m.11778G>A MT-ND4 Leber 遗传性视神经病变患者治疗效果的元分析。

IF 5.1 2区 医学 Q1 OPHTHALMOLOGY
Nancy J Newman, Valérie Biousse, Patrick Yu-Wai-Man, Valerio Carelli, Catherine Vignal-Clermont, François Montestruc, Magali Taiel, José-Alain Sahel
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引用次数: 0

摘要

我们的目的是评估携带m.11778G>A MT-ND4突变的勒伯遗传性视神经病变(LHON)患者的视觉疗效,这些患者未接受任何治疗(自然史),或接受了依地苯酮或来那度根诺帕韦克治疗。疗效结果包括从最低点的临床相关恢复(CRR)和最终最佳矫正视力(BCVA)。对于自然病史组和依地孕酮组,我们对文献和现有的临床/法规报告进行了系统性回顾。至于来那度金-酚帕沃韦克组,我们纳入了来自3期研究的所有数据。总体效应及其95%置信区间(CI)采用随机效应模型进行估算。在每项荟萃分析中,患者视力丧失时的平均年龄约为30岁,大部分(≥78%)为男性。未经治疗、经依地苯酮治疗和经来那度尼泊金诺帕韦克治疗的患者眼底CRR[95% CI]分别为17%[7%;30%](316眼)、31%[24%;40%](313眼)和59%[54%;64%](348眼)。在患者水平的CRR和最终BCVA中也观察到了这种疗效梯度。在所有评估的视觉结果中都存在疗效梯度,CRR的疗效比最终BCVA的疗效更明显,来那多吉酚帕沃韦基因疗法的疗效优于依地苯酮疗法,两者的疗效都优于疾病的自然病史。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Meta-analysis of treatment outcomes for patients with m.11778G>A MT-ND4 Leber hereditary optic neuropathy.

Our aim was to assess the visual outcomes of patients with Leber hereditary optic neuropathy (LHON) harboring the m.11778G>A MT-ND4 mutation who had no treatment (natural history) or received idebenone or lenadogene nolparvovec. Efficacy outcomes included clinically relevant recovery (CRR) from nadir and final best-corrected visual acuity (BCVA). For the natural history and idebenone groups, we performed a systematic review of the literature and available clinical/regulatory reports. For the lenadogene nolparvovec group, all data from phase 3 studies were included. The overall effect and its 95 % confidence interval (CI) were estimated using a random effects model. For each meta-analysis, patients had a mean age of approximately 30 years at vision loss and were mostly (≥78 %) men. The CRR from nadir [95 % CI] at eye level was 17 % [7 %; 30 %] (n=316 eyes), 31 % [24 %; 40 %] (n=313) and 59 % [54 %; 64 %] (n=348) in untreated, idebenone-treated and lenadogene nolparvovec-treated patients, respectively. This gradient of efficacy was also observed with CRR at the patient level and final BCVA. There was a gradient of efficacy in all assessed visual outcomes, more marked for CRR than for final BCVA, with lenadogene nolparvovec gene therapy superior to idebenone treatment, and both superior to the natural history of the disease.

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来源期刊
Survey of ophthalmology
Survey of ophthalmology 医学-眼科学
CiteScore
10.30
自引率
2.00%
发文量
138
审稿时长
14.8 weeks
期刊介绍: Survey of Ophthalmology is a clinically oriented review journal designed to keep ophthalmologists up to date. Comprehensive major review articles, written by experts and stringently refereed, integrate the literature on subjects selected for their clinical importance. Survey also includes feature articles, section reviews, book reviews, and abstracts.
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