目的:干细胞治疗遗传性视网膜疾病(IRD)是一种有前途的治疗方法。这项研究旨在定量检查干细胞治疗对IRD患者的有效性和安全性,包括色素性视网膜炎和Stargardt病(STGD)。
方法:我们搜索了PubMed,EMBASE,WebofScience,Cochrane图书馆数据库,和ClinicalTrials.gov网站。最新检索时间为2023年8月20日。主要结果是最佳矫正视力(BCVA)改善的比率和平均差异(MD)。根据给药途径和干细胞类型进行亚组分析。本研究在PROSPERO(CRD42022349271)注册。
结果:21项前瞻性研究,涉及382例患者(306例RP和76例STGD)的496只眼(404例RP和92例STGD),包括在这项研究中。对于RP,6个月和12个月时BCVA改善率分别为49%和30%,分别,治疗后6个月,手术眼的BCVA显着改善(MD=-0.12logMAR,95%CI.17至-0.06logMAR;P<0.001),而治疗后12个月无显著差异(MD=-0.06logMAR;95%CI-0.13至0.01logMAR;P=0.10)。对于STGD,6个月和12个月时BCVA改善率分别为60%和55%,分别,手术眼在6个月时BCVA显着改善(MD=-0.14logMAR,95%CI-0.22至-0.07logMAR;P=0.0002)和12个月(MD=-0.17logMAR,95%CI-0.29至-0.04logMAR;P=0.01)。亚组分析显示,脉络膜上腔注射干细胞可能对RP更有效。3项研究报告了11例与治疗相关的眼部不良事件,没有相关的全身不良事件。
结论:本研究提示干细胞治疗对于RP或STGD患者可能是有效和安全的。RP患者的长期视力改善可能有限。脉络膜上腔注射干细胞可能是RP患者的一种有希望的给药途径。受证据等级低的限制,未来需要大样本量的随机临床试验.
Stem cell therapy is a promising therapeutic approach for inherited retinal diseases (IRDs). This study aims to quantitatively examine the effectiveness and safety of stem cell therapy for patients with IRDs, including retinitis pigmentosa and Stargardt disease (STGD).
We searched PubMed, EMBASE, Web of Science, Cochrane Library databases, and the ClinicalTrials.gov website. The latest retrieval time was August 20, 2023. The primary outcomes were rates and mean difference (MD) of best-corrected visual acuity (BCVA) improvement. Subgroup analyses were conducted according to administration routes and stem cell types. This study was registered with PROSPERO (CRD42022349271).
Twenty-one prospective studies, involving 496 eyes (404 RP and 92 STGD) of 382 patients (306 RP and 76 STGD), were included in this study. For RP, the rate of BCVA improvement was 49% and 30% at 6 months and 12 months, respectively, and the BCVA was significantly improved in the operative eyes at 6 months post-treatment (MD = - 0.12 logMAR, 95% CI .17 to - 0.06 logMAR; P < 0.001), while there was no significant difference at 12 months post-treatment (MD = -0.06 logMAR; 95% CI - 0.13 to 0.01 logMAR; P = 0.10). For STGD, the rate of BCVA improvement was 60% and 55% at 6 months and 12 months, respectively, and the BCVA was significantly improved in the operative eyes at 6 months (MD = - 0.14 logMAR, 95% CI - 0.22 to - 0.07 logMAR; P = 0.0002) and 12 months (MD = - 0.17 logMAR, 95% CI - 0.29 to - 0.04 logMAR; P = 0.01). Subgroup analyses showed suprachoroidal space injection of stem cells may be more efficient for RP. Eleven treated-related ocular adverse events from three studies and no related systemic adverse events were reported.
This study suggests stem cell therapy may be effective and safe for patients with RP or STGD. The long-term vision improvement may be limited for RP patients. Suprachoroidal space injection of stem cells may be a promising administration route for RP patients. Limited by the low grade of evidence, large sample size randomized clinical trials are required in the future.