Myelodysplastic syndrome (MDS) is a group of bone marrow disorders caused by poorly formed or dysfunctional blood cells. People with MDS have low numbers of one or more types of blood cells, which can lead to anemia, bleeding problems, and a higher risk of infection and transformation to acute myeloid leukemia. While there is no cure for Myelodysplastic Syndrome Treatment, several treatment options are available to manage symptoms and slow disease progression.
Chemotherapy Drugs
Chemotherapy uses drugs to destroy fast-growing myelodysplastic cells in the bone marrow or blood. Two commonly used chemotherapy drugs for MDS are azacitidine and decitabine. They belong to a group of drugs called hypomethylating agents that treat MDS by modifying DNA. Treatment with azacitidine or decitabine can relieve MDS symptoms and potentially delay or prevent the need for blood transfusions or transformation to AML. Side effects may include low blood counts, nausea, vomiting, fatigue and bruising.
Growth Factors
Growth factors are man-made versions of proteins that stimulate bone marrow stem cells to mature and produce blood cells. Epoetin alfa (Epogen) and darbepoetin alfa (Aranesp) are erythropoiesis-stimulating agents that may raise hemoglobin levels and reduce transfusion needs in some MDS patients with low red blood cell counts. Thrombopoietin receptor agonists like eltrombopag (Promacta) and romiplostim (Nplate) can boost platelet levels in people whose MDS is causing low platelet counts. Growth factors are administered by injection under the skin or into a muscle.
Stem Cell Transplantation
A stem cell transplant, also called a bone marrow transplant, is currently the only potentially curative treatment for MDS. In this procedure, the patient receives high-dose chemotherapy and sometimes radiation to eliminate the abnormal bone marrow cells. Healthy stem cells are then infused through an intravenous line to rebuild the blood and immune system. Stem cells can come from the patient (autologous transplant) or a donor (allogeneic transplant). Transplants often have significant side effects and risks, so they are usually considered only for younger patients with high-risk or advanced MDS.
Iron Chelation Therapy
Many MDS patients require regular red blood cell transfusions to treat anemia. But repeated transfusions can cause iron overload and lead to organ damage over time. Iron chelation therapy uses medication like deferasirox (Jadenu), deferiprone (Ferriprox), or deferoxamine (Desferal) to bind iron in the body and help remove it through urine or stool. This helps prevent complications from iron buildup in vital organs such as the heart, liver and pancreas. Deferasirox is taken orally daily, while deferoxamine must be given through slow infusion by a pump. Side effects may include gastrointestinal upset or mild symptoms.
Supportive Care for Myelodysplastic Syndrome Treatment
Along with the main MDS treatments described, supporting general health and quality of life with care measures is an important part of management. These include blood transfusions to address low blood counts, antibiotics for infections, pain medication for discomfort, nutritional supplements, exercise as tolerated, and seeing a therapist to discuss feelings and concerns. Maintaining medical follow-up with regular examinations and blood tests helps doctors monitor disease progression and adjust treatment plans over time. In more advanced cases, palliative care may be recommended to focus on relief from distressing symptoms.
Clinical Trials
Clinical trials are research studies that test new MDS drugs or procedures. They offer access to experimental therapies not otherwise available and help advance future MDS treatment options. Eligibility depends on factors like age, risk group, disease stage, organ function and genetics. Patients interested in clinical trials should discuss feasibility and risks/benefits thoroughly with their doctor. Current trials are exploring cellular therapies using immune cells or stem cells, gene therapy modifying bone marrow cells, immunomodulatory drugs, and targeted therapies attacking specific MDS-related mutations at the molecular level.
No single treatment works best for every MDS patient. Myelodysplastic Syndrome Treatment options depend on factors like disease risk group, symptoms, age, medical history and patient preferences. Through properly managing MDS with a combination of therapies, healthcare providers aim to boost blood counts, decrease transfusion needs, minimize symptoms, delay progression to more advanced disease stages, and potentially cure the condition in eligible candidates. Ongoing research continues developing new therapeutic strategies against MDS.
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