The importance of genetic testing in the clinical management of patients with Marfan syndrome and related disorders

Dtsch Arztebl Int. 2008 Jul;105(27):483-91. doi: 10.3238/arztebl.2008.0483. Epub 2008 Jul 4.

Abstract

Introduction: Marfan syndrome and Marfan-related syndromes are part of a broad and overlapping spectrum of diseases that were originally defined on clinical grounds alone. They have in common a dramatically increased risk of life-threatening dissecting aortic aneurysms, which must be prevented by elective aortic replacement.

Methods: Selective review of the literature supplemented by own clinical experience.

Results: Marfan syndrome and Marfan-related syndromes are phenotypically highly variable. The full-blown clinical picture is not always present, and particular symptoms can be missing altogether. Accordingly, it is often very difficult to diagnose a specific syndrome in the individual patient. In many cases, only a combination of genetic tests and clinical assessment can settle the differential diagnosis, thus enabling better prognostication and better planning of preventive measures.

Discussion: The diagnosis and treatment of individual patients with Marfan syndrome and Marfan-related syndromes requires an interdisciplinary approach. This can only be achieved through a coordination of medical care with centralized record-keeping of all diagnostic findings.

Keywords: Marfan syndrome; aneurysm; aortic syndrome; genetic testing; mitral valve prolaps; molecular medicine.