Dubois R S, Rodgerson D O, Hambidge K M
Department of Pediatrics, Wayne State University, School of Medicine, Detroit, Michigan.
J Pediatr Gastroenterol Nutr. 1990 Jan;10(1):77-81. doi: 10.1097/00005176-199001000-00015.
Penicillamine is the drug of choice for the treatment of Wilson's disease, whatever the stage of the illness. Toxic manifestations may preclude the use of this life-saving drug in some patients and discontinuation of penicillamine therapy usually leads to death. We report our experience with Trientine in seven patients, aged 13 to 33 years, with Wilson's disease who developed toxic manifestations with penicillamine that required discontinuation of therapy. These include two with nephrosis, one with neutropenia, two with thrombocytopenia, and one each with a SLE-like and a Henoch-Schonlein-like syndrome. The patients were treated for periods from 6 weeks to 16 years with a dose of 0.5 to 2 g/day. Trientine proved to be an effective alternative copper chelating agent in the treatment of Wilson's disease in patients with penicillamine-induced neutropenia, thrombocytopenia, SLE, and nephrosis. No serious untoward side effects were noted.
青霉胺是治疗威尔逊氏病的首选药物，无论疾病处于何种阶段。毒性表现可能使一些患者无法使用这种救命药物，而停用青霉胺治疗通常会导致死亡。我们报告了7例年龄在13至33岁之间的威尔逊氏病患者使用曲恩汀的经验，这些患者在使用青霉胺时出现了毒性表现，需要停药。其中包括2例肾病患者、1例中性粒细胞减少症患者、2例血小板减少症患者，以及各1例出现系统性红斑狼疮样综合征和过敏性紫癜样综合征的患者。患者接受治疗的时间为6周至16年，剂量为每日0.5至2克。曲恩汀被证明是治疗因青霉胺引起的中性粒细胞减少症、血小板减少症、系统性红斑狼疮和肾病的威尔逊氏病患者的一种有效的替代铜螯合剂。未观察到严重的不良副作用。
