Controlled trial of pamidronate in children with types III and IV osteogenesis imperfecta confirms vertebral gains but not short-term functional improvement

Journal of Bone and Mineral Research

Volumn 20, Issue 6, 2005, Pages 977-986

  Letocha, Anne D ^b   Cintas, Holly L ^c   Troendle, James F ^b   Reynolds, James C ^c   Cann, Christopher E ^a   Chernoff, Edith J ^b   Hill, Suvimol C ^c   Gerber, Lynn H ^c   Marini, Joan C ^b  

^a Mindways Software Inc   (United States)

^b EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT   (United States)

^c NATIONAL INSTITUTES OF HEALTH   (United States)

Author keywords

Bisphosphonates; BMD; Osteogenesis imperfecta; Osteoporosis; Pamidronate

Indexed keywords

HUMAN GROWTH HORMONE; PAMIDRONIC ACID; RECOMBINANT GROWTH HORMONE;

ADOLESCENT; ARTICLE; BONE MASS; CHILD; CLINICAL ARTICLE; CLINICAL TRIAL; COMPUTER ASSISTED TOMOGRAPHY; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DUAL ENERGY X RAY ABSORPTIOMETRY; FEMALE; FRACTURE; HUMAN; MALE; MOTOR PERFORMANCE; MUSCLE STRENGTH; OSTEOGENESIS IMPERFECTA; PAIN; RANDOMIZED CONTROLLED TRIAL; SPINE RADIOGRAPHY; TREATMENT OUTCOME; VERTEBRA;

ADOLESCENT; ANTI-INFLAMMATORY AGENTS; BODY HEIGHT; BONE AND BONES; BONE DENSITY; BONE DEVELOPMENT; CHILD; CHILD, PRESCHOOL; DENSITOMETRY; DIPHOSPHONATES; FRACTURES, BONE; GROWTH HORMONE; HUMANS; OSTEOGENESIS IMPERFECTA; SPINE; TREATMENT OUTCOME;

EID: 19044396835     PISSN: 08840431     EISSN: None     Source Type: Journal    
DOI: 10.1359/JBMR.050109     Document Type: Article

References (44)

1. Byers PH, Cole WG 2002 Osteogenesis imperfecta. In: Royce PM, Steinmann B (eds.) Connective Tissue and Its Heritable Disorders, 2nd ed. Wiley-Liss, New York, NY, USA, pp. 385-430.
2. Marini JC, Chernoff EJ 2001 Osteogenesis imperfecta. In: Cassidy SB, Allanson JE (eds.) Management of Genetic Syndromes. Wiley-Liss, New York, NY, USA, pp. 281-300.
3. Prockop DJ, Kivirikko KI 1995 Collagens: Molecular biology, diseases, and potentials for therapy. Annu Rev Biochem 64:403-434.
4. Sillence DO, Senn A, Danks DM 1979 Genetic heterogeneity in Osteogenesis imperfecta. J Med Genet 16:101-116.
5. Glorieux FH, Rauch F, Plotkin H, Ward L, Travers R, Roughley P, Lalic L, Glorieux DF, Fassier F, Bishop NJ 2000 Type V osteogenesis imperfecta: A new form of brittle bone disease. J Bone Miner Res 15:1650-1658.
6. Glorieux FH, Ward LM, Rauch F, Lalic L, Roughley PJ, Travers R 2002 Osteogenesis imperfecta type VI: A form of brittle bone disease with a mineralization defect. J Bone Miner Res 17:30-38.
7. Ward LM, Rauch F, Travers R, Chabot G, Azouz EM, Lalic L, Roughley PJ, Glorieux FH 2002 Osteogenesis imperfecta type VII: An autosomal recessive form of bone disease. Bone 31:12-18.
8. Devogelaer J-P 2002 New uses of bisphosphonates: Osteogenesis imperfecta. Curr Opin Pharmacol 2:748-753.
9. Batch JA, Couper JJ, Rodda C, Cowell CT, Zacharin M 2003 Use of bisphosphonate therapy for osteoporosis in childhood and adolescence (position paper). J Paediatr Child Health 39:88-92.
10. Lindsay R 2002 Modeling the benefits of pamidronate in children with osteogenesis imperfecta. J Clin Invest 110:1239-1241.
11. Langman CB 2003 Editorial: Improvement of bone in patients with osteogenesis imperfecta treated with pamidronate - lessons from biochemistry. J Clin Endocrinol Metab 88:984-985.
12. Reid IR, Brown JP, Burckhardt P, Horowitz Z, Richardson P, Trechsel U, Widmer A, Devogelaer J-P, Kaufman J-M, Jaeger P, Body J-J, Meunier P-J 2002 Intravenous zoledronic acid in postmenopausal women with low bone mineral density. N Engl J Med 346:653-661.
13. Shaw NJ, Boivin CM, Crabtree NJ 2000 Intravenous pamidronate in juvenile osteoporosis. Arch Dis Child 83:143-145.
14. Orcel P, Beaudreuil J 2002 Bisphosphonates in bone diseases other than osteoporosis. Joint Bone Spine 69:19-27.
15. Reid IR 2003 Bisphosphonates: New indications and methods of administration. Curr Opin Rheumatol 15:458-463.
16. Zacharin M, O'Sullivan M 2000 Intravenous pamidronate treatment of polyostotic fibrous dysplasia associated with the McCune Albright syndrome. J Pediatr 137:403-409.
17. Bembi B, Parma A, Bottega M, Ceschel S, Zanatta M, Martini C, Ciana G 1997 Intravenous pamidronate treatment in osteogenesis imperfecta. J Pediatr 131:622-625.
18. Roldan EJ, Pasqualini T, Plantalech L 1999 Bisphosphonates in children with osteogenesis imperfecta may improve bone mineralization but not bone strength. Report of two patients. J Pediatr Endocrinol Metab 12:555-559.
19. Aström E, Soderhall S 1998 Beneficial effect of bisphosphonate during five years of treatment of severe osteogenesis imperfecta. Acta Paediatr 87:64-68.
20. Glorieux FH, Bishop NJ, Plotkin H, Chabot G, Lanoue G, Travers R 1998 Cyclic administration of pamidronate in children with severe osteogenesis imperfecta. N Engl J Med 339:947-952.
21. Gonzalez E, Pavia C, Ros J, Villaronga M, Valls C, Escola J 2001 Efficacy of low dose schedule pamidronate infusion in children with osteogenesis imperfecta. J Pediatr Endocrinol Metab 14:529-533.
22. Lee Y-S, Low S-L, Lim L-A, Loke K-Y 2001 Cyclic pamidronate infusion improves bone mineralisation and reduces fracture incidence in osteogenesis imperfecta. Eur J Pediatr 160:641-644.
23. Aström E, Soderhall S 2002 Beneficial effect of long term intravenous bisphosphonate treatment of osteogenesis imperfecta. Arch Dis Child 86:356-364.
24. Banerjee I, Shortland GJ, Evans WD, Gregory JW 2002 Osteogenesis imperfecta and intravenous pamidronate. Arch Dis Child 87:562-563.
25. Zacharin M, Bateman JF 2002 Pamidronate treatment of osteogenesis imperfecta-lack of correlation between clinical severity, age at onset of treatment, predicted collagen mutation and treatment response. J Pediatr Endocrinol Metab 15:163-174.
26. Falk MJ, Heeger S, Lynch KA, DeCaro KR, Bohach D, Gibson KS, Warman ML 2003 Intravenous bisphosphonate therapy in children with osteogenesis imperfecta. Pediatrics 111:573-578.
27. Arikoski P, Silverwood B, Tillmann V, Bishop NJ 2004 Intravenous pamidronate treatment in children with moderate to severe osteogenesis imperfecta: Assessment of indices of dual-energy X-ray absorptiometry and bone metabolic markers during the first year of therapy. Bone 34:539-546.
28. Plotkin H, Rauch F, Bishop NJ, Monpetit K, Ruck-Gibis J, Travers R, Glorieux FH 2000 Pamidronate treatment of severe osteogenesis imperfecta in children under 3 years of age. J Clin Endocrinol Metab 85:1846-1850.
29. Rauch F, Travers R, Plotkin H, Glorieux FH 2002 The effects of intravenous pamidronate on the bone tissue of children and adolescents with osteogenesis imperfecta. J Clin Invest 110:1293-1299.
30. Sakkers R, Kok D, Engelbert R, van Dongen A, Jansen M, Pruijs H, Verbeut A, Schweitzer D, Uiterwaal C 2004 Skeletal effects and functional outcome with olpadronate in children with osteogenesis imperfecta: A 2-year randomised placebo-controlled study. Lancet 363:1427-1431.
31. Cole DE, Carpenter TO, Gundberg CM 1985 Serum osteocalcin concentrations in children with metabolic bone disease. J Pediatr 106:770-776.
32. Cavanaugh DJ, Cann CE 1988 Brisk walking does not stop bone loss in postmenopausal women. Bone 9:201-204.
33. Gilsanz V, Gibbens DT, Roe TF, Carlson M, Senac MO, Boechat MI, Huang HK, Schulz EE, Libanati CR, Cann CE 1988 Vertebral bone density in children: Effect of puberty. Radiology 166:847-850.
34. Cintas HL, Siegel KL, Furst GP, Gerber LH 2003 Brief assessment of motor function: Reliability and concurrent validity of the gross motor scale. Am J Phys Med Rehabil 82:33-41.
35. Kendall FP, McCreary EK, Provance PG 1993 Muscles: Testing and Function, 4th ed. Lippincott, Williams & Wilkins, Baltimore, MD, USA.
36. Giraud F, Meunier PJ 2002 Effect of cyclical intravenous pamidronate therapy in children with osteogenesis imperfecta. Open-label study in seven patients. Joint Bone Spine 69:486-490.
37. Marini JC 2003 Do bisphosphonates make children's bones better or brittle? N Engl J Med
38. Marini JC, Kosloff KM, Uveges TE, Ty JM, Gronowicz G, Ledgard F, Goldstein SA 2003 Alendronate treatment of Brtl mouse model for osteogenesis imperfecta increases bone strength by increasing bone volume but fails to improve femoral brittleness or mineralization. J Bone Miner Res 18:S2;S287.
39. Zeitlin L, Rauch F, Plotkin H, Glorieux FH 2003 Height and weight development during four years of therapy with cyclical intravenous pamidronate in children and adolescents with osteogenesis imperfecta types I, III, and IV. Pediatrics 111:1030-1036.
40. Montpetit K, Plotkin H, Rauch F, Bilodeau N, Cloutier S, Rabzel M, Glorieux FH 2003 Rapid increase in grip force after start of pamidronate therapy in children and adolescents with severe osteogenesis imperfecta. Pediatrics 111:e601-e603.
41. Whyte MP, Wenkert D, Clements KL, McAlister WH, Mumm S 2003 Bisphosphonate-induced osteopetrosis. N Engl J Med 349:457-463.
42. Munns CF, Rauch F, Zeitlin L, Fassier F, Glorieux FH 2004 Delayed osteotomy but not fracture healing in pediatric osteogenesis imperfecta patients receiving pamidronate. J Bone Miner Res 19:1779-1786.
43. Rauch F, Travers R, Munns CF, Glorieux FH 2004 Sclerotic metaphyseal lines in a child treated with pamidronate: Histomorphometric analysis. J Bone Miner Res 19:1191-1193.
44. Uveges TE, Kozloff KM, Ty JM, Gronowicz G, Ledgard F, Goldstein SA, Marini JC 2004 Alendronate Treatment of the Brtl Mouse Model for Osteogenesis Imperfecta Improves Bone Geometry and Loading Before Fracture but Decreases Bone Material Quality and Alters Osteoblast Morphology. European Human Genetics Conference, Munich, Germany, June 2004.