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Hematopoietic stem cell transplantation for people with sickle cell disease.

镰状细胞病患者的造血干细胞移植.

  • 影响因子:6.1030
  • DOI:10.1002/14651858.CD007001.pub5
  • 作者列表:"Oringanje C","Nemecek E","Oniyangi O
  • 发表时间:2020-07-03
Abstract

BACKGROUND: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. This is an update of a previously published review. OBJECTIVES: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. SEARCH METHODS: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. We also searched trial registries for ongoing trials up to April 2020. Date of the most recent search of the Group's Haemoglobinopathies Trials Register: 09 December 2019. SELECTION CRITERIA:Randomized controlled and quasi-randomized trials 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. DATA COLLECTION AND ANALYSIS:No trials were eligible for inclusion in the review. MAIN RESULTS:We identified 12 potentially-eligible trials by the searches; we excluded 11 of these and the remaining trial is an ongoing trial that may be eligible for inclusion in a future version of the 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. We did not find any eligible randomized controlled trials assessing the benefit or risk of hematopoietic stem cell transplantations. However, there is an ongoing quasi-randomized trial comparing hematopoietic stem cell transplantation with standard care, 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.

摘要

背景: 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缺陷。主要的治疗干预措施包括预防和支持措施。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 (即血液形成) 干细胞),以纠正紊乱。这是以前发表的评论的更新。 目的: 确定干细胞移植是否可以改善生存率,预防镰状细胞病相关的症状和并发症。研究干细胞移植对镰状细胞病患者潜在长期获益的风险。 检索方法: 我们检索了Cochrane囊性纤维化和遗传性疾病组的血红蛋白病试验登记册,该试验登记册符合Cochrane中心对照试验登记册 (Central) 的电子检索 (Cochrane Library每期更新) 和MEDLINE的季度检索。我们还检索了截至2020年4月的试验注册中心。该组织血红蛋白病试验注册的最新搜索日期: 2019年12月9日。 选择标准: 比较任何干细胞移植方法的随机对照和半随机试验,或者相互比较,或者与任何预防或支持性干预 (例如g.定期输血,使用羟基脲,抗生素,止痛药,补充氧气) 在镰状细胞病患者中,无论镰状细胞病的类型,性别和设置。 数据收集和分析: 没有试验符合纳入审查的条件。 主要结果: 我们通过检索确定了12项潜在合格的试验; 我们排除了其中的11项,其余的试验是一项正在进行的试验,可能有资格纳入未来版本的综述. 作者的结论: 关于使用造血干细胞移植改善生存率和预防镰状细胞病相关症状和并发症的报道目前仅限于观察性和其他不太有力的研究。我们没有发现任何评估造血干细胞移植获益或风险的合格随机对照试验。然而,有一项正在进行的准随机试验将造血干细胞移植与标准治疗进行比较,因此,本系统综述确定需要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的风险,比较镰状细胞病患者的镰状细胞状态和疾病严重程度.

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翻译标题与摘要 下载文献
血红蛋白病方向

由于血红蛋白分子结构异常(异常血红蛋白病),或珠蛋白肽链合成速率异常(珠蛋白生成障碍性贫血,又称海洋性贫血)所引起的一组遗传性血液病。

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