Efficacy of teriparatide vs neridronate in adults with osteogenesis imperfecta type I: a prospective randomized international clinical study

Abstract

SummaryOsteogenesis imperfecta (OI) is an hereditary disease characterized by low bone mass, increased bone fragility, short stature, and skeletal deformities, few treatment options are currently available. Neridronate is an aminobisphosphonate, licensed in Italy for the treatment of OI and Paget's disease of bone. A characteristic property of neridronate is that it can be administered both intravenously and intramuscularly, providing an useful system for administration in homecare. Neridronate appears to increase Bone Mineral Density (BMD) in adults with OI and reduces bone resorption by inhibition of osteoclastic activity. Teriparatide (recombinant 1-34 N terminal sequence of human parathyroid hormone) is the first anabolic agent approved for the treatment of patients with osteoporosis and has been reported to increase bone formation by stimulating osteoblast differentiation, osteoblast function, and survival. The results of this study showed a promising role of teriparatide in the therapy of OI type I.

Osteogenesis Imperfecta: New Perspectives From Clinical and Translational Research

“…This showed increased BMD in mild OI, but was less effective in moderate‐to‐severe OI. In accordance with these findings, positive effects of teriparatide were observed in two studies that focused on adults with OI type I . It therefore appears that teriparatide can be useful for the treatment of mild OI in adults.…”

“…In accordance with these findings, positive effects of teriparatide were observed in two studies that focused on adults with OI type I. (105,106) It therefore appears that teriparatide can be useful for the treatment of mild OI in adults. However, the use of teriparatide in children is contraindicated, as studies in growing rats have shown an increased risk of osteogenic sarcoma.…”

Osteogenesis imperfecta and therapeutics

“…In patients with mild OI (OI type I), osteoblasts were shown to have a preserved anabolic response, although this was somewhat lower compared to that observed in postmenopausal osteoporotic women treated for the same length of time [174]. In mild OI, Teriparatide treatment increased serum levels of markers of bone formation, and improved hip and spine areal bone-mineral density and estimated vertebral strength [174–176]. Patients affected with more severe forms of OI (type III and IV), however, showed no benefits after 18 months of treatment with Teriparatide, indicating that its clinical use in OI may be useful only to patients with mild disease [175].…”

Special form of osteoporosis in a 53-year-old man

“…Bisphosphonates remain the mainstay of treatment in patients with moderate and severe forms of OI 15. However, there is a growing body of evidence that teriparatide may be helpful in patient with milder forms of OI 16 17…”