Bisphosphonate therapy for osteogenesis imperfecta

Cochrane Database of Systematic Reviews

Volumn 2014, Issue 7, 2014, Pages

  Dwan, Kerry ^a   Phillipi, Carrie A ^b   Steiner, Robert D ^c,d   Basel, Donald ^e  

^a UNIVERSITY OF LIVERPOOL   (United Kingdom)

^b OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^c MARSHFIELD CLINIC   (United States)

^d OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^e Division of Genetics MC 716   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALENDRONIC ACID; ALKALINE PHOSPHATASE; AMINO TERMINAL TELOPEPTIDE; ANALGESIC AGENT; BIOLOGICAL MARKER; BISPHOSPHONIC ACID DERIVATIVE; CREATININE; OLPADRONIC ACID; PAMIDRONIC ACID; PHOSPHONIC ACID DERIVATIVE; PLACEBO; RISEDRONIC ACID; ZOLEDRONIC ACID; BONE DENSITY CONSERVATION AGENT;

ARM FRACTURE; BODY HEIGHT; BONE DENSITY; BONE GROWTH; BONE LENGTH; BONE LEVEL; BONE METABOLISM; BONE PAIN; CRUSH TRAUMA; DRUG EFFICACY; DRUG SAFETY; DRUG USE; DUAL ENERGY X RAY ABSORPTIOMETRY; FEMUR; FRACTURE; HEARING; HIP; HISTOLOGY; HUMAN; INCIDENCE; LEG FRACTURE; LUMBAR SPINE; LUNG FUNCTION; MINERAL METABOLISM; MOTOR FUNCTION TEST; MUSCLE STRENGTH; OSTEOGENESIS IMPERFECTA; PAIN ASSESSMENT; PHYSICAL MOBILITY; PRIORITY JOURNAL; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; RISK REDUCTION; SELF CARE; SPINE INJURY; SUPINE POSITION; SYSTEMATIC REVIEW; TREATMENT DURATION; TREATMENT OUTCOME; WELLBEING; DRUG EFFECTS; FRACTURES, BONE; INTRAVENOUS DRUG ADMINISTRATION; META ANALYSIS; ORAL DRUG ADMINISTRATION;

ADMINISTRATION, ORAL; BONE DENSITY; BONE DENSITY CONSERVATION AGENTS; DIPHOSPHONATES; FRACTURES, BONE; HUMANS; INJECTIONS, INTRAVENOUS; OSTEOGENESIS IMPERFECTA; RANDOMIZED CONTROLLED TRIALS AS TOPIC;

EID: 84908435160     PISSN: None     EISSN: 1469493X     Source Type: Journal    
DOI: 10.1002/14651858.CD005088.pub3     Document Type: Review

References (70)

1. Adami S, Gatti D, Colapietro F, Fracassi E, Braga V, Rossini M, et al. Intravenous neridronate in adults with osteogenesis imperfecta. Journal of Bone and Mineral Research 2003;18(1):126-30.
2. Gatti D, Viapiana O, Lippolis I, Braga V, Prizzi R, Rossini M, et al. Intravenous bisphosphonate therapy increases radial width in adults with osteogenesis imperfecta. Journal of Bone and Mineral Research 2005;20(8):1323-6.
3. Barros ER, Saraiva GL, de Oliveira TP, Lazaretti-Castro M. Safety and efficacy of a 1-year treatment with zoledronic acid compared with pamidronate in children with osteogenesis imperfecta. Journal of Pediatric Endocrinology and Metabolism 2012;25(5-6):485-91.
4. Bishop N, Harrison R, Ahmed F, Shaw N, Eastell R, Campbell M, et al. A randomised controlled dose-ranging study of risedronate in children with moderate and severe osteogenesis imperfecta. Journal of Bone and Mineral Research 2010;25(1):32-40.
5. Bishop N, Adami S, Ahmed SF, Antón J, Arundel P, Burren CP, et al. Risedronate in children with osteogenesis imperfecta:a randomised, double-blind, placebo-controlled trial. Lancet 2013;382(9902):1424-32.
6. Chevrel G, Schott AM, Fontanges E, Charrin JE, Lina-Granade G, Duboeuf F, et al. Effects of oral alendronate on BMD in adult patients with osteogenesis imperfecta: a 3-year randomized placebo-controlled trial. Journal of Bone and Mineral Research 2006;21(2):300-6.
7. DiMeglio LA, Peacock M. Two-year clinical trial of oral alendronate versus intravenous pamidronate in children with osteogenesis imperfecta. Journal of Bone and Mineral Research 2006;21(1):132-40.
8. Dimeglio LA, Ford L, McClintock C, Peacock M. A comparison of oral and intravenous bisphosphonate therapy for children with osteogenesis imperfecta. Journal of Pediatric Endocrinology and Metabolism 2005;18(1):43-53.
9. Gatti D, Antoniazzi F, Prizzi R, Braga V, Rossini M, Tato L, et al. Intravenous neridronate in children with osteogenesis imperfecta: A randomized controlled study. Journal of Bone and Mineral Research 2005;20(5):758-63.
10. Letocha AD, Cintas HL, Troendle JF, Reynolds JC, Cann CE, ChernoffEJ, et al. 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 2005;20(6):977-86.
11. Rauch F, Munns CF, Land C, Cheung M, Glorieux FH. Risedronate in the treatment of mild pediatric osteogenesis imperfecta: a randomized placebo-controlled study. Journal of Bone and Mineral Research 2009;24(7):1282-9.
12. Sakkers R, Kok D, Engelbert R, van Dongen A, Jansen M, Pruijs H, et al. Skeletal effects and functional outcome with olpadronate in children with osteogenesis imperfecta: a 2-year randomised placebo-controlled study. Lancet 2004;363(9419):1427-31. [CRS-ID: 5500100000002556]
13. Seikaly MG, Kopanati S, Salhab N, Waber P, Patterson D, Browne R, et al. Impact of alendronate on quality of life in children with osteogenesis imperfecta. Journal of Pediatric Orthopaedics 2005;25(6):786-91.
14. Senthilnathan S, Walker E, Bishop NJ. Two doses of pamidronate in infants with osteogenesis imperfecta. Archives of Disease in Childhood 2008;93(5):398-400.
15. Glorieux FH, Rauch F, Ward LM, Smith P, Verbruggen N, Heydon N, et al. Alendronate in the treatment of pediatric osteogenesis imperfecta [abstract]. Journal of Bone and Mineral Research 2004;20 Suppl:S12. [CRS-ID: 5500100000003043]
16. Ward LM, Rauch F, Whyte MP, D'Astous J, Gates PE, Grogan D, et al. Alendronate for the treatment of pediatric osteogenesis imperfecta: a randomized placebo-controlled study. Journal of Clinical Endocrinology and Metabolism 2011;96(2):355-64.
17. Food, Drug Administration (FDA). Center for Drug Evaluation and Research. Statistical review and evaluation - clinical studies: Zometa (zoledronic acid) intravenous injection (i.v.) 4 μg lyophilized powder. Treatment of children with osteogenesis imperfecta (OI). http://www.fda.gov/downloads/drugs/developmentapprovalprocess/developmentresources/ucm072901.pdf (accessed 07 April 2014).
18. NCT00063479. Bisphosphonate treatment of osteogenesis imperfecta. http://clinicaltrials.gov/show/NCT00063479 (accessed 08 March 2013).
19. Antoniazzi F, Bertoldo F, Mottes M, Valli M, Sirpresi S, Zamboni G, et al. Growth hormone treatment in osteogenesis imperfecta with quantitative defect of type I collagen synthesis. Journal of Pediatrics 1996;129(3):432-9.
20. Antoniazzi F, Zamboni G, Lauriola S, Donadi L, Adami S, Tato L. Early bisphosphonate treatment in infants with severe osteogenesis imperfecta. Journal of Pediatrics 2006;149(2):174-9.
21. Antoniazzi F, Monti E, Venturi G, Franceschi R, Doro F, Gatti D, et al. GH in combination with bisphosphonate treatment in osteogenesis imperfecta. European Journal of Endocrinology 2010;163(3):479-87.
22. DiMeglio LA, Ford L, McClintock C, Peacock M. Intravenous pamidronate treatment of children under 36 months of age with osteogenesis imperfecta. Bone 2004;35(5):1038-45.
23. Gerber LH, Binder H, Berry R, Siegel KL, Kim H, Weintrob J, et al. Effect of withdrawal of bracing in matched pairs of children with osteogenesis imperfecta. Archives of Physical Medicine and Rehabilitation 1998;79(1):46-51.
24. Granda JL, Falva KA, Bullough PG. Phyrophosphate levels and magnesium oxide therapy in osteogenesis imperfecta. Clinical Orthopaedics and Related Research 1977;Jul-Aug(216):228-31.
25. NCT00131469. Study of Teriparatide (FORTEO) to Treat Adults With Osteogenesis Imperfecta (OI). http://clinicaltrials.gov/show/NCT00131469 (accessed 08 March 2013). [CRS-ID: 5500100000003940]
26. Nagamani S, Shapiro J, Veith S, Wang Y, Lapidus J, Reeder JL, et al. Teriparatide, the first anabolic agent for treatment of osteogenesis imperfecta improves bone mineral density at the hip and spine: a randomized, blinded, placebo-controlled trial [abstract]. Proceedings of the 63rd Annual Meeting of the American Society of Human Genetics; 2013 Oct 22-26; Boston. Boston: American Society of Human Genetics, 2013:27.
27. Ward LM, Denker AE, Porras A, Shugarts S, Kline W, Travers R, et al. Single-dose pharmacokinetics and tolerability of alendronate 35-and 70-milligram tablets in children and adolescents with osteogenesis imperfecta type I. Journal of Clinical Endocrinology and Metabolism 2005;90(7):4051-6.
28. Alanay Y, Avaygan H, Camacho N, Utine GE, Boduroglu K, Aktas D, et al. Mutations in the gene encoding the RER protein FKBP65 cause autosomal-recessive osteogenesis imperfecta. American Journal of Human Genetics 2010;86(4):551-9.
29. Barnes AM, Chang W, Morello R, Cabral WA, Weis MA, Eyre DR, et al. Deficiency of cartilage-associated protein in recessive lethal osteogenesis imperfecta. New England Journal of Medicine 2006;355(26):2757-64.
30. Becker J, Semler O, Gilissen C, Li Y, Bolz HJ, Giunta C, et al. Exome sequencing identifies truncating mutations in human SERPINF1 in autosomal-recessive osteogenesis imperfecta. American Journal of Human Genetics 2011;88(3):362-71.
31. Black DM, Cummings SR, Karpf DB, Cauley JA, Thompson DE, Nevitt MC, et al. Randomized trial of effect of alendronate on risk of fracture in women with existing vertebral fractures. Lancet 1996;348(9041):1535-41.
32. Byers PH, Wallis GA, Willing MC. Osteogenesis imperfecta: translation of mutation to phenotype. Journal of Medical Genetics 1991;28(7):433-42.
33. Byers PH, Steiner RD. Osteogenesis imperfecta. Annual Review of Medicine 1992;43:269-82.
34. Byers PH. Osteogenesis imperfecta: perspectives and opportunities. Current Opinion in Pediatrics 2000;12(6):603-9.
35. Byers 2013 [pers comm]. OI [personal communication]. Email to: D Basel 2013.
36. Cabral WA, Chang W, Barnes AM, Weis M, Scott MA, Leikin S, et al. Prolyl 3-hydroxylase 1 deficiency causes a recessive metabolic bone disorder resembling lethal/severe osteogenesis imperfecta. Nature Genetics 2007;39(3):359-65.
37. Camacho NP, Raggio CL, Doty SB, Root L, Zraick V, Ilg WA, et al. A controlled study of the effects of alendronate in a growing mouse model of osteogenesis imperfecta. Calcified Tissue International 2001;69(2):94-101.
38. Castillo H, Samson-Fang L, American Academy for Cerebral Palsy and Developmental Medicine Treatment Outcomes Committee Review Panel. Effects of bisphosphonates in children with osteogenesis imperfecta: an AACPDM systematic review. Developmental Medicine and Child Neurology 2009;51(1):17-29.
39. Cho TJ, Lee KE, Lee SK, Song SJ, Kim KJ, Jeon D, et al. A Single Recurrent Mutation in the 5'-UTR of IFITM5 Causes Osteogenesis Imperfecta Type V. American Journal of Human Genetics 2012;91(2):343-8.
40. Christiansen HE, Schwarze U, Pyott SM, AlSwaid A, Al Balwi M, Alrasheed S, et al. Homozygosity for a missense mutation in SERPINH1, which encodes the collagen chaperone protein HSP47, results in severe recessive osteogenesis imperfecta. American Journal of Human Genetics 2010;86(3):389-98.
41. Cole WG. Advances in Osteogenesis Imperfecta. Clinical Orthopaedics and Related Research 2002;401:6-16.
42. Connor JM, Connor RA, Sweet EM, Gibson AA, Patrick WJ, McNay MB, et al. Lethal neonatal chondrodysplasias in the West of Scotland 1970-1983 with a description of a thanatophoric, dysplasialike, autosomal recessive disorder, Glasgow variant. American Journal of Medical Genetics 1985;22(2):243-53.
43. Elbourne DR, Altman DG, Higgins JP, Curtin F, Worthington HV, Vail A. Meta-analyses involving cross-over trials: methodological issues. International Journal of Epidemiology 2002;31(1):140-9.
44. Erviti J, Alonso A, Oliva B, Gorricho J, López A, Timoner J, et al. Oral bisphosphonates are associated with increased risk of subtrochanteric and diaphyseal fractures in elderly women: a nested case-control study. http://bmjopen.bmj.com/content/3/1/e002091 (accessed 06 February 2013).
45. Food, Drug Administration (FDA). Background document for meeting of Advisory Committee for Reproductive Health Drugs and Drug Safety and Risk Management Advisory Committee. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/DrugSafetyandRiskManagementAdvisoryCommittee/UCM270958.pdf (accessed 07 March 2013).
46. Fisher JE, Rogers MJ, Halasy JM, Luckman SP, Hughes DE, Masarachia PJ, et al. Alendronate mechanism of action: geranylgeraniol, an intermediate in the mevalonate pathway, prevents inhibition of osteoclast formation, bone resorption, and kinase activation in vitro [abstract]. Proceedings of the National Academy of Sciences of the United States of America 1999;96:133-8.
47. Gatti D, Rossini M, Viapiana O, Povino MR, Liuzza S, Fracassi E, et al. Teriparatide treatment in adult patients with osteogenesis imperfecta type I. Calcified Tissue International 2013;93(5):448-52.
48. Glorieux FH, Rauch F, Plotkin H, Ward L, Travers R, Roughley P, et al. Type V osteogenesis imperfecta: a new form of brittle bone disease. Journal of Bone and Mineral Research 2000;15(9):1650-8.
49. Glorieux FH, Rauch F, Ward LM, Smith P, Verbruggen N, Heyden N, et al. Alendronate in the treatment of pediatric osteogenesis imperfecta [abstract]. Journal of Bone and Mineral Research 2004;20(Suppl):1043.
50. Higgins JPT, Thompson SG, Deeks JJ, Altman DG. Measuring inconsistency in meta-analyses. BMJ 2003;327(7414):557-60.
51. Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 [updated March 2011]. The Cochrane Collaboration 2011. Available from www.cochrane-handbook.org.
52. Higgins JPT, Altman DG (editors). Chapter 8: Assessing risk of bias in included studies. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
53. Jüni T, Altman, DG, Egger M. Systematic reviews in health care: Assessing the quality of controlled clinical trials. BMJ 2001;323(7303):42-6.
54. Kuurila K, Kaitila I, Johansson R, Grenman R. Hearing loss in Finnish adults with osteogenesis imperfecta: a nationwide survey. The Annals of Otology, Rhinology, and Laryngology 2002;111(10):939-46.
55. Lapunzina P, Aglan M, Temtamy S, Caparros-Martin JA, Valencia M, Leton R, et al. Identification of a frameshift mutation in Osterix in a patient with recessive osteogenesis imperfecta. American Journal of Human Genetics 2010;87(1):110-4.
56. Marini JC. Do bisphosphonates make children's bones better or brittle?. New England Journal of Medicine 2003;349(5):423-6.
57. Martínez-Glez V, Valencia M, Caparrós-Martín JA, Aglan M, Temtamy S, Tenorio J, et al. Identification of a mutation causing deficient BMP1/mTLD proteolytic activity in autosomal recessive osteogenesis imperfecta. Human Mutation 2012;33(2):343-50.
58. Osteogenesis Imperfecta Foundation. Facts about Osteogenesis Imperfecta. www.oif.org (accessed August 2008).
59. Orioli IM, Castilla EE, Scarano G, Mastroiacovo P. Effect of paternal age in achondroplasia, thanatophoric dysplasia, and osteogenesis imperfecta. American Journal of Medical Genetics 1995;59(2):209-17.
60. Pyott SM, Tran TT, Leistritz DF, Pepin MG, Mendelsohn NJ, Temme RT, et al. WNT1 mutations in families affected by moderately severe and progressive recessive osteogenesis imperfecta. American Journal of Human Genetics 2013;92(4):590-7.
61. Reid IR, Brown JP, Burckhardt P, Horowitz Z, Richardson P, Trechsel U, et al. Intravenous zoledronic acid in postmenopausal women with low bone mineral density. New England Journal of Medicine 2002;346(9):653-61.
62. Semler O, Garbes L, Keupp K, Swan D, Zimmermann K, Becker J, et al. A Mutation in the 5'-UTR of IFITM5 Creates an In-Frame Start Codon and Causes Autosomal-Dominant Osteogenesis Imperfecta Type V with Hyperplastic Callus. American Journal of Human Genetics 2012;91(2):349-57.
63. Sillence DO, Senn A, Danks DM. Genetic heterogeneity in osteogenesis imperfecta. Journal of Medical Genetics 1979;16(2):101-16.
64. Sillence D. Osteogenesis Imperfecta. International committee for constitutional disorders of the skeleton. Sydney, 2012.
65. Steiner RD, Adsit J, Basel D. Gene Reviews; COL1A1/2-Related Osteogenesis Imperfecta. http://www.ncbi.nlm.nih.gov/books/NBK1295/(Initial Posting: January 28, 2005; Last Update: February 14, 2013).
66. Thornton J, Ashcroft DM, Mughal MZ, Elliott RA, O'Neill TW, Symmons D. Systematic review of effectiveness of bisphosphonates in treatment of low bone mineral density and fragility fractures in juvenile idiopathic arthritis. Archives of Disease in Childhood 2006;91(9):753-61.
67. van Dijk FS, Nesbitt IM, Zwikstra EH, Nikkels PGJ, Piersma SR, Fratantoni SA, et al. PPIB mutations cause severe osteogenesis imperfecta. American Journal of Human Genetics 2009;85(4):521-7.
68. Whyte MP, Wenkert D, Clements KL, McAlister WH, Mumm S. Bisphosphonate-induced osteopetrosis. New England Journal of Medicine 2003;349(5):457-63.
69. Woo SB, Hellstein JW, Kalmar JR. Narrative [corrected] review: bisphosphonates and osteonecrosis of the jaws. Annals of Internal Medicine 2006;144(10):753-61.
70. Phillipi CA, Remmington T, Steiner RD. Bisphosphonate therapy for osteogenesis imperfecta. Cochrane Database of Systematic Reviews 2008, Issue 4. [DOI: 10.1002/14651858.CD005088.pub2]