{"title":"基于患者报告结果的电子监测系统,用于评估食管鳞状细胞癌患者术前免疫化疗联合或不联合短期化疗的安全性和有效性(ePRO-PICCRT):前瞻性单臂 II 期研究方案。","authors":"Shujie Huang, Hongfan Yu, Zijie Li, Yong Tang, Liling Luo, Junhan Wu, Xianglin Li, Qiuling Shi, Songxi Xie, Guibin Qiao","doi":"10.21037/jtd-24-274","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Radiation-associated adverse events (ADEs) in patients with esophageal squamous cell carcinoma (ESCC) remain a problem. Recent research has focused on reducing radiation-associated ADEs while maintaining efficacy, particularly through the combination of immune checkpoint inhibitors (ICIs) with chemotherapy. Patient-reported outcomes (PROs) have also emerged as reliable measures for monitoring treatment effectiveness and quality of life (QoL). This trial aims to investigate the feasibility of using patient-reported dysphagia relief to assess pathological response following neoadjuvant immunochemotherapy, as well as the safety and efficacy of neoadjuvant immunochemotherapy combined with short-course radiotherapy for patients with locally advanced ESCC.</p><p><strong>Methods: </strong>This study is designed as a prospective, single-arm, phase II study. Eligible ESCC patients will be invited to participate in this study. All participants will receive paclitaxel (albumin-bound) (260 mg/m<sup>2</sup>, day 1), carboplatin [area under the curve (AUC) 5; 5 mg/mL/min, day 1] or cisplatin [60 mg/m<sup>2</sup>, intravenous drip (ivdrip), day 1], and tislelizumab (200 mg, day 1) in the first treatment cycle. Early remission of dysphagia is defined as relief greater than 70% according to the dysphagia symptom score in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire esophagus-specific questionnaire (EORTC OES-18). The early remission group (Group A) will continue with the same regimen for two treatment cycles. The latent remission group will continue with one treatment cycle followed by neoadjuvant immunochemotherapy combined with short-course radiotherapy (radiotherapy 30 Gy/10 F). The primary objective is the pathological complete response (pCR) rate. Research data collection, storage, and management will be conducted in a web-based Real-World-Data Management Platform (RWDMP). Longitudinal data will be conducted by a linear mixed model with treatment effects, baseline factors influencing the endpoint as fixed effects, and the center as a random effect.</p><p><strong>Discussion: </strong>This study will provide evidence for using patient-reported dysphagia relief to evaluate pathological response after neoadjuvant immunochemotherapy in early remission (Group A) and to evaluate the safety and efficacy of combining immunochemotherapy with short-course radiotherapy in latent remission (Group B) among patients with ESCC. Limitations include the single-arm study design, small sample size, and the need for further exploration of the specific mechanism and mediator of early dysphagia remission's effect on immunochemotherapy effectiveness.</p><p><strong>Trial registration: </strong>This study is registered at Clinicaltrials.gov (NCT05596890).</p>","PeriodicalId":17542,"journal":{"name":"Journal of thoracic disease","volume":null,"pages":null},"PeriodicalIF":2.1000,"publicationDate":"2024-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11320245/pdf/","citationCount":"0","resultStr":"{\"title\":\"Electronic patient-reported outcome-based surveillance system to evaluate safety and efficacy of preoperative immunochemotherapy with or without short-term chemoradiation in patients with esophageal squamous cell carcinoma (ePRO-PICCRT): protocol for a prospective, single-arm, phase II study.\",\"authors\":\"Shujie Huang, Hongfan Yu, Zijie Li, Yong Tang, Liling Luo, Junhan Wu, Xianglin Li, Qiuling Shi, Songxi Xie, Guibin Qiao\",\"doi\":\"10.21037/jtd-24-274\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Radiation-associated adverse events (ADEs) in patients with esophageal squamous cell carcinoma (ESCC) remain a problem. Recent research has focused on reducing radiation-associated ADEs while maintaining efficacy, particularly through the combination of immune checkpoint inhibitors (ICIs) with chemotherapy. Patient-reported outcomes (PROs) have also emerged as reliable measures for monitoring treatment effectiveness and quality of life (QoL). This trial aims to investigate the feasibility of using patient-reported dysphagia relief to assess pathological response following neoadjuvant immunochemotherapy, as well as the safety and efficacy of neoadjuvant immunochemotherapy combined with short-course radiotherapy for patients with locally advanced ESCC.</p><p><strong>Methods: </strong>This study is designed as a prospective, single-arm, phase II study. Eligible ESCC patients will be invited to participate in this study. All participants will receive paclitaxel (albumin-bound) (260 mg/m<sup>2</sup>, day 1), carboplatin [area under the curve (AUC) 5; 5 mg/mL/min, day 1] or cisplatin [60 mg/m<sup>2</sup>, intravenous drip (ivdrip), day 1], and tislelizumab (200 mg, day 1) in the first treatment cycle. Early remission of dysphagia is defined as relief greater than 70% according to the dysphagia symptom score in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire esophagus-specific questionnaire (EORTC OES-18). The early remission group (Group A) will continue with the same regimen for two treatment cycles. The latent remission group will continue with one treatment cycle followed by neoadjuvant immunochemotherapy combined with short-course radiotherapy (radiotherapy 30 Gy/10 F). The primary objective is the pathological complete response (pCR) rate. Research data collection, storage, and management will be conducted in a web-based Real-World-Data Management Platform (RWDMP). Longitudinal data will be conducted by a linear mixed model with treatment effects, baseline factors influencing the endpoint as fixed effects, and the center as a random effect.</p><p><strong>Discussion: </strong>This study will provide evidence for using patient-reported dysphagia relief to evaluate pathological response after neoadjuvant immunochemotherapy in early remission (Group A) and to evaluate the safety and efficacy of combining immunochemotherapy with short-course radiotherapy in latent remission (Group B) among patients with ESCC. Limitations include the single-arm study design, small sample size, and the need for further exploration of the specific mechanism and mediator of early dysphagia remission's effect on immunochemotherapy effectiveness.</p><p><strong>Trial registration: </strong>This study is registered at Clinicaltrials.gov (NCT05596890).</p>\",\"PeriodicalId\":17542,\"journal\":{\"name\":\"Journal of thoracic disease\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":2.1000,\"publicationDate\":\"2024-07-30\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11320245/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of thoracic disease\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.21037/jtd-24-274\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/7/8 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"RESPIRATORY SYSTEM\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of thoracic disease","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.21037/jtd-24-274","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/7/8 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"RESPIRATORY SYSTEM","Score":null,"Total":0}
Electronic patient-reported outcome-based surveillance system to evaluate safety and efficacy of preoperative immunochemotherapy with or without short-term chemoradiation in patients with esophageal squamous cell carcinoma (ePRO-PICCRT): protocol for a prospective, single-arm, phase II study.
Background: Radiation-associated adverse events (ADEs) in patients with esophageal squamous cell carcinoma (ESCC) remain a problem. Recent research has focused on reducing radiation-associated ADEs while maintaining efficacy, particularly through the combination of immune checkpoint inhibitors (ICIs) with chemotherapy. Patient-reported outcomes (PROs) have also emerged as reliable measures for monitoring treatment effectiveness and quality of life (QoL). This trial aims to investigate the feasibility of using patient-reported dysphagia relief to assess pathological response following neoadjuvant immunochemotherapy, as well as the safety and efficacy of neoadjuvant immunochemotherapy combined with short-course radiotherapy for patients with locally advanced ESCC.
Methods: This study is designed as a prospective, single-arm, phase II study. Eligible ESCC patients will be invited to participate in this study. All participants will receive paclitaxel (albumin-bound) (260 mg/m2, day 1), carboplatin [area under the curve (AUC) 5; 5 mg/mL/min, day 1] or cisplatin [60 mg/m2, intravenous drip (ivdrip), day 1], and tislelizumab (200 mg, day 1) in the first treatment cycle. Early remission of dysphagia is defined as relief greater than 70% according to the dysphagia symptom score in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire esophagus-specific questionnaire (EORTC OES-18). The early remission group (Group A) will continue with the same regimen for two treatment cycles. The latent remission group will continue with one treatment cycle followed by neoadjuvant immunochemotherapy combined with short-course radiotherapy (radiotherapy 30 Gy/10 F). The primary objective is the pathological complete response (pCR) rate. Research data collection, storage, and management will be conducted in a web-based Real-World-Data Management Platform (RWDMP). Longitudinal data will be conducted by a linear mixed model with treatment effects, baseline factors influencing the endpoint as fixed effects, and the center as a random effect.
Discussion: This study will provide evidence for using patient-reported dysphagia relief to evaluate pathological response after neoadjuvant immunochemotherapy in early remission (Group A) and to evaluate the safety and efficacy of combining immunochemotherapy with short-course radiotherapy in latent remission (Group B) among patients with ESCC. Limitations include the single-arm study design, small sample size, and the need for further exploration of the specific mechanism and mediator of early dysphagia remission's effect on immunochemotherapy effectiveness.
Trial registration: This study is registered at Clinicaltrials.gov (NCT05596890).
期刊介绍:
The Journal of Thoracic Disease (JTD, J Thorac Dis, pISSN: 2072-1439; eISSN: 2077-6624) was founded in Dec 2009, and indexed in PubMed in Dec 2011 and Science Citation Index SCI in Feb 2013. It is published quarterly (Dec 2009- Dec 2011), bimonthly (Jan 2012 - Dec 2013), monthly (Jan. 2014-) and openly distributed worldwide. JTD received its impact factor of 2.365 for the year 2016. JTD publishes manuscripts that describe new findings and provide current, practical information on the diagnosis and treatment of conditions related to thoracic disease. All the submission and reviewing are conducted electronically so that rapid review is assured.