早产儿微创表面活性剂治疗两年后的结局：随机临床试验 OPTIMIST 的随访。

Two-Year Outcomes After Minimally Invasive Surfactant Therapy in Preterm Infants: Follow-Up of the OPTIMIST-A Randomized Clinical Trial.

机构信息

Menzies Institute for Medical Research, University of Tasmania, Hobart, Tasmania, Australia.

Department of Paediatrics, Royal Hobart Hospital, Hobart, Tasmania, Australia.

出版信息

JAMA. 2023 Sep 19;330(11):1054-1063. doi: 10.1001/jama.2023.15694.

DOI:10.1001/jama.2023.15694

PMID:37695601

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10495923/

Abstract

IMPORTANCE

The long-term effects of surfactant administration via a thin catheter (minimally invasive surfactant therapy [MIST]) in preterm infants with respiratory distress syndrome remain to be definitively clarified.

OBJECTIVE

To examine the effect of MIST on death or neurodevelopmental disability (NDD) at 2 years' corrected age.

DESIGN, SETTING, AND PARTICIPANTS: Follow-up study of a randomized clinical trial with blinding of clinicians and outcome assessors conducted in 33 tertiary-level neonatal intensive care units in 11 countries. The trial included 486 infants with a gestational age of 25 to 28 weeks supported with continuous positive airway pressure (CPAP). Collection of follow-up data at 2 years' corrected age was completed on December 9, 2022.

INTERVENTIONS

Infants assigned to MIST (n = 242) received exogenous surfactant (200 mg/kg poractant alfa) via a thin catheter; those assigned to the control group (n = 244) received sham treatment.

MAIN OUTCOMES AND MEASURES

The key secondary outcome of death or moderate to severe NDD was assessed at 2 years' corrected age. Other secondary outcomes included components of this composite outcome, as well as hospitalizations for respiratory illness and parent-reported wheezing or breathing difficulty in the first 2 years.

RESULTS

Among the 486 infants randomized, 453 had follow-up data available (median gestation, 27.3 weeks; 228 females [50.3%]); data on the key secondary outcome were available in 434 infants. Death or NDD occurred in 78 infants (36.3%) in the MIST group and 79 (36.1%) in the control group (risk difference, 0% [95% CI, -7.6% to 7.7%]; relative risk [RR], 1.0 [95% CI, 0.81-1.24]); components of this outcome did not differ significantly between groups. Secondary respiratory outcomes favored the MIST group. Hospitalization with respiratory illness occurred in 49 infants (25.1%) in the MIST group vs 78 (38.2%) in the control group (RR, 0.66 [95% CI, 0.54-0.81]) and parent-reported wheezing or breathing difficulty in 73 (40.6%) vs 104 (53.6%), respectively (RR, 0.76 [95% CI, 0.63-0.90]).

CONCLUSIONS AND RELEVANCE

In this follow-up study of a randomized clinical trial of preterm infants with respiratory distress syndrome supported with CPAP, MIST compared with sham treatment did not reduce the incidence of death or NDD by 2 years of age. However, infants who received MIST had lower rates of adverse respiratory outcomes during their first 2 years of life.

TRIAL REGISTRATION

anzctr.org.au Identifier: ACTRN12611000916943.

摘要

重要性

经细导管给予表面活性剂（微创表面活性剂治疗 [MIST]）治疗患有呼吸窘迫综合征的早产儿的长期影响仍需明确。

目的

研究 MIST 对校正 2 年时死亡或神经发育障碍（NDD）的影响。

设计、地点和参与者：在 11 个国家的 33 个三级新生儿重症监护病房进行的一项随机临床试验的随访研究，对临床医生和结局评估者进行了盲法。该试验纳入了 486 名胎龄为 25 至 28 周、接受持续气道正压通气（CPAP）支持的婴儿。2022 年 12 月 9 日完成了校正 2 年时的随访数据收集。

干预措施

MIST 组（n = 242）接受了 200mg/kg 猪肺磷脂的外源性表面活性剂（poractant alfa）经细导管给予；对照组（n = 244）接受了假处理。

主要结局和测量指标

校正 2 年时的关键次要结局为死亡或中重度 NDD。其他次要结局包括该复合结局的组成部分，以及校正 2 年内因呼吸疾病住院和父母报告的喘息或呼吸困难的情况。

结果

在随机分组的 486 名婴儿中，453 名有随访数据（中位胎龄，27.3 周；女性 228 名 [50.3%]）；434 名婴儿有关于关键次要结局的数据。MIST 组有 78 名婴儿（36.3%）死亡或发生 NDD，对照组有 79 名（36.1%）（风险差异，0%[95%CI，-7.6%至 7.7%]；相对风险 [RR]，1.0[95%CI，0.81-1.24]）；该结局的组成部分在组间无显著差异。次要呼吸结局有利于 MIST 组。MIST 组有 49 名婴儿（25.1%）因呼吸疾病住院，对照组有 78 名（38.2%）（RR，0.66[95%CI，0.54-0.81]），父母报告的喘息或呼吸困难分别为 73 名（40.6%）和 104 名（53.6%）（RR，0.76[95%CI，0.63-0.90]）。