EFFECT OF GLYCEROL TRIERUCATE ON CLINICAL COURSE OF ADRENOLEUKODYSTROPHY

三芥酸甘油酯对肾上腺脑白质营养不良临床病程的影响

• 批准号: 7378767
• 负责人: HUGO W MOSER
• 金额: $ 1.48万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-12-01 至 2006-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7378767
• 关键词: EFFECT; GLYCEROL; TRIERUCATE; CLINICAL; COURSE

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. A published report from our group has provided evidence that Lorenzo's oil (a 4:1 mixture of glyceryl trioleate and glyceryl trierucate), when administered to boys with X-ALD less than 6 years old, were neurologically asymptomatic and had a normal brain MRI, had a reduced risk of developing brain MRI and neurological abnormalities. However, the follow-up period for this study was relatively short and the method of assessing the changes of plasma VLCFA levels was not optimal. Because of these limitations we carried out additional studies of 89 patients with longer follow-up (6.9+/-2.7 years) and used a more sensitive method (length adjusted area under the curve) to assess the alterations in plasma very long chain fatty acid levels.

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。我们小组发表的一份报告提供了证据，证明Lorenzo油（三油酸甘油酯和三芥酸甘油酯的4：1混合物），当给予6岁以下的X-ALD男孩时，神经系统无症状，脑部MRI正常，发生脑部MRI和神经系统异常的风险降低。然而，这项研究的随访时间相对较短，评估血浆VLCFA水平变化的方法并不理想。由于这些局限性，我们对89例患者进行了额外的研究，随访时间更长（6.9+/-2.7年），并使用更敏感的方法（长度调整曲线下面积）来评估血浆极长链脂肪酸水平的变化。