples of G-CSF medications.
- Granulocyte-macrophage colony-stimulating factor (GM-CSF) helps the body produce many different types of white blood cells. Sargramostim (Leukine®) is a GM-CSF medication.
Platelet Growth Factors
Thrombopoietin (TPO) is a substance that helps the body produce platelets.
- Romiplostim (Nplate®) and eltrombopag (Promacta®) are drugs that act like TPO. These agents are being investigated as treatment for MDS patients who have low platelet counts. Currently, these drugs are FDA-approved for the treatment of thrombocytopenia (low platelet counts) in patients who have chronic immune thrombocytopenic purpura (ITP), and who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
- Although romiplostim and eltrombopag are not approved specifically for MDS, sometimes they can be helpful in patients with very low platelet counts.
Erythroid maturation agents
Erythroid maturation agents are used to treat anemia. They work by regulating red blood cell maturation.
- Luspatercept-aamt (Reblozyl®) is FDA approved for the treatment of anemia failing an erythropoiesis stimulating agent (ESA) and requiring 2 or more red blood cell (RBC) units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).
Limitations of Use: Reblozyl is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.
A low number of white blood cells can increase the risk of infection. In some cases, infection may be frequent or severe. Your treatment team will pay close attention to any infection or unexplained fever. If a bacterial infection is identified or suspected, antibiotics may be needed. Antiviral drugs may be used to treat certain viral infections.