Prescrire Int. 1999 Jun;8(41):67-70.
(1) Cysteamine bitartrate (also known as mercaptamine) is marketed for the treatment of cystinosis, a rare hereditary disease which, in its juvenile and infantile forms, progresses spontaneously to severe renal and ocular impairment, growth retardation with rickets, and involvement of many other organs. Only symptomatic treatments have been available until now. (2) The clinical file includes only non comparative trials. (3) Few children have been treated with cysteamine beyond the age of 10. Provided treatment is begun early, the main beneficial effects of cysteamine are maintenance of the growth curve and prevention of deterioration of renal lesions (an effect that varies from patient to patient). Oral cysteamine does not prevent the progression of ocular lesions. (4) Cysteamine has many potential adverse effects, some of which are serious (mainly gastrointestinal and neuropsychiatric disorders). Little is known of its long-term adverse effects. (5) The main disadvantage of this treatment is the need for four daily intakes (every 6 hours) and the sulphurous breath it causes. (6) Treatment with cysteamine is expensive.
（1）半胱胺酒石酸盐（也称为巯基乙胺）被用于治疗胱氨酸病，这是一种罕见的遗传性疾病，其青少年型和婴儿型会自发进展为严重的肾脏和眼部损害、伴有佝偻病的生长发育迟缓以及许多其他器官受累。到目前为止，仅有对症治疗方法。（2）临床档案仅包括非对照试验。（3）超过10岁接受半胱胺治疗的儿童很少。如果早期开始治疗，半胱胺的主要有益作用是维持生长曲线以及预防肾脏病变恶化（这种效果因患者而异）。口服半胱胺不能阻止眼部病变的进展。（4）半胱胺有许多潜在的不良反应，其中一些很严重（主要是胃肠道和神经精神障碍）。对其长期不良反应了解甚少。（5）这种治疗的主要缺点是需要每天服用四次（每6小时一次）以及会导致口臭。（6）半胱胺治疗费用昂贵。
