BackgroundChildren with osteogenesis imperfecta (OI) experience pain and impaired physical functioning. The longitudinal effect of cyclic bisphosphonate treatment on these symptoms has not been described. We serially evaluated pain and functioning in pediatric patients with OI treated with intravenous bisphosphonate therapy.MethodsPain and physical functioning were assessed at multiple time-points over two infusion cycles in 22 OI patients (median age 10 years [range 2–21 years]; 8 girls) receiving cyclic intravenous bisphosphonate therapy. Pain was assessed using the FACES® visual analogue scale; physical functioning, including self-care, was assessed using the PedsQL™ Generic Core inventory.ResultsPain scores decreased significantly immediately following infusion and remained reduced at 4 weeks post-infusion, increasing before and decreasing again after subsequent infusion (F = 25.00, p < 0.001). Physical functioning scaled scores improved 4 weeks after infusion and declined before subsequent infusion across patients (F = 10.87, p = 0.007). Exploratory analyses indicated significantly different effects between mild and moderate-severe OI types for pain, but not for physical functioning. No fractures occurred during the study.ConclusionIn children with OI, cyclic intravenous bisphosphonate therapy transiently reduces pain and improves functional abilities. Pain relief occurs immediately following infusion with functional improvements observed 4 weeks later. Both pain and physical functioning return to pretreatment levels by the subsequent infusion.
Concerns about a child's growth are one of the most common topics parents voice during general pediatric office visits and are a leading cause for referral to a pediatric endocrinologist. There are a variety of conditions that lead to short stature in children; however, in the absence of true pathology, idiopathic short stature and constitutional delay are the most frequent causes. This article reviews the general approach to evaluating the short child and clinical signs that should prompt further evaluation and referral. We also address the unique psychological issues that these children face and approaches to counseling families with a child with idiopathic short stature.
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