当前位置:科学网首页 > 小柯机器人 >详情
胎儿镜下气管球囊阻塞术治疗重度左膈疝胎儿可显著改善预后
作者:小柯机器人 发布时间:2021/6/9 15:28:36

比利时鲁汶大学医院Jan A. Deprest团队研究了胎儿手术治疗重度左膈疝对预后的影响。2021年6月8日出版的《新英格兰医学杂志》发表了这一最新研究成果。

观察性研究显示,胎儿镜下气管球囊阻塞术(FETO)可增加因左侧孤立性先天性膈疝导致的严重肺发育不良婴儿的生存率,但缺乏随机试验数据。

研究组在有FETO和其他类型产前手术经验的机构中进行了一项开放标签试验,招募单胎且胎儿有严重左侧孤立性先天性膈疝的孕妇,将其按1:1随机分组,分别在孕27-29周时接受FETO或预期护理。两组均接受标准产后护理。主要结局是婴儿存活到新生儿重症监护病房出院。研究组采用组-序贯设计和5个预先指定的中期分析,最大样本量为116名女性。

第三次中期分析后,研究组因疗效提前终止试验。在一项包括80名女性的意向治疗分析中,FETO组有40%(16/40)的婴儿存活出院,显著高于预期护理组的15%(6/40)。6个月生存率与出院生存率相同。FETO组的孕妇早产和产前胎膜破裂的发生率为47%,显著高于预期护理组(11%);早产发生率为75%,显著高于预期护理组(29%)。1例新生儿死亡发生在紧急分娩后,原因是胎镜下气囊取出导致胎盘撕裂,1例新生儿死亡原因是气囊取出失败。在一项包括11名其他参与者的分析中,在试验停止后有可用数据,FETO组婴儿的出院存活率为36%,高于预期护理组的14%。

研究结果表明,对于左侧孤立性严重先天性膈疝的胎儿,在妊娠27-29周时进行FETO,与预期护理相比,在生存到出院方面均有显著的临床获益,且这种益处持续到6个月大。但FETO增加了早产、胎膜破裂和早产的风险。

附:英文原文

Title: Randomized Trial of Fetal Surgery for Severe Left Diaphragmatic Hernia | NEJM

Author: Jan A. Deprest, M.D., Ph.D.,, Kypros H. Nicolaides, M.D.,, Alexandra Benachi, M.D., Ph.D.,, Eduard Gratacos, M.D., Ph.D.,, Greg Ryan, M.D.,, Nicola Persico, M.D., Ph.D.,, Haruhiko Sago, M.D., Ph.D.,, Anthony Johnson, M.D.,, Mirosaw Wielgo, M.D., Ph.D.,, Christoph Berg, M.D., Ph.D.,, Ben Van Calster, Ph.D.,, and Francesca M. Russo, M.D., Ph.D.

Issue&Volume: 2021-06-08

Abstract:

Background

Observational studies have shown that fetoscopic endoluminal tracheal occlusion (FETO) has been associated with increased survival among infants with severe pulmonary hypoplasia due to isolated congenital diaphragmatic hernia on the left side, but data from randomized trials are lacking.

Methods

In this open-label trial conducted at centers with experience in FETO and other types of prenatal surgery, we randomly assigned, in a 1:1 ratio, women carrying singleton fetuses with severe isolated congenital diaphragmatic hernia on the left side to FETO at 27 to 29 weeks of gestation or expectant care. Both treatments were followed by standardized postnatal care. The primary outcome was infant survival to discharge from the neonatal intensive care unit. We used a group-sequential design with five prespecified interim analyses for superiority, with a maximum sample size of 116 women.

Results

The trial was stopped early for efficacy after the third interim analysis. In an intention-to-treat analysis that included 80 women, 40% of infants (16 of 40) in the FETO group survived to discharge, as compared with 15% (6 of 40) in the expectant care group (relative risk, 2.67; 95% confidence interval [CI], 1.22 to 6.11; two-sided P=0.009). Survival to 6 months of age was identical to the survival to discharge (relative risk, 2.67; 95% CI, 1.22 to 6.11). The incidence of preterm, prelabor rupture of membranes was higher among women in the FETO group than among those in the expectant care group (47% vs. 11%; relative risk, 4.51; 95% CI, 1.83 to 11.9), as was the incidence of preterm birth (75% vs. 29%; relative risk, 2.59; 95% CI, 1.59 to 4.52). One neonatal death occurred after emergency delivery for placental laceration from fetoscopic balloon removal, and one neonatal death occurred because of failed balloon removal. In an analysis that included 11 additional participants with data that were available after the trial was stopped, survival to discharge was 36% among infants in the FETO group and 14% among those in the expectant care group (relative risk, 2.65; 95% CI, 1.21 to 6.09).

Conclusions

In fetuses with isolated severe congenital diaphragmatic hernia on the left side, FETO performed at 27 to 29 weeks of gestation resulted in a significant benefit over expectant care with respect to survival to discharge, and this benefit was sustained to 6 months of age. FETO increased the risks of preterm, prelabor rupture of membranes and preterm birth.

DOI: 10.1056/NEJMoa2027030

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa2027030

 

期刊信息

The New England Journal of Medicine:《新英格兰医学杂志》,创刊于1812年。隶属于美国麻省医学协会,最新IF:70.67
官方网址:http://www.nejm.org/
投稿链接:http://www.nejm.org/page/author-center/home