Fan Fenling, Davis Stacy, Burbridge Claire, Chin Kelly, Friberg Michael, Grünig Ekkehard, Hughes Melanie, Jansa Pavel, Linder Jörg, Rafalski Jennifer, Rodriguez Alvaro Agustin, Randall Jason A
First Affiliated Hospital of Xi'an Jiaotong University, Xi'an, Shaanxi, China.
Janssen Global Services, LLC, a Johnson & Johnson Company, Horsham, PA, USA.
Adv Ther. 2025 May;42(5):2298-2313. doi: 10.1007/s12325-025-03159-x. Epub 2025 Mar 18.
This study explored patient and clinician perspectives on a new fixed-dose combination of macitentan and tadalafil (M/T FDC) in a once-daily single tablet for treatment of pulmonary arterial hypertension (PAH).
Qualitative semi-structured interviews were conducted during the open-label period of the global, phase 3 A DUE clinical trial that evaluated M/T FDC. A subset of enrolled patients (N = 26) and site investigators (N = 18 clinicians) were interviewed. Patients received four tablets during double-blind treatment and could be in one of three arms (macitentan + placebo; tadalafil + placebo; M/T FDC + placebo) followed by M/T FDC (one tablet) during the open-label period. Patients and clinicians were asked to share their experience of pre-trial PAH medication, double-blind treatment, and open-label M/T FDC. Thematic analysis was conducted on blinded data.
Patients preferred the M/T FDC tablet (open-label) over the four tablets during double-blind treatment. Patients were satisfied with M/T FDC, highlighting its positive impact on their psychological well-being, through reducing stress associated with managing multiple pills. All patients indicated that having a single, once-a-day pill for PAH was more convenient and associated with greater treatment adherence. Clinicians highlighted that their patients have a high daily pill burden for PAH and other comorbidities, and prefer treatments with an oral mode of administration that reduce the number of daily pills required. Clinicians felt that M/T FDC would be well received in clinical practice and potentially assist in implementing guideline-recommended combination treatment of PAH.
In this qualitative analysis, all 26 patients and 18 clinicians provided positive feedback on M/T FDC treatment, which was consistent across countries. Reducing the number of pills needed to treat PAH, through use of single-tablet M/T FDC, is highly valued by patients and endorsed by clinicians, who both felt the single-tablet combination therapy could have a positive effect on patients' well-being and increase treatment adherence.
本研究探讨了患者和临床医生对一种新的马昔腾坦和他达拉非固定剂量复方制剂（M/T FDC）每日一次单片疗法治疗肺动脉高压（PAH）的看法。
在评估M/T FDC的全球3期A DUE临床试验的开放标签期进行了定性半结构化访谈。对部分入组患者（N = 26）和研究点研究者（N = 18名临床医生）进行了访谈。患者在双盲治疗期间服用四片药，可能处于三个治疗组之一（马昔腾坦+安慰剂；他达拉非+安慰剂；M/T FDC+安慰剂），然后在开放标签期服用M/T FDC（一片）。患者和临床医生被要求分享他们在试验前PAH用药、双盲治疗和开放标签M/T FDC治疗方面的体验。对盲态数据进行了主题分析。
与双盲治疗期间服用四片药相比，患者更喜欢开放标签的M/T FDC片。患者对M/T FDC感到满意，强调其对心理健康有积极影响，减少了与服用多片药相关的压力。所有患者均表示，PAH每日服用一片药更方便，且治疗依从性更高。临床医生强调，他们的患者因PAH和其他合并症每日服药负担很重，更喜欢口服给药方式且能减少每日所需药片数量的治疗方法。临床医生认为M/T FDC在临床实践中会受到欢迎，并可能有助于实施指南推荐的PAH联合治疗。
在这项定性分析中，所有26名患者和18名临床医生对M/T FDC治疗均给予了积极反馈，且各个国家的反馈一致。通过使用单片M/T FDC减少治疗PAH所需的药片数量，受到患者高度重视并得到临床医生认可，他们都认为单片联合疗法可能对患者的健康产生积极影响并提高治疗依从性。
