Preliminary development of proxy-rated quality-of-life scales for children and adults with Niemann-Pick type C.

Objectives: Niemann-Pick disease type C (NPC) is a rare life-limiting disease for which there is no cure. No scales currently exist to measure the impact of medication, physical therapy or clinical trials. The aim of this study was to develop age-appropriate Quality-of-Life (QoL) scales to measure t...

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Publicado en:Quality of Life Research Vol. 28; no. 11; pp. 3083 - 3093
Autores principales: Aston, Lydia, Shaw, Rachel, Knibb, Rebecca
Formato: research tables/charts Journal Article
Publicado: Springer Nature Nov2019
Acceso en línea:Ver este registro en EBSCOhost
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      dt: Nov2019
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      pub: Springer Nature
      place: New York, New York
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        atl: Preliminary development of proxy-rated quality-of-life scales for children and adults with Niemann-Pick type C.
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        au:
          Aston, Lydia
          Shaw, Rachel
          Knibb, Rebecca
        affil: Department of Psychology, School of Life and Health Sciences, Aston University, Aston Triangle, B4 7ET, Birmingham, UK
      sug:
        subj:
          Niemann-Pick Disease, Type C Psychosocial Factors
          Quality of Life Psychosocial Factors
          Child
          Adult
          Reproducibility of Results
          Female
          Parents Psychosocial Factors
          Disease Attributes
          Caregivers Psychosocial Factors
          Male
          Adolescence
          Questionnaires
          Human
          Child: 6-12 years
          Adult: 19-44 years
          Adolescent: 13-18 years
          Female
          Male
      ab: Objectives: Niemann-Pick disease type C (NPC) is a rare life-limiting disease for which there is no cure. No scales currently exist to measure the impact of medication, physical therapy or clinical trials. The aim of this study was to develop age-appropriate Quality-of-Life (QoL) scales to measure the impact of NPC on children and adults.Design: Scale development study using a phenomenological approach to data generation and analysis.Methods: Fourteen interviews were conducted with people living with NPC and/or their parents/carers. Themes were generated and examined against an existential-phenomenological theory of wellbeing. A matrix was constructed to represent the phenomenological insight gained on participants' subjective experiences and a bank of items that were related to their QoL was developed.Results: NPC quality-of-life questionnaires for children (NPCQLQ-C) and adults (NPCQLQ-A) proxy prototype scales were produced and completed by 23 parents/carers of children (child age mean = 8.61 years) and 20 parents/carers of adults (adult age = 33.4 years). Reliability analysis resulted in a 15-item NPCQLQ-C and a 30-item NPCQLQ-A, which showed excellent internal consistency, Cronbach's α = 0.925 and 0.947, respectively.Conclusion: The NPCQLQ-C and NPCQLQ-A are the first disease-specific QoL scales to be developed for people living with NPC. This novel approach to scale development values the experiential, real life impact of living with NPC and focused on the lived-experiences and impact on QoL. The scales will enable healthcare professionals and researchers to have a better understanding and quantifiable measurement of the impact of living with NPC on a patient's daily life.
      pubtype: Academic Journal
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        research
        tables/charts
        Journal Article
      ougenre: Article
    language: English
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