Response to Zoledronic Acid Infusion in Children With Fibrous Dysplasia.

Sujit Kumar Tripathy,Sandeep Velagada,Amit Kumar Satpathy,Rashmi Ranjan Das,Kanhaiyalal Agrawal,Shakti Swaroop,Debashree Priyadarshini

doi:10.3389/fped.2020.582316

Sujit Kumar Tripathy, Sandeep Velagada + Show 5 more

Open Access

https://doi.org/10.3389/fped.2020.582316

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Abstract

Objective: This retrospective study evaluated the outcome and safety of long-term treatment with zoledronic acid, in both polyostotic and mono-ostotic fibrous dysplasia (FD) of children.Methods: The case records of children and adolescents with symptomatic FD who received zoledronic acid (0.1 mg/kg IV infusion over 1 h) and have completed at least 2 years follow-up were analyzed. The relevant details were recorded in a predesigned chart. Clinical assessment [pain assessment by visual analog scale (VAS) and incidence of new fracture], radiological changes (cortical thickening, ossification, and decrease in the diameter of the osteolytic lesions) and biochemical parameters [alkaline phosphatase (ALP)] were used to evaluate the improvement.Results: The mean age of presentation was 9.1 years, with four males and six females. All patients had symptomatic FD in the lower limb with complaints of pain, tenderness, swelling, or deformity. Four children had associated pathological fracture. The radiological evaluation with bone scan revealed polyostotic involvement in eight patients and mono-ostotic involvement in two patients. Three patients had associated systemic features like café-au-lait spots or precocious puberty. The fracture united within 3 months and the radiological improvement was evident in the form of filling of the osteolytic defect. The pain score in six patients showed significant improvement (VAS < 3). The ALP level decreased to 544.12 ± 47.35 IU/L from an initial value of 895.75 ± 79.64 IU/L (p = 0.04) at 12 months. One patient had symptomatic hypocalcaemia after zoledronic acid infusion.Conclusion: The clinical and radiological response of zoledronic acid treatment in FD of children is promising. Further randomized control trials with a larger sample size are required to establish this drug as a first-line medical treatment in FD.

Highlights

Fibrous dysplasia (FD), called as “Lichtenstein–Jaffe disease” is a benign developmental disorder of the bone affecting solitary bone or multiple bones [1]
We report the clinical and radiological response of zoledronic acid in children and adolescent with FD
Between April 2014 and March 2019, 12 children with FD were treated with the zoledronic acid infusion

Summary

Introduction

Fibrous dysplasia (FD), called as “Lichtenstein–Jaffe disease” is a benign developmental disorder of the bone affecting solitary bone or multiple bones [1]. It accounts for 5–7% of the primary benign bone tumors [1]. More than half of the patients present before the third decade of life, and the PFD usually presents in the first decade of life [1,2,3]. MFD presents between the ages of 5–20 years. Many of the patients may not have any symptoms, and it may be an incidental finding [1, 3]

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