衡量治疗对甲状旁腺功能减退症成人患者症状的影响:PaTHway试验的发现。

IF 2.4 Q2 HEALTH CARE SCIENCES & SERVICES Journal of Patient-Reported Outcomes Pub Date : 2024-08-13 DOI:10.1186/s41687-024-00757-1
Meryl Brod, Kathryn M Pfeiffer, Jane F Beck, Alden Smith
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引用次数: 0

摘要

背景:甲状旁腺功能减退症是一种罕见的内分泌疾病:甲状旁腺功能减退症是一种罕见的内分泌疾病,常伴有严重的躯体和认知症状。本研究旨在了解PaTHway临床试验3期治疗药物TransCon PTH对患者整体、身体和认知甲状旁腺功能减退症体征/症状的影响,以及患者认为有意义的改善:对刚结束 PaTHway 试验盲期的患者进行了个人电话离职访谈。采用半结构化访谈指南,访谈重点是根据甲状旁腺功能减退症患者体验量表-症状(HPES-Symptom)中的症状清单,了解试验治疗对甲状旁腺功能减退症症状的影响。甲状旁腺机能减退症状的有意义变化通过 "患者对严重程度的总体印象"(PGIS)和 "患者对变化的总体印象"(PGIC)进行评估。受访者会被问及从开始试验治疗前到过去两周/目前的症状变化是否有意义。对访谈进行了录音和转录。根据经改编的基础理论方法,对访谈指南中涵盖的新概念和主题/次主题进行编码:19名患有甲状旁腺功能减退症的成年人参加了访谈,他们分别来自美国(13人,占68.4%)和加拿大(6人,占31.6%)。试验治疗组受访者的身体和认知症状明显改善。大多数在试验前报告有甲状旁腺功能减退症躯体症状的受试者在接受治疗后症状都有所改善,包括肌肉抽搐(100%,n = 15)、精力不足(92.9%,n = 13)、感觉疲劳(92.3%,n = 12)、肌肉无力(92.9%,n = 13)、无麻木感的刺痛(84.6%,n = 11)、睡眠障碍(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)。大多数在试验前报告有认知症状的参与者在接受治疗后症状有所改善,包括难以找到正确的词语(86.7%,n = 13)、难以集中注意力(93.3%,n = 14)、记忆困难(92.9%,n = 13)、难以清晰思考(85.7%,n = 12)和难以理解信息(83.3%,n = 10)。安慰剂组的参与者表示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-Symptom 中症状频率的改善是有意义的:研究结果表明,TransCon PTH治疗能有效改善参与者的躯体和认知甲状旁腺功能减退症症状,同时减轻与传统治疗相关的日常负担:https://clinicaltrials.gov/study/NCT04701203?term=NCT04701203&rank=1 。
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Measuring treatment impacts on symptoms in adults with hypoparathyroidism: findings from the PaTHway trial.

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.

Trial registration: NCT04701203 Registered: 06 January 2021. https://clinicaltrials.gov/study/NCT04701203?term=NCT04701203&rank=1 .

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来源期刊
Journal of Patient-Reported Outcomes
Journal of Patient-Reported Outcomes Health Professions-Health Information Management
CiteScore
3.80
自引率
7.40%
发文量
120
审稿时长
20 weeks
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