[Biphosphonates and osteogenesis imperfecta in children]

S Gandon-Laloum1

  • 1Service de chirurgie infantile, centre hospitalier régional universitaire de Caen, avenue de la Côte-de-Nacre, 14033 Caen cedex 9, France. gandonlaloum-s@chu-caen.fr

Insights

Biphosphonate therapy has improved managing children with osteogenesis imperfecta for over a decade. This review covers its use, safety, and long-term skeletal persistence, offering valuable insights for pediatric bone disorder treatment.

Area of Science:

  • Pediatric Orthopaedics
  • Pharmacology
  • Bone Metabolism

Background:

  • Osteogenesis imperfecta (OI) is a rare genetic disorder characterized by brittle bones.
  • Severe and moderate forms of OI significantly impact children's quality of life and mobility.
  • Traditional treatments like physiotherapy and surgery have limitations in managing severe OI.

Purpose of the Study:

  • To review the established role of biphosphonate therapy in managing pediatric osteogenesis imperfecta.
  • To detail the indications, administration protocols, and safety profile of biphosphonate treatment in children.
  • To discuss the long-term skeletal retention and efficacy of biphosphonates in pediatric OI patients.

Main Methods:

  • Systematic review of biphosphonate therapy in pediatric OI management.
  • Analysis of clinical data on efficacy, safety, and administration of biphosphonates.
  • Evaluation of studies reporting on the duration and long-term skeletal persistence of biphosphonates.

Main Results:

  • Biphosphonate therapy has been a key advancement in OI management for over 10 years.
  • Established protocols exist for indications, administration routes, and treatment duration.
  • Safety data indicates a generally favorable profile, but long-term skeletal persistence requires further investigation.

Conclusions:

  • Biphosphonate therapy is a cornerstone in the multidisciplinary management of pediatric osteogenesis imperfecta.
  • Understanding administration and safety is crucial for optimal patient outcomes.
  • Further research is needed to clarify the long-term skeletal fate and sustained benefits of biphosphonates in children.

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