Valerio Carelli, Patrick Yu-Wai-Man, Xavier Llòria, Magda Joana Silva, Thomas Klopstock
{"title":"依地苯酮起始时间对leber遗传性视神经病变视力的影响：leber研究的事后分析","authors":"Valerio Carelli, Patrick Yu-Wai-Man, Xavier Llòria, Magda Joana Silva, Thomas Klopstock","doi":"10.1111/aos.17006","DOIUrl":null,"url":null,"abstract":"<div>\n \n <section>\n \n <p><b>Aims/Purpose:</b> It is not yet established whether time of idebenone treatment initiation from symptom onset (T<sub>i</sub>) impacts the clinical outcome of patients with Leber hereditary optic neuropathy (LHON). LEROS, a Phase IV, open-label, international, natural-history controlled study (NCT02774005), assessed the long-term efficacy of idebenone in the treatment of LHON.<sup>1</sup> In this post-hoc analysis of LEROS study data, we report visual acuity (VA) endpoints by T<sub>i</sub>.</p>\n \n <p><b>Methods:</b> VA endpoints at last visit were assessed as previously described, in idebenone-treated eyes from the LEROS study with an observation time of 12–24 months (m) and a m.11778G > A mitochondrial DNA mutation.<sup>1</sup> In this post-hoc analysis, eyes were stratified by T<sub>i</sub>. No statistical comparisons were performed.</p>\n \n <p><b>Results:</b> Median [range, <i>n</i>] age at baseline was similar regardless of T<sub>i</sub> (0–3m: 32.2y [12.6–49.6, 10]; 3–6m: 36.2y [12.6–62.5, 21]; 6–9m: 37.4y [12.6–64.5, 20]; 9–12m: 33.0y [19.5–49.6, 19]; 12–24m: 32.4y [12.1–60.2, 44]; > 24m: 34.7y [12.4–62.4, 61]). VA endpoints at last visit were also similar regardless of T<sub>i</sub>; however, minor differences were observed. Eyes that were 3–6m from symptom onset at treatment initiation had the best median [interquartile range (IQR)] VA at last visit (0–3m: 1.27 [0.40–1.68]; 3–6m: 1.22 [0.30–1.80]; 6–9m: 1.40 [1.02–1.56]; 9–12m: 1.52 [0.98–1.60]; 12–24m: 1.39 [0.58–1.74]; > 24m: 1.32 [0.90–1.62]) and the best median [IQR] VA change from last visit to baseline (0–3m: 0.08 [-0.20–0.62]; 3–6m: -0.20 [-0.46–0.10]; 6–9m: -0.11 [-0.19–0.00]; 9–12m: -0.10 [-0.48– -0.04]; 12–24m: -0.09 [-0.47–0.00]; > 24m: -0.06 [-0.18–0.00]).</p>\n \n <p><b>Conclusions:</b> LEROS study data provides important insights into the impact of time of treatment initiation on VA outcome. However, given the small number of eyes and large variation in the data, caution must be taken in interpreting these results.</p>\n \n <p><b>References</b></p>\n \n <p>1. Yu-Wai-Man P <i>et al. Cell Rep Med</i>. 2024;5:101437.</p>\n </section>\n </div>","PeriodicalId":6915,"journal":{"name":"Acta Ophthalmologica","volume":"103 S284","pages":""},"PeriodicalIF":3.0000,"publicationDate":"2025-01-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/10.1111/aos.17006","citationCount":"0","resultStr":"{\"title\":\"Impact of time to idebenone initiation on visual acuity in leber hereditary optic neuropathy: Post hoc analysis of the leros study\",\"authors\":\"Valerio Carelli, Patrick Yu-Wai-Man, Xavier Llòria, Magda Joana Silva, Thomas Klopstock\",\"doi\":\"10.1111/aos.17006\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div>\\n \\n <section>\\n \\n <p><b>Aims/Purpose:</b> It is not yet established whether time of idebenone treatment initiation from symptom onset (T<sub>i</sub>) impacts the clinical outcome of patients with Leber hereditary optic neuropathy (LHON). LEROS, a Phase IV, open-label, international, natural-history controlled study (NCT02774005), assessed the long-term efficacy of idebenone in the treatment of LHON.<sup>1</sup> In this post-hoc analysis of LEROS study data, we report visual acuity (VA) endpoints by T<sub>i</sub>.</p>\\n \\n <p><b>Methods:</b> VA endpoints at last visit were assessed as previously described, in idebenone-treated eyes from the LEROS study with an observation time of 12–24 months (m) and a m.11778G > A mitochondrial DNA mutation.<sup>1</sup> In this post-hoc analysis, eyes were stratified by T<sub>i</sub>. No statistical comparisons were performed.</p>\\n \\n <p><b>Results:</b> Median [range, <i>n</i>] age at baseline was similar regardless of T<sub>i</sub> (0–3m: 32.2y [12.6–49.6, 10]; 3–6m: 36.2y [12.6–62.5, 21]; 6–9m: 37.4y [12.6–64.5, 20]; 9–12m: 33.0y [19.5–49.6, 19]; 12–24m: 32.4y [12.1–60.2, 44]; > 24m: 34.7y [12.4–62.4, 61]). VA endpoints at last visit were also similar regardless of T<sub>i</sub>; however, minor differences were observed. Eyes that were 3–6m from symptom onset at treatment initiation had the best median [interquartile range (IQR)] VA at last visit (0–3m: 1.27 [0.40–1.68]; 3–6m: 1.22 [0.30–1.80]; 6–9m: 1.40 [1.02–1.56]; 9–12m: 1.52 [0.98–1.60]; 12–24m: 1.39 [0.58–1.74]; > 24m: 1.32 [0.90–1.62]) and the best median [IQR] VA change from last visit to baseline (0–3m: 0.08 [-0.20–0.62]; 3–6m: -0.20 [-0.46–0.10]; 6–9m: -0.11 [-0.19–0.00]; 9–12m: -0.10 [-0.48– -0.04]; 12–24m: -0.09 [-0.47–0.00]; > 24m: -0.06 [-0.18–0.00]).</p>\\n \\n <p><b>Conclusions:</b> LEROS study data provides important insights into the impact of time of treatment initiation on VA outcome. However, given the small number of eyes and large variation in the data, caution must be taken in interpreting these results.</p>\\n \\n <p><b>References</b></p>\\n \\n <p>1. Yu-Wai-Man P <i>et al. Cell Rep Med</i>. 2024;5:101437.</p>\\n </section>\\n </div>\",\"PeriodicalId\":6915,\"journal\":{\"name\":\"Acta Ophthalmologica\",\"volume\":\"103 S284\",\"pages\":\"\"},\"PeriodicalIF\":3.0000,\"publicationDate\":\"2025-01-19\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://onlinelibrary.wiley.com/doi/10.1111/aos.17006\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Acta Ophthalmologica\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://onlinelibrary.wiley.com/doi/10.1111/aos.17006\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"OPHTHALMOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Acta Ophthalmologica","FirstCategoryId":"3","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1111/aos.17006","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"OPHTHALMOLOGY","Score":null,"Total":0}
Impact of time to idebenone initiation on visual acuity in leber hereditary optic neuropathy: Post hoc analysis of the leros study
Aims/Purpose: It is not yet established whether time of idebenone treatment initiation from symptom onset (Ti) impacts the clinical outcome of patients with Leber hereditary optic neuropathy (LHON). LEROS, a Phase IV, open-label, international, natural-history controlled study (NCT02774005), assessed the long-term efficacy of idebenone in the treatment of LHON.1 In this post-hoc analysis of LEROS study data, we report visual acuity (VA) endpoints by Ti.
Methods: VA endpoints at last visit were assessed as previously described, in idebenone-treated eyes from the LEROS study with an observation time of 12–24 months (m) and a m.11778G > A mitochondrial DNA mutation.1 In this post-hoc analysis, eyes were stratified by Ti. No statistical comparisons were performed.
Results: Median [range, n] age at baseline was similar regardless of Ti (0–3m: 32.2y [12.6–49.6, 10]; 3–6m: 36.2y [12.6–62.5, 21]; 6–9m: 37.4y [12.6–64.5, 20]; 9–12m: 33.0y [19.5–49.6, 19]; 12–24m: 32.4y [12.1–60.2, 44]; > 24m: 34.7y [12.4–62.4, 61]). VA endpoints at last visit were also similar regardless of Ti; however, minor differences were observed. Eyes that were 3–6m from symptom onset at treatment initiation had the best median [interquartile range (IQR)] VA at last visit (0–3m: 1.27 [0.40–1.68]; 3–6m: 1.22 [0.30–1.80]; 6–9m: 1.40 [1.02–1.56]; 9–12m: 1.52 [0.98–1.60]; 12–24m: 1.39 [0.58–1.74]; > 24m: 1.32 [0.90–1.62]) and the best median [IQR] VA change from last visit to baseline (0–3m: 0.08 [-0.20–0.62]; 3–6m: -0.20 [-0.46–0.10]; 6–9m: -0.11 [-0.19–0.00]; 9–12m: -0.10 [-0.48– -0.04]; 12–24m: -0.09 [-0.47–0.00]; > 24m: -0.06 [-0.18–0.00]).
Conclusions: LEROS study data provides important insights into the impact of time of treatment initiation on VA outcome. However, given the small number of eyes and large variation in the data, caution must be taken in interpreting these results.
References
1. Yu-Wai-Man P et al. Cell Rep Med. 2024;5:101437.
期刊介绍：
Acta Ophthalmologica is published on behalf of the Acta Ophthalmologica Scandinavica Foundation and is the official scientific publication of the following societies: The Danish Ophthalmological Society, The Finnish Ophthalmological Society, The Icelandic Ophthalmological Society, The Norwegian Ophthalmological Society and The Swedish Ophthalmological Society, and also the European Association for Vision and Eye Research (EVER).
Acta Ophthalmologica publishes clinical and experimental original articles, reviews, editorials, educational photo essays (Diagnosis and Therapy in Ophthalmology), case reports and case series, letters to the editor and doctoral theses.
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