Paeds Vivas · genetics-dysmorphology-and-metabolism
Marfan syndrome and heritable connective-tissue disorders — branching viva
Branching viva on Marfan syndrome and the heritable connective-tissue disorders: recognising the marfanoid phenotype, confirming with the revised Ghent nosology and FBN1 sequencing, applying the lifelong aortic-root surveillance and treatment plan, distinguishing Marfan from Loeys-Dietz, vascular Ehlers-Danlos, Beals and homocystinuria by gene and lens direction, and cascade testing the family.
On this page
Study tools
Target exams
Opening question
A 14-year-old with tall stature, a positive wrist-and-thumb sign, superotemporal lens subluxation and aortic-root dilation at the sinuses of Valsalva, with no family history. What is the unifying diagnosis, how is it confirmed under the revised Ghent nosology, and why is a karyotype not the confirmatory test? [2] [1]
References5ShowHide
- [1]Dietz H FBN1-related Marfan syndrome. GeneReviews, 1993.PMID 20301510
- [2]Loeys BL, Dietz HC, Braverman AC, Callewaert BL, De Backer J, Devereux RB, et al. The revised Ghent nosology for the Marfan syndrome. J Med Genet, 2010.PMID 20591885
- [3]Lacro RV, Dietz HC, Sleeper LA, Yetman AT, Bradley TJ, Colan SD, et al. Atenolol versus losartan in children and young adults with Marfan's syndrome. N Engl J Med, 2014.PMID 25405392
- [4]MacCarrick G, Black JH, Bowdin S, El-Hamamsy I, Frischmeyer-Guerrerio PA, Guerrerio AL, et al. Loeys-Dietz syndrome: a primer for diagnosis and management. Genet Med, 2014.PMID 24577266
- [5]Sacharow SJ, Levy HL Homocystinuria due to Cystathionine Beta-Synthase Deficiency. GeneReviews, 1993.PMID 20301697