Early bisphosphonate treatment in infants with severe osteogenesis imperfecta

New Aspects of Genetic Basis, Classification and Treatment of Osteogenesis Imperfecta: Literature Review

“…В про-спективном исследовании эффективности терапии нери-дронатом у младенцев с несовершенным остеогенезом продемонстрированы более высокие показатели физи-ческого развития и редкие эпизоды переломов костей в группе пациентов, у которых к лечению приступили сразу же после рождения, чем в группе детей, у которых терапия была начата после 6 мес жизни [42].…”

Current and emerging treatments for the management of osteogenesis imperfecta

“…In contrast, experience with BPs in children with various pathologies, such as juvenile osteoporosis, has been limited until recently and most of the current reports refer to small numbers of patients. [83][84][85][86] Following limited early studies, 87,88 several researchers have demonstrated that oral (olpadronate 89,90 and alendronate 91,92 ) or intravenous (pamidronate [93][94][95][96][97][98][99][100][101] and neridronate 102,103 ) BP treatment has a beneficial effect on children with severe OI. 104 In a large non-controlled observational study involving 30 children (aged 3 to 16 years) with severe OI, Glorieux and colleagues demonstrated that the cyclic administration of intravenous pamidronate improved clinical outcomes, decreased the incidence of fractures, reduced bone resorption, increased bone density and the size of vertebral bodies.…”

“…106 Despite unanswered questions concerning them, BP treatment does appear to have a strong positive effect on the morbidity of severe forms of OI and it seems reasonable to continue the treatment more extensively. 107 These studies [93][94][95][96][97][98][99][100][101][102][103] reported encouraging results and strongly indicate that BPs are useful in the symptomatic treatment of children with severe OI, moreover without serious adverse effects. As with earlier studies these current trials share a common limitation, namely they are all open trials, therefore these favorable results should be confirmed in double-blind controlled studies.…”

“…Indeed, promising results were reported in patients who received BPs in the first years of life. 80,103,105 When treatment is being considered, since the knowledge of the long term tolerability is very limited and not all countries have BP therapy authorized in children, after the treatment regimen is explained, it may be necessary for an informed consent to be obtained from the parents and, if appropriate, from the child. As for the choice of BP, there are groups that treat OI patients (including children with moderate to severe OI) with cyclical intravenous pamidronate, in cycles of three days, repeated every 2 to 4 months (with an annual dose of 9 mg/kg/year).…”

“…97,98 In our experience any advantage in terms of duration of treatment results from the cyclical intravenous administration of neridronate (which is the only BP authorized in children with OI in Italy) received as an infusion over 3 hours in a single day (the dose is subdivided in two days in children younger than 3 years) and is generally repeated every 3 months (with an annual dose of 8 mg/kg/year). 102,103 Zoledronic acid (an infusion given over 45 minutes for one day) has been recently used 108 although it is still undergoing clinical trials, in infants.…”