Growth Factors for Myelodysplastic Syndrome
Hematopoietic growth factors are hormone-like substances that stimulate bone marrow to produce blood cells. These substances occur naturally in the body, but scientists have found a way to make them outside of the body in large amounts. This allows patients to get these factors in larger doses than would be produced by their own body.
Shortages of blood cells cause most of the symptoms in people with myelodysplastic syndromes (MDS), and growth factors can help the blood counts to become more normal. The growth factors granulocyte colony stimulating factor ( G-CSF, Neupogen®, or filgrastim) and granulocyte macrophage-colony stimulating factor (GM-CSF, Leukine®, or sargramostim) can improve white blood cell production. These can benefit some MDS patients whose main problem is a shortage of white blood cells and who suffer from frequent infections. Pegfilgrastim (Neulasta®) is a long-acting form of G-CSF. It works in the same way but can be given less often.
Erythropoietin (Epo® or Procrit®), a growth factor that promotes red blood cell production, can help avoid red blood cell transfusions in some patients. Giving some patients both erythropoietin and G-CSF improves their response to the erythropoietin. Darbepoetin alfa (Aranesp®) is a long-acting form of erythropoietin. It works in the same way but was designed to be given less often.
A drug called oprelvekin (Neumega®, interleukin-11, or IL-11) can be used to stimulate platelet production after chemotherapy and in some other diseases. This drug can help increase the platelet counts of some MDS patients for a time, but then the counts go back down again. For most MDS patients, this drug is not very helpful.
More studies are under way to find the best way to predict which patients will benefit from growth factors and the best way to combine growth factors with each other and with other treatments, such as chemotherapy or hormones. Patients usually receive the growth factors through subcutaneous (under the skin) injections. Your health care team can give the injections, or you or your family members can learn to give them.
Androgens, or male hormones, can boost blood cell production that is abnormally low due to certain diseases. A few people with MDS may be helped by androgens but most do not improve. If no other treatment options are appropriate for a patient, some doctors recommend trying androgens. However, these hormones can cause side effects, such as liver problems or muscle cramps. In women, androgens can produce male features such as growth of facial and body hair and can increase the sex drive.
Last Medical Review: February 10, 2014 Last Revised: July 2, 2015