L-GLUTAMINE THERAPY FOR SICKLE CELL ANEMIA

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目及 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 L-谷氨酰胺是一种用于形成NAD的氨基酸，NAD是一种保护红细胞免受氧化损伤的物质。以前的数据表明，镰状细胞贫血患者可能会受益于补充氨基酸，L-谷氨酰胺。因此，我们研究的目的是评估这种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，旨在检查口服L-谷氨酰胺治疗对5岁及以上镰状细胞贫血患者的影响。入组的受试者将被随机分配至L-谷氨酰胺组或安慰剂组，并将被指导口服分配的粉末24周。研究药物剂量将逐渐减少，持续5周。然后，将有一个为期三周的洗脱期，之后他们将转换为其他研究药物（L-谷氨酰胺或安慰剂）。将监测临床和心理参数。独立设盲观察员将密切跟踪以下临床终点：疼痛危象的频率;住院天数;慢性疼痛水平和每日麻醉剂需求量;缺勤天数和/或缺课天数。还将监测不良事件。所获得的数据将是必不可少的了解L-谷氨酰胺在治疗镰状细胞性贫血的作用。

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.