Oringanje Chioma, Nemecek Eneida, Oniyangi Oluseyi
Institute of Tropical Disease Research and Prevention, University of Calabar Teaching Hospital, Calabar, Nigeria.
Cochrane Database Syst Rev. 2013 May 31(5):CD007001. doi: 10.1002/14651858.CD007001.pub3.
Sickle cell disease is a genetic disorder involving a defect in the red blood cells due to its sickled hemoglobin. The main therapeutic interventions include preventive and supportive measures. Hematopoietic stem cell transplantations are carried out with the aim of replacing the defective cells and their progenitors (hematopoietic (i.e. blood forming) stem cells) in order to correct the disorder.
To determine whether stem cell transplantation can improve survival and prevent symptoms and complications associated with sickle cell disease. To examine the risks of stem cell transplantation against the potential long-term gain for people with sickle cell disease.
We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Group's Haemoglobinopathies Trials Register complied from electronic searches of the Cochrane Central Register of Controlled Trials (CENTRAL) (updated each new issue of The Cochrane Library) and quarterly searches of MEDLINE.Unpublished work was identified by searching the abstract books of major conference proceedings and we conducted a search of the website: www.ClinicalTrials.gov.Date of the most recent search of the Group's Haemoglobinopathies Trials Register: 16 August 2012.
Randomized controlled and quasi-randomized studies that compared any method of stem cell transplantation with either each other or with any of the preventive or supportive interventions (e.g. periodic blood transfusion, use of hydroxyurea, antibiotics, pain relievers, supplemental oxygen) in people with sickle cell disease irrespective of the type of sickle cell disease, gender and setting.
No relevant trials were identified.
Ten trials were identified by the initial search and none for the update. None of these trials were suitable for inclusion in this review.
AUTHORS' CONCLUSIONS: Reports on the use of hematopoietic stem cell transplantation improving survival and preventing symptoms and complications associated with sickle cell disease are currently limited to observational and other less robust studies. No randomized controlled trial assessing the benefit or risk of hematopoietic stem cell transplantations was found. Thus, this systematic review identifies the need for a multicentre randomized controlled trial assessing the benefits and possible risks of hematopoietic stem cell transplantations comparing sickle status and severity of disease in people with sickle cell disease.
镰状细胞病是一种遗传性疾病，由于其镰状血红蛋白导致红细胞出现缺陷。主要治疗干预措施包括预防和支持性措施。进行造血干细胞移植的目的是替换有缺陷的细胞及其祖细胞（造血（即形成血液的）干细胞），以纠正该疾病。
确定干细胞移植是否能提高镰状细胞病患者的生存率，并预防与之相关的症状和并发症。权衡干细胞移植的风险与对镰状细胞病患者潜在的长期益处。
我们检索了Cochrane囊性纤维化和遗传性疾病研究组的血红蛋白病试验注册库，该注册库通过对Cochrane对照试验中央注册库（CENTRAL）（《Cochrane图书馆》每期更新）进行电子检索以及对MEDLINE进行季度检索汇编而成。通过检索主要会议论文集的摘要书籍来识别未发表的研究，并对网站www.ClinicalTrials.gov进行了检索。该研究组血红蛋白病试验注册库的最新检索日期为2012年8月16日。
随机对照和半随机研究，这些研究比较了镰状细胞病患者中任何干细胞移植方法彼此之间，或与任何预防或支持性干预措施（如定期输血、使用羟基脲、抗生素、止痛药、补充氧气）的效果，无论镰状细胞病的类型、性别和研究背景如何。
未识别出相关试验。
初始检索识别出10项试验，更新检索未识别出试验。这些试验均不适合纳入本综述。
目前，关于使用造血干细胞移植提高镰状细胞病患者生存率以及预防相关症状和并发症的报道仅限于观察性研究和其他可靠性较低的研究。未找到评估造血干细胞移植益处或风险的随机对照试验。因此，本系统评价表明需要开展一项多中心随机对照试验，评估造血干细胞移植的益处和可能风险，比较镰状细胞病患者的镰状状态和疾病严重程度。
