Lee Kyung-Ann, Kim Bo Young, Choi Sung Jae, Kim Seong-Kyu, Kim Sang-Hyon, Kim Hyun-Sook
Department of Internal Medicine, Soonchunhyang University School of Medicine, Seoul, South Korea.
Department of Internal Medicine, Gangneung Asan Hospital, Gangneung, South Korea.
Arch Rheumatol. 2020 Jan 13;35(3):366-375. doi: 10.46497/ArchRheumatol.2020.7771. eCollection 2020 Sep.
This study aims to assess the efficacy and safety of mycophenolate mofetil (MMF) on lung function and skin thickness in Korean patients with systemic sclerosis-interstitial lung disease (SSc-ILD) in a real-world setting.
This retrospective, medical chart-based study was performed at four centers in South Korea and included 34 patients (2 males, 32 females; median age 50.5 years; range, 25 to 72 years) with SSc-ILD. We investigated changes in forced vital capacity (FVC), diffusion capacity of the lung for carbon monoxide (DLCO), and modified Rodnan skin score (mRSS) according to MMF treatment for 24 months.
The mean dose and treatment duration of MMF were 1,338.2±439.0 mg/day and 13.1±9.3 months, respectively. Although FVC decreased significantly at 15 months, FVC and DLCO did not change significantly during treatment with MMF. Median mRSS decreased significantly from 17.5 (2-40) to 10.5 (2-40) units (p<0.0001). Thirteen patients (38.24%) discontinued treatment with MMF [treatment duration 8.00 (3.0-24.0) months]; the predominant cause of discontinuation was of economic nature (46.15%). No serious adverse events were reported.
This real-world based study supports the role of MMF in the stabilization of lung function and the improvement in skin thickness with an acceptable safety profile in patients with SSc. Economic burden is the main cause of discontinued treatment with MMF.
本研究旨在评估霉酚酸酯（MMF）在真实临床环境中对韩国系统性硬化症相关间质性肺病（SSc-ILD）患者肺功能和皮肤厚度的疗效及安全性。
本回顾性、基于病历的研究在韩国四个中心开展，纳入了34例SSc-ILD患者（2例男性，32例女性；中位年龄50.5岁；范围25至72岁）。我们调查了根据MMF治疗24个月后用力肺活量（FVC）、肺一氧化碳弥散量（DLCO）以及改良Rodnan皮肤评分（mRSS）的变化情况。
MMF的平均剂量和治疗时长分别为1338.2±439.0毫克/天和13.1±9.3个月。尽管FVC在15个月时显著下降，但在MMF治疗期间FVC和DLCO并未发生显著变化。mRSS中位数从17.5（2至40）单位显著降至10.5（2至40）单位（p<0.0001）。13例患者（38.24%）停止MMF治疗[治疗时长8.00（3.0至24.0）个月]；停药的主要原因是经济因素（46.15%）。未报告严重不良事件。
这项基于真实临床环境的研究支持MMF在稳定SSc患者肺功能以及改善皮肤厚度方面的作用，且安全性可接受。经济负担是停止MMF治疗的主要原因。
