Bisphosphonate Treatment for Children With Disabling Conditions

PM R, 2014 · DOI: 10.1016/j.pmrj.2013.10.009 · Published: May 1, 2014

Endocrinology Pediatrics Musculoskeletal Medicine

Simple Explanation

Bisphosphonates are a class of medications that increase bone mineral density by inhibiting bone resorption. They are synthetic analogs of pyrophosphate, an endogenous regulator of bone metabolism. In bisphosphonates, the central oxygen atom in pyrophosphate is replaced with a carbon atom

Study Duration

Not specified

Participants

Not specified

Evidence Level

Review

Key Findings

1
Data suggest short-term improvements in BMD, whereas effects on fracture risk remain an area of debate.
2
Early studies of pamidronate showed positive preliminary results, with reported improvement in pain, bone turnover markers, and the radiographic appearance of FD lesions
3
All studies demonstrated beneficial effects on BMD but were insufficiently powered to detect an effect on fracture incidence.

Research Summary

Assessment of BMD in children is challenging because of the limitations of DXA in evaluating the growing skeleton. Increasing evidence suggests that bisphosphonates may be beneficial for pediatric disorders of the skeleton; however, their routine use is limited by a lack of long-term safety and efficacy data. The decision to initiate treatment with bisphosphonates in children must be made using clinical judgment, with the clinician weighing the potential risks and benefits for each individual patient.

Practical Implications

Individualized Treatment Decisions

Clinicians must carefully weigh the risks and benefits of bisphosphonate treatment for each child, considering their specific condition and fracture risk.

Further Research Needed

Long-term safety and efficacy data are needed to guide the routine use of bisphosphonates in children with skeletal disorders.

Cautious Use in Specific Conditions

Bisphosphonates may be considered for children with significantly low BMD, fragility fractures, high fracture risk, and disabling bone pain unresponsive to conservative measures, or within clinical trials.

Study Limitations

1
Lack of long-term safety and efficacy data in children.
2
Knowledge gaps regarding optimal dosing, formulation, and target population for bisphosphonate use.
3
Limitations of DXA in evaluating the growing skeleton.

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