The impact of caring for an individual with aromatic l-amino acid decarboxylase (AADC) deficiency: a qualitative study and the development of a conceptual model.


Journal

Current medical research and opinion
ISSN: 1473-4877
Titre abrégé: Curr Med Res Opin
Pays: England
ID NLM: 0351014

Informations de publication

Date de publication:
10 2021
Historique:
pubmed: 15 7 2021
medline: 16 11 2021
entrez: 14 7 2021
Statut: ppublish

Résumé

Aromatic l-amino acid decarboxylase (AADC) deficiency is a rare neurological condition, associated with a wide range of symptoms and functional issues, such as profound motor impairment and learning disability. Most individuals with AADC deficiency are completely dependent on their caregivers. This study explored the impact of caring for an individual with AADC deficiency. Qualitative interviews were conducted with caregivers of individuals with AADC deficiency in Italy, Portugal, Spain and the United States. An interview guide was developed with input from clinical experts and caregivers and included questions on the impact of caring for an individual with AADC deficiency. Interviews were conducted by telephone/videoconference and were recorded and transcribed. Data were analysed using thematic analysis. Fourteen caregivers took part who provided care to 13 individuals with AADC deficiency aged 1-15 years. Caregivers reported that their lives centred around the individual with AADC deficiency, due to their need for 24-hour care and regular healthcare appointments. They reported both proximal impacts (impact on time, planning, physical health and emotional wellbeing), and distal impacts (impact on social/leisure activities, relationships, work and finances). These concepts and relationships were illustrated in a conceptual model. This is the first qualitative study to report on the experience of caring for an individual with AADC deficiency. Caregivers reported that caring had a substantial multifaceted impact on their lives. These findings highlight the importance of considering the caregiver experience when evaluating the burden of AADC deficiency.

Sections du résumé

BACKGROUND
Aromatic l-amino acid decarboxylase (AADC) deficiency is a rare neurological condition, associated with a wide range of symptoms and functional issues, such as profound motor impairment and learning disability. Most individuals with AADC deficiency are completely dependent on their caregivers. This study explored the impact of caring for an individual with AADC deficiency.
METHODS
Qualitative interviews were conducted with caregivers of individuals with AADC deficiency in Italy, Portugal, Spain and the United States. An interview guide was developed with input from clinical experts and caregivers and included questions on the impact of caring for an individual with AADC deficiency. Interviews were conducted by telephone/videoconference and were recorded and transcribed. Data were analysed using thematic analysis.
RESULTS
Fourteen caregivers took part who provided care to 13 individuals with AADC deficiency aged 1-15 years. Caregivers reported that their lives centred around the individual with AADC deficiency, due to their need for 24-hour care and regular healthcare appointments. They reported both proximal impacts (impact on time, planning, physical health and emotional wellbeing), and distal impacts (impact on social/leisure activities, relationships, work and finances). These concepts and relationships were illustrated in a conceptual model.
CONCLUSIONS
This is the first qualitative study to report on the experience of caring for an individual with AADC deficiency. Caregivers reported that caring had a substantial multifaceted impact on their lives. These findings highlight the importance of considering the caregiver experience when evaluating the burden of AADC deficiency.

Identifiants

pubmed: 34259573
doi: 10.1080/03007995.2021.1955668
doi:

Substances chimiques

Aromatic-L-Amino-Acid Decarboxylases EC 4.1.1.28

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1821-1828

Auteurs

Hanna Skrobanski (H)

Patient-Centred Outcomes Research, Acaster Lloyd Consulting Ltd, London, United Kingdom.

Kate Williams (K)

Patient-Centred Outcomes Research, Acaster Lloyd Consulting Ltd, London, United Kingdom.

Christian Werner (C)

Global Medical Affairs, PTC Therapeutics Germany GmbH, Frankfurt am Main, Germany.

Sian O'Neill (S)

Patient Engagement EMEA, PTC Therapeutics International Limited, Ireland.

Katharina Buesch (K)

Global Health Economics and Outcomes Research, PTC Therapeutics Switzerland GmbH, Steinhausen/Zug, Switzerland.

Sarah Acaster (S)

Patient-Centred Outcomes Research, Acaster Lloyd Consulting Ltd, London, United Kingdom.

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Classifications MeSH