Abbott Northwestern's Heart Hospital, Minneapolis Heart Institute, 800 East 28th Street, 2nd Floor, Minneapolis, MN, 55407, USA.
Cleveland Clinic, Cleveland, OH, USA.
BMC Cardiovasc Disord. 2021 Apr 21;21(1):201. doi: 10.1186/s12872-021-02008-3.
Impact of recurrent pericarditis (RP) on patient health-related quality of life (HRQoL) was evaluated through qualitative patient interviews and as an exploratory endpoint in a Phase 2 trial evaluating the efficacy and safety of rilonacept (IL-1α/IL-1β cytokine trap) to treat RP.
Qualitative interviews were conducted with ten adults with RP to understand symptoms and HRQoL impacts, and the 10-item Patient-Reported Outcomes Measurement Information System Global Health (PROMIS GH) v1.2 was evaluated to determine questionnaire coverage of patient experience. The Phase 2 trial enrolled participants with active symptomatic RP (A-RP, n = 16) and corticosteroid-dependent participants with no active recurrence at baseline (CSD-RP, n = 9). All participants received rilonacept weekly during a 6-week base treatment period (TP) plus an optional 18-week extension period (EP). Tapering of concomitant medications, including corticosteroids (CS), was permitted during EP. HRQoL was assessed using the PROMIS GH, and patient-reported pain and blood levels of c-reactive protein (CRP) were collected at Baseline and follow-up periods. A secondary, descriptive analysis of the Phase 2 trial efficacy results was completed using HRQoL measures to characterize both the impact of RP and the treatment effect of rilonacept.
Information from qualitative interviews demonstrated that PROMIS GH concepts are relevant to adults with RP. From the Phase 2 trial, both participant groups showed impacted HRQoL at Baseline (mean PROMIS Global Physical Health [GPH] and Global Mental Health [GMH], were lower than population norm average). In A-RP, GPH/MPH improved by end of base TP and were sustained through EP (similar trends were observed for pain and CRP). Similarly, in CSD-RP, GPH/MPH improved by end of TP and further improved during EP, during CS tapering or discontinuation, without disease recurrence (low pain scores and CRP levels continued during the TP and EP).
This is the first study demonstrating impaired HRQoL in RP. Rilonacept treatment was associated with HRQoL improvements using PROMIS GH scores. Maintained/improved HRQoL during tapering/withdrawal of CS without recurrence suggests that rilonacept may provide an alternative to CS.
ClinicalTrials.Gov; NCT03980522; 5 June 2019, retrospectively registered; https://clinicaltrials.gov/ct2/show/NCT03980522 .
通过对复发性心包炎（RP）患者进行定性访谈，并在评估 rilonacept（IL-1α/IL-1β细胞因子陷阱）治疗 RP 的疗效和安全性的 2 期试验中作为探索性终点，评估 RP 对患者健康相关生活质量（HRQoL）的影响。
对 10 名 RP 成人患者进行定性访谈，以了解症状和 HRQoL 影响，并评估 10 项患者报告的结局测量信息系统全球健康（PROMIS GH）v1.2 问卷，以确定问卷对患者体验的涵盖范围。2 期试验纳入了 16 名有活动性症状性 RP（A-RP）的参与者和 9 名基线时无活动性复发但依赖皮质类固醇（CS）的参与者。所有参与者在 6 周的基础治疗期（TP）内每周接受 rilonacept 治疗，外加可选的 18 周扩展期（EP）。在 EP 期间允许同时使用药物（包括皮质类固醇（CS））进行减药。使用 PROMIS GH 评估 HRQoL，并在基线和随访期间收集患者报告的疼痛和 C 反应蛋白（CRP）血水平。使用 HRQoL 测量值完成了 2 期试验疗效结果的二次描述性分析，以描述 RP 的影响和 rilonacept 的治疗效果。
定性访谈的信息表明，PROMIS GH 概念与 RP 成人患者相关。来自 2 期试验的数据显示，两组患者在基线时的 HRQoL 均受到影响（平均 PROMIS 全球生理健康[GPH]和全球心理健康[GMH]均低于人群平均水平）。在 A-RP 中，基础 TP 结束时 GPH/MPH 得到改善，并在 EP 期间保持（疼痛和 CRP 也观察到类似的趋势）。同样，在 CSD-RP 中，GPH/MPH 在 TP 结束时得到改善，并在 EP 期间进一步改善，同时 CS 逐渐减少或停药，且无疾病复发（TP 和 EP 期间持续低疼痛评分和 CRP 水平）。
这是第一项表明 RP 患者 HRQoL 受损的研究。rilonacept 治疗与 PROMIS GH 评分的 HRQoL 改善相关。在没有复发的情况下，CS 逐渐减少/停药期间维持/改善 HRQoL 表明 rilonacept 可能是 CS 的替代治疗方法。
ClinicalTrials.Gov；NCT03980522；2019 年 6 月 5 日，回顾性注册；https://clinicaltrials.gov/ct2/show/NCT03980522。
