Randomised investigation of effects of pentoxifylline on left-ventricular performance in idiopathic dilated cardiomyopathy

Background There is accumulating evidence that inflammatory cytokines have an important role in the pathogenesis of heart failure. Plasma concentrations of tumour necrosis factor α (TNF-α) are high in heart failure and have been correlated with the severity of symptoms. Pentoxifylline suppresses the production of TNF-α. This study aimed to assess the effects of pentoxifylline on left-ventricular function and functional class in patients with idiopathic dilated cardiomyopathy. Methods We undertook a single-centre, prospective, double-blind, randomised, placebo-controlled trial, in which 28 patients with idiopathic dilated cardiomyopathy were assigned pentoxifylline 400 mg three times daily or matching placebo. Clinical, echocardiographic, and radionuclide assessments were done at baseline and after 6 months of treatment. Primary endpoints were New York Heart Association (NYHA) functional class and left-ventricular function. Findings Baseline characteristics were similar in the two groups. Four patients died during the study period, all in the placebo group. After 6 months of treatment, the proportion of patients in NYHA functional class I or II was higher in the pentoxifylline group than in the placebo group (14/14 vs 10/14; p=0•01), and ejection fraction was higher in the pentoxifylline group than in the placebo group (mean 38•7% [SD 15•0] vs 26•8% [11•0], p=0•04). At 6 months, TNF-α plasma concentrations were significantly lower in the pentoxifylline-treated group than in the placebo group (2•1 [1•0] vs 6•5 [5•0] pg/mL, p=0•001). Interpretation Our results suggest that pentoxifylline improves symptoms and left-ventricular systolic function in patients with idiopathic dilated cardiomyopathy. These results must be confirmed in larger-scale trials.