自由文本回复的数据挖掘：在度普利尤单抗IIa期概念验证研究中分析患者对鼻息肉慢性鼻窦炎治疗观点的创新方法。
Data Mining of Free-Text Responses: An Innovative Approach to Analyzing Patient Perspectives on Treatment for Chronic Rhinosinusitis with Nasal Polyps in a Phase IIa Proof-of-Concept Study for Dupilumab.
作者信息
Khan Asif H, Abbe Adeline, Falissard Bruno, Carita Paulo, Bachert Claus, Mullol Joaquim, Reaney Matthew, Chao Jingdong, Mannent Leda P, Amin Nikhil, Mahajan Puneet, Pirozzi Gianluca, Eckert Laurent
机构信息
Sanofi, Chilly-Mazarin, France.
Centre de recherche en epidémiologie et santé des populations (CESP), INSERM U1018, Paris, France.
出版信息
Patient Prefer Adherence. 2021 Nov 19;15:2577-2586. doi: 10.2147/PPA.S320242. eCollection 2021.
PURPOSE
Patient perspective is an important and increasingly sought-after complement to clinical assessment. The aim of this study was to transcribe individual patients' experience of treatment in a dupilumab clinical trial through free-text responses with analysis using natural language processing (NLP) to obtain the unique perspective of patients on disease impact and unmet needs with existing treatment to inform future trial design.
PATIENTS AND METHODS
Patients with chronic rhinosinusitis with nasal polyps (CRSwNP) who were enrolled in a Phase IIa randomized controlled trial comparing dupilumab with placebo (NCT01920893) were invited to complete a self-assessment of treatment (SAT) tool at the end of treatment, asking, "What is your opinion on the treatment you had during the trial? What did you like or dislike about the treatment?" Free-text responses were analyzed for the overall cohort and according to treatment assignment using natural language processing including sentiment scoring. In a mixed-methods approach, quantitative patient-reported outcome (PRO) results were utilized to complement the qualitative analysis of free-text responses.
RESULTS
Of 60 patients enrolled in the study, 43 (71.6%) completed the SAT and responses from 37 patients were analyzed (placebo, n = 16; dupilumab, n = 21). Word analyses showed that the most common words were "smell," "improve," "staff," "great," "time," and "good." Across the whole cohort, "smell" was the most common symptom-related word. The words "smell" and "experience" were more likely to occur in patients treated with dupilumab. Patients treated with dupilumab also had more positive sentiment in their SAT responses than those who received placebo. The results from this qualitative analysis were reflected in quantitative PRO results.
CONCLUSION
"Smell" was important to patients with CRSwNP, highlighting its importance as a patient-centric efficacy outcome measure in the context of clinical trials in CRSwNP.
TRIAL REGISTRATION
ClinicalTrials.gov, NCT01920893. Registered 12 August 2013, https://www.clinicaltrials.gov/ct2/show/NCT01920893.
目的
患者视角是临床评估中一种重要且越来越受关注的补充。本研究的目的是通过自由文本回复转录度普利尤单抗临床试验中个体患者的治疗体验，并使用自然语言处理（NLP）进行分析，以获取患者对疾病影响和现有治疗未满足需求的独特视角，为未来试验设计提供信息。
患者与方法
邀请参加比较度普利尤单抗与安慰剂的IIa期随机对照试验（NCT01920893）的慢性鼻-鼻窦炎伴鼻息肉（CRSwNP）患者在治疗结束时完成一份治疗自我评估（SAT）工具，问题为：“你对试验期间接受的治疗有什么看法？你喜欢或不喜欢该治疗的哪些方面？”使用包括情感评分在内的自然语言处理对整个队列的自由文本回复进行分析，并根据治疗分配进行分析。采用混合方法，利用定量的患者报告结局（PRO）结果来补充自由文本回复的定性分析。
结果
在纳入研究的60例患者中，43例（71.6%）完成了SAT，对37例患者的回复进行了分析（安慰剂组，n = 16；度普利尤单抗组，n = 21）。词分析显示，最常见的词是“嗅觉”“改善”“工作人员”“很棒”“时间”和“良好”。在整个队列中，“嗅觉”是最常见的与症状相关的词。“嗅觉”和“体验”这两个词在接受度普利尤单抗治疗的患者中更有可能出现。接受度普利尤单抗治疗的患者在SAT回复中的积极情感也比接受安慰剂的患者更多。这种定性分析的结果反映在定量PRO结果中。
结论
“嗅觉”对CRSwNP患者很重要，突出了其作为CRSwNP临床试验中以患者为中心的疗效结局指标的重要性。
试验注册
ClinicalTrials.gov，NCT01920893。2013年8月12日注册，https://www.clinicaltrials.gov/ct2/show/NCT01920893。
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