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. 2017 Aug 28:11:2507-2515.
doi: 10.2147/DDDT.S141075. eCollection 2017.

Therapy with pamidronate in children with osteogenesis imperfecta

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

Therapy with pamidronate in children with osteogenesis imperfecta

Otilia Marginean et al. Drug Des Devel Ther. .

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: child; osteogenesis imperfecta; osteoporosis; pamidronate.

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Conflict of interest statement

Disclosure The authors report no conflicts of interest in this work.

Figures

Figure 1
Figure 1
Newborn with triangle face.
Figure 2
Figure 2
Short stature due to limb fractures with large head and barrel-shaped rib cage.
Figure 3
Figure 3
Dentinogenesis imperfecta with brown–yellow spots, malposition and friability.
Figure 4
Figure 4
The evolution of Z-score in patients before and after 3 years of treatment with pamidronate.
Figure 5
Figure 5
Scatter diagram of serum alkaline phosphatase values and the number of fractures in patients before treatment.
Figure 6
Figure 6
Scatter diagram between the serum alkaline phosphatase and number of fractures in patients after 3 years of treatment.
Figure 7
Figure 7
The evolution of serum bone alkaline phosphatase before and after 3-year period of treatment with pamidronate.
See this image and copyright information in PMC

References

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    1. Orphanet [webpage on the Internet] Osteogenesis Imperfecta. [Accessed February 2017]. Available from: http://www.orpha.net/consor/cgi-bin/OC_Exp.php?Expert=666.
    1. Ramachandran M, Gellman H, Achan P, et al. Osteogenesis Imperfecta. [Accessed February 2017]. webpage on the Internet. Available from: http://emedicine.medscape.com/article/1256726-overview.
    1. Leiden University Medical Center [database on the Internet] Osteogenesis Imperfecta Variant Database. [Accessed February 2017]. Available from: https://oi.gene.le.ac.uk.

MeSH terms