Quality of life in children and adolescents with Osteogenesis Imperfecta: a qualitative interview based study

Claire L Hill, Wendy O Baird, Stephen J Walters, Claire L Hill, Wendy O Baird, Stephen J Walters

Abstract

Background: Osteogenesis Imperfecta (OI) is a disease with varying severity affecting physical, social and emotional well-being of the child and their family. There is no existing evidence on how the OI population regard their quality of life (QoL). The main aim of this study was to determine how OI impacts on the quality of life and well-being of children and their family. It is the first stage of a larger project to develop a disease specific quality of life measure for children with OI.

Methods: Purposive sampling was used to cover the diversity of the OI population. Twenty-five qualitative interviews were undertaken with children (n = 10), parents (n = 10) and health professionals (n = 5). Interviews were digitally recorded and transcribed verbatim. Significant themes were identified, extracted and organised, undergoing framework analysis.

Results: Six main themes were identified; being safe and careful, reduced function, pain, fear, isolation, independence. There was a large amount of agreement between the three groups of interviewees, although discrepancies did occur between parents and children, with regard to the themes independence and fear.

Conclusions: This data presents the first step in developing items for a disease specific QoL measure for children with OI. Several of the themes uncovered showed similarity to other QoL measures, but the addition of being safe and careful, particularly in relation to fractures, demonstrated the need for a disease specific measure for children with OI.

Figures

Figure 1
Figure 1
Hypothesized working conceptual framework, based on clinical expert opinion, for the health rated quality of life outcomes for a new patient reported outcome for children with OI.

References

    1. Glorieux F. Osteogenesis Imperfecta. Best Practice and Research Clinical Rheumatology. 2008. doi:10.1016/j.berh.2007.12.012.
    1. Forlino A, Cabral WA, Barnes AM, Marini JC. New perspectives on oetsogenesis imperfecta. Nat Rev Endocrinol. 2011. doi:10.1038/nrendo.2011.81.
    1. DiMeglio LA, Ford L, McClintock C, Peacock M. Intravenous pamidronate treatment of children under 36 months of age with osteogenesis imperfecta. Bone. 2004. doi:10.1016/j.bone.2004.07.003.
    1. Plotkin H, Rauch F, Bishop NJ, Montpetit K, Ruck-Gibis J, Travers R, Glorieux FH. Pamidronate treatment of severe Osteogenesis Imperfecta in children under 3 years of age. J Clin Endocrinol Metab. 2000. doi:10.1210/jc.85.5.1846.
    1. Rauch F, Plotkin H, Travers R, Zeitlin L, Glorieux FH. Osteogenesis Imperfecta Types I, III, and IV: effect of pamidronate therapy on bone and mineral metabolism. J Clin Endocrinol Metab. 2003. doi:10.1210/jc.2002-021371.
    1. Sakkers R, Kok D, Engelbert R, van Dongen A, Jansen M, Pruijs H, Verbout AB, Schweitzer D, Uiterwaal C. Skeletal effects and functional outcome with olpadronate in children with osteogenesis imperfecta: a 2-year randomised placebo-controlled study. Lancet. 2004. doi:10.1016/S0140-6736(04)16101-1.
    1. Letocha AD, Cintas HL, Troendle JF, Reynolds JC, Cann CE, Chernoff EJ, Hill SC, Gerber LH, Marini JC. 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. doi:10.1359/JBMR.050109.
    1. Fisher JE, Rogers MJ, Halasy JM, Luckman SP, Hughes DE, Masarachia PJ. Alendronate mechanism of action: geranylgeraniol, an intermediate in the mevalonate pathway, prevents inhibition of osteoclast formation, bone resorption, and kinase activation in vitro [abstract] Proc Natl Acad Sci U S A. 1999;96:133–138. doi: 10.1073/pnas.96.1.133.
    1. Sillence D, Ault J. Osteogenesis Imperfecta – caring for children and adolescents. Resource document. Australian Osteogenesis Imperfecta Foundation. Accessed 23 October 2012.
    1. Seikaly MG, Kopanati S, Salhab N, Waber P, Patterson D, Browne R, Herring JA. Impact of alendronate on quality of life in children with osteogenesis imperfecta. J Pediatr Orthop. 2005. doi:10.1097/01.bpo.0000176162.78980.ed.
    1. Engelbert R, Gulmans V, Uiterwaal C, Helders P. Osteogenesis imperfecta in childhood: perceived competence in relation to impairment and disability. Arch Phys Med Rehabil. 2001. doi:10.1053/apmr.2001.23889.
    1. Kok DHJ, Sakkers RJB, Janse AJ, Pruijs HEH, Verbout AJ, Castelein RM, Engelbert RHH. Quality of life in children with osteogenesis imperfecta treated with oral bisphosphonates (Olpadronate): a 2-year randomized placebo-controlled trial. Eur J Pediatr. 2007. doi:10.1007/s00431-006-0399-2.
    1. Juniper E, Guyatt G, Feeney D, Griffith L, Ferrie P. Minimun skills required by children to complete health-related quality of life instruments for asthma: comparrison of measurement properties. Eur Respir J. 1997. doi:10.1183/09031936.97.10102285.
    1. Fayer P, Machin D. Quality of Life. The assessment, analysis and interpretation of patient-reported outcomes. 2. Chichester: John Wiley and sons Ltd.; 2007.
    1. Eiser C, Morse R. A review of measures of quality of life for children with chronic illness. Arch Dis Child. 2001. doi:10.1136/adc.84.3.205.
    1. Eiser C. Children’s quality of life measures. Arch Dis Child. 1997. doi:10.1136/adc.77.4.350.
    1. Brazier J, Ratcliffe J, Salomen J, Tsuchiya A. Measuring and valuing health benefits for economic evaluation. Oxford: Oxford University Press; 2007.
    1. Stevens KJ. Working with children to develop dimensions for a preference-based, generic, pediatric. Qual Health Res. 2010. doi:10.1177/1049732309358328.
    1. Solans M, Pane S, Estrada M, Serra-Sutton V, Berra S, Herdman M, Alonso J, Rajmil L. Health-related quality of life measurement in children and adolescents: a systematic review of generic and disease-specific instruments. Value Health. 2008. doi:10.1111/j.1524-4733.2007.00293.x.
    1. Eiser C, Morse R. Quality of life measures in chronic diseases of childhood. Health Technol Assess. 2001;5(4):1–162.
    1. Petrou S. Methodoligical issues raised by preference-based approaches to measuring the health status of children. Health Econ. 2003;12:697–702. doi: 10.1002/hec.775. doi:10.1002/hec.775.
    1. Grange A, Bekker H, Noyes J, Langley P. Adequacy of health-related quality of life measures in children under 5 years old: systematic review. J Adv Nurs. 2007. doi:10.1111/J.1365-2648.2007.04333.x [corrected] [published erratum appears in J ADV NURS 2007 Sep;59(6):657]
    1. Prout A. Representing children: reflections on the children 5–15 programme. Child Soc. 2001. doi:10.1002/chi.667.
    1. Bryman A. Social Research Methods. 2. Oxford: Oxford University Press; 2004.
    1. Ritchie JS, Spencer L. In: Analyzing qualitative data. Bryman A, Burgess R, editor. London: Routledge; 1994. Qualitative data analysis for applied policy research; pp. 173–194.
    1. Lacey A, Luff D. Qualitative data analysis. 2009. . Accessed 21st March 2013.
    1. Santos M, Barros L. Risk and resistance factors in OI patients - What is to be learn by health providers, 11th International Conference on Osteogenesis Imperfecta. Dubrovnik, Croatia: Croatian Paediatric Orthopaedic Society of Croatian Medical Association; 2011.
    1. Davis E, Waters E, Mackinnon A, Reddihough D, Graham HK, Mehmet-Radji O, Boyd R. Paediatric quality of life instruments: a review of the impact of the conceptual framework on outcomes. Dev Med Child Neurol. 2006. doi:10.1017/s0012162206000673.
    1. Muldoon M, Barger S, Flory J, Manuck SVP. What are quality of life measurements measuring? Br Med J. 1998;316:542–545. doi: 10.1136/bmj.316.7130.542.
    1. Upton P, Lawford J, Eiser C. Parent–child agreement across child health-related quality of life instruments: a review of the literature. Qual Life Res. 2008. doi:10.1007/s11136-008-9350-5.
    1. Waters E, Elise D, Gabriel MR, Peter R, Michael L, Saroj S. Quality of life instruments for children and adolescents with neurodisabilities: how to choose the appropriate instrument. Dev Med Child Neurol. 2009. doi:10.1111/j.1469-8749.2009.03324.x.
    1. Morris C, Liabo K, Wright P, Fitzpatrick R. Development of the Oxford ankle foot questionnaire: Finding out how children are affected by foot and ankle problems. Child: Care Health and Development; 2007. doi:10.1111/j.1365-2214.2007.00770.x.
    1. Theunissen N, Vogels T, Koopman H, Verrips G, Zwinderman K, Verloove-Vanhorick S, Wit JM. The proxy problem: child report versus parent report in health-related quality of life research. Qual Life Res. 1998. doi:10.1023/A:1008801802877.
    1. Starfield B, Riley A, Green B, Ensminger M, Ryan S, Kelleher K, Kim-Harris S, Johnston D, Vogel K. The adolescent child health and illness profile. A population-based measure of health. Med Care. 1995. doi:10.1097/00005650-199505000-00008.
    1. Vogels T, Verrips GHW, Verloove-Vanhorick SP, Fekkes M, Kamphuis RP, Koopman HM, Theunissen NCM, Wit JM. Measuring health-related quality of life in children: the development of the TACQOL parent form. Qual Life Res. 1998. doi:10.1023/A:1008848218806.
    1. Bullinger M, Schmidt S, Petersen C, The Disabkids G. Assessing quality of life of children with chronic health conditions and disabilities: a European approach. Int J Rehabil Res. 2002. doi:10.1097/00004356-200209000-00005.

Source: PubMed

Sponsors and Collaborators

Medical Conditions

Drug Interventions

3
Subscribe