Cyclic bisphosphonate therapy reduces pain and improves physical functioning in children with osteogenesis imperfecta

Area of Science:

• Pediatric Orthopedics
• Pharmacology
• Genetics

Background:

• Osteogenesis imperfecta (OI) significantly impacts children's quality of life through pain and functional limitations.
• The long-term effects of cyclic bisphosphonate treatment in pediatric OI patients remain under-described.
• This study investigates the serial impact of intravenous bisphosphonate therapy on pain and function in children with OI.

Purpose of the Study:

• To evaluate the longitudinal effects of cyclic intravenous bisphosphonate therapy on pain and physical functioning in pediatric patients with osteogenesis imperfecta (OI).
• To assess the temporal relationship between bisphosphonate infusions and changes in pain and functional status.
• To explore potential differences in treatment response based on OI severity.

Main Methods:

• A cohort of 22 pediatric patients with OI received cyclic intravenous bisphosphonate therapy.
• Pain was measured using the FACES® visual analogue scale.
• Physical functioning, including self-care, was assessed using the PedsQL™ Generic Core inventory over two infusion cycles.

Main Results:

• Pain scores significantly decreased immediately post-infusion, remaining reduced for 4 weeks before returning to baseline.
• Physical functioning scores improved 4 weeks after infusion, declining before the next cycle.
• No fractures were observed during the study period; exploratory analyses suggested differential pain responses in mild versus moderate-severe OI.

Conclusions:

• Cyclic intravenous bisphosphonate therapy provides transient pain reduction and functional improvement in children with OI.
• Pain relief is immediate post-infusion, with functional gains noted at 4 weeks.
• Both pain and function revert to pre-treatment levels by the time of the subsequent infusion, indicating a temporary therapeutic effect.