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以患者为中心探讨波生坦治疗硬皮病患者的症状负担:一项前瞻性单中心观察性研究。

A patient-centered approach to the burden of symptoms in patients with scleroderma treated with Bosentan: A prospective single-center observational study.

作者信息

Rezus Elena, Burlui Alexandra Maria, Gafton Bogdan, Stratulat Teodora Alexa, Zota Gabriela Rusu, Cardoneanu Anca, Rezus Ciprian

机构信息

Department of Rheumatology and Physiotherapy, 'Grigore T. Popa' University of Medicine and Pharmacy, 700115 Iasi, Romania.

Department of Medical Oncology-Radiotherapy, 'Grigore T. Popa' University of Medicine and Pharmacy, 700115 Iasi, Romania.

出版信息

Exp Ther Med. 2020 Mar;19(3):1739-1746. doi: 10.3892/etm.2019.8361. Epub 2019 Dec 20.

Abstract

Systemic sclerosis (SSc) is a rare and complex autoimmune disease associated with poor vital and functional outcomes. The functional hindrance in patients derives from various disease-specific manifestations, including Raynaud's phenomenon and digital ulcers (DUs). Bosentan is an endothelin receptor antagonist capable of preventing the appearance of new DUs in patients with scleroderma. The present study aimed to evaluate the effects of Bosentan on the severity of Raynaud's phenomenon, DU-related symptoms and functional impairment during the first year of treatment. A prospective study that included adult patients with SSc admitted to the Rheumatology Department between January 2016 and January 2017 that were candidates for Bosentan therapy, was performed. All patients were asked to evaluate the burden of symptoms secondary to Raynaud's and DUs using a visual analogue scale (VAS), whereas functional hindrance was assessed via Health Assessment Questionnaire Disability Index (HAQ-DI). The outcomes were assessed at baseline and every 3 months during 1 year of therapy. Among the 41 patients included initially, 2 participants discontinued the treatment after 1 month due to adverse events (elevation of liver enzymes). The study cohort exhibited a significant improvement in HAQ-DI, VAS-R and VAS-DU scores in response to Bosentan therapy over the 1-year follow-up period. Higher scores at baseline predicted a weaker treatment-related improvement, with the risk of a poor outcome being increased by 220% for VAS-R, 116% for VAS-DU, whereas no increase was observed for HAQ-DI. The post-treatment improvement in VAS-DU levels was associated with a better outcome for HAQ-DI (R=0.44; P=0.005). This association was not identified for VAS-R (R=0.24; P=0.137). Throughout the follow-up period, patients with dyspnea presented with significantly higher HAQ-DI scores compared with non-dyspneic patients. Bosentan therapy may indirectly influence functionality and quality of life in patients with scleroderma by reducing the burden of Raynaud's and DU-related symptoms. Nonetheless, patients with SSc with a decreased symptom burden at baseline exhibited improved outcomes.

摘要

系统性硬化症(SSc)是一种罕见且复杂的自身免疫性疾病,与较差的生命和功能预后相关。患者的功能障碍源于各种疾病特异性表现,包括雷诺现象和指端溃疡(DUs)。波生坦是一种内皮素受体拮抗剂,能够预防硬皮病患者新的指端溃疡出现。本研究旨在评估波生坦在治疗第一年对雷诺现象严重程度、与指端溃疡相关的症状及功能损害的影响。进行了一项前瞻性研究,纳入了2016年1月至2017年1月间被收治于风湿科且适合接受波生坦治疗的成年系统性硬化症患者。所有患者均被要求使用视觉模拟量表(VAS)评估雷诺现象和指端溃疡继发症状的负担,而功能障碍则通过健康评估问卷残疾指数(HAQ-DI)进行评估。在治疗的1年期间,于基线及每3个月对结局进行评估。最初纳入的41例患者中,2例参与者因不良事件(肝酶升高)在1个月后停止治疗。在1年的随访期内,研究队列在接受波生坦治疗后,HAQ-DI、VAS-R和VAS-DU评分均有显著改善。基线时较高的评分预示治疗相关改善较弱,VAS-R评分较差结局的风险增加220%,VAS-DU评分增加116%,而HAQ-DI评分未见增加。治疗后VAS-DU水平的改善与HAQ-DI更好的结局相关(R=0.44;P=0.005)。VAS-R未发现这种相关性(R=0.24;P=0.137)。在整个随访期内,与无呼吸困难的患者相比,有呼吸困难的患者HAQ-DI评分显著更高。波生坦治疗可能通过减轻雷诺现象和与指端溃疡相关症状的负担,间接影响硬皮病患者的功能和生活质量。尽管如此,基线时症状负担减轻的系统性硬化症患者结局有所改善。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f357/7027142/024bfb3cefeb/etm-19-03-1739-g00.jpg

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