COMPASSIONATE USE OF AN INTRAVENOUS FAT EMULSION COMPRISED OF FSIH OIL IN THE TR

在 TR 中善意地使用由 FSIH 油组成的静脉脂肪乳剂

• 批准号: 8166719
• 负责人: STEVEN A ABRAMS
• 金额: $ 1.26万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-12-01 至 2010-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8166719
• 关键词: Alkaline Phosphatase; Amino Acids; Bilirubin; Biochemical; Calories; Childhood; Cholestasis; Cirrhosis; Computer Retrieval of Information on Scientific Projects Database; Critical Illness; Development; Diet; Dose; Electrolytes; Emulsions; Enteral; Essential Fatty Acids; Fibrosis; Funding; Glucose; Grant; Hepatic; Histologic; Human; Infant; Infusion procedures; Injury; Institution; Intake; Intervention; Intestines; Intravenous; Intravenous Fat Emulsions; Length; Lipids; Lipoidosis; Low Birth Weight Infant; Macronutrients Nutrition; Malnutrition; Micronutrients; Monitor; Nutrient; Nutritional; Oils; Operative Surgical Procedures; Parenteral Nutrition; Patients; Population; Proteins; Protocols documentation; Regimen; Research; Research Personnel; Resources; Risk Factors; Safety; Secondary to; Sepsis; Serum; Source; Starvation; Steatohepatitis; Supplementation; Trace Elements; Transaminases; United States National Institutes of Health; Vitamins; Weaning; alternative treatment; base; gastrointestinal; premature

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. We propose to provide an omega-6 predominant lipid infusion (Omegaven) to patients with severe parenteral nutrition associated cholestasis (conjugated bilirubin > 6) on a compassionate use basis. We will monitor for safety parameters during the maximum 5 months of intervention. The Omegaven will be intravenously infused at a dose of 1gm/kg/day until the infant is weaned from parenteral nutrition and for no longer than 5 months. This is a continuation of protocol H-21344. To provide a mechanism for critically ill infants with parenteral nutrition (PN) associated cholestasis to receive Omegaven for compassionate use situations for which there are no satisfactory alternative treatments. Parenteral nutrition (PN) provides intravenous nutritional supplementation for patients unable to absorb adequate enteral nutrients secondary to insufficient intestinal length or function. PN contains the macronutrient building blocks of the human diet in their most elemental forms (amino acids and dextrose) and is commonly administered with a lipid emulsion to avoid essential fatty acid deficiency and to provide a calorically dense source of non-protein calories. In addition, PN contains the essential micronutrients (electrolytes, trace elements, and vitamins) to provide an optimal nutritional regimen. Before the development of PN in the late 1960 s, patients with insufficient gastrointestinal absorptive function commonly died of starvation and subsequent complications of malnutrition (1, 2). Today, more than 30,000 patients are permanently dependent on parenteral nutrition for survival. However, PN continues to be associated with hepatic injury that occurs at an unpredictable rate and includes both biochemical, i.e., elevated serum aminotransferase and alkaline phosphatase, and histologic alterations such as steatosis, steatohepatitis, lipidosis, cholestasis, fibrosis, and cirrhosis (3, 4). These abnormalities, which may worsen with the duration of PN administration, is more prevalent in the pediatric population. Additional risk factors for this condition include prematurity, low birth weight, long-term use of PN, the lack of concomitant enteral intake, sepsis, and multiple operative procedures (5).

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。子项目和 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以在其他 CRISP 条目中表示。列出的机构是 对于中心来说，它不一定是研究者的机构。 我们建议在同情使用的基础上，为患有严重肠外营养相关胆汁淤积（结合胆红素> 6）的患者提供以omega-6为主的脂质输注（Omegaven）。我们将在最长 5 个月的干预期间监测安全参数。 Omegaven 将以 1 克/公斤/天的剂量静脉输注，直至婴儿断奶，且持续时间不超过 5 个月。这是协议 H-21344 的延续。 为患有肠外营养（PN）相关胆汁淤积的危重婴儿提供一种机制，在没有令人满意的替代治疗的情况下，在同情使用情况下接受 Omegaven。 肠外营养（PN）为因肠道长度或功能不足而无法吸收足够肠内营养的患者提供静脉营养补充。 PN 含有人类饮食中最基本形式的大量营养素（氨基酸和葡萄糖），通常与脂肪乳剂一起服用，以避免必需脂肪酸缺乏，并提供热量密集的非蛋白质热量来源。此外，PN 还含有必需的微量营养素（电解质、微量元素和维生素），可提供最佳的营养方案。在 20 世纪 60 年代末出现 PN 之前，胃肠道吸收功能不足的患者通常死于饥饿和随后的营养不良并发症 (1, 2)。如今，超过 30,000 名患者永久依赖肠外营养生存。然而，PN 仍然与肝损伤相关，肝损伤的发生率不可预测，包括生化（即血清转氨酶和碱性磷酸酶升高）和组织学改变，如脂肪变性、脂肪性肝炎、脂质沉积、胆汁淤积、纤维化和肝硬化 (3, 4)。这些异常情况可能会随着 PN 给药时间的延长而恶化，在儿科人群中更为普遍。这种情况的其他危险因素包括早产、低出生体重、长期使用 PN、缺乏伴随的肠内摄入、败血症和多次手术程序 (5)。