Impact on diet quality and dietary burden of sapropterin dihydrochloride use in children with phenylketonuria : a 6 months follow-up report / Maria Gama ; orient. Anita MacDonald, Júlio César Rocha
Main Author Gama, Maria Inês Ferreira da Rocha Secondary Author MacDonald, AnitaRocha, Júlio César Language Inglês. Country Portugal. Publication Lisboa : NOVA Medical School, Universidade NOVA de Lisboa, 2023 Description 103 p. Dissertation Note or Thesis: Dissertação de Mestrado
Nutrição Humana e Metabolismo
2023
Faculdade de Ciências Médicas, Universidade NOVA de Lisboa
Abstract Abstract Introduction: Adjuvant treatments in Phenylketonuria (PKU), such as sapropterin dihydrochloride, aim to increase natural protein tolerance and/or ameliorate blood phenylalanine (Phe) levels in a subset of patients. Changes in feeding patterns and behaviors in sapropterin-responsive populations have not been widely reported in the literature. Our aim was to assess changes in food quality, mental health and burden of care in a PKU sapropterin-responsive cohort. Methods: In an observational, longitudinal study, questionnaires on food frequency, neophobia, anxiety and depression, impact on family and burden of care were applied to patients at baseline, 3- months and 6-months after sapropterin-responsiveness testing. Data on growth, nutritional intake and metabolic control were collected at all time-points. Results: 17 children (10.8 ± 4.2 years) reached the 6-months follow-up assessment. Patients body mass index (BMI) z-scores remained similar after drug initiation, while increasing natural protein (p = <0.001) and reducing protein substitute intake (p = 0.002). Blood Phe control was kept stable throughout the study (p = 0.731). Increases in regular cow’s milk (p = 0.001), meat/fish and eggs (p = 0.005), bread (p = 0.01) and pasta (p = 0.011) were seen, while decreasing intake of low-protein milk (p = 0.007), low-protein bread (p = 0.028) and low-protein pasta (p = <0.001). Anxiety (p = 0.016) and depression (p = 0.022) were significantly decreased in caregivers. As for impact on family, differences were observed for familial-social (p = 0.002) and personal strain (p = 0.001) subsets. On burden of care, the majority of caregivers considered dietary management to be easier but still restrictive. Before drug treatment more time was spent on PKU related tasks, but this decreased with time. Around half of caregivers spent more money on food shopping and the majority went out for meals more regularly. Fewer caregivers had to deny food choices on a daily basis to their children after 6-months on the drug. Conclusion: There were significant positive changes in food patterns, behaviors, and alleviation of burden of care in children with PKU and their families after 6-months on sapropterin treatment Topical name Dietary Patterns
Caregiver Burden
Phenylketonurias
Academic Dissertation Online Resources Click here to access the eletronic resource http://hdl.handle.net/10362/162747
| Item type | Current location | Call number | url | Status | Date due | Barcode |
|---|---|---|---|---|---|---|
| Documento Eletrónico | Biblioteca NMS|FCM online | RUN | http://hdl.handle.net/10362/162747 | Available | 20240031 |
Dissertação de Mestrado Nutrição Humana e Metabolismo 2023 Faculdade de Ciências Médicas, Universidade NOVA de Lisboa
Abstract Introduction: Adjuvant treatments in Phenylketonuria (PKU), such as sapropterin dihydrochloride, aim to increase natural protein tolerance and/or ameliorate blood phenylalanine (Phe) levels in a subset of patients. Changes in feeding patterns and behaviors in sapropterin-responsive populations have not been widely reported in the literature. Our aim was to assess changes in food quality, mental health and burden of care in a PKU sapropterin-responsive cohort. Methods: In an observational, longitudinal study, questionnaires on food frequency, neophobia, anxiety and depression, impact on family and burden of care were applied to patients at baseline, 3- months and 6-months after sapropterin-responsiveness testing. Data on growth, nutritional intake and metabolic control were collected at all time-points. Results: 17 children (10.8 ± 4.2 years) reached the 6-months follow-up assessment. Patients body mass index (BMI) z-scores remained similar after drug initiation, while increasing natural protein (p = <0.001) and reducing protein substitute intake (p = 0.002). Blood Phe control was kept stable throughout the study (p = 0.731). Increases in regular cow’s milk (p = 0.001), meat/fish and eggs (p = 0.005), bread (p = 0.01) and pasta (p = 0.011) were seen, while decreasing intake of low-protein milk (p = 0.007), low-protein bread (p = 0.028) and low-protein pasta (p = <0.001). Anxiety (p = 0.016) and depression (p = 0.022) were significantly decreased in caregivers. As for impact on family, differences were observed for familial-social (p = 0.002) and personal strain (p = 0.001) subsets. On burden of care, the majority of caregivers considered dietary management to be easier but still restrictive. Before drug treatment more time was spent on PKU related tasks, but this decreased with time. Around half of caregivers spent more money on food shopping and the majority went out for meals more regularly. Fewer caregivers had to deny food choices on a daily basis to their children after 6-months on the drug. Conclusion: There were significant positive changes in food patterns, behaviors, and alleviation of burden of care in children with PKU and their families after 6-months on sapropterin treatment
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