Division of Rheumatology, Department of Medicine, Thomas Jefferson University, Philadelphia, PA 19107, USA; Jefferson Institute of Molecular Medicine and Scleroderma Center, Thomas Jefferson University. Philadelphia, PA 19107, USA.
Division of Rheumatology, Department of Medicine, Thomas Jefferson University, Philadelphia, PA 19107, USA.
Semin Arthritis Rheum. 2020 Feb;50(1):135-139. doi: 10.1016/j.semarthrit.2019.06.012. Epub 2019 Jun 19.
Rapidly progressive diffuse cutaneous Systemic Sclerosis (rp-dcSSc) is associated with severe internal organ involvement and high mortality. Mycophenolate Mofetil (MMF) has been shown to halt the progression of rp-dcSSc cutaneous and pulmonary involvement in observational and randomized controlled trials, respectively. However, optimal MMF therapy duration has not been established. Here, we describe the clinical evolution of rp-dcSSc patients successfully treated with MMF following MMF therapy discontinuation or dose reduction.
Twenty-five patients with recent-onset (< 24 mo) rp-dcSSc received MMF as the only SSc disease-modifying therapy. Following MMF discontinuation or dose reduction to or below 1000 mg/day after an average of two years, the Modified Rodnan skin score (mRSS) and Pulmonary function tests (PFT) were serially evaluated for additional 5 years. MMF therapy was re-instituted if the mRSS increased by greater than 20% or if restrictive lung disease developed.
From nineteen patients serially evaluated following MMF discontinuation or dose reduction, five patients (26.3%) developed recurrence of rapid skin involvement with an average of 35.9% increase in mRSS from 7.8 to 10.6 points requiring MMF re-institution. Two of these patients also presented worsening respiratory symptoms and reduction of lung volumes in PFTs. Following MMF resumption, mRSS returned to baseline or stabilized and PFTs improved or stabilized. All these patients were maintained on high dose long term MMF treatment.
Recurrence of severe skin involvement occurred in 26.3% of patients with rp-dcSSc following MMF discontinuation or dose reduction, requiring prompt MMF therapy resumption. These findings confirm the therapeutic benefit of MMF in rp-dcSSc and suggest that MMF treatment should be maintained for longer than 2 years.
快速进展性弥漫性皮肤系统性硬化症（rp-dcSSc）与严重的内脏器官受累和高死亡率相关。霉酚酸酯（MMF）已被证明分别在观察性和随机对照试验中阻止 rp-dcSSc 皮肤和肺部受累的进展。然而，尚未确定最佳 MMF 治疗持续时间。在这里，我们描述了在 MMF 治疗停止或剂量减少后成功用 MMF 治疗的 rp-dcSSc 患者的临床演变。
25 例新近发病（<24 个月）的 rp-dcSSc 患者接受 MMF 作为唯一的 SSc 疾病修饰治疗。在平均两年后 MMF 停药或剂量减少至 1000mg/天以下后，连续评估改良罗德里根皮肤评分（mRSS）和肺功能检查（PFT），再评估 5 年。如果 mRSS 增加超过 20%或出现限制性肺疾病，则重新开始 MMF 治疗。
在 MMF 停药或剂量减少后连续评估的 19 例患者中，有 5 例（26.3%）出现快速皮肤受累复发，mRSS 平均增加 35.9%，从 7.8 增加到 10.6 分，需要重新开始 MMF 治疗。其中 2 例患者还出现呼吸症状恶化和 PFT 中肺容积减少。重新开始 MMF 治疗后，mRSS 恢复到基线或稳定，PFT 改善或稳定。所有这些患者都接受了高剂量长期 MMF 治疗。
在 MMF 停药或剂量减少后，26.3%的 rp-dcSSc 患者出现严重皮肤受累复发，需要及时重新开始 MMF 治疗。这些发现证实了 MMF 在 rp-dcSSc 中的治疗益处，并表明 MMF 治疗应持续超过 2 年。
