Gissen P, Preece M A, Willshaw H A, McKiernan P J
Liver Unit, Birmingham Children's Hospital, Birmingham, UK.
J Inherit Metab Dis. 2003;26(1):13-6. doi: 10.1023/a:1024011110116.
NTBC has revolutionized the management of tyrosinaemia type I, although animal experiments have shown that long-term administration may produce corneal opacities analogous to those in tyrosinaemia type II. We have assessed the prevalence of ocular side-effects in 11 tyrosinaemia type I patients on NTBC attending the Birmingham Children's Hospital. Despite high plasma tyrosine concentrations in some patients, they did not experience symptoms or signs of ocular toxicity.
NTBC彻底改变了I型酪氨酸血症的治疗方式,尽管动物实验表明长期给药可能会产生类似于II型酪氨酸血症的角膜混浊。我们评估了在伯明翰儿童医院接受NTBC治疗的11例I型酪氨酸血症患者眼部副作用的发生率。尽管一些患者血浆酪氨酸浓度很高,但他们并未出现眼部毒性的症状或体征。