Denosumab use in osteogenesis imperfecta: an update on therapeutic approaches

Majdoub Fatma; Ferjani Hanene Lassoued; Nessib Dorra Ben; Kaffel Dhia; Maatallah Kaouther; Hamdi Wafa

doi:10.6065/apem.2346058.029

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자료유형: 학술저널

저자정보: Majdoub Fatma (Rheumatology Department Kassab Orthopedics Institute Tunisia) Ferjani Hanene Lassoued (Rheumatology Department Kassab Orthopedics Institute Tunisia) Nessib Dorra Ben (Rheumatology Department Kassab Orthopedics Institute Tunisia) Kaffel Dhia (Rheumatology Department Kassab Orthopedics Institute Tunisia) Maatallah Kaouther (Rheumatology Department Kassab Orthopedics Institute Tunisia) Hamdi Wafa (Rheumatology Department Kassab Orthopedics Institute Tunisia)

저널정보: 대한소아내분비학회 Annals of Pediatirc Endocrinology & Metabolism Annals of Pediatric Endocrinology & Metabolism 제28권 제2호

발행연도: 2023.6

수록면: 98 - 106 (9page)

DOI: 10.6065/apem.2346058.029

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Osteogenesis imperfecta (OI) is an inherited skeletal disorder that leads to bone fragility and multiple fractures. Given advances in the genetic understanding of existing phenotypes and newly discovered mutations, therapeutic management of OI has become challenging. Denosumab, a monoclonal antibody that inhibits the interaction between the receptor activator of nuclear factor kappa B ligand (RANKL) and its receptor RANK, has been approved to treat postmenopausal osteoporosis and emerged as an important therapy for malignancies and other skeletal disorders, including pediatric skeletal conditions such as OI. This review summarizes information about denosumab therapy in OI by exploring its mechanisms of action, main indications, and safety and efficacy. Several case reports and small series have been published about the short-term use of denosumab in children with OI. Denosumab was considered a strong drug candidate for OI patients with bone fragility and a high risk of fracture, particularly for patients with the bisphosphonate (BP)-unresponsive OI-VI subtype. The evidence for denosumab's effects in children with OI indicates that it effectively improves bone mineral density but not fracture rates. A decrease in bone resorption markers was observed after each treatment. Safety was assessed by tracking the effects on calcium homeostasis and reporting side effects. No severe adverse effects were reported. Hypercalciuria and moderate hypercalcemia were reported, suggesting that BPs be used to prevent the bone rebound effect. In other words, denosumab can be used as a targeted intervention in children with OI. The posology and administration protocol require more investigation to achieve secure efficiency.

#Osteogenesis imperfecta #Denosumab #RANK ligand #RANK

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