Klein E, Hahn G M, Solomon J A, Kim U, Schwartz R A, Case R W, Herman H, Milgrom H, Holtermann O A
J Surg Oncol. 1979;11(2):85-8. doi: 10.1002/jso.2930110202.
Successful therapy of a genetic disorder, Ichthyosis Linearis Circumflexa, was achieved using a low dose systemic cyclophosphamide. Prior to such therapy, 80 to 90% of the body surface was affected; the use of antimitotic agents reduced the extent of the lesions to less than 15% of the body surface. As a result, the clinical status was changed from severely disabling to being compatible with a normal way of life. It is believed that this phenomenon is related to selective effects of cyclophosphamide, such as those on lymphocyte subpopulations. To our knowledge, successful chemotherapy for diseases of genetic or congenital origin has not been previously reported.
使用低剂量全身环磷酰胺成功治疗了一种遗传性疾病——回旋线状鱼鳞病。在这种治疗之前,80%至90%的体表受到影响;使用抗有丝分裂剂后,皮损范围缩小至体表的15%以下。结果,临床状况从严重致残转变为与正常生活方式相容。据信,这种现象与环磷酰胺的选择性作用有关,比如对淋巴细胞亚群的作用。据我们所知,此前尚未有关于遗传性或先天性疾病成功化疗的报道。