Cyclophosphamide versus mycophenolate mofetil in scleroderma interstitial lung disease (SSc-ILD) as induction therapy: a single-centre, retrospective analysis

Abstract

BackgroundScleroderma is a systemic autoimmune disease characterized mainly by skin manifestations and involvement of various visceral organs, especially the lungs. Lung involvement is the leading cause of mortality in patients with scleroderma. There are data to suggest that cyclophosphamide (CYC) and mycophenolate mofetil (MMF) are effective in the management of scleroderma interstitial lung disease (SSc-ILD) but no head to head comparative data are available to date.MethodsFor the last 3 years, patients with SSc-ILD have been treated at our centre by protocol-based administration of intravenous CYC and MMF. Results of lung function tests (spirometry) were recorded at baseline, 3 months and 6 months in every patient. The clinical records of patients with systemic sclerosis and significant ILD, who were not previously exposed to any immunosuppressant and were treated with MMF OR CYC, were reviewed. The efficacy of treatment was assessed by the change in forced vital capacity on spirometry.ResultsOf the total 57 patients included in the analysis, 34 were treated with MMF and 23 were treated with CYC. Mean duration of illness was 4.19 ± 2.82 years in the MMF and 6.04 ± 5.96 years in the CYC group. After 6 months of therapy, FVC increased by 10.84 ± 13.81 % in the CYC group and by 6.07 ± 11.92 % in the MMF group. This improvement from baseline was statistically significant in both groups (P < 0.01). The improvement was comparable with no statistically significant differences between groups (P = 0.373). There were no major adverse events reported in either arm.ConclusionBoth MMF and CYC were equally effective in stabilizing lung function in patients with scleroderma and ILD.

Pulmonary function tests as outcomes for systemic sclerosis interstitial lung disease

“…Interestingly, of the five studies that cited prior validation to justify their choice of PFT (% predicted FVC in four cases [69,111,114,149] and % predicted DLCO in one) [33], only the study using % predicted DLCO referenced an article with reported measures of validity. The four studies which chose their main PFT outcome measure based on its high sensitivity (in the case of DLCO) [11,14] or its high specificity (in the case of FVC) [128,169] either did not provide a citation to support this claim [169], referred to studies performed outside the field of SSc-ILD [11,14,128], or cited SSc studies with fewer than 20 patients [11,14].…”

A Real-World Experience of Mycophenolate Mofetil for Systemic Sclerosis: A Retrospective Multicenter Observational Study

“…4 In other reported studies, pulmonary function has also improved or stabilized after treatment with at least more than 2 g/day of MMF. [15][16][17][18] The SLS II study has shown an average peak improvement of FVC at 18-21 months. The Australian Scleroderma Cohort study has demonstrated that treatment with MMF was associated with stability of the SSc-ILD for up to 36 months.…”

Immunotherapy of systemic sclerosis

“…Several retrospective or openlabel prospective studies in SSc and SSc-ILD have shown that MMF improved skin score and stabilized pulmonary function. [35][36][37] More importantly, it improved a 5-year survival. Recently a randomized, double-blind, parallel group trial that compared 2 years of oral MMF (target dose 3gr/day) to oral CYP (2 mg/kg/ day) for 1 year followed by placebo was conducted.…”