Abstract
Objective: Malnutrition is a severe complication in Systemic Sclerosis (SSc) and it is associated with significant mortality. Notwithstanding, there is no defined screening or clinical pathway for patients, which is hampering effective management and limiting the opportunity for early intervention. Here we aim to identify a combined index predictive of malnutrition at 12 months using clinical data and specific serum adipokines.Methods: This was an international, multicentre observational study involving 159 SSc patients in two independent discovery (n = 98) and validation (n = 61) cohorts. Besides routine clinical and serum data at baseline and 12 months, Malnutrition Universal Screening Tool (MUST) score and serum concentration of leptin and adiponectin were measured for each participant at baseline. The endpoint of malnutrition was defined according to European Society of Clinical Nutrition and Metabolism (ESPEN) recommendation. Significant parameters from univariate analysis were tested in logistic regression analysis to identify the predictive index of malnutrition in the derivation cohort.Results: The onset of malnutrition at 12 months correlated with adiponectin, leptin and their ratio (A/L), MUST, clinical subset, disease duration, Scl70 and Forced Vital Capaciy (FVC). Logistic regression analysis defined the formula: −2.13 + (A/L*0.45) + (Scl70*0.28) as the best PREdictor of MAlnutrition in SSc (PREMASS) (AUC = 0.96; 95% CI 0.93, 0.99). PREMASS < −1.46 had a positive predictive value (PPV) > 62% and negative predictive value (NPV) > 97% for malnutrition at 12 months.Conclusion: PREMASS is a feasible index which has shown very good performance in two independent cohorts for predicting malnutrition at 12 months in SSc. The implementation of PREMASS could aid both in clinical management and clinical trial stratification/enrichment to target malnutrition in SSc.
Highlights
Systemic sclerosis (SSc) is a rare autoimmune disease characterized by microvascular injury, immune dysregulation and fibroblasts activation, leading to progressive skin and multiorgan fibrosis with potentially life-threatening complications (1)
No statistical differences were observed in BMI, Malnutrition Universal Screening Tool (MUST), adiponectin, and leptin serum levels between discovery cohort and validation cohort at baseline, nor when analyzed according to age or gender
The lack of significant differences in the adiponectin and leptin was important given the two methods used for analysis
Summary
Systemic sclerosis (SSc) is a rare autoimmune disease characterized by microvascular injury, immune dysregulation and fibroblasts activation, leading to progressive skin and multiorgan fibrosis with potentially life-threatening complications (1). There are no data on the efficacy of intervention in preventing or managing malnutrition since it has never been considered as main clinical outcome in randomized clinical trials for systemic sclerosis. This is in part due to the inability to define which patients are at risk of developing malnutrition in a time window targetable with a clinical trial, e.g., 12 months. Stratification for risk of malnutrition in SSc could aid both in clinical management and in the design of RCTs to determine efficacy in modifying this outcome
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