Bone Abstracts

Acute lymphoblastic leukemia (ALL) is the most frequent form of childhood malignancy, with a cure rate that now exceeds 80%. As survival improves, the clinical consequences of ALL and its treatment are increasingly recognized, with skeletal health emerging as an important care issue. The skeletal morbidity that arises in this setting falls into two main categories: osteoporosis (low trauma fractures) and osteonecrosis (in situ bone death). Osteoporosis and necrosis ha...

Biographical DetailsDr Leanne M Ward is an Associate Professor of Pediatrics at the University of Ottawa where she holds a Research Chair in Pediatric Bone Health. She is the Medical Director of the Pediatric Bone Health Clinical and Research Programs at the Children’s Hospital of Eastern Ontario (CHEO) and a pediatric endocrinologist within the Division of Endocrinology and Metaboli...

Osteoporosis in Duchenne Muscular Dystrophy (DMD) is arguably one of the most severe bone fragility conditions among children with chronic illnesses. This is hardly surprising, given the deleterious effects of the myopathy and glucocorticoid (GC) therapy on bone strength. The severity of the osteoporotic phenotype is highlighted by observations that 60% of boys will sustain long bone fractures during childhood and a third will present with back pain due to vertebral fractures ...

Biographical DetailsLeanne WardDr Leanne Ward is an Associate Professor of Pediatrics at the University of Ottawa where she has held a Research Chair in Pediatric Bone Health since 2010. She is the Medical Director of the Pediatric Bone Health Clinical and Research Programs at the Children’s Hospital of Eastern Ontario. Dr Ward’s research ...

Intravenous (IV) bisphosphonates (pamidronate, zoledronic acid and neridronate) are the mainstays of medical therapy for numerous pediatric bone diseases. While most frequently prescribed for hypercalcemic disorders and osteoporosis, their potent analgesic effects have also led to their use in fibrous dysplasia, osteonecrosis, sickle cell disease, chronic non-bacterial osteomyelitis, and complex regional pain syndrome. Bisphosphonates given IV are highly effective in preventin...

Background: Crohn’s disease (CD) is an inflammatory bowel disease which affects many organ systems including the skeleton. In children with CD, bone mineral density is frequently low. Bone metabolic abnormalities, including lower biochemical measures of bone turnover (NTX) as well as decreased bone formation indices at the tissue level have been reported. The aim of our study was to gain information on the bone matrix mineralization in CD.Methods: W...

Background: Hyperphosphatemic familial tumoral calcinosis (HFTC) is characterized by hyperphosphatemia and the formation of tumor-like extra-osseous calcifications. Tumors often necessitate surgical management although medical treatment may limit the need for surgical intervention. We present two cases of HFTC successfully managed with combination acetazolamide (ACTZ) and sevelamer carbonate.Case Report #1: A 5-year-old African boy with a loss-of-functio...

Duchenne muscular dystrophy (DMD) causes progressive muscle weakness and loss of ambulation. While glucocorticoid (GC) therapy improves motor function, many boys sustain fractures due to osteoporosis. Recently, i.v. bisphosphonate (i.v.BP) therapy has shown promise in the treatment of DMD-related osteoporosis. At the same time, bone histomorphometry revealed lowered bone volume and significant reductions in bone formation pre-i.v.BP treatment, and a further drop after 2 years&...

Objectives: We evaluated the efficacy and safety of intravenous zoledronic acid (IV ZA) in children with glucocorticoid-induced osteoporosis (GIOP) through a randomized, placebo (PBO)-controlled trial.Methods: In this multi-national Phase 3 trial (NCT00799266), children 5–17 years of age with GIOP and low-trauma vertebral fractures (VF) were randomized 1:1 to IV ZA 0.05 mg/kg or IV PBO every six months for one year. Changes in lumbar spine areal bon...

Objective: We compared the efficacy and safety of burosumab, a monoclonal antibody against FGF23, to conventional therapy [oral phosphate and active vitamin D (Pi/D)] in children with X-linked hypophosphatemia (XLH).Methods: In this Phase 3 trial (NCT02915705), 61 children with XLH (1-12 years-old) were randomized 1:1 after a 7-day Pi/D washout to receive burosumab starting at 0.8 mg/kg SC Q2W or reinitiate Pi/D optimally titrated by investigators. Eligi...