Corden B J, Schulman J D, Schneider J A, Thoene J G
Dev Pharmacol Ther. 1981;3(1):25-30. doi: 10.1159/000457418.
Cysteamine (2-aminoethanethiol) has been given orally to 19 patients with nephropathic cystinosis for periods of 8-24 months in doses ranging from 50 to 70 mg base/kg/day. Adverse reactions were noted in 3 patients early in the study when a rapidly increasing dosage schedule was followed. The reactions included hyperthermia, lethargy and rash. These reactions were not seen when patients were started on a very low dosage which was increased gradually at 3-week intervals to a level which depleted leukocytes of about 90% of their free cystine. All three reactions resolved within 24 h or cessation of therapy and in these cases successful readministration of drug was achieved. Chronic cysteamine administration to pediatric patients with cystinosis is feasible. The efficacy of this therapy is still being evaluated.
已对19例肾病型胱氨酸病患者口服半胱胺（2-氨基乙硫醇），疗程为8至24个月，剂量范围为50至70毫克碱基/千克/天。在研究早期，当采用快速增加剂量方案时，3例患者出现了不良反应。这些反应包括高热、嗜睡和皮疹。当患者从非常低的剂量开始，每隔3周逐渐增加到使白细胞游离胱氨酸消耗约90%的水平时，未出现这些反应。所有这三种反应在停药后24小时内消退，并且在这些病例中成功地重新给药。对患有胱氨酸病的儿科患者长期给予半胱胺是可行的。这种治疗方法的疗效仍在评估中。
