High Burden of Fracture and Musculoskeletal Disorders in Osteogenesis Imperfecta Patients
Researchers conducted a retrospective study of 5722 patients with osteogenesis imperfecta (OI) using the IQVIA PharMetrics Plus database. The study aimed to quantify fracture rate and occurrence of musculoskeletal disorders among OI patients of all ages and those treated with off-label therapies. The results showed that approximately 40% of patients with OI and 37% of those with off-label treatments had a fracture within one year of diagnosis or treatment. The annual average fracture rates were 0.84 and 0.98, respectively, compared to 3% fracture occurrence and a 0.03 annual fracture rate in matched non-OI comparators.
Key Takeaways:
- The study found a high burden of fracture and musculoskeletal disorders in OI patients, which are not effectively managed by current management strategies or off-label treatments.
- About 40% of patients with OI and 37% of those with off-label treatments had a fracture within one year of diagnosis or treatment.
- The annual average fracture rates of these groups were 0.84 and 0.98, respectively, compared to 3% fracture occurrence and a 0.03 annual fracture rate in matched non-OI comparators.
- Pediatric patients had the highest fracture occurrence and fracture rate, both among patients with OI and the subgroup of treated OI patients.
- Musculoskeletal disorders and diagnosed pain were more frequent in OI patients compared with the non-OI cohort, especially among pediatric age groups and adults.
Statistics:
- 5722 patients with OI were identified in the IQVIA PharMetrics Plus database.
- 2095 patients (55.7% female; 35.1% pediatric; 20% with OI-related treatments; 87.1% commercial-insured) were included in analyses.
- 40% of patients with OI had a fracture within one year of diagnosis.
- 37% of patients with off-label treatments had a fracture within one year of treatment.
- The annual average fracture rates were 0.84 for patients with OI and 0.98 for patients with off-label treatments.
- 3% fracture occurrence and a 0.03 annual fracture rate were observed in matched non-OI comparators.
Sources:
- NewsRx. New Findings from Ultragenyx Pharmaceutical Inc. Describe Advances in Osteogenesis Imperfecta (Real-world data of fracture rates and musculoskeletal disorders for patients living with osteogenesis imperfecta). Pediatrics Week. October 11, 2025; p 131.