Aplastic Anemia Drugs and Treatments | Aplastic Anemia and MDS International Foundation (AAMDSIF) Return to top.

Aplastic Anemia Drugs and Treatments

More drugs and treatments are available to treat bone marrow failure disease than ever before. Researchers constantly look for ways to enhance drugs already on the market and develop new ones with fewer side effects. Some drugs treat only one type of the disease, while others may be used to treat more than one type. Check with your doctor to find out which drugs are right for you and why.


Brand name: Campath
A monocolonal antibody - a medicine that is engineered to look for a specific substance in the body. Alemtuzumab attaches to and kills white blood cells called lymphocytes. In certain types of aplastic anemia, lymphocytes are responsible for attacking the bone marrow stem cells. Alemtuzumab is in clinical trials for treating aplastic anemia. It is approved by the U.S. Food and Drug Administration (FDA) for treating certain types of leukemia and is helpful in treating other conditions that require immunosuppressive therapy.


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.


The most common white blood cells are called neutrophils. They fight infection. Patients who don't have enough healthy neutrophils are said to have neutropenia. Because of their low white count, they may get infections easily and have trouble getting rid of infections.

Antithymocyte Globulin

Brand name: ATG, Atgam, Thymoglobulin
ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH.


A biosimilar is a type of biologic medication. The term “biosimilar” means that the medication is highly similar to the already approved brand name biologic (“originator” medication). Biosimilars are not generic versions of the original biologic.

Blood Clot Treatment

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.

Bone Marrow Transplant

A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers some patients the possibility of a cure. But for many patients, a BMT is not an option due to the risks and potential long-term side effects as an "imperfect cure".

Clinical Trials

Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study.


Brand name: Cytoxan
Cyclophosphamide is in a class of medications called alkylating agents. When used to treat cancer, it works by slowing or stopping the growth of cancer cells in your body. When cyclophosphamide is used to treat bone marrow failure, it works by suppressing your body's immune system.