文摘
摘要目的:评估的长期安全性和有效性lenadogene nolparvovec Leber世袭视神经病变患者的基因疗法(LHON)由于m.11778G >ND4突变。
背景:两三期临床研究,救援和扭转,评估的有效性和安全性intravitreal注入lenadogene nolparvovec。恢复是他们的长期随访研究。
设计/方法:在救援/逆转,所有患者接受lenadogene nolparvovec一只眼睛和虚假的注入另一只眼睛。96周后,患者表示愿意参与恢复3年,对应5年治疗后的管理。生活质量监控使用视觉函数questionnaire-25 (NEI VFQ-25)。
结果:72名患者完成救援或逆转,62年参加了恢复。患者大多是男性(79.0%),平均37.1(15.3)岁(SD)。治疗后5年,两国改善从最低点观察治疗后2年维护:绝对意味着变化(SD)从最低点BCVA−0.44 (0.46) LogMAR(+ 22.0字母)lenadogene nolparvovec-treated眼睛和−0.39 (0.36)LogMAR(+ 19.5字母)sham-treated眼睛。大多数病人(80.7%)有他们BCVA图表(即。在5年内,≤1.6 LogMAR)。临床相关的响应从低谷中观察到71.0%的患者。临床有意义的整体生活质量改善报告,意味着获得的7分从基线VFQ-25综合得分。安全性良好,期间观察到的类似于第一个2年。
结论:长期随访的MT-ND4-LHON患者单方面处理lenadogene nolparvovec证明持续改善BCVA双眼和改善生活质量治疗后5年。
披露:Biousse博士已经收到个人薪酬在500 - 4999美元的范围作为顾问Gensights生物。Biousse博士已经收到个人补偿的范围0 - 499美元为Neurophoenix担任顾问。Biousse博士的机构收到NIH的研究支持。Biousse博士已经收到相关出版物的出版版税卫生保健。Biousse博士已经收到相关出版物的出版版税卫生保健。Newman博士已经收到个人薪酬在10000 - 49999美元的范围为GenSight担任顾问。Newman博士已经收到个人薪酬在500 - 4999美元的范围为基耶西担任顾问。Newman博士已经收到个人薪酬在500 - 4999美元的范围为Neurophoenix担任顾问。Newman博士已经收到个人薪酬在500 - 4999美元的范围为斯托克担任顾问。Newman博士已经收到个人薪酬在500 - 4999美元的范围为贪欲担任顾问。 Dr. Newman has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Neurodiem. The institution of Dr. Newman has received research support from GenSight. The institution of Dr. Newman has received research support from Chiesi/Santhera. The institution of Dr. Newman has received research support from NINDS/NIH. Dr. Newman has received publishing royalties from a publication relating to health care. Dr. Newman has received publishing royalties from a publication relating to health care. Dr. Newman has received publishing royalties from a publication relating to health care. Patrick Yu-Wai-Man has received personal compensation in the range of $500-$4,999 for serving as a Consultant for GenSight Biologics. Dr. Carelli has received personal compensation in the range of $0-$499 for serving as a Consultant for Chiesi Farmaceutici. Dr. Carelli has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Chiesi Farmaceutici. Dr. Moster has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Gensight . Dr. Moster has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Viridian. Dr. Moster has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Kilcoyne and Nesbitt. Dr. Vignal-Clermont has nothing to disclose. Dr. Klopstock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for GenSight Biologics. Dr. Klopstock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Chiesi GmbH. Dr. Klopstock has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Chiesi GmbH. Dr. Klopstock has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Chiesi GmbH. Dr. Klopstock has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for GenSight Biologics. Dr. Sadun has received publishing royalties from a publication relating to health care. Dr. Sergott has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Dr. Sergott has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon Therapeutics. Dr. Sergott has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen. Magali Taiel has nothing to disclose. Jose-Alain Sahel has received stock or an ownership interest from Prophesee. Jose-Alain Sahel has received stock or an ownership interest from Pixium Vision. Jose-Alain Sahel has received stock or an ownership interest from Tilak Healthcare. The institution of Jose-Alain Sahel has received research support from LabEx LIFESENSES (ANR-10-LABX-65). The institution of Jose-Alain Sahel has received research support from IHU FOReSIGHT (ANR-18-IAHU-01). The institution of Jose-Alain Sahel has received research support from ERC Synergy "HELMHOLTZ".
