ICCBH2017 Poster Presentations (1) (209 abstracts)
1Murdoch Childrens Research Institute, Melbourne, Victoria, Australia; 2Monash University, Melbourne, Victoria, Australia; 3Royal Childrens Hospital, Melbourne, Victoria, Australia; 4Deakin University, Melbourne, Victoria, Australia.
Objectives: Intravenous bisphosphonates have been used in children for various primary and secondary bone fragility disorders for three decades but beyond osteogenesis imperfecta, there is very limited information published in relation to outcomes. We report the experience at the Royal Childrens Hospital (RCH), Melbourne using Zoledronic acid (ZA), describing outcomes based on the underlying condition for which treatment was given, with the aim of informing future protocols and guidelines for bisphosphonate use.
Methods: A retrospective review of all RCH patients administered at least one dose of intravenous ZA from 20022015 was undertaken. All outcome data was collected from existing hospital medical records.
Results: In the 13 years of ZA use, 325 children had at least one dose of ZA; 12 of these were excluded from study inclusion due to either missing documentation regarding use of ZA or indications of extreme prematurity, renal osteodystrophy or intra-arterial calcification. Children included in this review were not taking any other bisphosphonates. Of the 313 patients, Table 1 describes preliminary data outcomes grouped for each condition; more analysis is currently being done on changes per year and after first year of treatment. Mild to moderate first dose acute phase reaction occurred in most with symptomatic acute day 2 hypocalcaemia in several who had steroid induced osteoporosis.
Indication for ZA use | n | Outcomes |
Bone abnormality indications | ||
Avascular necrosis (AVN) | 36 | 3/36 collapse within 12 months |
Bone metastases | 8 | 4/8 significant pain relief |
Fibrous dysplasia | 11 | 8/11 significant pain relief, no lesion size change |
Non-union | 3 | 2/3 complete union within 6 months, 1/3 partial healing at 12 months |
Massive sacral bone erosion by neurofibroma | 1 | Significant pain relief but no size change |
Aneurysmal bone cysts | 4 | 2/4 significant reduction in lesion size after 12 months |
Chronic recurrent multifocal osteomyelitis (prior to availability of biology) | 3 | 2/3 significant pain relief and no major change in lesion size |
Other indications | ||
Osteogenesis imperfecta | 117 | Median 73.4% (Interquartile range 35%, 97%) increase in lumbar spine bone mineral density from baseline after 112 years of treatment |
Osteoporosis inclusive of steroid dependent non DMD neuromuscular diseases | 57 | Median 44.2% (Interquartile range 23%, 72%) increase in lumbar spine bone mineral density from baseline after 110 years of treatment |
Duchenne Muscular Dystrophy (DMD) | 16 | Median 21% (Interquartile range 11%, 32%) increase in lumbar spine bone mineral density from baseline after 17 years of treatment |
Immobility | 55 | Median 39% (Interquartile range 24%, 57%) increase in lumbar spine bone mineral density from baseline over 110 years of treatment |
Hypercalcaemia | 2 | Normalization of serum calcium, with reactive hypocalcaemia for 2448 hours 24 days post dose |
Conclusion: Zoledronic acid demonstrated a good efficacy profile, with improved bone density for osteoporotic conditions, significant pain relief in all treated bone abnormality indications, and stabilization of lesion size with reduced incidence of bone collapse in AVN.
Disclosure: The authors declared no competing interests