Moser H W
Kennedy Krieger Institute, Baltimore, MD, USA.
J Inherit Metab Dis. 1995;18(4):435-47. doi: 10.1007/BF00710055.
Our laboratory has identified nearly 2000 patients with X-linked adrenoleukodystrophy (ALD) and conducted therapeutic trials in groups of patients who represent the major phenotypes. We report recent results of dietary therapy with a mixture of glyceryl trioleate and glyceryl trierucate oil, also referred to as Lorenzo's Oil, in the asymptomatic and childhood cerebral phenotypes. Fifty-three patients started this therapy at a mean age of 7.5 years at a time when they were free of neurological symptoms. Although analysis of data is hampered by the lack of a concurrent control group, follow-up studies after 39 months of therapy suggest that subsequent neurological involvement was less frequent and less severe than anticipated from historical controls. Retrospective analysis of the effect of the oil in patients with the severe childhood cerebral phenotype indicates that there was a slight but statistically significant slowing of clinical progression and delay of death.
我们实验室已识别出近2000名患有X连锁肾上腺脑白质营养不良（ALD）的患者，并在代表主要表型的患者群体中进行了治疗试验。我们报告了使用甘油三油酸酯和甘油三芥酸酯油混合物（也称为洛伦佐油）对无症状和儿童脑型表型进行饮食治疗的近期结果。53名患者在平均年龄7.5岁且无神经症状时开始这种治疗。尽管由于缺乏同期对照组而妨碍了数据分析，但治疗39个月后的随访研究表明，随后发生神经受累的频率和严重程度低于历史对照的预期。对严重儿童脑型表型患者使用该油的效果进行回顾性分析表明，临床进展略有但在统计学上有显著减缓，且死亡延迟。
