Androgen therapy is suitable for chronic or mild aplastic anemia, and the effective rate is 50%~60%. The mechanism of action is to improve the level of erythropoietin in vivo and directly promote erythropoiesis. Testosterone propionate and stanozolol are commonly used in China. Testosterone propionate 50~ 100mg is injected intramuscularly once a day or every other day. Stanozolol (2~4mg) is taken orally. The course of treatment is not less than 4 months. Some patients may have drug dependence, so maintenance treatment should be carried out after remission to reduce recurrence. The main side effects of androgen therapy are masculinization, liver function damage and water and sodium retention, and the injection has local induration and suppuration.
3. immunosuppressive therapy for aplastic anemia involves immune factors, which is the theoretical basis of immunosuppressive therapy. The commonly used immunosuppressants include antithymocyte globulin (ATG) or antilymphocyte globulin (ALG) and cyclosporine. They are mainly used for acute or severe aplastic anemia, and the effective rate is 50%~70% when applied alone or sequentially. The effect of combined medication is better than that of single medication. One drug is ineffective, and about half of the patients can still be effective after switching to another. ATG or ALG are heterogeneous proteins, with side effects such as allergic reaction and serum sickness. Cyclosporine is harmful to liver and kidney. It is reported abroad that the long-term side effects of immunosuppressive therapy are acquired clonal diseases, such as PNH and MDS, but it is rare in China. Other immunosuppressants used to treat severe aplastic anemia in clinic include high-dose methylprednisone and high-dose gamma globulin. Cyclosporine fails to treat androgen.
Allogeneic bone marrow transplantation is suitable for young patients with acute or severe aplastic anemia (< 40 years old) who have HLA-matched donors. 50%~70% of the patients can survive for a long time after transplantation. The main factors affecting the curative effect of allogeneic bone marrow transplantation are rejection and graft-versus-host disease. The rejection rate of patients with repeated blood transfusion is high, so preoperative blood transfusion should be avoided. Unrelated umbilical cord blood stem cell transplantation has been successfully reported for severe aplastic anemia.
Cytokines Hematopoietic cytokines currently used in clinic include erythropoietin (which is not recommended at present, because the EPO concentration of aplastic anemia patients is increased), granulocyte colony stimulating factor and granulocyte-monocyte colony stimulating factor. The effect of hematopoietic stimulating factor alone in the treatment of severe aplastic anemia is not exact, and the combination with immunosuppressive therapy may improve the curative effect.
Other drugs, including traditional Chinese medicine and some drugs for improving microcirculation (hematopoietic microenvironment), are mostly used to treat chronic aplastic anemia. Although it has been reported frequently in China, its value needs to be further evaluated because of the lack of strict prospective randomized case-control research data.
Generally speaking, aplastic anemia still belongs to the category of refractory hematological diseases. The prognosis of aplastic anemia is different according to its classification. Before effective treatment, the prognosis of severe aplastic anemia is bad, and most patients die within one year. The main causes of death are intracranial hemorrhage and serious infection. With the clinical application of effective therapies such as bone marrow transplantation and immunosuppressive therapy, the prognosis of severe aplastic anemia has been greatly improved. Chronic aplastic anemia progresses slowly, and about 70%~80% of patients can get well after treatment.