Growth Factors and Similar Medicines for Myelodysplastic Syndromes

Shortages of blood cells (red blood cells, white blood cells, or platelets) cause most of the symptoms in people with myelodysplastic syndromes (MDS). Hematopoietic growth factors can often help bring the blood counts closer to normal.

Hematopoietic growth factors are hormone-like substances that help bone marrow make new blood cells. These substances occur naturally in the body, but scientists have found ways to make large amounts of them in the lab. Patients can get these factors in larger doses than would be made by their own body.

Other medicines that raise blood cell counts in different ways might also be helpful for some people.

Patients usually receive growth factors and similar drugs through subcutaneous (under the skin) injections. Your health care team can give the injections, or you or your family members might be able to learn to give them at home.

Red blood cell growth factors

• Epoetin (Epogen or Procrit) is a manmade version of the growth factor erythropoietin, which promotes red blood cell production. It can help some patients avoid red blood cell transfusions. Giving some patients both epoetin and G-CSF (see "White blood cell growth factors") can improve their response to the epoetin.
• Darbepoetin alfa (Aranesp) is a long-acting form of epoetin. It works in the same way but was designed to be given less often.
• Luspatercept (Reblozyl) isn’t a manmade version of a natural growth factor, but it is another medicine that can help the body make more healthy red blood cells. Known as a red blood cell maturation agent, this drug affects TGF-β proteins in the bone marrow. TGF-β proteins normally help control how quickly new cells in the bone marrow mature into functioning red blood cells, so that there aren’t too many or too few of them in the body. By acting on specific TGF-β proteins, luspatercept helps the bone marrow make more healthy, full grown red blood cells.

White blood cell growth factors

• Granulocyte colony stimulating factor (G-CSF, filgrastim, or Neupogen) and granulocyte macrophage-colony stimulating factor (GM-CSF, sargramostim, or Leukine) can improve white blood cell production. These are not used routinely to prevent infections, but they can help some MDS patients whose main problem is a shortage of white blood cells and who have frequent infections.
• Pegfilgrastim (Neulasta) is a long-acting form of G-CSF. It works in the same way but can be given less often.

Platelet growth factors

• Drugs called thrombopoietin-receptor agonists, such as romiplostim (Nplate) and eltrombopag (Promacta) might help some people with MDS who have very low platelet levels, although this is still being studied.
• A drug called oprelvekin (interleukin-11, IL-11, or Neumega) can be used to raise platelet counts after chemotherapy and in some other diseases. But for most MDS patients, this drug has not been found to be very helpful.

Studies are under way to find the best way to predict which patients will be helped by growth factors and similar drugs, as well as the best way to combine growth factors with each other and with other treatments, such as chemotherapy.