Measuring GPPGA, Pain, Symptom, and Quality of Life Index Scores in Untreated Generalized Pustular Psoriasis: Results from the Placebo Group of the Effisayil-2 Trial

Bruce Strober, Arash Mostaghimi, Milan J Anadkat, C. Thoma, Ming Tang, Jason Guercio, M. Lebwohl
{"title":"Measuring GPPGA, Pain, Symptom, and Quality of Life Index Scores in Untreated Generalized Pustular Psoriasis: Results from the Placebo Group of the Effisayil-2 Trial","authors":"Bruce Strober, Arash Mostaghimi, Milan J Anadkat, C. Thoma, Ming Tang, Jason Guercio, M. Lebwohl","doi":"10.25251/skin.8.supp.361","DOIUrl":null,"url":null,"abstract":"Introduction & Objectives: Generalized pustular psoriasis (GPP) is a chronic inflammatory, and potentially life-threatening skin disease, characterized by flares of sterile pustules. Spesolimab is approved to treat GPP flares in adults. Effisayil 2 (NCT04399837) evaluated the efficacy and safety of subcutaneous spesolimab in preventing GPP flares. Here, we report on the underlying disease burden of untreated GPP by longitudinally analyzing patients in the placebo group who did not experience a GPP flare. \nMaterials & Methods: Eligible patients with a history of GPP were randomized (1:1:1:1) to receive one of three subcutaneous spesolimab regimens or placebo for 48 weeks. Patients were assessed for measures of chronic disease burden through the use of Generalized Pustular Psoriasis Physician Global Assessment (GPPPGA) Total Score, Pain Visual Analog Scale (VAS), and Psoriasis Symptom Scale (PSS) at baseline and at 4-week intervals during the trial, and Dermatology Quality of Life Index (DLQI) at baseline and weeks 4, 8, 12, 24, 36, and 48. \nResults: 16/31 placebo-treated patients experienced a GPP flare over the 48-week observational period of the trial, defined as an increase in GPPPGA score by ≥2 from baseline and the pustular component of GPPPGA ≥2. Of the remaining (“non-flaring”) 15 patients, 40% (6/15) had at least one GPPPGA Total Score value of 2 (skin not clear or almost clear); 4/6 reported such score at ≥4 visits. Pain scores ranged from 0 to 92.47, with 47% (7/15) and 20% (3/15) of patients having at least one “moderate” and “severe” VAS score over 48 weeks, respectively. Most of the 7 patients’ pain scores fluctuated with episodic peaks and valleys. 47% (7/15) and 13% (2/15) of patients had at least one “moderate” and “severe” PSS score, respectively. “Moderate” and “very large” effect on quality of life was reported, at least once, in 67% (10/15) and 40% (6/15) of patients, respectively. \nConclusion: Acute flare was reported in more than 50% (16/31) of patients in the placebo group over 48 weeks. Despite not meeting the trial’s definition of GPP flare, most of the 15 “non-flaring” placebo-treated patients showed clear evidence of underlying GPP disease activity – nearly half did not have “clear” or “almost clear” skin and had moderate pain and symptoms; a small subset reported severe pain and symptoms. Majority of patients experienced a moderate to very large impact on quality of life over the 48 weeks. These findings suggest that untreated GPP negatively affects patients even in the absence of acute flare events.","PeriodicalId":22013,"journal":{"name":"SKIN The Journal of Cutaneous Medicine","volume":"338 9","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-03-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"SKIN The Journal of Cutaneous Medicine","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.25251/skin.8.supp.361","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0

Abstract

Introduction & Objectives: Generalized pustular psoriasis (GPP) is a chronic inflammatory, and potentially life-threatening skin disease, characterized by flares of sterile pustules. Spesolimab is approved to treat GPP flares in adults. Effisayil 2 (NCT04399837) evaluated the efficacy and safety of subcutaneous spesolimab in preventing GPP flares. Here, we report on the underlying disease burden of untreated GPP by longitudinally analyzing patients in the placebo group who did not experience a GPP flare. Materials & Methods: Eligible patients with a history of GPP were randomized (1:1:1:1) to receive one of three subcutaneous spesolimab regimens or placebo for 48 weeks. Patients were assessed for measures of chronic disease burden through the use of Generalized Pustular Psoriasis Physician Global Assessment (GPPPGA) Total Score, Pain Visual Analog Scale (VAS), and Psoriasis Symptom Scale (PSS) at baseline and at 4-week intervals during the trial, and Dermatology Quality of Life Index (DLQI) at baseline and weeks 4, 8, 12, 24, 36, and 48. Results: 16/31 placebo-treated patients experienced a GPP flare over the 48-week observational period of the trial, defined as an increase in GPPPGA score by ≥2 from baseline and the pustular component of GPPPGA ≥2. Of the remaining (“non-flaring”) 15 patients, 40% (6/15) had at least one GPPPGA Total Score value of 2 (skin not clear or almost clear); 4/6 reported such score at ≥4 visits. Pain scores ranged from 0 to 92.47, with 47% (7/15) and 20% (3/15) of patients having at least one “moderate” and “severe” VAS score over 48 weeks, respectively. Most of the 7 patients’ pain scores fluctuated with episodic peaks and valleys. 47% (7/15) and 13% (2/15) of patients had at least one “moderate” and “severe” PSS score, respectively. “Moderate” and “very large” effect on quality of life was reported, at least once, in 67% (10/15) and 40% (6/15) of patients, respectively. Conclusion: Acute flare was reported in more than 50% (16/31) of patients in the placebo group over 48 weeks. Despite not meeting the trial’s definition of GPP flare, most of the 15 “non-flaring” placebo-treated patients showed clear evidence of underlying GPP disease activity – nearly half did not have “clear” or “almost clear” skin and had moderate pain and symptoms; a small subset reported severe pain and symptoms. Majority of patients experienced a moderate to very large impact on quality of life over the 48 weeks. These findings suggest that untreated GPP negatively affects patients even in the absence of acute flare events.
测量未经治疗的泛发性脓疱型银屑病患者的 GPPGA、疼痛、症状和生活质量指数得分:Effisayil-2 试验安慰剂组的结果
简介和目标:泛发性脓疱型银屑病(GPP)是一种慢性炎症性皮肤病,有可能危及生命,其特点是无菌性脓疱复发。斯派索利单抗已被批准用于治疗成人 GPP 病发。Effisayil 2(NCT04399837)评估了皮下注射斯派索利单抗预防GPP复发的有效性和安全性。在此,我们通过纵向分析安慰剂组中未出现 GPP 复发的患者,报告未经治疗的 GPP 潜在疾病负担。材料与方法:有 GPP 病史的合格患者随机(1:1:1:1:1)接受三种皮下注射斯派索利单抗方案中的一种或安慰剂治疗 48 周。在基线和试验期间每隔 4 周使用泛发性脓疱型银屑病医师总体评估 (GPPPGA) 总分、疼痛视觉模拟量表 (VAS) 和银屑病症状量表 (PSS),在基线和第 4、8、12、24、36 和 48 周使用皮肤病生活质量指数 (DLQI) 对患者的慢性疾病负担进行评估。结果在为期48周的试验观察期内,16/31名接受过安慰剂治疗的患者出现了GPP复发,GPP复发的定义是GPPPGA评分比基线增加≥2分,且GPPPGA中的脓疱部分≥2分。在其余("非剥脱")15 名患者中,40% 的患者(6/15)至少有一次 GPPPGA 总分值为 2(皮肤不透明或几乎透明);4/6 的患者在≥4 次就诊时报告了此类分数。疼痛评分从 0 到 92.47 不等,分别有 47% (7/15)和 20% (3/15)的患者在 48 周内至少有一次 VAS 评分为 "中度 "和 "重度"。在这 7 名患者中,大多数人的疼痛评分都会出现偶发性的波峰和波谷。47%(7/15)和 13%(2/15)的患者分别有至少一次 "中度 "和 "重度 "PSS 评分。67%(10/15)和 40%(6/15)的患者分别报告了至少一次对生活质量的 "中度 "和 "极大 "影响。结论安慰剂组超过 50%(16/31)的患者在 48 周内出现急性复发。尽管不符合试验对 GPP 复发的定义,但安慰剂治疗的 15 名 "非复发 "患者中,大多数都有明确的证据显示潜在的 GPP 疾病活动--近一半的患者皮肤不 "透明 "或 "几乎透明",并伴有中度疼痛和症状;一小部分患者伴有严重疼痛和症状。大多数患者的生活质量在 48 周内受到中度到非常大的影响。这些发现表明,即使没有急性发作事件,未经治疗的 GPP 也会对患者产生负面影响。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
自引率
0.00%
发文量
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信