The following list of treatments are the most common. Some treatments have been used alone, but more frequently are used in varying combinations. Which treatment is chosen is dependent upon the severity of the patient's AA, the overall condition of the patient, whether a bone marrow match is available, etc. All are basically immunosuppressant therapies since AA is believed to be a result of the immune system going out of control and attacking the body's own bone marrow. Namely, the lymphocytes (a portion of the white blood cells) start destroying the stem cells even if they are good, thus the lymphocytes are targeted to be suppressed by medication.
Anti-thymocyte globulin (ATG) - A serum derived from horses or rabbits. The horse version, Atgam, was developed before the rabbit serum, Thymoglobulin. It is administered via IV, usually over a specific number of days while the patient is in the hospital. Close supervision is required during administration. According to Eric Lax author of Life and Death on 10 West, ATG was first introduced in the 1970's and used to treat aplastic anemia patients by ULCA doctors.
Anti-lymphocyte globulin (ALG) - This term appears to be used interchangeably with ATG. ALG may be a more general name than ATG. ATG and ALG both involves suppressing the lymphocytes. However, the term ATG is more specific in that it refers more specificly to the T-cells. The lymphocytes are classified as B-lymphocytes or T-lymphocytes. The T-lymphocytes are believed to circulate through the thymus, thus it is labelled "T" cell for thymus.
Cyclosporin - Derived from a fungus. Administered orally in either capsule form or liquid form. Usually used in combination with either ATG or ALG to increase the immunosuppression action. Occasionally it is administered alone without ATG or ALG, but not frequently. Cyclosporin is also used with bone marrow transplant recipients.
Mycophenolate Mofetil (MMF) - Also known as Cellcept. Immunosuppressant administered orally. Has been used in combination with Cyclosporin, however, is milder or is considered less toxic than Cyclosporin. The hope is that it can be used in place of Cyclosporin for less side effects.
Prednisone - Steroid. Used to counteract graft vs. host disease in bone marrow transplant recepients and/or to boost blood counts.
Neupogin - Drug administered via IV or intramuscular injection for the purpose of boosting the white count. (Growth factor)
Bone Marrow Transplant (BMT) - The replacement of the patient's bone marrow with the bone marrow of a matched donor. This is a very complicated process which will most likely include some of the aboved mentioned drugs and more. Radiation and/or chemotherapy are used to condition a patient for a bone marrow transplant by wiping out the patient's bone marrow before transpant is performed. The first successful bone marrow transplants were performed in the late 1960's by teams headed by Drs. George Mathe and E. Donnall Thomas. UCLA's first transplant was done in 1973. For anyone considering undergoing a bone marrow transplant, I recommend reading Life and Death on 10 West by Eric Lax, 100 Days, My Unexpected Journey from Doctor to Patient by Dr. David Biro, Survivor, Taking Control of Your Fight Against Cancer, by Laura Landro, and Time on Fire, by Evan Handler.
Cyclophosphamide (Cytoxan) - An alkylating agent created from mustaard gas. It is generally known as Cytoxan or Neosar in the United States and Endoxan in Germany. Cyclophosphamide was introduced clinically in 1958 and soon became the most widely used alkylating agent in the world. It is often used in high doses in leukemia, but can cause blood deficiencies, hair loss, nausea, and vomiting. It can also cause bleeding in the urinary tract, which may require medical intervention. Patients taking this drug must drink plenty of water and urinate frequently. Other side effects include mouth sores, discoloration of the nails and skin, lung disease and heart damage.
(All alkylating agents should
be regarded as carcinogenic. Leukemias and other secondary cancers
are fairly common among survivors. Up to ten percent of patients
who survive ovarian cancer succumb to second cancers caused by these drugs.
Alkylating agents can also cause serious lung injury and have been known
to cause mutations and fetal malformations in experimental animals.)
These are the basic treatments,
however, for those willing to experiment with other drugs on your already
compromised immune system, there are clinical trials available too.
Various antibiotics are generally used throughout periods when the absolute
neutrophil count is low. For some patients, the above treatments
do not immediately eliminate the need for supportive blood transfusions,
thus the patient usually starts chelation to remove excess iron accumulated
from the blood transfusions. Desferal is the chelation agent used
for iron overload in the U.S. and can be administered in various ways--IV,
subcutaneous injection, or intramuscular injections. In some other
countries such as Germany, an oral chelation agent is currently available.
Menstruating females are usually prescribed birth control pills or hormone
pills of some sort to prevent menstruation.
Another avenue that is persued, but not frequently used alone, is supportive care. Supportive care is the maintaining of the patient while he/she is waiting for results of their chosen treatments. Frequently, AA patients need blood product transfusions to maintain life. Red blood cells and platelets are transfused until the patient's body starts producing enough of its own blood for the patient's survival.