Thomas S Mir
University Heart Center, Germany
Title: Angiotension II Inhibitor therapy in pediatric patients with Marfan-Syndrome (MFS) - Update after six years of experience with particular regard to tolerability
Biography
Biography: Thomas S Mir
Abstract
Introduction: MFS is an inherited connective tissue disorder in which aortic root dilatation remains the significant indicator for morbidity and mortality. After recent publication of the pediatric multicenter study and publication of our mono-centric data concerning the effectiveness and tolerability of therapy with ARB vs. beta blocker (BB) we would like to present an actual update after six years of experience. Methods: We identified 126 children with confirmed MFS. Indication for prophylaxis was found in 64 patients and was subjected to standardized diagnostic program including echocardiography, MRI and clinical examination. We examined the effectiveness of therapy with ARB (n=44) on the growth of the sinus valsalvae (SV) with comparison of z-scores of SV before treatment and during follow up and compared these data to patients treated with BB (n=20) only. Results: Treatment by ARB and BB leads to significant reduction of SV dilation (p<0.05). The deviation of SV enlargement from normal as expressed by the rate of change in z-scores was significantly reduced by a mean difference of −0.57±0.65 z-scores (p<0.05) under ARB therapy and by a mean difference of −0.42±0.55 z-scores (p<0.05) under BB therapy. The effect of ARB and BB on aortic root dilation is similar in both groups (p>0.05). Therapy with BB was discontinued in 20% (4/20). No discontinuation in the ARB group (0/34). Conclusion: The prophylactic effect of ARB and BB in pediatric patients with MFS is similar but tolerability of ARB is clearly superior. In addition inhibition of TGF-ß signaling by ARB which is supposed to contribute to the pathogenesis of MFS has to consider.