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Aplastic Anemia & MDS: Treatment Options

There is no definitive cure for Bone Marrow Failure Diseases. If the case is not severe, your doctor will be sending you for blood tests regularly to monitor your blood count (CBC). Supportive care for people with more severe cases who can not tolerate or do not want a bone marrow or stem cell transplant will involve blood transfusions, usually of packed red blood cells (PRBC) and platelets (for clotting). Growth factors may be given to help the body create blood cells. In severe aplastic anemia, the bone marrow has not produced enough white blood cells (WBC) to protect against infection, so you will be advised to avoid crowds and people with minor ailments like the common cold. An otherwise harmless infection can land you in the hospital to receive intravenous antibiotic therapy. Increasingly, these antibiotics can be given at home by home care nurses once intravenous access has been established. Too many transfusions can trigger immune system response against transfused platelets and excess iron build up from RBC transfusions can damage organs.

Too many transfusions also decrease the chances that a bone marrow or stem cell transplant will be effective. Medicine is really complex, and each person responds differently to treatment, so have patience with your doctor! They really do care about you and are trying to figure out the best course of care for you. Sometimes directing your care can be like sailing a ship through a narrow, rocky channel in a storm with fog. Hematologists (affectionately known as "hem/onc" for Hematology/Oncology) are the specialists who treat aplastic anemia and MDS.

Immunosuppressive therapy is the next treatment option. Scientists theorize that aplastic anemia is caused by the immune system attacking the bone marrow, so the immune system itself is attacked with a combination of drugs. The hope is that this will enable the bone marrow to start producing blood cells again. The long term side effects of this type of therapy are the risk of developing a secondary problem (MDS) later in life. When treatment gets this complex, it is imperative to insist that your doctor discuss implications of each type of therapy with you openly. If you are still not clear on what your options are and what the effects of a proposed treatment are, you may want to seek a second or even third opinion. This is where your insurance company can help you by referring you to an expert in hematology, usually at a tertiary care center.

BMT (bone marrow transplant) and Stem Cell Transplants are the next treatment options. You will want to work with the medical center’s financial planner and your insurer to work out coverage issues. When I checked last year, a stem cell transplant was running about $300,000. If someone related to you is able to donate the cells or marrow, the costs go down. The National Marrow Donor Program is an excellent resource for planning a transplant and selecting a center. Spend some time exploring their website for a wealth of information on this diagnosis!
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