Chew Emily Y, Clemons Traci E, Peto Tunde, Sallo Ferenc B, Ingerman Avner, Tao Weng, Singerman Lawrence, Schwartz Steven D, Peachey Neal S, Bird Alan C
National Eye Institute, National Institutes of Health, Bethesda, Maryland.
The EMMES Corporation, Rockville, Maryland.
Am J Ophthalmol. 2015 Apr;159(4):659-666.e1. doi: 10.1016/j.ajo.2014.12.013. Epub 2014 Dec 19.
To evaluate the safety and tolerability of intraocular delivery of ciliary neurotrophic factor (CNTF) using an encapsulated cell implant for the treatment of macular telangiectasia type 2.
An open-label safety trial conducted in 2 centers enrolling 7 participants with macular telangiectasia type 2.
The participant's more severely affected eye (worse baseline visual acuity) received the high-dose implant of CNTF. Patients were followed for a period of 36 months. The primary safety outcome was a change in the parameters of the electroretinogram (ERG). Secondary efficacy outcomes were changes in visual acuity, en face measurements of the optical coherence tomography of the disruption in the ellipsoid zone, and microperimetry when compared with baseline.
The ERG findings demonstrated a reduction in the amplitude of the scotopic b-wave in 4 participants 3 months after implantation (month 3). All parameters returned to baseline values by month 12 and remained so at month 36 with no clinical impact on dark adaptation. There was no change in visual acuity compared with baseline. The area of the defect as measured functionally by microperimetry and structurally by the en face OCT imaging of the ellipsoid zone loss appeared unchanged from baseline.
The intraocular delivery of CNTF in the encapsulated cell implant appeared to be safe and well tolerated in eyes with macular telangiectasia type 2. Further evaluation in a randomized controlled clinical trial is warranted to test for efficacy.
评估使用封装细胞植入物眼内递送睫状神经营养因子（CNTF）治疗2型黄斑毛细血管扩张症的安全性和耐受性。
在2个中心进行的一项开放标签安全性试验，纳入7名2型黄斑毛细血管扩张症患者。
患者受影响更严重的眼睛（基线视力较差）接受高剂量CNTF植入物。对患者进行36个月的随访。主要安全结局是视网膜电图（ERG）参数的变化。次要疗效结局是与基线相比，视力、椭圆体区破坏的光学相干断层扫描的正面测量结果以及微视野检查的变化。
ERG结果显示，4名参与者在植入后3个月（第3个月）暗视b波振幅降低。到第12个月时，所有参数均恢复到基线值，在第36个月时保持不变，对暗适应无临床影响。与基线相比，视力没有变化。通过微视野检查功能测量和通过椭圆体区丢失的正面OCT成像结构测量的缺损面积与基线相比似乎没有变化。
在2型黄斑毛细血管扩张症眼中，封装细胞植入物眼内递送CNTF似乎是安全的且耐受性良好。有必要在随机对照临床试验中进行进一步评估以测试疗效。
