酶替代疗法治疗1例严重Fabry肾衰竭的1年随访研究

• 作 者: Tsambaos D. Chroni E. 罗素菊
• 机 构: Department of Dermatol ogy, School of Medicine, University of Patras, PO Box 1413, Rio-Patras 26504,
• 出 处: 世界核心医学期刊文摘(皮肤病学分册)
• 发表日期: 2005年05期
• 关键词: 酶替代疗法 随访研究 临床反应 神经精神症状 耐受性 牛乳 血液透析 肾脏 生活质量 Fabry

We present here the course of clinical response of a 53-yearold haemodialysed Fabry patient who received recombinant human α-galactosidase A at a dose of 1 mg/kg every other week over a period of 1 year. The therapy was well tolerated by the patient, who revealed an impressive favourable cutaneous, gastrointestina l, neurological and psychiatric response and a dramatic improvement in his quali ty of life,but no improvement in cardiac and renal function.

We present here the course of clinical response of a 53-yearold haemodialysed Fabry patient who received recombinant human α-galactosidase A at a dose of 1 mg / kg every other week over a period of 1 year. The therapy was well tolerated by the patient , who revealed an impressive favorarable cutaneous, gastrointestina l, neurological and psychiatric response and a dramatic improvement in his quali ty of life, but no improvement in cardiac and renal function.