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A myelodysplastic syndrome (MDS) is one of a group of cancers in which blood cells in the bone marrow do not mature, and as a result, do not develop into healthy blood cells. [3] Early on, no symptoms typically are seen. [3] Later, symptoms may include fatigue, shortness of breath, bleeding disorders, anemia, or frequent infections. [3]
As there are a number of drawbacks brought by frequent transfusions, directly treating the cause of anemia (e.g. myelodysplastic syndrome), if available, remains the optimal choice of treatment. [3] Hematopoietic stem cell transplantation is a treatment for thalassemia that minimizes the need of transfusion in long term.
Midostaurin, sold under the brand name Rydapt by Novartis, is a multi-targeted protein kinase inhibitor that has been investigated for the treatment of acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and advanced systemic mastocytosis.
Azacitidine is indicated for the treatment of myelodysplastic syndrome, [4] for which it received approval by the U.S. Food and Drug Administration (FDA) on 19 May 2004. [11] [4] [12] In two randomized controlled trials comparing azacitidine to supportive treatment, 16% of subjects with myelodysplastic syndrome who were randomized to receive azacitidine had a complete or partial normalization ...
Lenalidomide, sold under the brand name Revlimid among others, is a medication used to treat multiple myeloma, smoldering myeloma, and myelodysplastic syndromes (MDS). [8] For multiple myeloma, it is a first line treatment, and is given with dexamethasone. [8] It is taken by mouth. [8]
A hypomethylating agent (or demethylating agent [1]) is a drug that inhibits DNA methylation: the modification of DNA nucleotides by addition of a methyl group.Because DNA methylation affects cellular function through successive generations of cells without changing the underlying DNA sequence, treatment with a hypomethylating agent is considered a type of epigenetic therapy.
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