Doctors will sometimes refer to certain treatments as supportive care. The goal of supportive care is to help you manage the symptoms of your disease. These treatments do not treat the underlying cause of the disease.
In general, supportive care includes the following:
- Blood transfusions to raise blood cell counts
- Antibiotics to treat infection
- Iron chelation therapy to treat iron overload, a side effect of getting many red blood cell transfusions
Growth factors may also be used as supportive care. Growth factors are naturally occurring hormones produced your body. They cause your bone marrow to produce more blood cells. Red cell growth factor is most commonly used in bone marrow failure patients. It is called erythropoietin or EPO for short. Man made forms of erythropoietin come in the brand names Epogen and Procrit. Darbapoietin is similar to EPO, and comes in the brand name Aranesp. White cell growth factors and platelet growth factors are also available and used in some patients.
For each drug, doctors have a set of criteria they use to measure a complete response, also called a full response. This criteria differs depending on the specific drug being used and the disease being treated.
For patients with bone marrow failure diseases, a complete response is typically blood counts that improve and remain at or near normal after treatment. For patients being treated for MDS, this might also include no blast cells in the blood and a normal number of blasts cells in the bone marrow.
A partial response is less than a full response and better than no response. It typically includes blood counts that are at least halfway between where they started and normal.
Ask your doctor for the criteria he or she has used to define a “full response” and a “partial response” to the treatment you have been given.
Doctors will sometimes refer to certain drugs as “active treatment” or “active drug therapy.” Active treatments are drugs used to treat the underlying cause of the bone marrow failure. These drugs are typically approved by the U.S. Food and Drug Administration (FDA) to treat a specific disease. It is important to note that active treatments do not cure the disease. The only cure for a bone marrow failure disease is a bone marrow/stem cell transplant.
Watchful waiting is an approach your doctor might suggest if your blood counts are not too low and your symptoms are not too bad. Your doctor will want to closely monitor your condition without giving any treatment, unless symptoms appear or change. This approach may be recommended in early stages of aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH).
The main components of blood are:
- Red blood cells (RBCs), which carry oxygen throughout the body
- White blood cells (WBCs), which fight infection
- Platelets, which protect against easy bleeding by helping your blood to clot
- Plasma, the liquid part of blood that carries blood cells, nutrients, wastes, hormones, and many other substances
Abnormalities in any of these components or in related cells or tissues can cause a blood disorder. The disorder and its symptoms depend on precisely what’s gone wrong.
Some blood disorders—including leukemia, lymphoma, myeloma, myelodysplastic syndrome, and myeloproliferative neoplasms—cause unusually low or high levels of blood cells. In some cases the blood cells don’t form or mature as they should and cannot carry out their normal functions.
- A low level of RBCs is called anemia. A high level is called polycythemia.
- A low level of WBCs is called leukopenia. A high level is called leukocytosis.
- A low level of platelets is called thrombocytopenia. A high level is called thrombocytosis.
Other blood disorders have to do with a deficiency of substances the body needs, such as clotting factors in hemophilia, or with the presence of unwanted substances in the blood, such as abnormal proteins in amyloidosis. There are many other specific types of blood disorders.