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Assessment of the Efficacy of An Osteopathic Treatment in Infants with Biomechanical Impairments to Suckling
Published on: February 5, 2019
Zoledronic Acid Treatment in Infants and Toddlers with Osteogenesis Imperfecta is Safe and Effective: A Tertiary Care
Angad Kumar1, Uma K Saikia1, Ashok K Bhuyan1
1Department of Endocrinology, Gauhati Medical College and Hospital, Guwahati, Assam, India.
Insights
Zoledronic acid (ZA) effectively treats osteogenesis imperfecta (OI) in children under five, reducing fractures and pain while improving height and motor skills. This safe and convenient therapy offers a promising alternative for managing OI in young patients.
Area of Science:
- Pediatric Endocrinology
- Bone Metabolism
- Genetic Disorders
Background:
- Osteogenesis imperfecta (OI) is a genetic disorder affecting bone quality, leading to fractures, developmental delays, and pain.
- Pamidronate is an established treatment, but zoledronic acid (ZA) is emerging for OI management.
Purpose of the Study:
- To evaluate the efficacy and safety of zoledronic acid (ZA) in treating osteogenesis imperfecta (OI) in children under five years of age.
Main Methods:
- A prospective observational study involving children under five with OI treated with intravenous ZA (0.05 mg/kg every six months).
- Monitoring included clinical variables (pain, fractures, height, motor development), biochemical parameters, and adverse events over two years.
- Clinical Severity Score (CSS) was assessed at baseline and after two years.
Main Results:
- Two-year ZA treatment significantly reduced fracture rates (p < 0.001) and alleviated pain (p < 0.001).
- Improvements were observed in ambulation (p = 0.005) and height standard deviation score (p < 0.05).
- Significant improvement in CSS and only mild, transient adverse effects (flu-like symptoms, asymptomatic hypocalcemia) were noted.
Conclusions:
- Zoledronic acid (ZA) is an effective and safe treatment for osteogenesis imperfecta (OI) in infants and young children.
- ZA offers a more convenient therapeutic option compared to pamidronate for this pediatric population.
Context:
Osteogenesis imperfecta (OI) is a genetic disorder of the extracellular matrix of bone characterized by low bone mass manifesting as frequent fractures, delayed motor development, pain, and impaired quality of life. The intravenous bisphosphonate, pamidronate is an established treatment for OI. Recently, zoledronic acid (ZA) has been used for the management of OI.
Aim:
To assess the efficacy and safety of ZA in children below five years of age with OI.
Settings And Design:
A hospital-based prospective observational study.
Methods And Material:
Patients with OI aged less than five years attending our centre were treated with intravenous ZA at a dose of 0.05 mg/kg every six months. Subjects were closely monitored for clinical and biochemical variables, adverse events, and new-onset fractures. The response to therapy was assessed by monitoring clinical variables including the degree of bony pains, number of fractures, height/length standard deviation score (SDS), and motor developmental milestones. All patients were analysed at baseline and at the end of two years for biochemical parameters and clinical severity score (CSS) as proposed by Aglan et al. with modifications.
Results:
After two years of treatment, OI patients showed a significant decline in the rate of fractures (p < 0.001), improvement in ambulation (p = 0.005), alleviation of pain (p < 0.001), and improvement in height SDS (p < 0.05). There was a significant improvement in CSS after two years of therapy. Apart from mild flu-like symptoms and mild asymptomatic hypocalcaemia immediately post-infusion, no other adverse effect was noted.
Conclusion:
ZA therapy in infants and children below five years of age with OI was effective and safe and a more convenient alternative to pamidronate.
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