Division of Otolaryngology, Children's Hospital of Philadelphia, 3500 Civic Center Boulevard, 5th Floor, Philadelphia, PA, 19104, USA.
Division of Pediatric Otolaryngology-HNS, Cincinnati Children's Hospital Medical Center, 3333 Burnet Ave, Cincinnati, OH, 45229, USA.
Int J Pediatr Otorhinolaryngol. 2023 Sep;172:111631. doi: 10.1016/j.ijporl.2023.111631. Epub 2023 Jun 23.
Comorbidities such as chronic lung disease and gastroesophageal reflux (GERD), prematurity, and numerous other conditions may impact the success of LTR. Single-center studies are limited in terms of patient numbers and may be underpowered.
To analyze the impact of specific comorbidities on the operation-specific and overall surgical success of LTR in a large multicenter cohort and validate a predictive model for surgical success.
A large retrospective multicenter 10-year review was undertaken to validate the data of a previous single-center study (Wertz et al. Laryngoscope 2020) which identified specific predictive comorbidities which impacted LTR outcomes. A Monte Carlo simulation based on the previous data set suggested that 300-400 cases would be needed to optimize the statistical power of a Bayesian model developed from the single-center data to predict surgical success. An IRB-approved data-sharing agreement was executed for 4 large U.S.
A virtual REDCap® data entry form inquired about patient characteristics that best predicted surgical success in the single-center model. These included demographics, surgical approaches, cardiac, airway, genetic, endocrine, musculoskeletal, gastrointestinal, and pulmonary comorbidities; details of the surgical procedures, and results of esophagogastroduodenoscopy (EGD), esophageal pH/impedance and flexible bronchoscopy with bronchioalveolar lavage (BAL) were included. Surgical success defined as successful decannulation or resolution of airway symptoms was recorded as single surgery success and overall success following open surgical revision surgery. Multivariate Bayesian analysis, logistical regression, and Kaplan-Meier analysis were performed.
542 patients were identified, including 165 from the single-center study and an additional 377 patients from the multicenter group. The median age was 36 months at the time of the most recent surgery. 70.9% of the LTRs were double-staged procedures. The overall success rate was 86.4% and operation-specific success rate was 69.2%. The specific comorbidities and aerodigestive test results that impacted success based on univariate analysis included staging, bronchiectasis, pulmonary hypertension, GERD, ASD, PDA, grade of stenosis, advanced levels of stenosis, Trisomy 21, MRSA, prior open surgery at another hospital, and gross appearance on EGD. Bayesian model averaging with backward selection was used to validate and refine a predictive model for surgical success with favorable receiver operating curve characteristics - AUC values of 0.827 for single surgery success and 0.797 for overall success.
With over 500 patients reviewed, this was the largest multicenter study of LTR to date, which elucidated the impact of comorbidities on success with LTR and was able to improve upon the predictive modeling based on single-center data. Patient factors are most critical in the outcome of LTR. Stage and levels of stenosis, as well as pulmonary and GI conditions most strongly impact the likelihood of success. Future prospective case-control studies will be performed to further optimize the current model for outcome prediction and patient management.
慢性肺部疾病和胃食管反流病（GERD）、早产以及许多其他疾病等合并症可能会影响喉气管重建术（LTR）的成功。单中心研究在患者数量方面存在局限性，可能存在效能不足的问题。
在一个大型多中心队列中分析特定合并症对 LTR 手术特定和总体手术成功率的影响，并验证手术成功率的预测模型。
进行了一项大型回顾性多中心 10 年研究，验证了之前单中心研究（Wertz 等人，《喉镜》2020 年）的数据，该研究确定了影响 LTR 结果的特定预测性合并症。基于先前数据集的蒙特卡罗模拟表明，需要 300-400 例病例，才能优化从单中心数据开发的贝叶斯模型预测手术成功率的统计能力。为 4 个美国大型中心执行了经机构审查委员会批准的数据共享协议：虚拟 REDCap®数据输入表单询问了在单中心模型中最佳预测手术成功率的患者特征。这些特征包括人口统计学、手术方法、心脏、气道、遗传、内分泌、肌肉骨骼、胃肠道和肺部合并症；手术细节以及食管胃十二指肠镜检查（EGD）、食管 pH/阻抗和支气管肺泡灌洗（BAL）的结果。成功拔管或气道症状缓解定义为单次手术成功，经开放式手术修正后的总体手术成功。进行了多变量贝叶斯分析、逻辑回归和 Kaplan-Meier 分析。
共确定了 542 例患者，其中 165 例来自单中心研究，另有 377 例来自多中心组。最近一次手术时的中位年龄为 36 个月。70.9%的 LTR 为双阶段手术。总体成功率为 86.4%，手术特定成功率为 69.2%。基于单变量分析，影响成功的特定合并症和呼吸道测试结果包括分期、支气管扩张症、肺动脉高压、GERD、房间隔缺损、动脉导管未闭、狭窄程度分级、高级别狭窄、21 三体、耐甲氧西林金黄色葡萄球菌、另一家医院的先前开放性手术，以及 EGD 的大体外观。使用向后选择的贝叶斯模型平均法验证和改进了手术成功率的预测模型，具有良好的接收者操作曲线特征——单次手术成功率的 AUC 值为 0.827，总体成功率的 AUC 值为 0.797。
在对 500 多例患者进行回顾后，这是迄今为止最大的 LTR 多中心研究，阐明了合并症对 LTR 成功的影响，并能够基于单中心数据改进预测模型。患者因素是 LTR 结果的关键。分期和狭窄程度以及肺部和胃肠道状况对成功的可能性影响最大。未来将进行前瞻性病例对照研究，以进一步优化当前的模型，用于预测结果和患者管理。
