Angiotensin II Inhibitor Therapy in Pediatric Patients with Marfan Syndrome- Update after Six Years of Experience with Focus on Tolerability

Introduction: Marfan syndrome MFS is an inherited connective tissue disorder. Aortic root dilatation remains the significant indicator for morbidity and mortality. After recent publication of the pediatric multicenter study and the publication of our data the year before concerning the effectiveness and tolerability Angiotensin II inhibitor (ARB) therapy versus β-blocker (BB) we now present an actual update after six years of experience.

Methods: We identified 120 pediatric patients with confirmed MFS. 56 patients showed indication for medical prophylaxis. All patients were subjected to standardized diagnostics including echocardiography, MRI and Ghent Classification. We examined the effectiveness of ARB (n = 36) on growth of 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 with ARB and BB leads to significant reduction of SV dilatation (p < 0.05). The deviation of SV enlargement from normal to augmented was significantly reduced under ARB therapy by a mean difference of −0.57 ± 0.65 z-scores (p < 0.05) and under BB therapy with −0.42 ± 0.55 z-scores (p < 0.05). The prophylactic effect of ARB and BB on aortic root dilatation is similar (p < 0.05). Therapy with BB was discontinued in 20% of treated patients due to intolerance. There was no discontinuation in the ARB group.

Conclusion: The prophylactic effect of ARB and BB in pediatric MFS patients is similar but tolerability of ARB is clearly superior. Furthermore inhibition of TGF-β signaling by ARB which is supposed to contribute to the pathogenesis of MFS has to be considered. Clinical study results to confirm this hypothesis are still missing.