Vitreoretinal Service, Addenbrooke's Hospital, Cambridge University Hospitals NHS Foundation Trust, Cambridge, United Kingdom.
Centre for Applied Medical Statistics (CAMS), University of Cambridge, Cambridge, United Kingdom.
Ophthalmology. 2014 Aug;121(8):1588-97. doi: 10.1016/j.ophtha.2014.02.022. Epub 2014 May 1.
The Stickler syndromes are the most common causes of inherited and childhood retinal detachment; however, no consensus exists regarding the effectiveness of prophylactic intervention. We evaluate the long-term safety and efficacy of the Cambridge prophylactic cryotherapy protocol, a standardized retinal prophylactic treatment developed to prevent retinal detachment arising from giant retinal tears in type 1 Stickler syndrome.
Retrospective comparative case series.
Four hundred eighty seven patients with type 1 Stickler syndrome.
Time to retinal detachment was compared between patients who received bilateral prophylaxis and untreated controls, with and without individual patient matching. Patients receiving unilateral prophylaxis (after fellow eye retinal detachment) were similarly compared with an appropriate control subgroup. Individual patient matching ensured equal age and follow-up between groups and that an appropriate control (who had not suffered a retinal detachment before the age at which their individually matched treatment patient underwent prophylactic treatment) was selected. Matching was blinded to outcome events. Individual patient matching protocols purposely weighted bias against the effectiveness of treatment. All treatment side effects are reported.
Time to retinal detachment and side effects occurring after prophylactic treatment.
The bilateral control group (n = 194) had a 7.4-fold increased risk of retinal detachment compared to the bilateral prophylaxis group (n = 229) (hazard ratio [HR], 7.40; 95% confidence interval [CI], 4.53-12.08; P<0.001); the matched bilateral control group (n = 165) had a 5.0-fold increased risk compared to the matched bilateral prophylaxis group (n = 165) (HR, 4.97; 95% CI, 2.82-8.78; P<0.001). The unilateral control group (n = 104) had a 10.3-fold increased risk of retinal detachment compared to the unilateral prophylaxis group (n = 64) (HR, 10.29; 95% CI, 4.96-21.36; P<0.001); the matched unilateral control group (n = 39) had a 8.4-fold increased risk compared to the matched unilateral prophylaxis group (n = 39) (HR, 8.36; 95% CI, 3.24-21.57; P<0.001). No significant long-term side effects occurred.
In the largest global cohort of type 1 Stickler syndrome patients published, all analyses indicate that the Cambridge prophylactic cryotherapy protocol is safe and markedly reduces the risk of retinal detachment.
斯特奇-韦伯综合征是遗传性和儿童期视网膜脱离最常见的原因；然而，对于预防性干预的效果尚无共识。我们评估了剑桥预防性冷冻疗法方案的长期安全性和有效性，该方案是一种标准化的视网膜预防性治疗方法，旨在预防 1 型斯特奇-韦伯综合征中巨大视网膜裂孔引起的视网膜脱离。
回顾性对比病例系列。
487 名 1 型斯特奇-韦伯综合征患者。
比较接受双侧预防治疗的患者与未接受治疗的对照组之间的视网膜脱离时间，同时进行了有和没有个体患者匹配的比较。同样，对接受单侧预防治疗（在对侧眼视网膜脱离后）的患者与适当的对照组亚组进行了比较。个体患者匹配确保了组间的年龄和随访时间相等，并选择了一个合适的对照组（在接受个体化匹配治疗的患者接受预防性治疗之前，未发生视网膜脱离）。匹配对结果事件进行了盲法处理。个体患者匹配方案有意偏向于治疗的有效性。报告了所有治疗的副作用。
视网膜脱离时间和预防性治疗后发生的副作用。
双侧对照组（n=194）发生视网膜脱离的风险是双侧预防组（n=229）的 7.4 倍（风险比[HR]，7.40；95%置信区间[CI]，4.53-12.08；P<0.001）；匹配后的双侧对照组（n=165）的风险是匹配后的双侧预防组（n=165）的 5.0 倍（HR，4.97；95%CI，2.82-8.78；P<0.001）。单侧对照组（n=104）发生视网膜脱离的风险是单侧预防组（n=64）的 10.3 倍（HR，10.29；95%CI，4.96-21.36；P<0.001）；匹配后的单侧对照组（n=39）发生视网膜脱离的风险是匹配后的单侧预防组（n=39）的 8.4 倍（HR，8.36；95%CI，3.24-21.57；P<0.001）。没有发生明显的长期副作用。
在已发表的最大的 1 型斯特奇-韦伯综合征患者全球队列中，所有分析均表明剑桥预防性冷冻疗法方案是安全的，并显著降低了视网膜脱离的风险。
