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Abstract：
BACKGROUND: Hypoparathyroidism is a rare endocrine disease frequently associated with serious physical and cognitive symptoms. This study\'s purpose was to understand the impacts of the phase 3 PaTHway clinical trial treatment, TransCon PTH, on patients\' overall, physical, and cognitive hypoparathyroidism signs/symptoms and what patients consider meaningful improvement.
METHODS: Individual telephone exit interviews were conducted with patients who recently completed the PaTHway trial blinded period. Using a semi-structured interview guide, interviews focused on trial treatment impact on hypoparathyroidism symptoms following the symptom list in the Hypoparathyroidism Patient Experience Scale-Symptom (HPES-Symptom). Meaningful changes in hypoparathyroidism symptoms were assessed with the Patient Global Impression of Severity (PGIS) and Patient Global Impression of Change (PGIC) measures. Interviewees were probed on the meaningfulness of reported changes in symptoms from prior to starting trial treatment to the past 2 weeks/current time. Interviews were audiotaped and transcribed. Transcripts were coded for emerging concepts and themes/subthemes covered in the interview guide based on an adapted grounded theory approach.
RESULTS: Nineteen adults with hypoparathyroidism participated in interviews in the United States (n = 13, 68.4%) and Canada (n = 6, 31.6%). Marked improvements in physical and cognitive symptoms were described among trial treatment group respondents. The majority of participants who reported experiencing hypoparathyroidism physical symptoms pre-trial indicated symptom improvement with treatment, including muscle twitching (100%, n = 15), low energy (92.9%, n = 13), feeling tired (92.3%, n = 12), muscle weakness (92.9%, n = 13), tingling without numbness (84.6%, n = 11), trouble sleeping (92.3%, n = 12), muscle cramping (92.3%, n = 12), tingling with numbness (92.3%, n = 12), muscle spasms (100%, n = 12), and pain (90.9%, n = 10). Most participants who reported experiencing cognitive symptoms pre-trial reported symptom improvement with treatment, including difficulty finding the right words (86.7%, n = 13), difficulty concentrating (93.3%, n = 14), trouble remembering (92.9%, n = 13), trouble thinking clearly (85.7%, n = 12), and difficulty understanding information (83.3%, n = 10). Those in the placebo group reported limited or no improvement. The vast majority of participants affirmed that the improvements they experienced in symptom frequency on the PGIS/PGIC and HPES-Symptom were meaningful.
CONCLUSIONS: Findings indicate that TransCon PTH treatment improved participants\' physical and cognitive hypoparathyroidism symptoms in meaningful ways, while reducing the daily burden associated with conventional therapy.
BACKGROUND: NCT04701203 Registered: 06 January 2021. https://clinicaltrials.gov/study/NCT04701203?term=NCT04701203&rank=1 .
METHODS: Individual telephone exit interviews were conducted with patients who recently completed the PaTHway trial blinded period. Using a semi-structured interview guide, interviews focused on trial treatment impact on hypoparathyroidism symptoms following the symptom list in the Hypoparathyroidism Patient Experience Scale-Symptom (HPES-Symptom). Meaningful changes in hypoparathyroidism symptoms were assessed with the Patient Global Impression of Severity (PGIS) and Patient Global Impression of Change (PGIC) measures. Interviewees were probed on the meaningfulness of reported changes in symptoms from prior to starting trial treatment to the past 2 weeks/current time. Interviews were audiotaped and transcribed. Transcripts were coded for emerging concepts and themes/subthemes covered in the interview guide based on an adapted grounded theory approach.
RESULTS: Nineteen adults with hypoparathyroidism participated in interviews in the United States (n = 13, 68.4%) and Canada (n = 6, 31.6%). Marked improvements in physical and cognitive symptoms were described among trial treatment group respondents. The majority of participants who reported experiencing hypoparathyroidism physical symptoms pre-trial indicated symptom improvement with treatment, including muscle twitching (100%, n = 15), low energy (92.9%, n = 13), feeling tired (92.3%, n = 12), muscle weakness (92.9%, n = 13), tingling without numbness (84.6%, n = 11), trouble sleeping (92.3%, n = 12), muscle cramping (92.3%, n = 12), tingling with numbness (92.3%, n = 12), muscle spasms (100%, n = 12), and pain (90.9%, n = 10). Most participants who reported experiencing cognitive symptoms pre-trial reported symptom improvement with treatment, including difficulty finding the right words (86.7%, n = 13), difficulty concentrating (93.3%, n = 14), trouble remembering (92.9%, n = 13), trouble thinking clearly (85.7%, n = 12), and difficulty understanding information (83.3%, n = 10). Those in the placebo group reported limited or no improvement. The vast majority of participants affirmed that the improvements they experienced in symptom frequency on the PGIS/PGIC and HPES-Symptom were meaningful.
CONCLUSIONS: Findings indicate that TransCon PTH treatment improved participants\' physical and cognitive hypoparathyroidism symptoms in meaningful ways, while reducing the daily burden associated with conventional therapy.
BACKGROUND: NCT04701203 Registered: 06 January 2021. https://clinicaltrials.gov/study/NCT04701203?term=NCT04701203&rank=1 .
摘要：
背景：甲状旁腺功能减退症是一种罕见的内分泌疾病，常伴有严重的身体和认知症状。本研究的目的是了解3期PATHway临床试验治疗的影响，TransConPTH,对病人来说，总的来说，物理，和认知甲状旁腺功能减退体征/症状以及患者认为有意义的改善。
方法：对最近完成PATHway试验盲期的患者进行单独的电话退出访谈。使用半结构化面试指南，根据甲状旁腺功能减退症患者体验量表-症状(HPES-症状)中的症状列表,访谈重点关注试验治疗对甲状旁腺功能减退症症状的影响.使用患者严重程度整体印象（PGIS）和患者整体变化印象（PGIC）评估甲状旁腺功能减退症状的有意义变化。受访者调查了从开始试验治疗前到过去2周/当前时间报告的症状变化的意义。面试被录音和转录。根据适应的扎根理论方法，为面试指南中涵盖的新兴概念和主题/子主题对成绩单进行了编码。
结果：在美国（n=13，68.4％）和加拿大（n=6，31.6％），有19名甲状旁腺功能减退的成年人参加了访谈。试验治疗组受访者描述了身体和认知症状的明显改善。大多数报告经历甲状旁腺功能减退的身体症状的参与者在试验前表明症状改善与治疗，包括肌肉抽搐(100%，n=15),低能耗（92.9%，n=13),感到疲倦（92.3%，n=12),肌肉无力（92.9%，n=13),刺痛无麻木(84.6%，n=11),睡眠困难(92.3%，n=12),肌肉痉挛（92.3%，n=12),麻木刺痛(92.3%，n=12),肌肉痉挛（100%，n=12),和疼痛（90.9%，n=10)。大多数报告在试验前出现认知症状的参与者报告说，治疗后症状有所改善，包括很难找到合适的单词(86.7%，n=13),难以集中注意力(93.3%，n=14),记忆困难(92.9%，n=13),思考问题(85.7%，n=12),和难以理解信息(83.3%，n=10)。安慰剂组报告的改善有限或没有改善。绝大多数参与者确认，他们在PGIS/PGIC和HPES症状的症状频率方面所经历的改善是有意义的。
结论：研究结果表明，TransConPTH治疗可以有效改善参与者的身体和认知甲状旁腺功能减退症状，同时减少与常规治疗相关的日常负担。
背景：NCT04701203注册时间：2021年1月6日。https://clinicaltrials.gov/study/NCT04701203?term=NCT04701203&rank=1.
方法：对最近完成PATHway试验盲期的患者进行单独的电话退出访谈。使用半结构化面试指南，根据甲状旁腺功能减退症患者体验量表-症状(HPES-症状)中的症状列表,访谈重点关注试验治疗对甲状旁腺功能减退症症状的影响.使用患者严重程度整体印象（PGIS）和患者整体变化印象（PGIC）评估甲状旁腺功能减退症状的有意义变化。受访者调查了从开始试验治疗前到过去2周/当前时间报告的症状变化的意义。面试被录音和转录。根据适应的扎根理论方法，为面试指南中涵盖的新兴概念和主题/子主题对成绩单进行了编码。
结果：在美国（n=13，68.4％）和加拿大（n=6，31.6％），有19名甲状旁腺功能减退的成年人参加了访谈。试验治疗组受访者描述了身体和认知症状的明显改善。大多数报告经历甲状旁腺功能减退的身体症状的参与者在试验前表明症状改善与治疗，包括肌肉抽搐(100%，n=15),低能耗（92.9%，n=13),感到疲倦（92.3%，n=12),肌肉无力（92.9%，n=13),刺痛无麻木(84.6%，n=11),睡眠困难(92.3%，n=12),肌肉痉挛（92.3%，n=12),麻木刺痛(92.3%，n=12),肌肉痉挛（100%，n=12),和疼痛（90.9%，n=10)。大多数报告在试验前出现认知症状的参与者报告说，治疗后症状有所改善，包括很难找到合适的单词(86.7%，n=13),难以集中注意力(93.3%，n=14),记忆困难(92.9%，n=13),思考问题(85.7%，n=12),和难以理解信息(83.3%，n=10)。安慰剂组报告的改善有限或没有改善。绝大多数参与者确认，他们在PGIS/PGIC和HPES症状的症状频率方面所经历的改善是有意义的。
结论：研究结果表明，TransConPTH治疗可以有效改善参与者的身体和认知甲状旁腺功能减退症状，同时减少与常规治疗相关的日常负担。
背景：NCT04701203注册时间：2021年1月6日。https://clinicaltrials.gov/study/NCT04701203?term=NCT04701203&rank=1.
