caa a22 4500 605463441 CHVBK 20210128100253.0 cr unu---uuuuu 210128e20150901xx s 000 0 eng 10.1007/s00774-014-0618-2 doi (NATIONALLICENCE)springer-10.1007/s00774-014-0618-2 Beneficial effects of intravenous pamidronate treatment in children with osteogenesis imperfecta under 24months of age [Elektronische Daten] [Kirsten Kusumi, Rose Ayoob, Sasigarn Bowden, Susan Ingraham, John Mahan] Osteogenesis imperfecta (OI) is an inherited disorder characterized by bone fragility and low bone mass. Low bone density and fracture is a cause of morbidity. Limited data exists on bisphosphonate treatment in patients under 24months of age. The objective of the study was to examine the safety and efficacy of pamidronate in children under 24months with OI. To do so, we carried out a retrospective chart review and analysis of OI patients started on intravenous pamidronate under 24months of age. Pamidronate was administered in three-day cycles. Growth, the number of fractures, and lumbar bone mineral densities were recorded both prior to and after treatment initiation. A total of 18 patients were reviewed. Five were classified as OI type I, seven were type III, and six were type IV. The mean age at treatment initiation was 12months (range 11days to 23months). The mean lumbar z score at baseline was −3.63, which improved to −1.53 at one year (P<0.01) and 0.79 (P<0.01) at the end of the study. The fracture rate improved from 68 fractures in 209months (0.32 fractures/patient-month) before treatment to 41 fractures in 1,248months (0.03 fractures/patient-month) post-treatment (P<0.05). Height standard deviation score (SDS) was conserved from baseline to end of study (−2.12±2.45 vs. −2.45±2.73) (P=0.05) with an average follow-up of 73months. The only adverse effect recorded in six infants was fever during the initial pamidronate infusion. Treatment with intravenous pamidronate is safe, significantly improves lumbar bone mineral density (L-BMD), and reduces fracture rates in young infants with OI while preserving linear growth. The Japanese Society for Bone and Mineral Research and Springer Japan, 2014 Pediatrics nationallicence Osteogenesis imperfecta nationallicence Treatment nationallicence Growth nationallicence Kusumi Kirsten Division of Nephrology, Department of Pediatrics, Nationwide Children's Hospital/The Ohio State University, 700 Children's Dr., 43205, Columbus, OH, USA aut Ayoob Rose Division of Nephrology, Department of Pediatrics, Nationwide Children's Hospital/The Ohio State University, 700 Children's Dr., 43205, Columbus, OH, USA aut Bowden Sasigarn Division of Endocrinology, Department of Pediatrics, Nationwide Children's Hospital/The Ohio State University, Columbus, OH, USA aut Ingraham Susan Division of Nephrology, Department of Pediatrics, Nationwide Children's Hospital/The Ohio State University, 700 Children's Dr., 43205, Columbus, OH, USA aut Mahan John Division of Nephrology, Department of Pediatrics, Nationwide Children's Hospital/The Ohio State University, 700 Children's Dr., 43205, Columbus, OH, USA aut Journal of Bone and Mineral Metabolism Springer Japan 33/5(2015-09-01), 560-568 0914-8779 33:5<560 2015 33 774 https://doi.org/10.1007/s00774-014-0618-2 text/html Onlinezugriff via DOI BK010053 XK010053 XK010000 Metadata rights reserved Springer special CC-BY-NC licence nationallicence 1 research-article jats NATIONALLICENCE NATIONALLICENCE NL-springer NATIONALLICENCE 856 40 https://doi.org/10.1007/s00774-014-0618-2 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Kusumi Kirsten Division of Nephrology, Department of Pediatrics, Nationwide Children's Hospital/The Ohio State University, 700 Children's Dr., 43205, Columbus, OH, USA aut NATIONALLICENCE 700 1- Ayoob Rose Division of Nephrology, Department of Pediatrics, Nationwide Children's Hospital/The Ohio State University, 700 Children's Dr., 43205, Columbus, OH, USA aut NATIONALLICENCE 700 1- Bowden Sasigarn Division of Endocrinology, Department of Pediatrics, Nationwide Children's Hospital/The Ohio State University, Columbus, OH, USA aut NATIONALLICENCE 700 1- Ingraham Susan Division of Nephrology, Department of Pediatrics, Nationwide Children's Hospital/The Ohio State University, 700 Children's Dr., 43205, Columbus, OH, USA aut NATIONALLICENCE 700 1- Mahan John Division of Nephrology, Department of Pediatrics, Nationwide Children's Hospital/The Ohio State University, 700 Children's Dr., 43205, Columbus, OH, USA aut NATIONALLICENCE 773 0- Journal of Bone and Mineral Metabolism Springer Japan 33/5(2015-09-01), 560-568 0914-8779 33:5<560 2015 33 774