环磷酰胺治疗系统性硬化症相关间质性肺病：硬皮病肺研究 I 和 II 的比较。
Cyclophosphamide for Systemic Sclerosis-related Interstitial Lung Disease: A Comparison of Scleroderma Lung Study I and II.
机构信息
From the Department of Medicine and Department of Radiology, University of California, Los Angeles, David Geffen School of Medicine, Los Angeles, California; Department of Biomathematics, University of California, Los Angeles, California; Department of Medicine, University of Michigan Medical School, Ann Arbor, Michigan, USA; Department of Rheumatology, Oslo University Hospital, Oslo, Norway.
E.R. Volkmann, MD, MS, Assistant Professor, Department of Medicine, University of California, Los Angeles, David Geffen School of Medicine; D.P. Tashkin, MD, Emeritus Professor, Department of Medicine, University of California, Los Angeles, David Geffen School of Medicine; M. Sim, PhD, Associate Professor, Department of Medicine, University of California, Los Angeles, David Geffen School of Medicine; N. Li, PhD, Associate Professor, Department of Biomathematics, University of California, Los Angeles; D. Khanna, MD, MS, Department of Medicine, Professor, University of Michigan Medical School; M.D. Roth, MD, Professor, Department of Medicine, University of California, Los Angeles, David Geffen School of Medicine; P.J. Clements, MD, MPH, Professor, Department of Medicine, University of California, Los Angeles, David Geffen School of Medicine; A.M. Hoffmann-Vold, MD, PhD, Postdoctoral Candidate, Department of Rheumatology, Oslo University Hospital; D.E. Furst, MD, Emeritus Professor, Department of Medicine, University of California, Los Angeles, David Geffen School of Medicine; G. Kim, PhD, Associate Professor, Department of Radiology, University of California, Los Angeles, David Geffen School of Medicine; J. Goldin, MD, PhD, Professor, Department of Radiology, University of California, Los Angeles, David Geffen School of Medicine; R.M. Elashoff, PhD, Distinguished Professor, Department of Biomathematics, University of California, Los Angeles.
出版信息
J Rheumatol. 2019 Oct;46(10):1316-1325. doi: 10.3899/jrheum.180441. Epub 2019 Feb 15.
OBJECTIVE
To compare safety and efficacy outcomes between the cyclophosphamide (CYC) arms of Scleroderma Lung Study (SLS) I and II.
METHODS
Participants enrolled in the CYC arms of SLS I (n = 79) and II (n = 69) were included. SLS I and II randomized participants to oral CYC for 1 year and followed patients for an additional year off therapy (in SLS II, patients received placebo in Year 2). Eligibility criteria for SLS I and II were nearly identical. Outcomes included the forced vital capacity (FVC%)-predicted and DLCO%-predicted (measured every 3 mos) and quantitative radiographic extent of interstitial lung disease (measured at 1 and 2 yrs for SLS I and SLS II, respectively). Joint models were created to evaluate the treatment effect on the course of the FVC/DLCO over 2 years while controlling for baseline disease severity.
RESULTS
SLS I and II CYC participants had similar baseline characteristics. After adjusting for baseline disease severity, there was no difference in the course of the FVC%-predicted (p = 0.535) nor the DLCO%-predicted (p = 0.172) between the SLS I and II CYC arms. In both groups, treatment with CYC led to a significant improvement in the FVC%-predicted from 3 to 12 months, but no significant improvement beyond this point. Treatment with CYC had no effect on the DLCO for either group.
CONCLUSION
Treatment with 1 year of oral CYC led to similar improvements in lung function in both SLS I and II, although the effects were not sustained following cessation of CYC. These results suggest that increasing the duration of ILD therapy may improve outcomes for patients with systemic sclerosis-ILD.
目的
比较硬皮病肺研究（SLS）I 期和 II 期环磷酰胺（CYC）组的安全性和疗效结果。
方法
纳入 SLS I 期（n=79）和 II 期（n=69）CYC 组的参与者。SLS I 和 II 期将参与者随机分配接受口服 CYC 治疗 1 年，然后在停药后再随访 1 年（在 SLS II 期，患者在第 2 年接受安慰剂）。SLS I 和 II 期的入选标准几乎相同。结果包括用力肺活量（FVC）%预测值和 DLCO%-预测值（每 3 个月测量一次）以及定量放射学间质性肺病程度（分别在 SLS I 和 SLS II 期的 1 年和 2 年进行测量）。创建联合模型来评估治疗对 FVC/DLCO 病程的影响，同时控制基线疾病严重程度。
结果
SLS I 和 II 期 CYC 组参与者具有相似的基线特征。在调整基线疾病严重程度后，SLS I 和 II 期 CYC 组的 FVC%-预测值（p=0.535）和 DLCO%-预测值（p=0.172）的病程无差异。在两组中，CYC 治疗在 3 至 12 个月内显著改善了 FVC%-预测值，但此后没有进一步改善。CYC 治疗对两组的 DLCO 均无影响。
结论
1 年口服 CYC 治疗在 SLS I 和 II 期均导致肺功能相似的改善，尽管在停止 CYC 后，效果并未持续。这些结果表明，增加间质性肺病治疗的持续时间可能会改善系统性硬皮病-间质性肺病患者的结局。
Zotero 插件