Intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta started under three years of age

M. B. Alcausin, J. Briody, V. Pacey, J. Ault, M. McQuade, C. Bridge, R. H H Engelbert, D. O. Sillence, C. F. Munns

Research output: Contribution to journalArticleResearchpeer-review

Abstract

Objective: Evaluate clinical outcome of early cyclic intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta (OI), commenced before three years of age. Methods: A retrospective review of 17 patients with moderate-to-severe OI. Development, anthropometry, fracture history, bone mineral density (BMD) and biochemistry were collected at baseline, 12 and 24 months. Results: Four had OI type I, eleven had type III, one OI-FKBP10 type and one OI type V. Mean age at start of pamidronate was 14 ± 11 months. Pamidronate ranged from 6 to 12 mg/kg/year. No adverse reaction apart from fever and vomiting was noted. Long bone fracture decreased from a mean of 10.4/year to 1.2/year after 12 months and 1.4/year after 24 months (p = 0.02). Lumbar spine age- and height-matched BMD Z-scores increased (p < 0.005). Sixteen with vertebral compression fractures at baseline all showed improved vertebral shape (p < 0.001). Concavity index, likewise, improved (p < 0.005). Motor milestones compared to historical data show earlier attainment in rolling over, crawling, pulling to stand and walking independently but not sitting. Conclusion: Cyclic intravenous pamidronate, started under 3 years of age in children with moderate-to-severe OI, was well tolerated and associated with an increase in lumbar spine BMD, reduced fracture frequency, vertebral remodelling and attainment of motor milestones at an earlier age.

LanguageEnglish
Pages333-340
Number of pages8
JournalHormone Research in Paediatrics
Volume79
Issue number6
DOIs
Publication statusPublished - Jul 2013
Externally publishedYes

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pamidronate
Osteogenesis Imperfecta
Bone Density
Spine
Compression Fractures
Anthropometry
Bone Fractures
Therapeutics
Biochemistry
Walking
Vomiting
Fever

Cite this

Alcausin, M. B. ; Briody, J. ; Pacey, V. ; Ault, J. ; McQuade, M. ; Bridge, C. ; Engelbert, R. H H ; Sillence, D. O. ; Munns, C. F. / Intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta started under three years of age. In: Hormone Research in Paediatrics. 2013 ; Vol. 79, No. 6. pp. 333-340.
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title = "Intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta started under three years of age",
abstract = "Objective: Evaluate clinical outcome of early cyclic intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta (OI), commenced before three years of age. Methods: A retrospective review of 17 patients with moderate-to-severe OI. Development, anthropometry, fracture history, bone mineral density (BMD) and biochemistry were collected at baseline, 12 and 24 months. Results: Four had OI type I, eleven had type III, one OI-FKBP10 type and one OI type V. Mean age at start of pamidronate was 14 ± 11 months. Pamidronate ranged from 6 to 12 mg/kg/year. No adverse reaction apart from fever and vomiting was noted. Long bone fracture decreased from a mean of 10.4/year to 1.2/year after 12 months and 1.4/year after 24 months (p = 0.02). Lumbar spine age- and height-matched BMD Z-scores increased (p < 0.005). Sixteen with vertebral compression fractures at baseline all showed improved vertebral shape (p < 0.001). Concavity index, likewise, improved (p < 0.005). Motor milestones compared to historical data show earlier attainment in rolling over, crawling, pulling to stand and walking independently but not sitting. Conclusion: Cyclic intravenous pamidronate, started under 3 years of age in children with moderate-to-severe OI, was well tolerated and associated with an increase in lumbar spine BMD, reduced fracture frequency, vertebral remodelling and attainment of motor milestones at an earlier age.",
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Alcausin, MB, Briody, J, Pacey, V, Ault, J, McQuade, M, Bridge, C, Engelbert, RHH, Sillence, DO & Munns, CF 2013, 'Intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta started under three years of age', Hormone Research in Paediatrics, vol. 79, no. 6, pp. 333-340. https://doi.org/10.1159/000351374

Intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta started under three years of age. / Alcausin, M. B.; Briody, J.; Pacey, V.; Ault, J.; McQuade, M.; Bridge, C.; Engelbert, R. H H; Sillence, D. O.; Munns, C. F.

In: Hormone Research in Paediatrics, Vol. 79, No. 6, 07.2013, p. 333-340.

Research output: Contribution to journalArticleResearchpeer-review

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T1 - Intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta started under three years of age

AU - Alcausin, M. B.

AU - Briody, J.

AU - Pacey, V.

AU - Ault, J.

AU - McQuade, M.

AU - Bridge, C.

AU - Engelbert, R. H H

AU - Sillence, D. O.

AU - Munns, C. F.

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N2 - Objective: Evaluate clinical outcome of early cyclic intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta (OI), commenced before three years of age. Methods: A retrospective review of 17 patients with moderate-to-severe OI. Development, anthropometry, fracture history, bone mineral density (BMD) and biochemistry were collected at baseline, 12 and 24 months. Results: Four had OI type I, eleven had type III, one OI-FKBP10 type and one OI type V. Mean age at start of pamidronate was 14 ± 11 months. Pamidronate ranged from 6 to 12 mg/kg/year. No adverse reaction apart from fever and vomiting was noted. Long bone fracture decreased from a mean of 10.4/year to 1.2/year after 12 months and 1.4/year after 24 months (p = 0.02). Lumbar spine age- and height-matched BMD Z-scores increased (p < 0.005). Sixteen with vertebral compression fractures at baseline all showed improved vertebral shape (p < 0.001). Concavity index, likewise, improved (p < 0.005). Motor milestones compared to historical data show earlier attainment in rolling over, crawling, pulling to stand and walking independently but not sitting. Conclusion: Cyclic intravenous pamidronate, started under 3 years of age in children with moderate-to-severe OI, was well tolerated and associated with an increase in lumbar spine BMD, reduced fracture frequency, vertebral remodelling and attainment of motor milestones at an earlier age.

AB - Objective: Evaluate clinical outcome of early cyclic intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta (OI), commenced before three years of age. Methods: A retrospective review of 17 patients with moderate-to-severe OI. Development, anthropometry, fracture history, bone mineral density (BMD) and biochemistry were collected at baseline, 12 and 24 months. Results: Four had OI type I, eleven had type III, one OI-FKBP10 type and one OI type V. Mean age at start of pamidronate was 14 ± 11 months. Pamidronate ranged from 6 to 12 mg/kg/year. No adverse reaction apart from fever and vomiting was noted. Long bone fracture decreased from a mean of 10.4/year to 1.2/year after 12 months and 1.4/year after 24 months (p = 0.02). Lumbar spine age- and height-matched BMD Z-scores increased (p < 0.005). Sixteen with vertebral compression fractures at baseline all showed improved vertebral shape (p < 0.001). Concavity index, likewise, improved (p < 0.005). Motor milestones compared to historical data show earlier attainment in rolling over, crawling, pulling to stand and walking independently but not sitting. Conclusion: Cyclic intravenous pamidronate, started under 3 years of age in children with moderate-to-severe OI, was well tolerated and associated with an increase in lumbar spine BMD, reduced fracture frequency, vertebral remodelling and attainment of motor milestones at an earlier age.

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