Patient-Centred Outcomes Research, Acaster Lloyd Consulting Ltd, London, United Kingdom.
Global Medical Affairs, PTC Therapeutics Germany GmbH, Frankfurt am Main, Germany.
Curr Med Res Opin. 2021 Oct;37(10):1821-1828. doi: 10.1080/03007995.2021.1955668. Epub 2021 Aug 1.
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.
芳香族 l-氨基酸脱羧酶(AADC)缺乏症是一种罕见的神经疾病,与广泛的症状和功能问题相关,例如严重的运动障碍和学习障碍。大多数 AADC 缺乏症患者完全依赖他们的照顾者。本研究探讨了照顾 AADC 缺乏症患者的影响。
对意大利、葡萄牙、西班牙和美国的 AADC 缺乏症患者的照顾者进行了定性访谈。访谈指南由临床专家和照顾者共同制定,包括照顾 AADC 缺乏症患者的影响的问题。通过电话/视频会议进行访谈,并对访谈进行录音和转录。使用主题分析对数据进行分析。
共有 14 名照顾者参与,他们照顾了 13 名年龄在 1-15 岁的 AADC 缺乏症患者。照顾者报告说,他们的生活以 AADC 缺乏症患者为中心,因为他们需要 24 小时护理和定期医疗保健预约。他们报告了近端影响(对时间、计划、身体健康和情绪健康的影响)和远端影响(对社交/休闲活动、人际关系、工作和财务的影响)。这些概念和关系在一个概念模型中得到了说明。
这是第一项报告照顾 AADC 缺乏症患者经历的定性研究。照顾者报告说,照顾对他们的生活有很大的多方面影响。这些发现强调了在评估 AADC 缺乏症的负担时,考虑照顾者体验的重要性。