Identifying the concepts in outcome measures of clinical trials on osteogenesis imperfecta using the International Classification of Functioning, Disability and Health - version for children and youth
DOI:
https://doi.org/10.5935/0104-7795.20140027Keywords:
Osteogenesis Imperfecta, Outcome Assessment (Health Care), International Classification of Functioning, Disability and HealthAbstract
The biopsychosocial model from the International Classification of Functioning, Disability, and Health (ICF) has been used as a reference in clinical practice to identify and analyze the functioning components in outcome measures. Objective: The objectives of this study were to identify the concepts contained in outcome measures of clinical trials on Osteogenesis Imperfecta, to analyze how these concepts are linked with the ICF - Children and Youth version (ICF-CY) and describe what the functioning components are that are assessed in these studies. Method: Randomized controlled trials on children with diagnoses of Osteogenesis Imperfecta carried out between 2000 and 2013 were selected using MedLine and Cochrane. The outcome measures were extracted and the concepts contained in the outcome measures were linked to the ICF-CY. Results: Fourteen trials were included. The concepts of clinical and technical measures and of one health assessment instrument (Pediatric Evaluation of Disability Inventory - PEDI) were identified. The concepts of clinical and technical measures were linked to the ICF-CY Body Functions and Structures component. The PEDI concepts were linked to the Body Functions and especially to Activity and Participation. Conclusion: Using the linking of the concepts of outcome measures to the ICF-CY it was possible to verify that clinical trials on Osteogenesis Imperfecta assessed mainly the Body Functions and Body Structures component. Assessments of Activity and Participation and contextual factors are scarce on these studies. More research is necessary on the effects of interventions on these components.
Downloads
References
Engelbert RHH, Custers JWH, van der Net J, van der Graaf Y, Beemer FA, Helders PJM. Functional outcome in osteogenesis imperfecta: disability profiles using the PEDI. Pediatric Phys Ther. 1997;9(1):18-22. DOI: http://dx.doi.org/10.1097/00001577199700910-00004
CIF: Classificação Internacional de Funcionalidade, Incapacidade e Saúde. São Paulo: EDUSP; 2003.
Brockow T, Cieza A, Kuhlow H, Sigl T, Franke T, Harder M, et al. Identifying the concepts contained in outcome measures of clinical trials on musculoskeletal disorders and chronic widespread pain using the International Classification of Functioning, Disability and Health as a reference. J Rehabil Med. 2004;(44 Suppl):30-6.
Silva Drummond A, Ferreira Sampaio R, Cotta Mancini M, Noce Kirkwood R, Stamm TA. Linking the Disabilities of Arm, Shoulder, and Hand to the International Classification of Functioning, Disability, and Health. J Hand Ther. 2007;20(4):336-43. DOI: http://dx.doi.org/10.1197/j.jht.2007.07.008
CIF-CJ: Classificação Internacional de Funcionalidade, Incapacidade e Saúde: versão crianças e jovens. São Paulo: EDUSP; 2011.
Stucki G, Cieza A, Ewert T, Kostanjsek N, Chatterji S, Ustün TB. Application of the International Classification of Functioning, Disability and Health (ICF) in clinical practice. Disabil Rehabil. 2002;24(5):281-2. DOI: http://dx.doi.org/10.1080/09638280110105222
Cieza A, Brockow T, Ewert T, Amman E, Kollerits B, Chatterji S, et al. Linking health-status measurements to the international classification of functioning, disability and health. J Rehabil Med. 2002;34(5):205-10. DOI: http://dx.doi.org/10.1080/165019702760279189
Cieza A, Geyh S, Chatterji S, Kostanjsek N, Ustün B, Stucki G. ICF linking rules: an update based on lessons learned. J Rehabil Med. 2005;37(4):212-8. DOI: http://dx.doi.org/10.1080/16501970510040263
Van Brussel M1, Takken T, Uiterwaal CS, Pruijs HJ, Van der Net J, Helders PJ, et al. Physical training in children with osteogenesis imperfecta. J Pediatr. 2008;152(1):111-6. DOI: http://dx.doi.org/10.1016/j.jpeds.2007.06.029
Haley SM, Coster WJ, Ludlow LH, Haltiwanger JT, Andrellos PJ. Pediatric Evaluation Disability Inventory (PEDI) development, standardization and administrations manual. Boston: PEDI Research Group, New England Medical Center Hospitals; 1992.
Mancini MC. Inventário de avaliação pediátrica de incapacidade (PEDI): manual da versão brasileira adaptada. Belo Horizonte: UFMG; 2005.
Kok DH, Sakkers RJ, Janse AJ, Pruijs HE, Verbout AJ, Castelein RM, et al. Quality of life in children with osteogenesis imperfecta treated with oral bisphosphonates (Olpadronate): a 2-year randomized placebo-controlled trial. Eur J Pediatr. 2007;166(11):1155-61. DOI: http://dx.doi.org/10.1007/s00431-006-0399-2
Antoniazzi F, Monti E, Venturi G, Franceschi R, Doro F, Gatti D, et al. GH in combination with bisphosphonate treatment in osteogenesis imperfecta. Eur J Endocrinol. 2010;163(3):479-87. DOI: http://dx.doi.org/10.1530/EJE-10-0208
Dimeglio LA, Ford L, McClintock C, Peacock M. A comparison of oral and intravenous bisphosphonate therapy for children with osteogenesis imperfecta. J Pediatr Endocrinol Metab. 2005;18(1):43-53. DOI: http://dx.doi.org/10.1515/JPEM.2005.18.1.43
Rauch F, Munns CF, Land C, Cheung M, Glorieux FH. Risedronate in the treatment of mild pediatric osteogenesis imperfecta: a randomized placebocontrolled study. J Bone Miner Res. 2009;24(7):1282-9. DOI: http://dx.doi.org/10.1359/jbmr.090213
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. DOI: http://dx.doi.org/10.1016/S0140-6736(04)16101-1
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. J Clin Endocrinol Metab. 2011;96(2):355-64. DOI: http://dx.doi.org/10.1210/jc.2010-0636
Antoniazzi F, Zamboni G, Lauriola S, Donadi L, Adami S, Tatò L. Early bisphosphonate treatment in infants with severe osteogenesis imperfecta. J Pediatr. 2006;149(2):174-9. DOI: http://dx.doi.org/10.1016/j.jpeds.2006.03.013
Bishop N, Harrison R, Ahmed F, Shaw N, Eastell R, Campbell M, et al. A randomized, controlled dose-ranging study of risedronate in children with moderate and severe osteogenesis imperfecta. J Bone Miner Res. 2010;25(1):32-40. DOI: http://dx.doi.org/10.1359/jbmr.090712
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. J Clin Endocrinol Metab. 2005;90(7):4051-6. DOI: http://dx.doi.org/10.1210/jc.2004-2054
DiMeglio LA, Peacock M. Two-year clinical trial of oral alendronate versus intravenous pamidronate in children with osteogenesis imperfecta. J Bone Miner Res. 2006;21(1):132-40.
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. J Pediatr Endocrinol Metab. 2012;25(5-6):485-91. DOI: http://dx.doi.org/10.1515/jpem-2012-0016
Letocha AD, Cintas HL, Troendle JF, Reynolds JC, Cann CE, Chernoff EJ, et al. Controlled trial of pamidronate in children with types III and IV osteogenesis imperfecta confirms vertebral gains but not short-term functional improvement. J Bone Miner Res. 2005;20(6):977-86. DOI: http://dx.doi.org/10.1359/JBMR.050109
Gatti D, Antoniazzi F, Prizzi R, Braga V, Rossini M, Tatò L, et al. Intravenous neridronate in children with osteogenesis imperfecta: a randomized controlled study. J Bone Miner Res. 2005;20(5):758-63. DOI: http://dx.doi.org/10.1359/JBMR.041232
Moreira CLM, Lima MAF, Cardoso MHC, Gomes Junior SCS, Lopes PB, Llerena Junior JC. Determinantes da marcha independente na osteogênese imperfeita. Acta Ortop Bras. 2011;19(5):312-5. DOI: http://dx.doi.org/10.1590/S1413-78522011000500010
Vargus-Adams J. Understanding function and other outcomes in cerebral palsy. Phys Med Rehabil Clin N Am. 2009;20(3):567-75. DOI: http://dx.doi.org/10.1016/j.pmr.2009.04.002
Björck-Åkesson E, Wilder J, Granlund M, Pless M, Simeonsson R, Adolfsson M, et al. The International Classification of Functioning, Disability and Health and the version for children and youth as a tool in child habilitation/early childhood intervention--feasibility and usefulness as a common language and frame of reference for practice. Disabil Rehabil. 2010;32 Suppl 1:S125-38.
Palisano RJ. A collaborative model of service delivery for children with movement disorders: a framework for evidence-based decision making. Phys Ther. 2006;86(9):1295-305. DOI: http://dx.doi.org/10.2522/ptj.20050348
Downloads
Published
Issue
Section
License
Copyright (c) 2014 Acta Fisiátrica

This work is licensed under a Creative Commons Attribution-NonCommercial-ShareAlike 4.0 International License.