ABSTRACT
Introducción. La exostosis múltiple hereditaria es una enfermedad poco frecuente autosómica dominante caracterizada por presencia de múltiples proyecciones óseas. Objetivo. Analizar factores asociados a la calidad de vida relacionada con la salud (CVRS) en niños >2 años y en adultos en seguimiento en un hospital de pediatría de alta complejidad de Argentina. Población y métodos. Estudio transversal de una cohorte en seguimiento. La CVRS se midió con Pediatric Quality of Life Inventory (PedsQL) y Short Form Health Survey (SF-36). Se registró sexo, edad, características sociodemográficas, estatura, radiología, alteración de eje y función de miembros, presencia de dolor y malignización. Se clasificó la gravedad según Pedrini y col. Se realizaron pruebas paramétricas, no paramétricas y análisis de regresión. Resultados. Se incluyeron 66 casos (47 niños y 19 adultos). Relación sexo masculino/femenino: 1,7/1. Mediana de edad: 13,4 años (r: 2,2155,3). Presentaron dolor 30 de 47 niños y 17 de 19 adultos. Si se considera la edad ósea adulta (o cierre epifisario) como punto de corte para definir el estado de adulto, 11 de 37 niños y 18 de 27 adultos presentaron forma grave de enfermedad, y se observó baja estatura en 2 de 38 niños y en 9 de 27 adultos. El valor promedio del componente físico de CVRS en niños fue 65,9 (DE: 22,5) y, en adultos, 27,2 (RIC: 18,5-34,7). La presencia de dolor y la gravedad clínica se asoció significativamente a menor CVRS tanto en niños como en adultos. Conclusiones. En este estudio se observó que el dolor y la gravedad de la enfermedad tuvieron un efecto negativo en la CVRS.
Introduction. Hereditary osteochondromatosis is an uncommon, autosomal, dominant condition characterized by the presence of multiple bone growths. Objective. To analyze factors associated with health-related quality of life (HRQoL) among children > 2 years and adults receiving follow-up at a tertiary care children's hospital in Argentina. Population and methods. Cross-sectional study of a follow-up cohort. HRQoL was measured using the Pediatric Quality of Life Inventory (PedsQL) and the Short Form Health Survey (SF36). Sex, age, sociodemographic characteristics, height, radiology, axis alteration and limb function, presence of pain, and malignant change were recorded. Severity was classified as per Pedrini et al. Parametric and non-parametric tests and regression analysis were done. Results. A total of 66 cases (47 children and 19 adults) were included. Male/female ratio: 1.7/1. Median age: 13.4 years (r: 2.21-55.3). Pain was observed in 30/47 children and in 17/19 adults. Considering the adult bone age (or epiphyseal closure) as the cutoff point to define adult status, 11/37 children and 18/27 adults had a severe disease and 2/38 children and 9/27 adults had short stature. The average value of the physical component of HRQoL in children was 65.9 (SD: 22.5) and, in adults, 27.2 (IQR: 18.534.7). The presence of pain and clinical severity were significantly associated with a lower HRQoL, both in children and adults. Conclusions. This study found that pain and disease severity had a negative effect on HRQoL.
Subject(s)
Humans , Child, Preschool , Child , Adolescent , Quality of Life , Osteochondromatosis , Pain , Severity of Illness Index , Cross-Sectional Studies , Surveys and QuestionnairesABSTRACT
INTRODUCTION: Hereditary osteochondromatosis is an uncommon, autosomal, dominant condition characterized by the presence of multiple bone growths. OBJECTIVE: To analyze factors associated with health-related quality of life (HRQoL) among children > 2 years and adults receiving follow-up at a tertiary care children's hospital in Argentina. POPULATION AND METHODS: Cross-sectional study of a follow-up cohort. HRQoL was measured using the Pediatric Quality of Life Inventory® (PedsQL) and the Short Form Health Survey (SF- 36). Sex, age, sociodemographic characteristics, height, radiology, axis alteration and limb function, presence of pain, and malignant change were recorded. Severity was classified as per Pedrini et al. Parametric and non-parametric tests and regression analysis were done. RESULTS: A total of 66 cases (47 children and 19 adults) were included. Male/female ratio: 1.7/1. Median age: 13.4 years (r: 2.21-55.3). Pain was observed in 30/47 children and in 17/19 adults. Considering the adult bone age (or epiphyseal closure) as the cutoff point to define adult status, 11/37 children and 18/27 adults had a severe disease and 2/38 children and 9/27 adults had short stature. The average value of the physical component of HRQoL in children was 65.9 (SD: 22.5) and, in adults, 27.2 (IQR: 18.5- 34.7). The presence of pain and clinical severity were significantly associated with a lower HRQoL, both in children and adults. CONCLUSIONS: This study found that pain and disease severity had a negative effect on HRQoL.
Introducción. La exostosis múltiple hereditaria es una enfermedad poco frecuente autosómica dominante caracterizada por presencia de múltiples proyecciones óseas. OBJETIVO: Analizar factores asociados a la calidad de vida relacionada con la salud (CVRS) en niños >2 años y en adultos en seguimiento en un hospital de pediatría de alta complejidad de Argentina. Población y métodos. Estudio transversal de una cohorte en seguimiento. La CVRS se midió con Pediatric Quality of Life Inventory® (PedsQL) y Short Form Health Survey (SF-36). Se registró sexo, edad, características sociodemográficas, estatura, radiología, alteración de eje y función de miembros, presencia de dolor y malignización. Se clasificó la gravedad según Pedrini y col. Se realizaron pruebas paramétricas, no paramétricas y análisis de regresión. RESULTADOS: Se incluyeron 66 casos (47 niños y 19 adultos). Relación sexo masculino/femenino: 1,7/1. Mediana de edad: 13,4 años (r: 2,21- 55,3). Presentaron dolor 30 de 47 niños y 17 de 19 adultos. Si se considera la edad ósea adulta (o cierre epifisario) como punto de corte para definir el estado de adulto, 11 de 37 niños y 18 de 27 adultos presentaron forma grave de enfermedad, y se observó baja estatura en 2 de 38 niños y en 9 de 27 adultos. El valor promedio del componente físico de CVRS en niños fue 65,9 (DE: 22,5) y, en adultos, 27,2 (RIC: 18,5-34,7). La presencia de dolor y la gravedad clínica se asoció significativamente a menor CVRS tanto en niños como en adultos. CONCLUSIONES: En este estudio se observó que el dolor y la gravedad de la enfermedad tuvieron un efecto negativo en la CVRS.
Subject(s)
Osteochondromatosis , Quality of Life , Adolescent , Adult , Child , Cross-Sectional Studies , Female , Humans , Male , Pain , Severity of Illness Index , Surveys and QuestionnairesABSTRACT
BACKGROUND: To validate the Argentinean Spanish version of the PedsQL 4.0 Generic Core Scales in Argentinean children and adolescents with chronic conditions and to assess the impact of socio-demographic characteristics on the instrument's comprehensibility and acceptability. Reliability, and known-groups, and convergent validity were tested. METHODS: Consecutive sample of 287 children with chronic conditions and 105 healthy children, ages 2-18, and their parents. Chronically ill children were: (1) attending outpatient clinics and (2) had one of the following diagnoses: stem cell transplant, chronic obstructive pulmonary disease, HIV/AIDS, cancer, end stage renal disease, complex congenital cardiopathy. Patients and adult proxies completed the PedsQL 4.0 and an overall health status assessment. Physicians were asked to rate degree of health status impairment. RESULTS: The PedsQL 4.0 was feasible (only 9 children, all 5 to 7 year-olds, could not complete the instrument), easy to administer, completed without, or with minimal, help by most children and parents, and required a brief administration time (average 5-6 minutes). People living below the poverty line and/or low literacy needed more help to complete the instrument. Cronbach Alpha's internal consistency values for the total and subscale scores exceeded 0.70 for self-reports of children over 8 years-old and parent-reports of children over 5 years of age. Reliability of proxy-reports of 2-4 year-olds was low but improved when school items were excluded. Internal consistency for 5-7 year-olds was low (alpha range = 0.28-0.76). Construct validity was good. Child self-report and parent proxy-report PedsQL 4.0 scores were moderately but significantly correlated (rho = 0.39, p < 0.0001) and both significantly correlated with physician's assessment of health impairment and with child self-reported overall health status. The PedsQL 4.0 discriminated between healthy and chronically ill children (72.72 and 66.87, for healthy and ill children, respectively, p = 0.01), between different chronic health conditions, and children from lower socioeconomic status. CONCLUSION: Results suggest that the Argentinean Spanish PedsQL 4.0 is suitable for research purposes in the public health setting for children over 8 years old and parents of children over 5 years old. People with low income and low literacy need help to complete the instrument. Steps to expand the use of the Argentinean Spanish PedsQL 4.0 include an alternative approach to scoring for the 2-4 year-olds, further understanding of how to increase reliability for the 5-7 year-olds self-report, and confirmation of other aspects of validity.
