High-Dose Cyclophosphamide To Treat Severe Aplastic Anemia. Ann Intern Med. 2001;135:S42. doi: 10.7326/0003-4819-135-7-200110020-00002
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Published: Ann Intern Med. 2001;135(7):S42.
Aplastic anemia is a disease of the bone marrow. Bone marrow is the material in the center of bones that produces blood cells. Stem cells are the cells in the bone marrow that become red blood cells (cells that carry oxygen), white blood cells (cells that fight infection), and platelets (cells that help blood to clot). In aplastic anemia, the bone marrow does not produce stem cells, resulting in abnormally low numbers of all blood cells. Aplastic anemia is difficult to treat and can be fatal. The best hope for curing the disease has been treatment with the drug cyclophosphamide to destroy the abnormal bone marrow, followed by replacement with normal bone marrow (bone marrow transplantation). Unfortunately, many patients do not have appropriate bone marrow donors, and bone marrow transplantation has many serious complications. An earlier report suggested that some people with aplastic anemia can be free of the disease for long periods after treatment with only high-dose cyclophosphamide and do not require bone marrow transplantation.
To further study the safety and results of high-dose cyclophosphamide without bone marrow transplantation in the treatment of severe aplastic anemia.
The researchers studied 19 patients with severe aplastic anemia at three hospitals.
The researchers treated the patients with high-dose cyclophosphamide alone and then contacted them regularly to see how they did. Patients with complete remission of aplastic anemia had normal blood counts for their age. Patients with partial remission did not need transfusions, and their white blood counts were out of the danger zone for infection.
On average, it took 49 days for the number of white blood cells to rise above the danger zone for infection. Two years after treatment, 73% of the patients no longer needed transfusions. At 50 months, 65% were in complete remission. Five years after treatment, no patient with complete remission had had relapse or developed another bone marrow disorder.
The study did not include a comparison group of patients who were treated with bone marrow transplantation in addition to cyclophosphamide. The study excluded patients who were older than 70 years of age and those who had abnormal kidney function, heart failure, infection, or another cancer. Thus, the results may not apply to these types of patients.
High-dose cyclophosphamide without bone marrow transplantation may be a useful treatment option for patients with aplastic anemia who cannot have bone marrow transplantation. This treatment option deserves further study.
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Hematology/Oncology, Red Cell Disorders.
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