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Is Myelodysplastic Syndrome A Fatal Disease?

Myelodysplastic syndrome is a group of hematologic disorder that arises from dysplasia in myeloid hematopoietic stem cell lineage in the bone marrow. It is mostly a disease of the elderly with a median age of around 70 years and only 10% patients are found to be below the age of 50 years. The dysplasia of myeloid hematopoietic cell lineage causes anomaly in differentiation and maturation of cells, which leads to reduced number of peripheral blood cells (red blood cells, granulocytes, platelets). The cytopenia causes symptoms of anemia, neutropenia and/or thrombocytopenia. Myelodysplastic syndrome, not only leads to cytopenias, but also transforms to acute myeloid leukemia (AML) in 20-25% of the patients. Secondary acute myeloid leukemia has a poorer prognosis than de novo acute myeloid leukemia.

Is Myelodysplastic Syndrome A Fatal Disease?

The clinical course of the disease is highly variable and has a median survival of several years to few months (8.8-0.8 years) depending on the stage and severity of the disease. The median survival time is only about 30 months from the time of diagnosis. The cure rate is 30-50% only. The complications associated with the disease include bleeding, mucositis, infections and graft versus host diseases. Therefore, myelodysplastic syndrome is a highly fatal disease in high risk group patients. This mandates the timely diagnosis and treatment of the individuals. For low risk group patients, the survival is comparable to their age-matched healthy counterparts.

The diagnosis of myelodysplastic syndrome is made on the basis of blood tests, which may reveal anemia, neutropenia or thrombocytopenia. It is important to rule out the common causes of anemia, such as folic acid or vitamin B12 deficiency, iron deficiency and hemolysis. Patients with a history of prior chemotherapy or radiation therapy have an increased likelihood (around 10%) of developing myelodysplastic syndrome. It is important to do careful cytomorphological blood evaluation along with bone marrow analysis to reach a definitive diagnosis. This should be done by an experienced hematologist or pathologist.(1)

Treatment Of Myelodysplastic Syndrome

The treatment of myelodysplastic syndrome depends on the severity of the disease, whether the patient is low risk patient or high risk patient. Supportive care is the gold standard for low risk patients with a goal to maintain the quality of life for these patients. The goal for high risk patients is to prolong the life expectancy, in addition to restoring the quality of life. Since patients suffer from anemia, thrombocytopenia and neutropenia, the mainstay of treatment is the transfusion of red blood cells (Hb <8-9 g/dl), platelets (when platelet count is in single figures) and to provide antibiotic therapy in cases of infection.

In low risk patients with RBC transfusion of more than 20-25 cycles can lead to iron overload in the body; therefore, it is important to treat iron overload with iron chelation therapy. Deferoxamine and deferasirox are two chelating agents used for this purpose. Erythropoietins are also used in low risk patients who have erythropoietin concentration <500 U/l, in addition to lenalidomide in low to intermediate risk patients who are positive for del(5q) to free their requirement of transfusion.

Allogenic stem cell transplantation is the only therapeutic cure for patients with high risk myelodysplastic disorder. However, the treatment is only befitting for patients <70 years without any other comorbidities or in special cases patients <70 years. Therefore, the role of transplantation in patients with myelodysplastic syndrome is limited as most of the patients are above 70 years of age. In low to intermediate risk patients, transplantation is done only when signs of disease progression are noted. However, high risk patients benefit from early transplantation. Nevertheless, there are increased relapse rates of around 10-40%, in addition to high mortality rate with hematopoietic stem cell transplantation. High risk patients are given cytotoxic chemotherapy in the form of immunosuppression, and demethylating agents (azacitidine, decitabine).(1)(2)

It is also important to provide antibiotic therapy for patients with infections and avoid prophylactic antibiotics; however, in selected cases, prophylactic antibiotic therapy is necessary.


  1. Germing U, Kobbe G, Haas R, Gattermann N. Myelodysplastic syndromes: diagnosis, prognosis, and treatment. Dtsch Arztebl Int. 2013;110(46):783–790. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3855821/
  2. https://emedicine.medscape.com/article/207347-guidelines#g4

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Sheetal DeCaria, M.D.
Sheetal DeCaria, M.D.
Written, Edited or Reviewed By: Sheetal DeCaria, M.D. This article does not provide medical advice. See disclaimer
Last Modified On:September 22, 2021

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