Abstract：
UNASSIGNED: Allogeneic hematopoietic stem cell transplant (HSCT) and gene therapy (GT) are two potentially curative approaches for sickle cell disease (SCD), but they have never been compared in clinical trials.
UNASSIGNED: To compare the safety and efficacy of HSCT and GT to assist clinicians and patients in making informed treatment decisions.
UNASSIGNED: Phase I-III clinical trials and case reports/series were included. Regimens included HSCT from all stem cell sources, lentiviral gene therapy, and gene editing, with any conditioning regimen. We searched Medline and EMBASE databases as of 1st June 2020 for studies reporting HSCT and GT outcomes in SCD. The Newcastle-Ottawa scale was used to assess the risk of bias. Descriptive statistics and post-hoc imputation for standard deviations of mean change in FEV1 and FVC were performed.
UNASSIGNED: In total, 56 studies (HSCT, n = 53; GT, n = 3) representing 1,198 patients met inclusion criteria (HSCT, n = 1,158; GT, n = 40). Length of follow-up was 3,881.5 and 58.7 patient-years for HSCT and GT, respectively. Overall quality of evidence was low, with no randomized controlled trials identified. Two-year overall survival for HSCT was 91%; mortality was 2.5% for GT. Acute chest syndrome and vaso-occlusive episodes were reduced post-HSCT and GT. Meta-analysis was not possible due to lack of comparator and heterogeneity in outcome measures reporting. Very few studies reported post-transplant end-organ function. Six secondary malignancies (5 post-HSCT, 1 post-GT) were reported.
UNASSIGNED: Reporting of SCD-related complications and patient-important outcomes is lacking for both strategies. We advocate for standardized reporting to better compare outcomes within and between treatment groups.
UNASSIGNED: To compare the safety and efficacy of HSCT and GT to assist clinicians and patients in making informed treatment decisions.
UNASSIGNED: Phase I-III clinical trials and case reports/series were included. Regimens included HSCT from all stem cell sources, lentiviral gene therapy, and gene editing, with any conditioning regimen. We searched Medline and EMBASE databases as of 1st June 2020 for studies reporting HSCT and GT outcomes in SCD. The Newcastle-Ottawa scale was used to assess the risk of bias. Descriptive statistics and post-hoc imputation for standard deviations of mean change in FEV1 and FVC were performed.
UNASSIGNED: In total, 56 studies (HSCT, n = 53; GT, n = 3) representing 1,198 patients met inclusion criteria (HSCT, n = 1,158; GT, n = 40). Length of follow-up was 3,881.5 and 58.7 patient-years for HSCT and GT, respectively. Overall quality of evidence was low, with no randomized controlled trials identified. Two-year overall survival for HSCT was 91%; mortality was 2.5% for GT. Acute chest syndrome and vaso-occlusive episodes were reduced post-HSCT and GT. Meta-analysis was not possible due to lack of comparator and heterogeneity in outcome measures reporting. Very few studies reported post-transplant end-organ function. Six secondary malignancies (5 post-HSCT, 1 post-GT) were reported.
UNASSIGNED: Reporting of SCD-related complications and patient-important outcomes is lacking for both strategies. We advocate for standardized reporting to better compare outcomes within and between treatment groups.
摘要：
未经证实：异基因造血干细胞移植（HSCT）和基因治疗（GT）是治疗镰状细胞病（SCD）的两种潜在方法。但从未在临床试验中进行过比较。
UNASSIGNED：比较HSCT和GT的安全性和有效性，以帮助临床医生和患者做出明智的治疗决定。
未经评估：包括I-III期临床试验和病例报告/系列。方案包括来自所有干细胞来源的HSCT，慢病毒基因疗法，和基因编辑，任何调理方案。我们在截至2020年6月1日的Medline和EMBASE数据库中搜索了报告SCD中HSCT和GT结果的研究。纽卡斯尔-渥太华量表用于评估偏倚风险。对FEV1和FVC的平均变化的标准偏差进行描述性统计和事后填补。
未经批准：总共，56项研究（HSCT，n=53；GT，n=3)，代表1198例患者符合纳入标准(HSCT，n=1,158；GT，n=40)。HSCT和GT的随访时间为3,881.5和58.7患者年，分别。总体证据质量较低，没有确定的随机对照试验。HSCT的两年总生存率为91%；GT的死亡率为2.5%。HSCT和GT后，急性胸部综合征和血管闭塞发作减少。荟萃分析是不可能的，因为缺乏比较和结果测量报告的异质性。很少有研究报道移植后的终末器官功能。六个继发性恶性肿瘤（5个HSCT后，1post-GT)被报道。
UNASSIGNED：两种策略均缺乏SCD相关并发症和患者重要结局的报告。我们提倡标准化报告，以更好地比较治疗组内部和治疗组之间的结果。
UNASSIGNED：比较HSCT和GT的安全性和有效性，以帮助临床医生和患者做出明智的治疗决定。
未经评估：包括I-III期临床试验和病例报告/系列。方案包括来自所有干细胞来源的HSCT，慢病毒基因疗法，和基因编辑，任何调理方案。我们在截至2020年6月1日的Medline和EMBASE数据库中搜索了报告SCD中HSCT和GT结果的研究。纽卡斯尔-渥太华量表用于评估偏倚风险。对FEV1和FVC的平均变化的标准偏差进行描述性统计和事后填补。
未经批准：总共，56项研究（HSCT，n=53；GT，n=3)，代表1198例患者符合纳入标准(HSCT，n=1,158；GT，n=40)。HSCT和GT的随访时间为3,881.5和58.7患者年，分别。总体证据质量较低，没有确定的随机对照试验。HSCT的两年总生存率为91%；GT的死亡率为2.5%。HSCT和GT后，急性胸部综合征和血管闭塞发作减少。荟萃分析是不可能的，因为缺乏比较和结果测量报告的异质性。很少有研究报道移植后的终末器官功能。六个继发性恶性肿瘤（5个HSCT后，1post-GT)被报道。
UNASSIGNED：两种策略均缺乏SCD相关并发症和患者重要结局的报告。我们提倡标准化报告，以更好地比较治疗组内部和治疗组之间的结果。
