TREATMENT WITH PAMIDRONATE IN CHILDREN AND ADOLESCENTS WITH OSTEOGENESIS IMPERFECTA IN SANTA CATARINA

• DOI: https://doi.org/10.22533/at.ed.1594842416098

• Palavras-chave: Osteogenesis Imperfecta, pamidronate, bone fractures, children, quality of life

• Keywords: Osteogenesis Imperfecta, pamidronate, bone fractures, children, quality of life

• Abstract:

  Objectives: The aim of this study was to evaluate the clinical, laboratory and epidemiological profile of patients with OI undergoing early treatment with pamidronate compared to those who did not receive early treatment, at a Reference Center for Osteogenesis Imperfecta (CROI) in Santa Catarina. Methods: We retrospectively collected data from the medical records of patients with OI seen and treated with pamidronate between March 2005 and December 2022, resulting in 81 participants. Information such as age at start of treatment, clinical characteristics, laboratory parameters and number of fractures were analyzed. Results: It was observed that 48.2% of patients started treatment before 2 years, and 80.2% of patients before 6 years. The rate of fractures/year showed a median of 3.89 before and 0.53 after the start of treatment for all ages. In addition, patients who started treatment before the age of 2 showed a reduction in the median fracture rate/year from 33.97 before to 0.97 after starting treatment, with a p-value < 0.001. The median serum alkaline phosphatase for all ages went from 307.5 U/L before to 168 U/L after starting treatment, with statistical significance in patients who started treatment before the age of 6. Conclusion: Pamidronate was effective in reducing the fracture rate/year and serum alkaline phosphatase. It was found that, in all the types of OI analyzed, the earlier the start of treatment, the better the outcomes.