Comparative Treatment Outcomes for Patients With Idiopathic Subglottic Stenosis
Comparative Treatment Outcomes for Patients With Idiopathic Subglottic Stenosis
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DOI：
10.1001/jamaoto.2019.3022
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发表时间：
2020-01-01
影响因子：
7.8
通讯作者：
Francis, David O.
中科院分区：
文献类型：
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作者：
Gelbard, Alexander;Anderson, Catherine;Francis, David O.
本研究对810例特发性声门下狭窄患者进行队列研究，比较了三种最常见的手术治疗方法的结果：内镜扩张、内镜切除配合辅助药物治疗或环气管切除术。特发性声门下狭窄（iSGS）的3种最常见手术入路的结果是什么？在这项队列研究中，810例iSGS患者接受了三种最常见的手术治疗中的一种，在3年的研究期间，23%的患者接受了复发性手术治疗，但复发率因治疗方式而异（环气管切除术，1%；内镜切除配合辅助药物治疗，12%；内镜扩张，28%）。在成功治疗的患者中，接受环气管切除术的患者报告的生活质量最高，但围手术期风险最大，长期语音预后最差。这些结果表明，将参与的罕见病社区与外科医生网络整合起来，以促进快速和细致的治疗比较是可行的；研究结果可能有助于为iSGS的治疗决策提供信息。重要性罕见病的手术治疗比较是困难的，其次是患者的地理分布。幸运的是，新兴技术有望减少这些研究障碍。目的前瞻性比较特发性声门下狭窄症（iSGS）的3种最常见手术入路的治疗效果。设计、环境和参与者在这项为期3年的国际前瞻性多中心队列研究中，纳入了810例未经治疗、新诊断或先前治疗过的iSGS患者，这些患者在接受外科手术（内镜扩张[ED]、内镜切除配合辅助药物治疗[ERMT]或环气管切除术[CTR]）后入院。患者从北美气道协作组织和Facebook在线iSGS社区的临床实践中招募。主要终点为从首次手术到复发手术的天数。次要终点包括使用临床COPD（慢性阻塞性肺疾病）问卷（CCQ）、语音障碍指数-10 （VHI-10）、饮食评估测试-10 （EAT-10）、12项简表第2版（SF-12v2）和术后并发症的生活质量。结果810例患者中，女性798例（98.5%），白人787例（97.2%），中位年龄50岁（四分位数间距43-58岁）。主要手术为ED （n = 603; 74.4%）、ERMT （n = 121; 14.9%）和CTR （n = 86; 10.6%）。总体而言，在3年的研究期间，185例（22.8%）患者有过复发性手术，但复发因手术方式而异（CTR， 1例[1.2%]；ERMT， 15例[12.4%]；ED， 169例[28.0%]）。加权、倾向评分匹配、Cox比例风险回归模型显示ED低于ERMT（风险比[HR]， 3.16; 95% CI, 1.8-5.5）。在治疗成功且无复发的患者中，接受CTR治疗的患者在入组后360天CCQ评分最高（0.75分），SF-12v2评分最高（54分），VHI-10评分最低（13分），围手术期风险最高。结论和相关性在这项纳入810例iSGS患者的队列研究中，内镜扩张是最常用的iSGS手术入路，与其他手术相比，其复发率更高。环气管切除术提供了最持久的结果，但显示出最大的围手术期风险和最差的长期语音结果。与ED相比，内窥镜切除联合药物治疗与更好的疾病控制相关，与声带功能的关联最小。这些结果可用于告知个别患者的治疗决策。
This cohort study of 810 patients with idiopathic subglottic stenosis compares the outcomes of the 3 most common surgical treatment procedures: endoscopic dilation, endoscopic resection with adjuvant medication, or cricotracheal resection.Question What are the outcomes of the 3 most common surgical approaches for idiopathic subglottic stenosis (iSGS)? Findings In this cohort study of 810 patients with iSGS who underwent 1 of the 3 most common surgical treatments, 23% of patients underwent a recurrent surgical procedure during the 3-year study period, but recurrence differed by modality (cricotracheal resection, 1%; endoscopic resection with adjuvant medical therapy, 12%; and endoscopic dilation, 28%). Among successfully treated patients, those who underwent cricotracheal resection reported the highest quality of life but the greatest perioperative risk and worst long-term voice outcomes. Meaning These results show the feasibility of integrating an engaged rare disease community with a network of surgeons to facilitate rapid and nuanced treatment comparisons; findings may help inform treatment decision-making in iSGS.Importance Surgical treatment comparisons in rare diseases are difficult secondary to the geographic distribution of patients. Fortunately, emerging technologies offer promise to reduce these barriers for research. Objective To prospectively compare the outcomes of the 3 most common surgical approaches for idiopathic subglottic stenosis (iSGS), a rare airway disease. Design, Setting, and Participants In this international, prospective, 3-year multicenter cohort study, 810 patients with untreated, newly diagnosed, or previously treated iSGS were enrolled after undergoing a surgical procedure (endoscopic dilation [ED], endoscopic resection with adjuvant medical therapy [ERMT], or cricotracheal resection [CTR]). Patients were recruited from clinician practices in the North American Airway Collaborative and an online iSGS community on Facebook. Main Outcomes and Measures The primary end point was days from initial surgical procedure to recurrent surgical procedure. Secondary end points included quality of life using the Clinical COPD (chronic obstructive pulmonary disease) Questionnaire (CCQ), Voice Handicap Index-10 (VHI-10), Eating Assessment Test-10 (EAT-10), the 12-Item Short-Form Version 2 (SF-12v2), and postoperative complications. Results Of 810 patients in this cohort, 798 (98.5%) were female and 787 (97.2%) were white, with a median age of 50 years (interquartile range, 43-58 years). Index surgical procedures were ED (n = 603; 74.4%), ERMT (n = 121; 14.9%), and CTR (n = 86; 10.6%). Overall, 185 patients (22.8%) had a recurrent surgical procedure during the 3-year study, but recurrence differed by modality (CTR, 1 patient [1.2%]; ERMT, 15 [12.4%]; and ED, 169 [28.0%]). Weighted, propensity score-matched, Cox proportional hazards regression models showed ED was inferior to ERMT (hazard ratio [HR], 3.16; 95% CI, 1.8-5.5). Among successfully treated patients without recurrence, those treated with CTR had the best CCQ (0.75 points) and SF-12v2 (54 points) scores and worst VHI-10 score (13 points) 360 days after enrollment as well as the greatest perioperative risk. Conclusions and Relevance In this cohort study of 810 patients with iSGS, endoscopic dilation, the most popular surgical approach for iSGS, was associated with a higher recurrence rate compared with other procedures. Cricotracheal resection offered the most durable results but showed the greatest perioperative risk and the worst long-term voice outcomes. Endoscopic resection with medical therapy was associated with better disease control compared with ED and had minimal association with vocal function. These results may be used to inform individual patient treatment decision-making.