The Brod Group, 219 Julia Ave., Mill Valley, CA, 94941, USA.
Ascendis Pharma, Inc., 1000 Page Mill Road, Palo Alto, CA, 94304, USA.
J Patient Rep Outcomes. 2024 Aug 13;8(1):94. doi: 10.1186/s41687-024-00757-1.
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.
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.
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.
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.
NCT04701203 Registered: 06 January 2021. https://clinicaltrials.gov/study/NCT04701203?term=NCT04701203&rank=1 .
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 TransCon PTH 对患者总体、身体和认知甲状旁腺功能减退症体征/症状的影响，以及患者认为何种改善有意义。
对最近完成 PaTHway 试验盲法期的患者进行了个体电话退出访谈。使用半结构化访谈指南，访谈重点是试验治疗对 Hypoparathyroidism Patient Experience Scale-Symptom（HPES-Symptom）症状列表中甲状旁腺功能减退症症状的影响。使用患者总体严重程度印象量表（PGIS）和患者总体变化印象量表（PGIC）来评估甲状旁腺功能减退症症状的有意义变化。受访者被询问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有意义程度。访谈进行了录音和转录。根据改编的扎根理论方法，对转录本进行了新兴概念和访谈指南中涵盖的主题/子主题的编码。
19 名患有甲状旁腺功能减退症的成年人参加了在美国（n=13，68.4%）和加拿大（n=6，31.6%）进行的访谈。试验治疗组受访者描述了身体和认知症状的明显改善。大多数在试验前报告有甲状旁腺功能减退症身体症状的参与者表示治疗后症状有所改善，包括肌肉抽搐（100%，n=15）、能量低下（92.9%，n=13）、疲倦感（92.3%，n=12）、肌肉无力（92.9%，n=13）、无麻木刺痛感（84.6%，n=11）、睡眠困难（92.3%，n=12）、肌肉痉挛（92.3%，n=12）、麻木刺痛感（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表示治疗后症状有所改善，包括难以找到合适的词语（86.7%，n=13）、注意力集中困难（93.3%，n=14）、记忆力差（92.9%，n=13）、思维清晰困难（85.7%，n=12）和理解信息困难（83.3%，n=10）。安慰剂组的报告改善有限或没有改善。绝大多数参与者肯定他们在 PGIS/PGIC 和 HPES-Symptom 上的症状频率改善是有意义的。
研究结果表明，TransCon PTH 治疗以有意义的方式改善了参与者的身体和认知甲状旁腺功能减退症症状，同时减轻了与传统治疗相关的日常负担。
NCT04701203 注册：2021 年 1 月 6 日。https://clinicaltrials.gov/study/NCT04701203?term=NCT04701203&rank=1。
