Chabannon C, Molina L, Pégourié-Bandelier B, Bost M, Léger J, Hollard D
Service d'Hématologie Clinique, Centre Hospitalier, Grenoble, France.
Cancer. 1994 Jun 15;73(12):3073-80. doi: 10.1002/1097-0142(19940615)73:12<3073::aid-cncr2820731228>3.0.co;2-#.
Treatment of myelodysplastic syndromes (MDSs) remains unsatisfactory. A limited number of reports provide contradictory information on whether danazol, a synthetic androgen, may be useful in patients with MDS.
Between 1984 and 1992, 76 patients were treated with danazol (Danatrol, Winthrop) in an open nonrandomized study. Clinical status, blood counts, differential marrow cell counts, transfusion requirements, and liver enzymes were monitored at 3-month intervals during and 6 months after discontinuation of therapy. The authors present the retrospective analysis of this cohort of patients.
This study shows a limited usefulness of danazol in patients with MDS. Blood counts were not significantly changed during danazol administration; neither were transfusion requirements. Overall survival and the rate of leukemic transformation were not affected when compared with 50 untreated patients or with data available in the literature. An increase in platelet numbers in four patients and in hemoglobin level in one patient was observed; these patients were unremarkable and were not transfused before initial danazol therapy; therefore, changes in blood counts were of limited clinical significance. The search for subgroups of patients likely to have a favorable response was unsuccessful. Side effects of danazol were limited. In addition, this study provides information on the frequency of antiplatelet antibodies and other autoantibodies in patients with MDS.
This study does not support a positive effect of danazol in patients with MDS during long term follow-up. Anecdotal positive evolution in a few patients cannot be formally ascribed to danazol. These results should be interpreted by comparison with previous publications that report on small numbers of patients with limited follow-up. Based on these data, the authors cannot recommend the systematic use of danazol in MDS.
骨髓增生异常综合征（MDS）的治疗效果仍不尽人意。关于合成雄激素达那唑对MDS患者是否有用，仅有少数报告提供了相互矛盾的信息。
在1984年至1992年间，一项开放性非随机研究中，76例患者接受了达那唑（Danatrol，Winthrop公司生产）治疗。在治疗期间每3个月以及停药后6个月监测患者的临床状况、血细胞计数、骨髓细胞分类计数、输血需求和肝酶。作者对该队列患者进行了回顾性分析。
本研究表明达那唑对MDS患者的作用有限。服用达那唑期间血细胞计数无显著变化；输血需求也未改变。与50例未治疗患者或文献中的数据相比，总生存率和白血病转化率均未受影响。观察到4例患者血小板数量增加，1例患者血红蛋白水平升高；这些患者情况不显著，在初始达那唑治疗前未接受输血；因此，血细胞计数的变化临床意义有限。寻找可能有良好反应的患者亚组未成功。达那唑的副作用有限。此外，本研究提供了MDS患者抗血小板抗体和其他自身抗体频率的信息。
本研究不支持达那唑在MDS患者长期随访中有积极作用。少数患者的轶事性积极进展不能正式归因于达那唑。这些结果应与之前报告少量患者且随访有限的出版物进行比较来解释。基于这些数据，作者不推荐在MDS中系统性使用达那唑。
