Aplastic anemia is a form of bone marrow failure. Marrow, the soft, fatty tissue inside bones, is the place where new blood cells are formed. In aplastic anemia, the bone marrow does not produce new cells, leaving the body susceptible to bleeding and infection. Researchers have discovered that most cases of severe aplastic anemia happen when the body’s immune system attacks its own tissues and organs. Although aplastic anemia is not a cancer, its treatment can be similar to therapies used to treat leukemias and lymphomas.
Experts at The Johns Hopkins Kimmel Cancer are nationally and internationally recognized as leaders in the research and treatment of aplastic anemia and other blood disorders.
The decision to treat patients with aplastic anemia is based on disease severity. Patients are seen in a multidisciplinary clinic at the Center for Bone Marrow Failures, where patients with difficult cases are discussed by a group of experts who work together...
Find information about open aplastic anemia studies offered at the Johns Hopkins Kimmel Cancer Center.
Bone Marrow Transplant
A bone marrow transplant gives a patient healthy stem cells – immature cells that grow into different types of blood cells. These stem cells come from marrow, the soft, fatty tissue inside the bones, replacing marrow that is not working properly. Aplastic anemia was one of the first diseases for which bone marrow transplantation was found to be effective.
In this therapy, the patient's non-functioning bone marrow is destroyed with drugs and/or radiation and replaced with donor bone marrow from a compatible donor, usually a sibling or other family member. Bone marrow transplants from unrelated donors or donors that do not match a patient’s bone marrow characteristics are usually saved for severe aplastic anemia or very severe aplastic anemia patients who have not responded to drug therapy first.
When successful, bone marrow transplant is often a cure for aplastic anemia with few incidences of relapse. However, it is a very intensive therapy, and serious immune complications and infections are not unusual. Other non-life-threatening effects of bone marrow transplantation include sterility and temporary hair loss. Patients under age 40 are the best candidates. Cure rates decline significantly in those over 40. (Learn more about the Johns Hopkins Bone Marrow Transplant Program.)
Transfusion delivers new blood, including red blood cells and platelets, to a patient through a narrow tube placed in a blood vessel. Transfusions can quickly stabilize aplastic anemia patients with severe blood cell deficiencies, but it is very rare for a patient to achieve long-term recovery using this form of treatment alone.
In some patients, bone marrow transplants may not be an option. Therapy with drugs that suppress the immune system, such as anti-thymocyte globulin (ATG) and cyclosporine may be recommended instead. Another drug for severe aplastic anemia, called eltrombopag, has only a 20% response rate and even in those who respond may relapse.
While drug therapy is not a cure for aplastic anemia, it may partially restore blood cell production. However, up to 50% of patients relapse or develop other blood-forming malignancies or disorders.
A central venous catheter – a tube that carries medicines to a vein in the chest – may be helpful for aplastic anemia patients who require frequent transfusions or infused medications. Patients with deficiencies in certain blood elements may need infused blood products, such as packed red blood cells to correct or avoid heart and lung complications. Platelet infusions may be needed to prevent spontaneous bleeding. Because fungal and bacterial infections are a major cause of death in patients with severe aplastic anemia, patients may require therapy with antibiotics, or with antiviral or antifungal drugs.
Clinical trials test new drugs and new ways of treating patients with aplastic anemia. Our experts conduct these studies to find better treatments. Many clinical trials become the next standard of care. To determine if a trial is right for you, make an appointment for a consultation at 410.955.8964.