Outcomes and Adverse Effects of Voretigene Neparvovec Treatment for Biallelic RPE65-Mediated Inherited Retinal Dystrophies in a Cohort of Patients from a Single Center.
Outcomes and Adverse Effects of Voretigene Neparvovec Treatment for Biallelic RPE65-Mediated Inherited Retinal Dystrophies in a Cohort of Patients from a Single Center.
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DOI：
10.3390/biom13101484
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发表时间：
2023-10-05
期刊：
影响因子：
5.5
通讯作者：
中科院分区：
文献类型：
--
作者：
我们的研究评估了voretigene neparvovec （VN）基因治疗rpe65介导的遗传性视网膜营养不良（IRDs）的形态学和功能结果以及副作用，在牛津眼科医院的12只眼睛（6名患者）中，平均随访时间为8.2个月（范围1-12）。所有患者在基因治疗后1个月主观视力均有改善。最佳矫正视力（BCVA）保持稳定(基线：1.28（±0.71）vs.末次随访：1.46（±0.60）；P = 0.25)。平均白色全场刺激测试（FST）有改善趋势(基线：- 4.41（±10.62）dB，末次随访：- 11.98（±13.83）dB；P = 0.18)。视网膜中央厚度和黄斑体积未见变化。副作用包括轻度眼内炎症（两只眼睛）和白内障（四只眼睛）。10只眼睛出现视网膜萎缩（8只轻微，2只严重），但在随访期间没有影响FST测量。3例（6眼）患者眼压升高；4只眼（2例）需要青光眼手术。在我们的队列中，VN治疗的总体安全性和有效性与之前的VN临床试验一致，除了我们的队列中视网膜萎缩的发生率更高和IOP升高。这表明IOP升高和视网膜萎缩可能比以前报道的更为常见。
Our study evaluated the morphological and functional outcomes, and the side effects, of voretigene neparvovec (VN) gene therapy for RPE65-mediated inherited retinal dystrophies (IRDs) in 12 eyes (six patients) at the Oxford Eye Hospital with a mean follow-up duration of 8.2 (range 1–12) months. All patients reported a subjective vision improvement 1 month after gene therapy. Best-corrected visual acuity (BCVA) remained stable (baseline: 1.28 (±0.71) vs. last follow-up: 1.46 (±0.60); p = 0.25). Average white Full-Field Stimulus Testing (FST) showed a trend towards improvement (baseline: −4.41 (±10.62) dB vs. last follow-up: −11.98 (±13.83) dB; p = 0.18). No changes in central retinal thickness or macular volume were observed. The side effects included mild intraocular inflammation (two eyes) and cataracts (four eyes). Retinal atrophy occurred in 10 eyes (eight mild, two severe) but did not impact FST measurements during the follow-up period. Increased intraocular pressure (IOP) was noted in three patients (six eyes); four eyes (two patients) required glaucoma surgery. The overall safety and effectiveness of VN treatment in our cohort align with previous VN clinical trials, except for the higher occurrence of retinal atrophy and increased IOP in our cohort. This suggests that raised IOP and retinal atrophy may be more common than previously reported.
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影响因子：
8.1
作者：
Levi, Sarah R.;Oh, Jin Kyun;de Carvalho Jr, Jose Ronaldo Lima;Mahajan, Vinit B.;Tsang, Stephen H.;Sparrow, Janet R.
通讯作者：
Sparrow, Janet R.
影响因子：
4.1
作者：
Stingl, Krunoslav;Kempf, Melanie;Bartz-Schmidt, Karl U.;Dimopoulos, Spyridon;Reichel, Felix;Jung, Ronja;Kelbsch, Carina;Kohl, Susanne;Kortuem, Friederike Charlotte;Nasser, Fadi;Peters, Tobias;Wilhelm, Barbara;Wissinger, Bernd;Wozar, Fabian;Zrenner, Eberhart;Fischer, M. Dominik;Stingl, Katarina
通讯作者：
Stingl, Katarina
Voretigene Neparvovec 基因治疗可改善患有 Leber 先天性黑蒙的学龄前儿童的视力并部分恢复电生理功能。
影响因子：
4.7
作者：
Gerhardt, Maximilian J.;Priglinger, Claudia S.;Rudolph, Guenther;Hufendiek, Karsten;Framme, Carsten;Jaegle, Herbert;Salchow, Daniel J.;Anschuetz, Andreas;Michalakis, Stylianos;Priglinger, Siegfried G.
通讯作者：
Priglinger, Siegfried G.
影响因子：
3.8
作者：
Blonde L;Khunti K;Harris SB;Meizinger C;Skolnik NS
通讯作者：
Skolnik NS
影响因子：
168.9
作者：
Maguire, Albert M.;High, Katherine A.;Auricchio, Alberta;Wright, J. Fraser;Pierce, Eric A.;Testa, Francesco;Mingozzi, Federico;Bennicelli, Jeannette L.;Ying, Gui-shuang;Rossi, Settimio;Fulton, Ann;Marshall, Kathleen A.;Banfi, Sandra;Chung, Daniel C.;Morgan, Jessica I. W.;Hauck, Bernd;Zelenaia, Olga;Zhu, Xiaosong;Raffini, Leslie;Coppieters, Frauke;De Baere, Elfride;Shindler, Kenneth S.;Volpe, Nicholas J.;Surace, Enrico M.;Acerra, Carmela;Lyubarsky, Arkady;Redmond, T. Michael;Stone, Edwin;Sun, Junwei;McDonnell, Jennifer Wellman;Leroy, Bart P.;Simonelli, Francesca;Bennett, Jean
通讯作者：
Bennett, Jean