- Oral presentation
- Open Access
Losartan improves clinical outcome in Camurati Engelmann Disease
© Ayyavoo et al; licensee BioMed Central Ltd. 2013
- Published: 3 October 2013
- Amino Acid Substitution
- Aortic Root
- Transform Growth Factor Beta
- Growth Factor Beta
We hypothesized that losartan would help in achieving clinical remission in CED (Camurati Engelmann Disease) patients by blocking TGFB1(transforming growth factor beta 1) with fewer side-effects than steroids.
CED characterised by progressive diaphyseal dysplasia is associated with debilitating bone pain in the limbs, muscle weakness, fatiguability and waddling gait. Clinical manifestations are due to mutations in the TGFB1 gene leading to its over-expression and effect on bone. Losartan is an antagonist of TGFB1 and it slows the progress of aortic root dilatation inMarfan’s syndrome by blocking the over-expression of TGFB1. Steroids which have long been used for treatment of CED and been linked to long term side effects including those on growth, blood pressure and spinal osteoporosis.
Age at analysis(years)
Cumulative pain score
6 minute walk test(metres)
Total body fat%
Losartan improves the quality of life in children with CED by reducing the bone pain along with improvement in their activity levels, fat & muscle mass, without major effects on growth, blood pressure and spinal osteoporosis.
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