Van Hengstum M, Steenbergen J, Haanen C
Br J Haematol. 1979 Mar;41(3):323-33. doi: 10.1111/j.1365-2141.1979.tb05865.x.
Twenty-eight unselected patients with histologically proven aplastic anaemia were electively treated with anabolic steroids (75-150 mg orally q.d.) Additional supportive treatment with blood cell components and antibiotics was given if indicated. Response to therapy was defined as favourable if after 3 months of anabolic therapy overt bleeding tendency had disappeared, there was no need for transfusion therapy, a spontaneous increase of haemoglobin had occurred of greater than 3 g/dl above the initial level, and a platelet rise of twofold the initial count (up to at least greater than 30 x 10(9) /L) had occurred. Of 22 patients evaluable for the results of long-term (greater than 3 months) anabolic treatment, six showed a partial response and eleven responded favourably. These 11 are all alive at the end of the study. Five of these patients proved to be anabolic steroid-dependent. The 50% actuarial survival is approximately 4 years after diagnosis, which compares favourably with the best published results from bone marrow transplantation for aplastic anaemia. It is concluded that anabolic therapy in aplastic anaemia should be tried for 2-3 months before the bone marrow transplantation or immunosuppressive therapy is taken into consideration.
28例经组织学证实的再生障碍性贫血患者接受了合成代谢类固醇的选择性治疗（口服75 - 150毫克，每日一次）。如有指征，给予血细胞成分和抗生素的额外支持性治疗。如果在合成代谢治疗3个月后，明显的出血倾向消失，无需输血治疗，血红蛋白自发升高超过初始水平3克/分升以上，血小板计数升至初始计数的两倍（至少大于30×10⁹/L），则将治疗反应定义为良好。在可评估长期（大于3个月）合成代谢治疗结果的22例患者中，6例有部分反应，11例反应良好。在研究结束时，这11例患者均存活。其中5例患者被证明对合成代谢类固醇有依赖性。诊断后50%的精算生存率约为4年，与已发表的再生障碍性贫血骨髓移植最佳结果相比具有优势。得出的结论是，在考虑进行骨髓移植或免疫抑制治疗之前，应尝试对再生障碍性贫血进行2 - 3个月的合成代谢治疗。
