ABSTRACT
OBJECTIVE: To evaluate prospectively the efficacy of bisphosphonate treatment in infants with severe forms of osteogenesis imperfecta (OI). STUDY DESIGN: Of 10 children (6 females) with OI type III, 5 (group A) started treatment (2 mg/kg neridronate administered intravenously for 2 consecutive days, every 3 months) just after diagnosis at birth and 5 (group B) after 6 months. Ten untreated children, matched for sex, age, and clinical severity of OI, constituted a historical control group (group C). We measured weight, length, and number of fractures every 3 months and serum and urinary levels of calcium, phosphorus, creatinine, serum alkaline phosphatase, 25-hydroxyvitamin D, insulin-like growth factor I, parathyroid hormone, and osteocalcin, urinary type I collagen N-terminal telopeptide, and lateral radiography of vertebral column every 6 months. RESULTS: Group A had better growth and a lower incidence of fractures than groups B and C in the first 6 months of treatment. In the second 6 months, both groups A and B had lower fracture rates than group C. After 12 months of therapy, osteocalcin and insulin-like growth factor I levels significantly increased only in group A. The urinary Ca/Cr ratio and N-terminal telopeptide/Cr ratio significantly declined only in treated patients. Vertebral body area and the structure of vertebral bodies improved in all treated patients, but especially in group A. CONCLUSIONS: Cyclical neridronate treatment, started just after diagnosis at birth, had positive effects on growth and fracture rate.
Subject(s)
Diphosphonates/therapeutic use , Osteogenesis Imperfecta/diagnosis , Osteogenesis Imperfecta/drug therapy , Absorptiometry, Photon , Anthropometry , Calcium/blood , Calcium/urine , Drug Administration Schedule , Early Diagnosis , Female , Follow-Up Studies , Fractures, Bone/epidemiology , Humans , Incidence , Infant , Infant, Newborn , Lumbar Vertebrae/diagnostic imaging , Male , Osteocalcin/blood , Osteocalcin/urine , Osteogenesis Imperfecta/epidemiology , Phosphates/blood , Phosphates/urine , Prevalence , Prospective Studies , Severity of Illness Index , Time Factors , Treatment OutcomeABSTRACT
Pediatric patients with Graves' disease (n=26) were studied longitudinally by magnetic resonance imaging of the orbits, allowing an assessment of the enlargement of the extraocular muscles and orbital volume variations. The positive outcome of Graves' ophthalmopathy correlated with low TRAb (autoantibodies to thyroid-stimulating hormone receptor) titers at diagnosis and during follow-up and with prepubertal condition at diagnosis.