EFFECT OF GLYCEROL TRIERUCATE ON CLINICAL COURSE OF ADRENOLEUKODYSTROPHY

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。我们小组发表的一份报告提供了证据，证明Lorenzo's oil（三油酸甘油酯和三酸甘油酯的4:1混合物）用于6岁以下的X-ALD男孩，神经系统无症状，脑MRI正常，患脑MRI和神经系统异常的风险降低。然而，本研究随访时间较短，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，随访时间较长（6.9+/-2.7年），并使用更灵敏的方法（曲线下长度调整面积）来评估血浆超长链脂肪酸水平的变化。

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.