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Fatigue and quality of life in citrin deficiency during adaptation and compensation stage.
Okano, Yoshiyuki; Kobayashi, Kyoko; Ihara, Kenji; Ito, Tetsuya; Yoshino, Makoto; Watanabe, Yoriko; Kaji, Shunsaku; Ohura, Toshihiro; Nagao, Masayoshi; Noguchi, Atsuko; Mushiake, Sotaro; Hohashi, Naohiro; Hashimoto-Tamaoki, Tomoko.
Afiliación
  • Okano Y; Department of Genetics, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, 663-8501, Japan. okano@med.osaka-cu.ac.jp
Mol Genet Metab ; 109(1): 9-13, 2013 May.
Article en En | MEDLINE | ID: mdl-23453692
Citrin-deficient children and adolescents between adult-onset type II citrullinemia and neonatal intrahepatic cholestasis by citrin deficiency do not have clear clinical features except for unusual diet of high-fat, high-protein, and low-carbohydrate food. The aims of the present study are to characterize fatigue and quality of life (QOL) in citrin-deficient patients during adaptation and compensation stage, and to define the relationship between fatigue and QOL. The study subjects were 55 citrin-deficient patients aged 1-22years (29 males) and 54 guardians. Fatigue was evaluated by self-reports and proxy-reports of the PedsQL Multidimensional Fatigue Scale. QOL was evaluated by the PedsQL Generic Core Scales. Both scale scores were significantly lower in child self-reports (p<0.01 and p<0.05, respectively) and parent proxy-reports (p<0.01 and p<0.01, respectively) than those of healthy children. Citrin-deficient patients with scores of 50 percentile or less of healthy children constituted 67.5% of the sample for the Fatigue Scale and 68.4% for the Generic Core Scales. The PedsQL Fatigue Scale correlated with the Generic Core Scales for both the patients (r=0.56) and parents reports (r=0.71). Assessments by the patients and their parents showed moderate agreement. Parents assessed the condition of children more favorably than their children. The study identified severe fatigue and impaired QOL in citrin-deficient patients during the silent period, and that such children perceive worse fatigue and poorer QOL than those estimated by their parents. The results stress the need for active involvement of parents and medical staff in the management of citrin-deficient patients during the silent period.
Asunto(s)

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Adaptación Fisiológica / Citrulinemia / Metabolismo de los Hidratos de Carbono / Fatiga Tipo de estudio: Prognostic_studies Aspecto: Patient_preference Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2013 Tipo del documento: Article País de afiliación: Japón Pais de publicación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Adaptación Fisiológica / Citrulinemia / Metabolismo de los Hidratos de Carbono / Fatiga Tipo de estudio: Prognostic_studies Aspecto: Patient_preference Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2013 Tipo del documento: Article País de afiliación: Japón Pais de publicación: Estados Unidos