未经证实:神经营养性角膜病变(NK)和角膜缘干细胞缺乏症(LSCD)的发病率很高,需要积极的治疗以防止永久性视力丧失。Cenegermin,重组人神经生长因子,2018年被联邦药物管理局批准用于治疗NK。
UNASSIGNED:确定cenegermin在治疗与NK相关的LSCD中的疗效和安全性。
未经评估:前瞻性队列研究。
UNASSIGNED:在这项前瞻性开放标签研究中纳入了常规治疗失败的诊断为LSCD和NK的患者。患者接受cenegermin治疗8周。主要目的是确定治疗后异常上皮的面积是否减少。角膜感觉,视敏度(VA),还评估了LSCD严重程度。
未经证实:本研究纳入了5例患者的6只眼。Cenegermin显著改善了6只眼中5只异常角膜上皮的面积,在初次就诊时测量角膜总面积的73%,在最后一次就诊时测量角膜总面积的48%(P=0.036)。所有患者的角膜感觉都得到了改善,Cochet-Bonnet在初次和最终就诊时测得14.7和26.7毫米,分别(P=.009)。6只眼睛中有4只的VA有所改善,平均初始logMARVA为1.67,最终logMARVA为1.19(P=0.045)。最后,使用Aravena评分系统改进了LSCD评分;然而,差异无统计学意义(P=.14).一名患者在基线时出现上皮缺损,在治疗后解决了。没有患者由于不良反应而退出研究。
UNASSIGNED:Cenegermin有效改善了角膜上皮,VA,先前治疗失败的LSCD和NK患者的角膜感觉。需要进一步的研究以更好地了解解剖学变化,并通过更大的随机对照试验证实我们的结果。
UNASSIGNED:该研究已在ClinicalTrials.gov注册,标识符为NCT04552730(https://clinicaltrials.gov/ct2/show/NCT04552730)。
UNASSIGNED: Neurotrophic keratopathy (NK) and limbal stem cell deficiency (LSCD) have high morbidity and require aggressive management to prevent permanent vision loss. Cenegermin, a recombinant human nerve growth factor, was approved by the Federal Drug Administration in 2018 for the treatment of NK.
UNASSIGNED: To determine the efficacy and safety of cenegermin in the treatment of LSCD associated with NK.
UNASSIGNED: Prospective cohort
study.
UNASSIGNED: Patients diagnosed with LSCD and NK who had failed conventional treatment were enrolled in this prospective open-label
study. Patients were treated with cenegermin for 8 weeks. The primary objective was to determine whether the area of abnormal epithelium decreased following treatment. Corneal sensation, visual acuity (VA), and LSCD severity were also evaluated.
UNASSIGNED: Six eyes of 5 patients were included in the
study. Cenegermin significantly improved the area of abnormal corneal epithelium in 5 of 6 eyes, measuring 73% of total corneal area at the initial visit and 48% at the final visit (P = .036). Corneal sensation improved in all patients, Cochet-Bonnet aesthesiometry measured 14.7 and 26.7 mm at the initial and final visit, respectively (P = .009). VA improved in 4 out of 6 eyes, with mean initial logMAR VA of 1.67 and final logMAR VA of 1.19 (P = .045). Finally, LSCD grading improved using the Aravena scoring system; however, this difference was not statistically significant (P = .14). One patient presented with an epithelial defect at baseline, which resolved following treatment. No patient withdrew from the
study due to adverse effects.
UNASSIGNED: Cenegermin effectively improved the cornea epithelium, VA, and corneal sensation in patients with LSCD and NK who had failed prior treatment. Further studies are necessary to better understand the anatomical changes and to confirm our results with a larger randomized control
trial.
UNASSIGNED: The study was registered at ClinicalTrials.gov with identifier NCT04552730 (https://clinicaltrials.gov/ct2/show/NCT04552730).