ICCBH2019 Poster Presentations (1) (226 abstracts)
The safety and efficacy of denosumab versus zoledronic acid in the treatment of pediatric osteoporosis: a randomized controlled pilot trial
Marie-Eve Robinson 1 , Jinhui Ma 2 , Nasrin Khan 3 , Karine Khatchadourian 4 , Marika Pagé 3 , Victor Konji 3 , Mary Ann Matzinger 5 , Nazih Shenouda 5 , Jacob L Jaremko 6 , Caroline Zuijdwijk 4 , Stefan Jackowski 3 , David Saleh 7 , Lynn MacLeay 3 , Kerry Siminoski 8 & Leanne M Ward 4
1Shriners Hospital for Children - Canada, McGill University, Montreal, Canada; 2Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Canada; 3Children’s Hospital of Eastern Ontario Research Institute, Ottawa, Canada; 4Department of Pediatrics, University of Ottawa, Ottawa, Canada; 5Department of Medical Imaging, Children’s Hospital of Eastern Ontario and University of Ottawa, Ottawa, Canada; 6Department of Radiology and Diagnostic Imaging, University of Alberta, Edmonton, Canada; 7Department of Pediatrics, Queen’s University, Kingston, Canada; 8Department of Radiology and Diagnostic Imaging and Department of Medicine, University of Alberta, Edmonton, Canada.
Objectives: Denosumab (Dmab) is a monoclonal antibody targeting RANKL administered by sub-cutaneous injection. Given its convenient mode of administration, our goal was to assess the safety and efficacy of Dmab compared to intravenous zoledronic acid (ZA) in pediatric osteoporosis.
Methods: In this one-year pilot study (NCT02632916), children 4–16 years with low-trauma fractures due to osteoporosis were randomized 1:1 to receive ZA 0.025 mg/kg or Dmab 1 mg/kg every 6 months (total of 3 doses), followed by calcium supplementation for at least 10 days following each dose. The primary outcome was the proportion of children with at least one episode of hypocalcemia at 48-hours post-dosing. 12-month changes in bone mineral density Z-scores (BMDZ), Spinal Deformity Index (SDI), and the incidence of long bone fractures were assessed with blinding to treatment. Adverse events (AEs) were reported. As a pilot study, all measures but the primary outcome were reported descriptively.
Results: Ten children were enrolled in and completed the study (4 with Duchenne muscular dystrophy and 1 with juvenile osteoporosis in each group, all boys). 80% on ZA had at least one episode of asymptomatic hypocalcemia vs none on Dmab (P=0.01). The mean ± standard deviation (SD) changes in BMDZ in the ZA versus Dmab groups were: lumbar spine (areal) +1.1±0.7 vs +0.4±0.4; hip (areal) +0.7±0.5 vs +0.02±0.2; tibia (volumetric) −0.2±1.7 vs −0.05±0.5). At baseline, the median SDI on ZA was 2 (range 0, 6) vs 0 (range 0, 1) on Dmab, and remained unchanged in both groups at 12 months. Two children sustained low-trauma non-VF (1 radius, 2 femur) on ZA compared to none on Dmab. AEs deemed related to the study drug occurred in 100% on ZA versus 40% on Dmab, none of which were serious. First exposure AEs occurred in 100% of patients on ZA compared to none on Dmab.
Conclusion: In this pilot study, hypocalcemic episodes and side effects were fewer on Dmab compared to ZA, and vertebral fractures stabilized in both groups. These data support further study of Dmab in children with osteoporosis.
Disclosure: Leanne Ward has been a consultant to and participated in clinical trials with Novartis Pharmaceuticals and Amgen Inc.
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