Elimination of Pain and Improvement of Exercise Capacity in Camurati-Engelmann Disease With Losartan

Background: Camurati-Engelmann disease (CED) is a rare disorder, with approximately 250 described cases in the literature. Treatment options are limited and have been suboptimal so far. Patient and Methods: A prepubertal girl aged 9 years was diagnosed with CED. Treatment with losartan was initiated...

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Published in:The journal of clinical endocrinology and metabolism 2014-11, Vol.99 (11), p.3978-3982
Main Authors: Ayyavoo, Ahila, Derraik, José G. B, Cutfield, Wayne S, Hofman, Paul L
Format: Article
Language:English
Subjects:
Angiotensin II Type 1 Receptor Blockers - therapeutic use
Biological and medical sciences
Camurati-Engelmann Syndrome - complications
Camurati-Engelmann Syndrome - drug therapy
Child
Endocrinopathies
Exercise
Feeding. Feeding behavior
Female
Fundamental and applied biological sciences. Psychology
Humans
Losartan - therapeutic use
Medical sciences
Pain - drug therapy
Pain - etiology
Treatment Outcome
Vertebrates: anatomy and physiology, studies on body, several organs or systems
Vertebrates: endocrinology
Citations: Items that cite this one
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Summary:Background: Camurati-Engelmann disease (CED) is a rare disorder, with approximately 250 described cases in the literature. Treatment options are limited and have been suboptimal so far. Patient and Methods: A prepubertal girl aged 9 years was diagnosed with CED. Treatment with losartan was initiated at a daily dose of 0.75 mg/kg. Over a period of 12 weeks, the dose was gradually increased to 1.0 mg/kg/d. The patient was reviewed in clinic regularly and underwent thorough clinical assessments 9, 17, and 38 months after treatment initiation. Results: The patient experienced marked clinical improvements with losartan. In particular, losartan treatment led to the complete elimination of the previously severe and incapacitating pain, with an increased ability to walk and perform physical activities. There was also a considerable improvement in body composition with increased lean and adipose tissue. Notably, the improvement in fat deposition had not been previously observed with other treatments in CED. Hematology, liver, and renal function tests were within normal ranges at presentation and remained so over the course of treatment. Conclusions: In light of our findings, losartan may be a useful option in CED management.
ISSN:0021-972X
1945-7197
DOI:10.1210/jc.2014-2025