Therapies

There are many therapies and approaches doctors use to treat bone marrow failure disease patients. Some treatments are used for several different diseases. Others are used only for aplastic anemia, MDS or PNH. Every person's condition is unique, and each situation is different. That's why your health care team must look carefully at your specific case before recommending what's right for you.

Androgens

Androgens are natural male hormones that can cause your bone marrow to make more red blood cells. This can improve anemia. Androgens are sometimes used to treat aplastic anemia and PNH.

Blood Clot Therapy

Blood clots are very dangerous. If you have a blood clot, you need to get help right away. Patients with PNH are at an increased risk of getting serious blood clots. Here are some ways blood clots are treated.

Blood Transfusions

A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia, MDS or PNH will receive at least one blood transfusion. When you receive a blood transfusion, parts of blood from a donor are put into your bloodstream. This can help some patients with low blood counts.

Chemotherapy

If you have MDS and have a high-risk or intermediate-2 risk IPSS score, you have a higher risk of developing Acute Myelogenous Leukemia (AML). To prevent this from happening, your doctor may prescribe intensive chemotherapy in order to kill bone marrow cells that have an abnormal size, shape, or look.

Eculizumab

Eculizumab (Soliris ®) is the only drug approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Evaluation Agency (EMEA) to treat PNH. It works by making your complement system less active.

Growth Factors

Growth factors are naturally occurring hormones in your body that signal your bone marrow to make more of certain types of blood cells. Man-made growth factors may be given to some people with bone marrow failure diseases to help increase red blood cell, white blood cell or platelet counts.

Immunosuppressive Therapy

Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts.

Iron Chelation

Iron chelation therapy is the main treatment used when you have a condition called iron overload. Iron overload means you have too much iron in your body. This can be a problem for people who get lots of red blood cell transfusions.

MDS Drug Therapy

There are three medicines approved in the U.S. to treat MDS. Azacitidine (Vidaza®) and decitabine (Dacogen®) are approved to treat both low- and high-risk patients with all sub-types of MDS. Lenalidomide (Revlimid®) is approved for transfusion-dependent MDS patients with isolated del(5q) and with a low or intermediate-1 risk IPSS score.

Prednisone

Prednisone a man-made version of a hormone produced naturally by the adrenal gland that can help reduce inflammation and stop allergic responses. It is prescribed when the body is not producing enough of this chemical on its own.