(RxWiki News) In the United States, there is only one approved aplastic anemia drug today. Doctors pondered whether a potent medication approved in Europe may better treat anemia.
ATGAM was found to improve blood cell counts and improve survival better than Thymoglobulin, the only medication approved in Europe, Japan and Latin America.
"Get regular blood counts to monitor anemia."
Dr. Susan Shurin, acting director of the National Heart, Lung, and Blood Institute, said the study was important because it compared the effectiveness of two drugs that work in a similar manner. She emphasized that doctors and patients need to know what the most effective treatment is for aplastic anemia, which can be life-threatening.
Aplastic anemia is a rare disorder usually seen in children and young adults. The disease destroys bone marrow and lowers the amount of functional blood cells in the body. Often the bone marrow is ruined by the patient's own immune system. Severe cases can be fatal.
Thymoglobulin had been reported to be effective in patients who did not respond, or who relapsed after ATGAM treatment. Both drugs are known as antithymocyte globulins or ATGs, and work primarily by suppressing the immune system.
ATGAM is derived from the blood of horses injected with human blood cells, while Thymoglobulin is derived from the blood of rabbits immunized in a similar fashion. Positive results with relapsed patients and preferred use in kidney transplants suggested that Thymoglobulin might be more effective than ATGAM as a first therapy for aplastic anemia.
Researchers enrolled 120 patients between the ages of 2 and 77. Half were assigned to ATGAM while the remainder took Thymoglobulin.
After six months, 68 percent of patients taking ATGAM had improved blood cell counts, while only 37 percent taking Thymoglobulin improved. Survival also was different three years after treatment began. Of the ATGAM patients, 96 survived, while only 76 percent of the Thymoglobulin patients were still alive.
The findings could have implications for treatment in other countries while ATGAM is not currently available. It may also lead researchers to identify which patients would most benefit from each of the medications, since certain medications may better treat certain patient subgroups.
The research was funded by the National Heart, Lung, and Blood Institute, a part of the National Institutes of Health.
The study will be published in the New England Journal of Medicine.