Oral minocycline for the treatment of retinitis pigmentosa-associated cystoid macular edema: results of a phase I/II clinical trial.
Oral minocycline for the treatment of retinitis pigmentosa-associated cystoid macular edema: results of a phase I/II clinical trial.
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口服米诺环素治疗色素性视网膜炎相关黄斑囊样水肿：I/II 期临床试验的结果。
DOI：
10.1007/s00417-023-05986-6
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发表时间：
2023
期刊：
影响因子：
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通讯作者：
Cukras,CatherineA
中科院分区：
文献类型：
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作者：
Dave,AmishaD;Chen,KatherineG;Chiang,TrentTsun-Kang;Singaravelu,Janani;Alvarez,JasonA;Wong,WaiT;Cukras,CatherineA
目的视网膜色素变性（RP）相关性黄斑囊样水肿（CME）的病因与视网膜神经炎症和小胶质细胞活化有关。米诺环素是FDA批准用于抗微生物适应症的药物，也抑制小胶质细胞活化和炎症介质的表达。本研究探讨了口服米诺环素作为RP相关CME.MethodsA单中心，前瞻性，开放标签I/II期临床试验的主要治疗的安全性和有效性与RP相关CME招募5名参与者。参与者在开始口服米诺环素100 mg，每日两次，持续12个月之前进行了导入评估。主要结果变量包括最佳矫正视力（BCVA）和视网膜中央子场厚度（CST）的变化，使用光谱域光学相干断层扫描测量相对于平均治疗前measurement.ResultsThe研究药物的耐受性良好，不与任何严重的不良事件。在研究眼（6个月时为+ 0.7 ± 4.1个字母，12个月时为− 1.1 ± 1.7个字母）或合格对侧眼（6个月时为− 0.3 ± 3.4个字母，12个月时为− 0.3 ± 4.6个字母）中均未观察到平均BCVA较研究基线的显著变化（所有比较p> 0.05）。然而，CST相对于基线的平均百分比变化随着治疗进行性降低（6个月和12个月时降低：研究眼3.9和9.8%;合格对侧眼1.4和7.7%）。考虑所有眼睛（n= 10），6个月和12个月时CST平均百分比下降为2.7 ± 9.5%（p= 0.39）和8.7 ± 9.5%结论口服米诺环素12个月以上与平均BCVA无显著变化和平均CST小幅但进行性降低相关。2014年）。
PurposeThe etiology of retinitis pigmentosa (RP)–associated cystoid macular edema (CME) has been related to retinal neuroinflammation and microglial activation. Minocycline, a drug FDA-approved for anti-microbial indications, also inhibits microglial activation and expression of inflammatory mediators. This study investigates the safety and efficacy of oral minocycline as primary treatment for RP-associated CME.MethodsA single-center, prospective, open-label phase I/II clinical trial enrolled five participants with RP-associated CME. Participants had lead-in assessments prior to the initiation of oral minocycline 100 mg twice daily for 12 months. Main outcome variables included changes in best-corrected visual acuity (BCVA) and retinal central subfield thickness (CST) measured using spectral domain optical coherence tomography relative to mean of pre-treatment measurements.ResultsThe study drug was well tolerated and not associated with any severe adverse events. No significant changes in mean BCVA from study baseline were noted in either the study eye (+ 0.7 ± 4.1 letters at 6 months, − 1.1 ± 1.7 letters at 12 months) or the qualifying fellow eye (− 0.3 ± 3.4 letters at 6 months, − 0.3 ± 4.6 letters at 12 months) (p> 0.05 for all comparisons). Mean percentage changes in CST from baseline however decreased progressively with treatment (decreases at 6 and 12 months: study eyes 3.9 and 9.8%; qualifying fellow eyes 1.4 and 7.7%). Considering all eyes (n= 10), mean percentage CST decrease at 6 and 12 months was 2.7 ± 9.5% (p= 0.39) and 8.7 ± 9.5% (p= 0.02) respectively.ConclusionOral minocycline administration over 12 months was associated with no significant changes in mean BCVA and a small but progressive decrease in mean CST.Trial registrationNCT02140164 (05/2014).