Study of the use of zoledronic acid in pediatric patients with osteogenesis imperfecta at the Fernandes Figueira Institute (IFF/Fiocruz)

Authors

DOI:

https://doi.org/10.33448/rsd-v15i1.50474

Keywords:

Bisphosphonates, Bones fractures, Osteogenesis imperfecta, Zoledronic acid, Drug therapy.

Abstract

Osteogenesis Imperfecta (OI) is a rare genetic disorder characterized by bone fragility and wide clinical variability. This study aimed to evaluate the use of zoledronic acid in pediatric patients with OI treated at the Fernandes Figueira Institute (IFF/Fiocruz), describing the clinical profile, therapeutic response, and safety. This retrospective study was conducted between April 2024 and August 2025 and was based on the medical record review of 40 patients aged 2 to 18 years. An equal distribution between sexes was observed, with higher frequency of OI types I, IV, and III. Intravenous pamidronate was the most frequently used initial bisphosphonate, followed by oral alendronate as a transition strategy, while zoledronic acid was subsequently incorporated into the therapeutic regimen of all patients. Adverse events were more common with pamidronate (32.5%) than with zoledronic acid (17.5%), including fever, bone pain, headache, and gastrointestinal symptoms. Before zoledronic acid use, all patients had a history of fractures; after its introduction, only 12.5% reported new episodes, demonstrating a marked reduction. Subjective perception of clinical improvement was recorded in 7.5% of patients. NTX (n=3) and DEXA (n=2) results were insufficient for conclusions regarding bone remodeling or mineral density. Despite limitations related to the scarcity of complementary exams, the findings suggest clinical benefit of zoledronic acid, reinforcing its potential to reduce fractures and improve OI management in pediatric patients within the Brazilian public health context.

References

Barros, E. R., Saraiva, G. L., de Oliveira, T. P., & Lazaretti-Castro, M. (2012). Safety and efficacy of a 1-year treatment with zoledronic acid compared with pamidronate in children with osteogenesis imperfecta. Journal of Pediatric Endocrinology & Metabolism, 25(5–6), 485–491. https://doi.org/10.1515/jpem-2012-0016

Bishop, N., Arundel, P., Clark, E., Dimitri, P., Farr, J., Jones, G., & Ward, L. M. (2016). Fracture prediction and the definition of osteoporosis in children and adolescents: The ISCD 2013 pediatric official positions. Journal of Clinical Densitometry, 17(2), 275–280. https://doi.org/10.1016/j.jocd.2014.01.004

Cestari, C. G., et al. (2022). Osteogênese imperfeita: Revisão de literatura e relato de casos. Revista Unilago, 2(1).

Forlino, A., & Marini, J. C. (2016). Osteogenesis imperfecta. The Lancet, 387(10028), 1657–1671. https://doi.org/10.1016/S0140-6736(15)00728-X

George, S., et al. (2015). Short-term safety of zoledronic acid in young patients with bone disorders: An extensive institutional experience. The Journal of Clinical Endocrinology & Metabolism, 100(11), 4163–4170. https://doi.org/10.1210/jc.2015-2680

Glorieux, F. H., Bishop, N. J., Plotkin, H., Chabot, G., Lanoue, G., & Travers, R. (1998). Cyclic administration of pamidronate in children with severe osteogenesis imperfecta. The New England Journal of Medicine, 339(14), 947–952. https://doi.org/10.1056/NEJM199810013391402

Kumar, A., Saikia, U. K., Bhuyan, A. K., Baro, A., & Prasad, S. G. (2023). Zoledronic acid treatment in infants and toddlers with osteogenesis imperfecta is safe and effective: A tertiary care centre experience. Indian Journal of Endocrinology and Metabolism, 27(3), 255–259. https://doi.org/10.4103/ijem.ijem_268_22

Land, C., Rauch, F., Montpetit, K., Ruck-Gibis, J., & Glorieux, F. H. (2006). Effect of intravenous pamidronate therapy on functional abilities and level of ambulation in children with osteogenesis imperfecta. The Journal of Pediatrics, 148, 456–460. https://doi.org/10.1016/j.jpeds.2005.10.041

Lima, M. A. F. D., et al. (2014). Contradições das políticas públicas voltadas para doenças raras: O exemplo do programa de tratamento da osteogênese imperfeita no SUS. Ciência & Saúde Coletiva, 19(2), 475–480. https://doi.org/10.1590/1413-81232014192.15582012

Luís, V. L. F. (2013). Osteogenesis imperfecta: Diagnóstico, tratamento e prognóstico (Dissertação de mestrado). Universidade do Porto.

Mahmoud, I., Bouden, S., Sahli, M., Rouached, L., Ben Tekaya, A., Tekaya, R., Saidane, O., & Abdelmoula, L. (2024). Efficacy and safety of intravenous zoledronic acid in the treatment of pediatric osteogenesis imperfecta: A systematic review. Journal of Pediatric Orthopaedics Part B, 33(3), 283–289. https://doi.org/10.1097/BPB.0000000000001104

Melo, P. C. (2015). Osteogênese imperfeita – “Ossos de cristal”: Revisão bibliográfica (Dissertação de mestrado). Universidade da Beira Interior.

Ministério da Saúde. (2022). Protocolo clínico e diretrizes terapêuticas da osteogênese imperfeita (Portaria Conjunta nº 17, de 8 de setembro de 2022).

Montedor, T. S., et al. (2023). Osteogênese imperfeita: Uma revisão literária. Contribuciones a las Ciencias Sociales, 16(9), 18683–18696. https://doi.org/10.55905/revconv.16n.9-288

Otaify, G. A., et al. (2020). Zoledronic acid in children with osteogenesis imperfecta and Bruck syndrome: A 2-year prospective observational study. Osteoporosis International, 27(1), 81–92. https://doi.org/10.1007/s00198-015-3216-9

Paiva, D. F., et al. (2018). Percepções de pessoas com osteogênese imperfeita acerca das intervenções terapêuticas ocupacionais e possibilidades de cuidado. Cadernos Brasileiros de Terapia Ocupacional, 26(2), 399–407. https://doi.org/10.4322/2526-8910.ctoAO1135

Pereira, A. S., et al. (2018). Metodologia da pesquisa científica [e-book]. Editora da UFSM.

Rauch, F., et al. (2006). Pamidronate in children and adolescents with osteogenesis imperfecta: Effect of treatment discontinuation. The Journal of Clinical Endocrinology & Metabolism, 91(4), 1268–1274. https://doi.org/10.1210/jc.2005-2413

Ribeiro, V. C. C. (2021). Osteogênese imperfeita: Novas tecnologias na prevenção de fraturas – Uma revisão sistemática de literatura (Dissertação de mestrado). Universidade Federal da Bahia.

Sánchez-Sánchez, L. M. (2015). Ácido zoledrónico (zolendronato) en niños con osteogénesis imperfecta. Gaceta Médica de México, 152(2), 164–168.

Shitsuka, R., et al. (2014). Matemática fundamental para a tecnologia (2ª ed.). Editora Érica.

Vuorimies, I., et al. (2011). Zoledronic acid treatment in children with osteogenesis imperfecta. Journal of Bone and Mineral Research, 26(9), 2187–2194. https://doi.org/10.1159/000323368

Ward, L., et al. (2016). Bone health and osteoporosis in children and adolescents. Pediatrics, 138(4), e20160296. https://doi.org/10.1542/peds.2016-0296

Published

2026-01-05

Issue

Section

Health Sciences

How to Cite

Study of the use of zoledronic acid in pediatric patients with osteogenesis imperfecta at the Fernandes Figueira Institute (IFF/Fiocruz). Research, Society and Development, [S. l.], v. 15, n. 1, p. e0615150474, 2026. DOI: 10.33448/rsd-v15i1.50474. Disponível em: https://rsdjournal.org/rsd/article/view/50474. Acesso em: 22 jan. 2026.