Borrelli Enrico, Berni Alessandro, Cascavilla Maria Lucia, Barresi Costanza, Battista Marco, Lari Giorgio, Reibaldi Michele, Bandello Francesco, Barboni Piero
From the Vita-Salute San Raffaele University, Milan (E.B., A.B., M.L.C., C.B., M.B., G.L., F.B., P.B.); IRCCS San Raffaele Scientific Institute, Milan (E.B., A.B., M.L.C., C.B., M.B., G.L., F.B., P.B.).
From the Vita-Salute San Raffaele University, Milan (E.B., A.B., M.L.C., C.B., M.B., G.L., F.B., P.B.); IRCCS San Raffaele Scientific Institute, Milan (E.B., A.B., M.L.C., C.B., M.B., G.L., F.B., P.B.).
Am J Ophthalmol. 2023 Mar;247:35-41. doi: 10.1016/j.ajo.2022.11.004. Epub 2022 Nov 12.
To assess the relationship of demographics, clinical characteristics and structural optical coherence tomography (OCT) findings to long-term visual outcomes in patients with Leber hereditary optic neuropathy (LHON) treated with idebenone.
Retrospective, interventional, noncomparative clinical cohort study.
In this study, a total of 17 participants (34 eyes) with LHON treated with idebenone therapy within 1 year after disease onset and 2 years (24 months) of regular follow-ups were retrospectively enrolled. At baseline, structural OCT volume scans of the macula and optic nerve were reviewed to measure metrics reflecting neuronal loss (ie, macular ganglion cell and inner plexiform layer [GC-IPL] and peripapillary retinal nerve fiber layer [RNFL] thicknesses). Stepwise multiple regression analyses were computed to assess associations between final best-corrected visual acuity (BCVA) at 2 years and change in BCVA from baseline at 2 years as dependent variables with demographics, clinical characteristics, and OCT metrics at baseline (visit before the initiation of treatment).
The BCVA was 1.6±0.8 logMAR (Snellen VA of ∼20/800) at baseline (visit before the initiation of treatment) and 1.0±0.7 logMAR (Snellen VA of 20/200) at the 2-year follow-up visit (P < .0001). Mean±SD change in BCVA from baseline at 2 years was -51.9%±35.9%. In multivariable analysis, the strongest associations with final BCVA were with baseline BCVA (P = .012), superior macular GC-IPL thickness (P = .044), superotemporal macular GC-IPL thickness (P = .010), and inferotemporal macular GC-IPL thickness (P = .015). Similarly, the strongest associations with delta BCVA were with superior macular GC-IPL thickness (P = .045), superotemporal macular GC-IPL thickness (P = .047), and inferotemporal macular GC-IPL thickness (P = .030).
We identified OCT biomarkers associated with long-term (ie, 2-year) visual outcomes in patients with LHON treated with idebenone therapy in the first year after disease onset. Thinning of the GC-IPL in the superior and temporal parafoveal regions was associated with worse long-term visual outcomes in these patients.
评估在接受艾地苯醌治疗的Leber遗传性视神经病变（LHON）患者中，人口统计学特征、临床特征和光学相干断层扫描（OCT）结构检查结果与长期视觉预后的关系。
回顾性、干预性、非对比临床队列研究。
本研究回顾性纳入了17例（34只眼）在疾病发作后1年内接受艾地苯醌治疗且进行了2年（24个月）定期随访的LHON患者。在基线时，对黄斑和视神经的OCT结构容积扫描进行评估，以测量反映神经元丢失的指标（即黄斑神经节细胞和内丛状层[GC-IPL]以及视乳头周围视网膜神经纤维层[RNFL]厚度）。进行逐步多元回归分析，以评估2年时最终最佳矫正视力（BCVA）以及2年时BCVA相对于基线的变化作为因变量与人口统计学特征、临床特征和基线时（治疗开始前的就诊）的OCT指标之间的关联。
基线时（治疗开始前的就诊）BCVA为1.6±0.8 logMAR（约20/800的Snellen视力），2年随访时为1.0±0.7 logMAR（20/200的Snellen视力）（P <.0001）。2年时BCVA相对于基线的平均±标准差变化为-51.9%±35.9%。在多变量分析中，与最终BCVA关联最强的是基线BCVA（P =.012）、上方黄斑GC-IPL厚度（P =.044）、颞上黄斑GC-IPL厚度（P =.010）和颞下黄斑GC-IPL厚度（P =.015）。同样，与BCVA变化量关联最强的是上方黄斑GC-IPL厚度（P =.045）、颞上黄斑GC-IPL厚度（P =.047）和颞下黄斑GC-IPL厚度（P =.030）。
我们确定了在疾病发作后第一年接受艾地苯醌治疗的LHON患者中与长期（即2年）视觉预后相关的OCT生物标志物。上方和颞侧旁中央凹区域的GC-IPL变薄与这些患者较差的长期视觉预后相关。
