Development of the APBD-SQ, a novel patient-reported outcome for health-related quality of life in adult polyglucosan body disease.
J Neurol Sci
; 464: 123168, 2024 Sep 15.
Article
em En
| MEDLINE
| ID: mdl-39121524
ABSTRACT
Adult polyglucosan body disease (APBD) is a rare autosomal recessive glycogen storage disorder that leads to slowly progressive multi-organ dysfunction in adulthood. A novel disease-specific patient-reported outcome measure was created and administered to assess symptom burden and health-related quality of life (HR-QOL) in APBD. Thirty-six participants between 30 and 79 years of age (83% ≥60 years, 56% male) completed the anonymous questionnaire independently or with a caregiver proxy (75% self-report). Unemployment predicted an 18.3 (95% CI 2.8, 33.8; p = 0.028) higher composite disease severity score and a 28.8 (95% CI 8.2, 49.4; p = 0.010) higher composite HR-QOL score. Use of one or more assistive devices also predicted a 29.3 (95% CI 8.3, 50.4; p = 0.011) higher composite disease severity score and a 41.8 (95% CI 10.9, 72.8; p = 0.013) higher composite HR-QOL score. Proxy survey completion predicted a 19.4 (95% CI 4.1, 34.7; p = 0.020) higher composite disease severity score compared to self-report. Older age at survey completion predicted a 27.4 higher composite HR-QOL score (95% CI 2.5, 52.4; p = 0.039) for participants in their sixties compared to those between 30 and 59 years old. The development of the Adult Polyglucosan Body Disease questionnaire on Symptom burden and health-related Quality of life (APBD-SQ) marks an important stride forward in capturing the patient experience as a tool for disease monitoring and future research.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Qualidade de Vida
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Doença de Depósito de Glicogênio
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Medidas de Resultados Relatados pelo Paciente
Limite:
Adult
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Aged
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Female
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Humans
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Male
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Middle aged
Idioma:
En
Revista:
J Neurol Sci
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J. neurol. sci
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Journal of the neurological sciences
Ano de publicação:
2024
Tipo de documento:
Article
País de afiliação:
Estados Unidos