ORAL L GLUTAMINE THERAPY FOR SICKLE CELL ANEMIA

镰状细胞病是一组以异常红细胞存在为特征的遗传性血红蛋白疾病。这种疾病的并发症可能在生命早期就开始出现，如胎儿畸形、脑血管意外、感染和血管闭塞的痛苦危机。目前的治疗（骨髓移植除外）通常仅限于预防感染和处理并发症（缓解疼痛、预防脱水、治疗感染、输血）。对镰状细胞病有效治疗方法的研究总体上令人失望。虽然某些新方法，如基因治疗，很有希望，但它们仍处于发展的早期阶段。骨髓移植在镰状细胞病中的应用需要评估风险/收益问题。虽然使用这种治疗方式有可能治愈，但手术的发病率和死亡率仍然很高。显然，开发利用新的作用机制的新治疗方式是必要的。在非洲人后裔中，镰状细胞病的患者人数相对较多，为了减轻这些患者的痛苦，迫切需要一种药物或治疗方法来预防镰状细胞病的发病率和死亡率。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，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。

The sickle cell diseases are a group of hereditary hemoglobin disorders characterized by presence of abnormal red cells. The complications of this disease may start early in life, such as fetal deformity, brain blood vessel accidents, infection and vaso-oclusion painful crises. Current therapy (with the exception of bone marrow transplantation) is usually limited to the prevention of infections and management of complications (pain relief, prevention of dehydration, treatment of infections, blood transfusions). The search for effective treatments for sickle cell disease has been generally disappointing. While certain new approaches, such as gene therapy, are promising, they are still in early stages of development. The utility of bone marrow transplantation in sickle cell disease has to be assessed for risk/benefit issues. While cure is possible when this treatment modality is used, the morbidity and mortality of the procedure is still very high. Clearly, development of new treatment modalities exploiting novel mechanisms of action is necessary. In order to relieve those who are affected by this disease, which is a relatively large population among people of African descent, an agent or method of therapy is urgently needed to prevent morbidity and mortality of sickle cell disease. 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 this research is to evaluate the efficacy of this amino acid in treatment of sickle cell anemia.