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What is the treatment for mydeoplastic syndrome (MDS)?

Q: My 60-year-old aunt is suffering from MDS (mydeoplastic syndrome), which is a blood related disorder. Is there any cure available? She is under treatment but her platelet count keeps on decreasing after few days, due to which she has to undergo platelet transfusion number of times.

A:Myelodysplastic syndrome (MDS) refers to a heterogeneous group of closely related haematologic disorders in which an early blood-forming cell (stem cell) is affected resulting in ineffective blood cell production. The mutated cell predominates in the bone marrow and suppresses healthy stem cells, which form normal blood. This leads to anaemia (red cells), neutropaenia (white cells) and thrombocytopaenia (platelets). What causes the cell injury is unknown but the factors implicated include cytotoxic chemotherapy for some other malignancy, radiation exposure, viral infection, chemical exposure, or genetic predisposition. The peripheral blood and bone marrow show changes in cell count (cytopaenia) and cell morphology. Cytogenetic studies of the bone marrow cells may show mutations in 48-64% cases. The standard treatment is constantly evolving but basically comprises treating the symptoms to improve quality of life. Supportive therapy, including transfusions of the cells that are low (red cells and platelets), and treatment of infections are the mainstay though new drugs like azacitidine have been introduced and trials are going on with promising drugs such as Revimid. These measures are temporary and more long-term measures are necessary to stimulate the patients' bone marrow production of mature blood cells. Bone marrow cell production can be stimulated by using agents called haematopoietic growth factors such as erythropoietin (for anaemia) and granulocyte colony-stimulating factor (for neutropaenia). Disease transition to acute leukaemia may be checked by the use of the drug azacitidine. Bone marrow transplantation is used in patients with poor prognoses or late-stage MDS who are aged 55 years or younger and have an available donor. As most patients are elderly and only a few young MDS patients will have a matched donor, the use of this modality is limited. As patients with MDS have varying clinical features and outcomes, a method has been devised to stratify the patients according to their prognosis. This is called staging and treatment is based on the severity and stage of the disease. This takes into account the cell count (haemoglobin, total leukocyte count and platelet count), the number of blast cells present in the bone marrow, and cytogenetics. Patients with good prognoses include those with single or mild cytopaenias, normal chromosomes or a single chromosomal abnormality and less than 10% blasts in the bone marrow. These patients have mean survival of 18-24 months or longer. Patients with pancytopaenia requiring RBC or platelet transfusions, patients with chromosome 7 or multiple abnormalities, and patients with greater than 10% blasts in the bone marrow have a 6- to 12-month survival rate. You need to discuss with the treating haematologist, as he/she would be best placed to guide and advise you.


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