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Therapy with pamidronate in children with osteogenesis imperfecta

Authors Marginean O, Tamasanu RC, Mang N, Mozos I, Brad GF

Received 4 May 2017

Accepted for publication 27 June 2017

Published 28 August 2017 Volume 2017:11 Pages 2507—2515

DOI https://doi.org/10.2147/DDDT.S141075

Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Lucy Goodman

Peer reviewer comments 2

Editor who approved publication: Dr James Janetka

Otilia Marginean,1 Raluca Corina Tamasanu,1 Niculina Mang,1 Ioana Mozos,2,3 Giorgiana Flavia Brad1

1First Department of Pediatrics, 2Department of Functional Sciences, 3Center for Translational Research and Systems Medicine, “Victor Babes” University of Medicine and Pharmacy, Timisoara, Romania

Abstract: Osteogenesis imperfecta (OI) is a genetic disease characterized by excessive bone fragility with fractures consecutive to minor trauma. Considering lack of standardization of therapy with pamidronate in children, it was our aim to present our experience over a period of 10 years regarding evolution and treatment in patients diagnosed with osteoporosis and OI. Nine patients diagnosed with OI were admitted to the First Pediatric Clinic, Timisoara. They were investigated (clinical, biomarkers of bone metabolism and imaging studies), and a quality-of-life questionnaire was used to evaluate the impact of OI. Treatment was performed with pamidronate 1 mg/kg/cycle, every 3 months. The patients were evaluated every 3 months. The most frequent was type III (three patients), and two patients were diagnosed with type II, while the other patients were diagnosed with other forms such as types IV, V, VI and VIII. The clinical expression was polymorphic, and the number of fractures was variable. Bone pain ameliorated just after the first cycle of pamidronate, while the activity and mobility increased quickly. Osteodensitometry in children over 12 years showed a decreased bone mineral density (BMD) with a significant improvement after treatment. The values of the bone alkaline phosphatase and osteocalcin changed after the antiresorptive treatment, and the quality of life of the children and their family improved. Treatment with pamidronate is beneficial for the patient, family and society, increases mobility and bone density, improves quality of life and reduces family dependence in children with OI.

Keywords: osteoporosis, child, osteogenesis imperfecta, pamidronate

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