L-GLUTAMINE THERAPY FOR SICKLE CELL ANEMIA

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。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。先前的数据显示，补充氨基酸l -谷氨酰胺可能对镰状细胞性贫血患者有益。因此，我们研究的目的是评估这种氨基酸在治疗镰状细胞性贫血中的疗效。这是一项前瞻性、随机、双盲交叉临床试验，旨在研究口服l -谷氨酰胺治疗对5岁及以上镰状细胞性贫血患者的影响。被招募的受试者将被随机分配到l -谷氨酰胺组或安慰剂组，并被指示口服指定的粉末24周。研究用药剂量将逐渐减少5周。然后，将有三周的洗脱期，之后他们将切换到另一种研究药物（l -谷氨酰胺或安慰剂）。临床和心理参数将被监测。一名独立的盲法观察者将密切关注以下临床终点；痛苦危机的频率；住院天数；慢性疼痛程度及每日需要量；旷工和/或旷课天数。不良事件也将被监测。获得的数据对于理解l -谷氨酰胺在镰状细胞性贫血治疗中的作用至关重要。

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.