Background/Purpose:  Interstitial lung disease occurs in over 80% of patients with scleroderma. Cyclophosphamide is the only treatment proven to benefit scleroderma lung disease in a large randomized placebo-controlled trial. However, the clinical benefit is modest and potential adverse effects are a major concern. Mycophenolate mofetil (MMF) has been reported to stabilize scleroderma lung disease in case series from academic institutions. However, patient characteristics may vary from the academic to community setting and the relevance of these reports to the broader rheumatology community is uncertain. Our purpose is to analyze the effect of MMF in scleroderma lung disease from a community-based rheumatology clinic. 

Methods:  Patients who fulfilled the 2013 ACR/EULAR classification criteria for scleroderma were identified in a community-based rheumatology practice.  All patients included had evidence of scleroderma lung disease and treatment with MMF. Exclusion criteria were patients treated for < 4months,  >14 months between baseline data and initiation of MMF, or >24 months after initiation of MMF for follow-up data.  Lung functions, CT scans, and clinical symptoms were analyzed at baseline and post treatment.  A total of thirteen patients were identified who met the above criteria. A paired student t-test was performed to assess pre and post MMF treatment change in forced vital capacity (FVC) and diffusion capacity for carbon monoxide (DLCO). 

Results:  A total of 22 patients met inclusion criteria. Of these 22 patients, 1 was deceased from pulmonary arterial hypertension, 2 stopped treatment before follow-up due to severe diarrhea, and 6 did not have timely baseline or follow-up data. This left 13 patients for analysis who were treated an average of 10 months with MMF. There was no significant difference between baseline and post treatment values for FVC or DLCO (p=0.89, p =0.76, respectively).  The mean % predicted FVC pre and post MMF was 72 and 72 respectively. The mean % predicted DLCO pre and post MMF was 66 and 67 respectively. There were only two individuals who experienced a significant decline in FVC from baseline to follow up as defined by national criteria (absolute change in FVC of 10%).Baseline and post treatment CT scans were also evaluated. Of the 13 patients, 9 had stable CT findings, 2 worsened, and 2 improved. Clinical symptoms of cough and dyspnea were all stable or improved on MMF therapy.

Conclusion:  MMF appears effective in preventing progression of scleroderma lung disease in a community-based setting. Compared to historical controls, we would have expected worsening lung function in the absence of treatment as seen in the placebo arm of the Scleroderma Lung Study. Limitations to our study include the lack of a standard protocol for individual rheumatologists to record symptoms, clinical findings, or standard intervals for pulmonary function tests and CT scans. Also, pre and post treatment CT scans were not universally interpreted by the same radiologist. These limitations were expected given the nature of rheumatology practice outside of academic centers. Our study supports the use of MMF for scleroderma lung disease in the community-based setting.

---

« Back to 2014 ACR/ARHP Annual Meeting

ACR Meeting Abstracts - https://acrabstracts.org/abstract/treatment-of-scleroderma-associated-lung-disease-with-mycophenolate-mofetil-a-community-based-study/