不同剂量口服和眼用普萘洛尔治疗早产儿视网膜病变的比较:网络 Meta 分析

IF 3.4 3区 医学 Q1 PEDIATRICS
Pediatric Drugs Pub Date : 2024-09-01 Epub Date: 2024-08-07 DOI:10.1007/s40272-024-00647-5
Amparo Ortiz-Seller, Pablo Martorell, Patricia Roselló, Esteban Morcillo, José L Ortiz
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引用次数: 0

摘要

目的:普萘洛尔治疗早产儿视网膜病变(ROP)的有效性和安全性仍存在争议。这项网络荟萃分析(NMA)的重点是,是否可以根据不同剂量水平的普萘洛尔治疗早产儿视网膜病变的疗效,以阶段进展为主要结果,以出现加号病变和是否需要使用抗血管内皮生长因子(anti-VEGFs)或激光治疗为次要终点,对普萘洛尔的疗效进行排序:从截至 2024 年 4 月的数据库中检索了 14 项研究(10 项随机对照试验、3 项单臂试验和 1 项回顾性观察研究),涉及 474 名接受口服或眼用普萘洛尔治疗的患者。采用 Meta-insight 和基于模型的 NMA 评估普萘洛尔的剂量-反应关系。对研究进行了模型拟合度、偏倚风险和证据可信度的评估。效应大小以几率比(OR)和 95% 可信区间(CrI)确定:贝叶斯分析显示,在 ROP 进展的晚期(2-3 期;S23)给予普萘洛尔与在早期(0-1 期;S01)给予普萘洛尔相比,效果呈改善趋势。在S23期口服普萘洛尔1.5和2毫克/千克/天的OR值分别为0.13(95% CrI 0.04-0.37)和0.16(95% CrI 0.04-0.61),而在S01期口服普萘洛尔1.5和2毫克/千克/天的OR值分别为0.28(95% CrI 0.02-2.96)和0.78(95% CrI 0.14-4.43)。同样,眼用普萘洛尔 0.2% 在 S23 期的 OR 值为 0.37(95% CrI 0.09-1.00),而 S01 期的 OR 值为 0.64(95% CrI 0.21-2.04)。累积排名曲线下表面(SUCRA)分析证实,口服普萘洛尔 1.5-2 mg/kg 的概率值最佳,其次是眼用普萘洛尔 0.2%,均为 S23。基于模型的 NMA 显示,口服普萘洛尔的剂量-反应呈非线性,晚期用药比早期用药具有更大的最大效应。在次要终点方面,口服普萘洛尔 1.5 毫克/千克/天后,S23 期病情恶化(OR 0.14;95% CrI 0.02-0.84)、需要抗血管内皮生长因子(OR 0.23;95% CrI 0.05-0.93)和激光(OR 0.16;95% CrI 0.02-1.10)的风险值较低,眼用普萘洛尔 0.2% 的风险值也较低,SUCRA 分析也得出了类似的结果。较低剂量(0.5-1.0 毫克/千克/天)的口服普萘洛尔仍具有疗效。不良事件的威胁按与对照组的风险差异估算,眼用普萘洛尔0.2%和口服普萘洛尔0.5毫克/千克/天没有差异,口服普萘洛尔1.0和1.5毫克/千克/天的风险略有增加,口服普萘洛尔2.0毫克/千克/天的风险差异最大(0.06;95% CI -0.01至0.13):结论:普萘洛尔治疗 ROP 可降低疾病进展和需要额外治疗的风险,但安全性是一个潜在问题。普萘洛尔滴眼液(0.2%)与口服普萘洛尔一样有效。尽管如此,由于现有研究数量少、质量低,因此证据有限。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Comparison of Different Doses of Oral and Ocular Propranolol for Retinopathy of Prematurity: A Network Meta-Analysis.

Comparison of Different Doses of Oral and Ocular Propranolol for Retinopathy of Prematurity: A Network Meta-Analysis.

Objective: The efficacy and safety of propranolol for retinopathy of prematurity (ROP) remain under debate. This network meta-analysis (NMA) focuses on whether a ranking may be established for different dose levels of propranolol as treatment of ROP in terms of stage progression as the primary outcome, with appearance of plus disease and need for anti-vascular endothelial growth factors (anti-VEGFs) or laser therapy as secondary endpoints.

Methods: Fourteen studies (10 randomised controlled trials, three single-arm trials and one retrospective observational study) of 474 patients treated with oral or ocular propranolol were retrieved from databases up to April 2024. Meta-insight and model-based NMA were undertaken to evaluate the propranolol dose-response relationship. Studies were evaluated for model fit, risk of bias and Confidence of evidence In Network Meta-Analysis (CINeMA). Effect sizes were determined as odds ratio (OR) with 95% credible interval (CrI).

Results: Bayesian analysis showed a trend towards improved effects for propranolol given at late stages (stages 2-3; S23) of ROP progression compared with its administration at earlier stages (stages 0-1; S01). OR values for oral propranolol 1.5 and 2 mg/kg/day given at S23 were 0.13 (95% CrI 0.04-0.37) and 0.16 (95% CrI 0.04-0.61), respectively, while given at S01 were 0.28 (95% CrI 0.02-2.96) and 0.78 (95% CrI 0.14-4.43), respectively. Similarly, OR of eye propranolol 0.2% at S23 was 0.37 (95% CrI 0.09-1.00) versus an S01 OR of 0.64 (95% CrI 0.21-2.04). Surface under the cumulative ranking curve (SUCRA) analyses confirmed best probability values for oral propranolol 1.5-2 mg/kg followed by eye propranolol 0.2%, all at S23. Model-based NMA showed nonlinearity in the dose-response for oral propranolol with a trend to greater maximal effect for its administration at late versus early stages. For secondary endpoints, lower risk values were found with oral propranolol 1.5 mg/kg/day at S23 for progression to plus disease (OR 0.14; 95% CrI 0.02-0.84) and need for anti-VEGFs (OR 0.23; 95% CrI 0.05-0.93) and laser (OR 0.16; 95% CrI 0.02-1.10) therapies also followed by eye propranolol 0.2%, and a similar profile was obtained with SUCRA analysis. Lower doses (0.5-1.0 mg/kg/day) of oral propranolol retained efficacy. Threat of adverse events was estimated as risk difference versus control with no difference for eye propranolol 0.2% and oral propranolol 0.5 mg/kg/day, modest increases of risk for oral propranolol 1.0 and 1.5 mg/kg/day and the highest risk difference for oral propranolol 2.0 mg/kg/day (0.06; 95% CI -0.01 to 0.13).

Conclusion: A diminished risk of disease progression and need for additional treatment was obtained with propranolol in ROP, but safety is a potential concern. Propranolol eye micro-drops (0.2%) can be as efficacious as oral propranolol. Nonetheless, the evidence is limited due to the paucity and quality of the available studies.

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来源期刊
Pediatric Drugs
Pediatric Drugs PEDIATRICS-PHARMACOLOGY & PHARMACY
CiteScore
7.20
自引率
0.00%
发文量
54
审稿时长
>12 weeks
期刊介绍: Pediatric Drugs promotes the optimization and advancement of all aspects of pharmacotherapy for healthcare professionals interested in pediatric drug therapy (including vaccines). The program of review and original research articles provides healthcare decision makers with clinically applicable knowledge on issues relevant to drug therapy in all areas of neonatology and the care of children and adolescents. The Journal includes: -overviews of contentious or emerging issues. -comprehensive narrative reviews of topics relating to the effective and safe management of drug therapy through all stages of pediatric development. -practical reviews covering optimum drug management of specific clinical situations. -systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by the PRISMA statement. -Adis Drug Reviews of the properties and place in therapy of both newer and established drugs in the pediatric population. -original research articles reporting the results of well-designed studies with a strong link to clinical practice, such as clinical pharmacodynamic and pharmacokinetic studies, clinical trials, meta-analyses, outcomes research, and pharmacoeconomic and pharmacoepidemiological studies. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Pediatric Drugs may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.
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