比利时鲁汶大学医院Jan A. Deprest团队研究了胎儿手术治疗中度左侧膈疝的预后。相关论文于2021年6月8日发表于《新英格兰医学杂志》上。

胎儿镜下气管球囊堵塞术（FETO）可提高因左侧孤立性先天性膈疝导致的严重肺发育不全婴儿的出生后生存率，但其对中度疾病婴儿的影响仍缺乏资料。

在这项开放标签试验中，研究组在多家有胎儿和其他类型产前手术经验的中心进行了研究，招募单胎且胎儿左侧中度孤立性先天性膈疝的孕妇，将其按1：1随机分组，分别在孕30-32周时接受FETO或预期护理。这两种治疗方法都遵循标准化的产后护理。主要结局是婴儿从新生儿重症监护室（NICU）出院的存活率和6个月大时不补充氧气的存活率。

在涉及196名女性的意向治疗分析中，FETO组98名婴儿中有62名（63%）存活出院，预期护理组98名婴儿中49名（50%）。6个月大时，FETO组中有53名（54%）可不吸氧存活，预期护理组中有43名（44%）。FETO组孕妇早产和产前胎膜破裂的发生率为44%，显著高于预期护理组（12%）；早产发生率为64%，显著高于预期护理组（22%），但FETO与任何其他严重的孕产妇并发症无关。共发生2例无明显原因的胎儿死亡（每组1例）和1例与球囊摘除相关的新生儿死亡。

研究结果表明，对于左侧中度先天性膈疝的胎儿，妊娠30-32周时进行FETO与预期护理相比，并未改善新生儿存活到出院或6个月时不吸氧生存率，反而增加了早产、胎膜破裂和早产的风险。

附：英文原文

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

Author: Jan A. Deprest, M.D., Ph.D.,, Alexandra Benachi, M.D., Ph.D.,, Eduard Gratacos, M.D., Ph.D.,, Kypros H. Nicolaides, M.D.,, Christoph Berg, M.D., Ph.D.,, Nicola Persico, M.D., Ph.D.,, Michael Belfort, M.D., Ph.D.,, Glenn J. Gardener, M.D., Ph.D.,, Yves Ville, M.D., Ph.D.,, Anthony Johnson, M.D.,, Francesco Morini, M.D., Ph.D.,, Mirosaw Wielgo, M.D., Ph.D.,, Ben Van Calster, Ph.D.,, and Philip L.J. DeKoninck, M.D., Ph.D.

Issue&Volume: 2021-06-08

Abstract:

Background

Fetoscopic endoluminal tracheal occlusion (FETO) has been associated with increased postnatal survival among infants with severe pulmonary hypoplasia due to isolated congenital diaphragmatic hernia on the left side, but data are lacking to inform its effects in infants with moderate disease.

Methods

In this open-label trial conducted at many centers with experience in FETO and other types of prenatal surgery, we randomly assigned, in a 1:1 ratio, women carrying singleton fetuses with a moderate isolated congenital diaphragmatic hernia on the left side to FETO at 30 to 32 weeks of gestation or expectant care. Both treatments were followed by standardized postnatal care. The primary outcomes were infant survival to discharge from a neonatal intensive care unit (NICU) and survival without oxygen supplementation at 6 months of age.

Results

In an intention-to-treat analysis involving 196 women, 62 of 98 infants in the FETO group (63%) and 49 of 98 infants in the expectant care group (50%) survived to discharge (relative risk , 1.27; 95% confidence interval [CI], 0.99 to 1.63; two-sided P=0.06). At 6 months of age, 53 of 98 infants (54%) in the FETO group and 43 of 98 infants (44%) in the expectant care group were alive without oxygen supplementation (relative risk, 1.23; 95% CI, 0.93 to 1.65). The incidence of preterm, prelabor rupture of membranes was higher among women in the FETO group than among those in the expectant care group (44% vs. 12%; relative risk, 3.79; 95% CI, 2.13 to 6.91), as was the incidence of preterm birth (64% vs. 22%, respectively; relative risk, 2.86; 95% CI, 1.94 to 4.34), but FETO was not associated with any other serious maternal complications. There were two spontaneous fetal deaths (one in each group) without obvious cause and one neonatal death that was associated with balloon removal.

Conclusions

This trial involving fetuses with moderate congenital diaphragmatic hernia on the left side did not show a significant benefit of FETO performed at 30 to 32 weeks of gestation over expectant care with respect to survival to discharge or the need for oxygen supplementation at 6 months. FETO increased the risks of preterm, prelabor rupture of membranes and preterm birth.

DOI: 10.1056/NEJMoa2026983

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa2026983