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- W2186291990 abstract "In systemic sclerosis (SSc), there is no proven treatment to prevent disease progression. In a recent meta-analysis of three randomised controlled trials (RCTs) and six open prospective studies on cyclophosphamide (CYC), no significant changes in lung function were observed. However, CYC is associated with an improvement of Mahler’s dyspnea index, short form-36 (physical and mental domains), and health-related quality of life, contributing to the amelioration of patients’ functional status. Further RCTs on early SSc are needed to assess the real efficacy of CYC in inducing remission and increasing survival. In a previous issue of Arthritis Research & Therapy, Nannini and colleagues [1] addressed the topic by a well-conducted meta-analysis and concluded that cyclophosphamide (CYC) treatment does not induce a statistically significant improvement in lung function in systemic sclerosis (SSc) interstitial lung disease (ILD). This meta-analysis pooled the data from three randomised controlled trials (RCTs) and six observational studies and focused on the changes in pulmonary functional parameters (forced vital capacity [FVC] and diffusing lung capacity for carbon monoxide [DLCO]) of SSc patients treated with CYC for at least 1 year [1]. Such a conclusion sets a negative scenario in which CYC should not be considered as an ‘anchor’ drug for the treatment of SSc and ILD. In SSc, no drug or combination of drugs has been demonstrated to modify disease progression. In both RCTs versus placebo, the trend toward improvement in FVC suggests that the efficacy of CYC on lung function is limited, as also corroborated by a mild effect size (Table 1) [2,3]. This negative opinion may not reflect what is going on in a ‘real life’ scenario, when other efficacy parameters are considered. Actually, the scleroderma lung study group reported, after 12 months, a significant reduction of Mahler’s dyspnea index in CYC-treated patients when compared with placebo (P <0.0001) and a statistically significant amelioration of short form-36 (SF-36) in each domain: mental (P = 0.006), emotional (P = 0.005), general health status (P = 0.003), and vitality (P = 0.009) [4]. Despite the negligible value of effect size for all of these scale domains, minimum clinically important difference (which represents the smallest improvement in the score of a health-related quality of life measurement instrument which patients perceive as beneficial) was significantly higher for all measures in CYC-treated patients [5]. The improvement of dyspnea and reduction of skin thickness may have a favourable effect not only on functional status but also on psychological aspects, as demonstrated by the analysis of each SF-36 domain." @default.
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- W2186291990 date "2009-01-01" @default.
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- W2186291990 title "Editorial Cyclophosphamide in systemic sclerosis: still in search of a 'real life' scenario" @default.
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