Progression of Stargardt Disease as Determined by Fundus Autofluorescence Over a 24-Month Period (ProgStar Report No. 17).
Progression of Stargardt Disease as Determined by Fundus Autofluorescence Over a 24-Month Period (ProgStar Report No. 17).
复制标题
通过眼底自发荧光确定的 Stargardt 病在 24 个月内的进展情况（ProgStar 报告第 17 号）。
DOI：
10.1016/j.ajo.2023.02.003
复制
发表时间：
2023
影响因子：
4.2
通讯作者：
ProgstarStud
中科院分区：
文献类型：
--
作者：
Strauss,RupertW;Ho,Alexander;Jha,Anamika;Fujinami,Kaoru;Michaelides,Michel;Cideciyan,ArturV;Audo,Isabelle;Birch,DavidG;Sadda,Srinivas;Ip,Michael;West,Sheila;Schönbach,EtienneM;Kong,Xiangrong;Scholl,HendrikPN;ProgstarStud
目的评价眼底自体荧光（FAF）引起的Stargardt病萎缩性病变的进展率。DESIGNInternational，多中心，前瞻性队列研究。方法从9个研究中心招募了259名年龄≥6岁的ABCA4基因致病变异患者，随访时间超过24个月。每6个月取一次FAF图像，定量测定自身荧光明显减弱（DDAF）和自身荧光减弱（DAF）的面积。进度率由以时间为自变量的线性混合模型估计。结果259名受试者共488只眼（88.8%为双眼）被纳入研究，432只眼的图像被随访24个月。DDAF的总体估计进展为0.74 mm2 /y （95% CI 0.64-0.85， P< 0.05）。在单因素分析中，24个月期间的DAF为0.64 mm2 /y （95% CI 0.57-0.71）。生长速率强烈依赖于基线病变面积。平方根转化后，DDAF的生长速率与基线病灶半径无关(P=。11)，而DAF的生长速率呈相关性（P< 0.05）。0001)。基因型未发现显著影响DDAF或DAF病变的生长速度。结论sfaf可作为一种方便的监测工具，并可作为减缓疾病进展的介入性临床试验的合适终点。基线时的DDAF和DAF病变大小是病变面积增长的有力预测因素，可以通过平方根变换部分解释。
PURPOSETo estimate the progression rate of atrophic lesions in Stargardt disease derived from fundus autofluorescence (FAF).DESIGNInternational, multicenter, prospective cohort study.METHODSA total of 259 participants aged≥ 6 years with disease-causing variants in the ABCA4 gene were enrolled from 9 centers and followed over a 24-month period. FAF images were obtained every 6 months, and areas of definitely decreased autofluorescence (DDAF) and decreased autofluorescence (DAF) were quantified. Progression rates were estimated from linear mixed models with time as the independent variable.RESULTSA total of 488 study eyes of 259 participants (88.8% with both eyes) were enrolled and images from 432 eyes were followed for 24 months. The overall estimated progression of DDAF was 0.74 mm 2/y (95% CI 0.64-0.85, P<. 0001) and that of DAF was 0.64 mm 2/y (95% CI 0.57-0.71) over a 24-month period in univariate analysis. Growth rates were strongly dependent on baseline lesion area. After square root transformation, the DDAF growth rate was not dependent on baseline lesion radius (P=. 11), whereas the DAF growth rate was dependent (P<. 0001). Genotype was not found to significantly impact the growth rate of DDAF or DAF lesions.CONCLUSIONSFAF may serve as a convenient monitoring tool and suitable end point for interventional clinical trials that aim to slow disease progression. DDAF and DAF lesion sizes at baseline are strong predicting factors for lesion area growth and can be partially accounted for by square root transformation.