Evaluation of a Modified Pamidronate Protocol for the Treatment of Osteogenesis Imperfecta

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• 作者：Telma Palomo ; Maria C. Andrade ; Barbara S. E. Peters…
• 关键词：Pamidronate ; Safety ; Renal function ; Osteogenesis imperfecta ; Efficacy
• 刊名：Calcified Tissue International
• 出版年：2016
• 出版时间：January 2016
• 年：2016
• 卷：98
• 期：1
• 页码：42-48
• 全文大小：493 KB
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• 作者单位：Telma Palomo (1) (5)
  Maria C. Andrade (2)
  Barbara S. E. Peters (3)
  Fernanda A. Reis (4)
  João Tomás A. Carvalhaes (2)
  Francis H. Glorieux (5)
  Frank Rauch (5)
  Marise Lazaretti-Castro (1)
  
  1. Bone and Mineral Unit, Division of Endocrinology, Universidade Federal de São Paulo, São Paulo, Brazil
  5. Shriners Hospital for Children, 1529 Cedar Avenue, Montreal, QC, H3G 1A6, Canada
  2. Department of Pediatric Nephrology, Universidade Federal de São Paulo, São Paulo, Brazil
  3. Department of Nutrition, Public Health School, USP, São Paulo, Brazil
  4. Department of Radiology, Universidade Federal de São Paulo, São Paulo, Brazil
  
• 刊物类别：Biomedical and Life Sciences
• 刊物主题：Life Sciences
  Biochemistry
  Endocrinology
  Orthopedics
  Cell Biology
  
• 出版者：Springer New York
• ISSN：1432-0827

文摘

Intravenous pamidronate is widely used to treat children with osteogenesis imperfecta (OI). In a well-studied protocol (‘standard protocol’), pamidronate is given at a daily dose of 1 mg per kg body weight over 4 h on 3 successive days; infusion cycles are repeated every 4 months. Here, we evaluated renal safety of a simpler protocol for intravenous pamidronate infusions (2 mg per kg body weight given in a single infusion over 2 h, repeated every 4 months; ‘modified protocol’). Results of 18 patients with OI types I, III, or IV treated with the modified protocol for 12 months were compared to 18 historic controls, treated with standard protocol. In the modified protocol, mild transient post-infusion increases in serum creatinine were found during each infusion but after 12 months serum creatinine remained similar from baseline [0.40 mg/dl (SD: 0.13)] to the end of the study [0.41 mg/dl (SD: 0.11)] (P = 0.79). The two protocols led to similar changes in serum creatinine during the first pamidronate infusion [modified protocol: +2 % (SD: 21 %); standard protocol: −3 % (SD: 8 %); P = 0.32]. Areal lumbar spine bone mineral density Z-scores increased from −2.7 (SD: 1.5) to −1.8 (SD: 1.4) with the modified protocol, and from −4.1 (SD: 1.4) to −3.1 (SD: 1.1) with standard protocol (P = 0.68 for group differences in bone density Z-score changes). The modified pamidronate protocol is safe and may have similar effects on bone density as the standard pamidronate protocol. More studies are needed with longer follow-up to prove anti-fracture efficacy. Keywords Pamidronate Safety Renal function Osteogenesis imperfecta Efficacy