{"title":"新胚芽素治疗神经营养性角膜病变相关角膜缘干细胞缺乏症的观察研究。","authors":"Alejandro Arboleda, Christopher N Ta","doi":"10.1177/25158414221134598","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Objectives: </strong>To determine the efficacy and safety of cenegermin in the treatment of LSCD associated with NK.</p><p><strong>Design: </strong>Prospective cohort study.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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 (<i>P</i> = .036). Corneal sensation improved in all patients, Cochet-Bonnet aesthesiometry measured 14.7 and 26.7 mm at the initial and final visit, respectively (<i>P</i> = .009). VA improved in 4 out of 6 eyes, with mean initial logMAR VA of 1.67 and final logMAR VA of 1.19 (<i>P</i> = .045). Finally, LSCD grading improved using the Aravena scoring system; however, this difference was not statistically significant (<i>P</i> = .14). One patient presented with an epithelial defect at baseline, which resolved following treatment. No patient withdrew from the study due to adverse effects.</p><p><strong>Conclusions: </strong>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.</p><p><strong>Registration: </strong>The study was registered at ClinicalTrials.gov with identifier NCT04552730 (https://clinicaltrials.gov/ct2/show/NCT04552730).</p>","PeriodicalId":23054,"journal":{"name":"Therapeutic Advances in Ophthalmology","volume":" ","pages":"25158414221134598"},"PeriodicalIF":2.3000,"publicationDate":"2022-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/74/17/10.1177_25158414221134598.PMC9638689.pdf","citationCount":"1","resultStr":"{\"title\":\"Observational study of cenegermin for the treatment of limbal stem cell deficiency associated with neurotrophic keratopathy.\",\"authors\":\"Alejandro Arboleda, Christopher N Ta\",\"doi\":\"10.1177/25158414221134598\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>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.</p><p><strong>Objectives: </strong>To determine the efficacy and safety of cenegermin in the treatment of LSCD associated with NK.</p><p><strong>Design: </strong>Prospective cohort study.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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 (<i>P</i> = .036). Corneal sensation improved in all patients, Cochet-Bonnet aesthesiometry measured 14.7 and 26.7 mm at the initial and final visit, respectively (<i>P</i> = .009). VA improved in 4 out of 6 eyes, with mean initial logMAR VA of 1.67 and final logMAR VA of 1.19 (<i>P</i> = .045). Finally, LSCD grading improved using the Aravena scoring system; however, this difference was not statistically significant (<i>P</i> = .14). One patient presented with an epithelial defect at baseline, which resolved following treatment. No patient withdrew from the study due to adverse effects.</p><p><strong>Conclusions: </strong>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.</p><p><strong>Registration: </strong>The study was registered at ClinicalTrials.gov with identifier NCT04552730 (https://clinicaltrials.gov/ct2/show/NCT04552730).</p>\",\"PeriodicalId\":23054,\"journal\":{\"name\":\"Therapeutic Advances in Ophthalmology\",\"volume\":\" \",\"pages\":\"25158414221134598\"},\"PeriodicalIF\":2.3000,\"publicationDate\":\"2022-11-04\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/74/17/10.1177_25158414221134598.PMC9638689.pdf\",\"citationCount\":\"1\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Therapeutic Advances in Ophthalmology\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1177/25158414221134598\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2022/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q2\",\"JCRName\":\"OPHTHALMOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Therapeutic Advances in Ophthalmology","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1177/25158414221134598","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2022/1/1 0:00:00","PubModel":"eCollection","JCR":"Q2","JCRName":"OPHTHALMOLOGY","Score":null,"Total":0}
引用次数: 1
摘要
背景:神经营养性角膜病变(NK)和角膜缘干细胞缺乏症(LSCD)发病率高，需要积极治疗以防止永久性视力丧失。Cenegermin是一种重组人神经生长因子，于2018年获得美国联邦药物管理局(Federal Drug Administration)批准用于治疗NK。目的:探讨根芽素治疗NK相关性LSCD的疗效和安全性。设计:前瞻性队列研究。方法:诊断为LSCD和NK的常规治疗失败的患者被纳入这项前瞻性开放标签研究。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、视力(VA)和LSCD严重程度也进行了评估。结果:5例患者6只眼纳入研究。genegermin显著改善了6只眼中5只眼的角膜上皮异常面积，首次就诊时占角膜总面积的73%，最后一次就诊时占角膜总面积的48% (P = 0.036)。所有患者的角膜感觉均有改善，初次和最后一次就诊时的Cochet-Bonnet美学测量值分别为14.7和26.7 mm (P = 0.009)。6只眼中有4只眼的VA改善，平均初始logMAR VA为1.67，最终logMAR VA为1.19 (P = 0.045)。最后，采用Aravena评分系统对LSCD评分进行改进;然而，这种差异没有统计学意义(P = .14)。一名患者在基线时出现上皮缺陷，治疗后消失。没有患者因不良反应退出研究。结论:genegermin可有效改善LSCD和NK患者先前治疗失败的角膜上皮、VA和角膜感觉。需要进一步的研究来更好地了解解剖变化，并通过更大的随机对照试验来证实我们的结果。注册:该研究在ClinicalTrials.gov注册，识别码为NCT04552730 (https://clinicaltrials.gov/ct2/show/NCT04552730)。
Observational study of cenegermin for the treatment of limbal stem cell deficiency associated with neurotrophic keratopathy.
Background: 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.
Objectives: To determine the efficacy and safety of cenegermin in the treatment of LSCD associated with NK.
Design: Prospective cohort study.
Methods: 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.
Results: 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.
Conclusions: 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.
Registration: The study was registered at ClinicalTrials.gov with identifier NCT04552730 (https://clinicaltrials.gov/ct2/show/NCT04552730).
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