Division of Neonatology and Pediatric Intensive Care, Children's University Hospital of Geneva and University of Geneva, Geneva, Switzerland.
Faculty of Medicine, University of Melbourne, Melbourne, Australia.
Pediatr Res. 2024 Jan;95(1):251-256. doi: 10.1038/s41390-023-02785-x. Epub 2023 Aug 31.
Prophylactic low-dose hydrocortisone (HC) was found to improve survival without bronchopulmonary dysplasia (BPD) in extremely preterm infants. However, appropriately adjusting for baseline risks of BPD or death might substantially increase the precision of the HC effect size.
We conducted a secondary analysis of the PREMILOC trial. The treatment effect was evaluated on the primary endpoint through a covariance analysis ANCOVA, adjusting for the baseline covariates using a mixed linear model. Several sensitivity analyses were conducted to assess the potential heterogeneity of the treatment effect across centers and subpopulations.
The interaction between treatment group and baseline risk for BPD or death was not statistically significant (p = 0.498). After adjusting for the patient's probability of BPD-free survival using baseline predictors alone, the HC treatment exhibited a highly significant effect (OR [95% CI] = 2.053 [1.602-2.501], p = 0.002), with a number needed to treat NNT [95% CI] = 5.8 [4.1-23.0]. Despite a weak interaction with sex, we found a lack of heterogeneity in the treatment effect across specific subpopulations.
In the PREMILOC trial, the beneficial effect of prophylactic HC versus placebo on BPD-free survival in extremely preterm neonates was found to be greater when adjusted to baseline risks of BPD or death.
EudraCT number 2007-002041-20, ClinicalTrial.gov number NCT00623740.
Prophylactic low-dose hydrocortisone (HC) provided past evidence of a beneficial effect in improving survival without BPD in infants born extremely preterm. Adjustment for baseline risks of BPD or death might substantially increase the precision of the HC effect size. The beneficial effect of prophylactic HC vs placebo on BPD-free survival in extremely preterm neonates was found to be greater when adjusted to baseline risks of BPD or death. We evidenced a lack of heterogeneity in the treatment effect in specific subpopulations despite some weak interaction with sex.
预防性低剂量氢化可的松（HC）可改善极早产儿支气管肺发育不良（BPD）的生存率，但适当调整 BPD 或死亡的基线风险可能会大大提高 HC 效果大小的精度。
我们对 PREMILOC 试验进行了二次分析。通过协方差分析（ANCOVA）评估治疗效果，使用混合线性模型根据基线协变量调整治疗效果。进行了几项敏感性分析，以评估治疗效果在中心和亚人群之间的潜在异质性。
治疗组与 BPD 或死亡的基线风险之间的相互作用无统计学意义（p=0.498）。单独使用基线预测因素调整患者 BPD 无生存概率后，HC 治疗具有显著效果（OR[95%CI]=2.053[1.602-2.501]，p=0.002），治疗所需人数 NNT[95%CI]=5.8[4.1-23.0]。尽管与性别存在弱相互作用，但我们发现治疗效果在特定亚人群中没有异质性。
在 PREMILOC 试验中，与安慰剂相比，极早产儿预防性 HC 对 BPD 无生存的有益效果在调整 BPD 或死亡的基线风险后发现更大。
EudraCT 编号 2007-002041-20，ClinicalTrials.gov 编号 NCT00623740。
预防性低剂量氢化可的松（HC）过去曾证明在改善极早产儿无 BPD 的生存率方面具有有益效果。调整 BPD 或死亡的基线风险可能会大大提高 HC 效果大小的精度。与安慰剂相比，极早产儿预防性 HC 对 BPD 无生存的有益效果在调整 BPD 或死亡的基线风险后发现更大。尽管与性别存在一些弱相互作用，但我们发现治疗效果在特定亚人群中没有异质性。
