Abstract
Osteoporosis in the youth most often accompanies severe illness, such as inborn errors of metabolism, or therapy affecting bone metabolism (e.g. steroid). Calcium supplementation, sodium fluoride, vitamin D3 and derivatives, calcitonin, while effective in relenting the progression of the disease, are not satisfactory as to recover from conspicuous loss of bone mass. By contrast, increasing experience is being collected in the use of bisphosphonates, a class of molecules able to inhibit osteoclastic activity of bone resorption. We have treated with 3amino Ihydroxy propylidene 1, Ibisphosphonate (APD) 5 patients aged 6.5 to 20 years, with severe osteoporosis: two had primitive bone disease (Gaucher's disease, familial hypophosphatasia), one was affected by cystic fibrosis, two received steroid (for rheumatoid arthritis and after bone marrow transplantation for acute leukemia). Two of them had experienced spontaneous fractures, the others had mostly limb and/or back pain. The calcium content of bone had been assessed by means of computed densitometry of lumbar vertebrae: the figure was reduced to 4-125 mg/ml of bone (normal range for age approx. 150-200). The APD was administered intravenously, 5-7.5 mg/day for 5 days, then 10 mg once every three weeks, Moderate hypocalcemia observed after the first infusions was prevented by oral calcium supplementation, 500-1500 mg/day for a few days following each infusion. After a follow-up ranging 5-12 months, the vertebral CT scan showed an increase of calcium content by 26 - 1325 %, the figure attaining normality in two cases: the best result was obtained in the boy with the lowest pretreatment value. Symptoms and regional radiographs also showed comparable improvement. Side effects were self-limited fever following infusions in one patient, and transient calf pain in another, We conclude that APD can be effectively and safely used in children and adolescents to cure severe osteoporosis, both primitive and iathrogenic.
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Marinoni, S., Bembi, B., Tonini, G. et al. THERAPY WITH AMINOHYDROXYPROPYLIDENE BISPHOSPHONATE IN YOUNG PATIENTS WITH SEVERE OSTEOPOROSIS. Pediatr Res 33 (Suppl 5), S80 (1993). https://doi.org/10.1203/00006450-199305001-00461
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DOI: https://doi.org/10.1203/00006450-199305001-00461