L-GLUTAMINE THERAPY FOR SICKLE CELL ANEMIA

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 L-谷氨酰胺是一种氨基酸，用于形成NAD，一种保护红细胞免受氧化损伤的物质。此前的数据显示，镰状细胞性贫血患者可以通过补充氨基酸L-谷氨酰胺受益。因此，本研究的目的是评价该氨基酸治疗镰状细胞性贫血的疗效。这是一项前瞻性、随机、双盲交叉临床试验，旨在考察口服L-谷氨酰胺治疗5岁及以上镰状细胞性贫血患者的疗效。登记的受试者将被随机分为L-谷氨酰胺组或安慰剂组，并被指示口服指定的粉末，为期24周。研究中的药物剂量将逐渐减少，为期5周。然后，将有三周的洗脱期，之后他们将被切换到其他研究药物(L-谷氨酰胺或安慰剂)。将监测临床和心理参数。一名独立的盲人观察员将密切关注以下临床终点；痛苦危象的频率；住院天数；慢性疼痛程度和每日麻醉剂需求量；工作天数和/或缺课天数。不良事件也将受到监测。这些数据将是理解L-谷氨酰胺在治疗镰状细胞性贫血中的作用所必需的。

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. L-glutamine is an amino acid that is utilized in the formation of NAD, a substance that protects red blood cells from oxidative damage. Previous data showed that sickle cell anemia patients may benefit by supplementation with the amino acid, L-glutamine. Therefore, the purpose of our research is to evaluate the efficacy of this amino acid in the treatment of sickle cell anemia. This is a prospective, randomized, double blind crossover clinical trial to examine the effects of oral L-glutamine therapy on sickle cell anemia patients aged 5 years of age and older. The enrolled subjects will be randomized to either L-glutamine or placebo and will be instructed to take that assigned powder orally for 24 weeks. There will be a gradual tapering of the study medication dose for 5 weeks. Then, there will be a three-week washout period after which they will be switched to the other study medication (L-glutamine or placebo). Clinical and psychological parameters will be monitored. An independent blinded observer will follow closely the following clinical endpoints; frequency of painful crises; number of hospitalization days; level of chronic pain and amount of daily requirement for narcotics; number of work days and/or school days missed. Adverse events will also be monitored. The data obtained will be essential in understanding the role of L-glutamine in the therapy of sickle cell anemia.