Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication
Lived Experiences of Families of Children With Spinal Muscular Atrophy Type 1: A Qualitative Investigation Into Feeding and Communication
Spinal Muscular Atrophy Type 1 (SMA )Type 1 is a severe, early-onset neuromuscular condition that typically leads to profound weakness and impaired bulbar function-affecting swallowing, feeding, speech, and airway protection. Historically, bulbar decline contributed significantly to early morbidity and mortality.
The advent of disease-modifying therapies (DMTs) such as nusinersen, zolgensma and risdiplam (also known as Spinraza, Zolgensma, and Evrysdi) sinersinhas altered the clinical course of SMA Type 1, with emerging evidence of motor improvement and increased survival. However, the impact of these therapies on bulbar function remains poorly understood, and standardised tools for its assessment are lacking.
Qualitative research which uses interviews with parents and carers offers an opportunity to capture nuanced caregiver perspectives, identify meaningful functional outcomes, and explore daily lived experiences in a way quantitative tools currently cannot.
This study will investigate the lived experiences of families managing feeding and communication in children with SMA Type 1.
The research will also aim to
1 Identify emotional, social issues experienced by families and practical support needs related to feeding and communication.
2. Provide insights that can inform healthcare interventions and support
Visão geral do estudo
Status
Condições
Descrição detalhada
Methodology This study will adopt a qualitative phenomenological approach to gain in-depth insights into the experiences of families of children with SMA Type 1. It is anticipated that the study will interview 10-15 participants, the numbers will be determined by how many parents need to be interviewed to reach the point of saturation i.e. the point at which no new themes arise. The interviews will be aim to be inductive and discursive.
Prior to the interview parents will have received the information leaflet and consent form. A broad interview guide will be used to enable the interviewer to ensure all areas are covered within each interview, whilst providing flexibility to ensure all participant feedback is gathered. The interviews will be recorded digitally.
Participants The study will be advertised on the SMAUK website and their other social media platforms. Families will be invited to contact the CI if they are interested in taking part. Participants will be caregivers (parents, guardians) of children diagnosed with SMA Type 1.
The CI will then contact the potential participants and share the full information sheet and consent form. The participant will then complete and return the consent form and the PI will contact them to arrange a time for the interview. Interviews are expected to last approximately 60 minutes.
Data Collection The CI will carry out semi-structured interviews conducted via video conferencing, focusing on feeding and communication experiences. Interviews will be recorded, and questions will be based upon a broad interview guide to ensure all key areas are covered whilst being flexible to enable participants to share their experiences.
Data Analysis Recordings from interviews will be transcribed using Thematic Analysis (Braun and Clarke 2006)Thematic analysis software will be used to support the identification of recurring patterns and themes. Coding consistency will be checked by another researcher who will recode an anonymised random sample of the transcriptions.
Ethical Considerations: Informed consent will be obtained from all participants. The study will adhere to ethical guidelines to ensure confidentiality, voluntary participation, and sensitivity to emotional distress.
Tipo de estudo
Inscrição (Estimado)
Contactos e Locais
Contato de estudo
- Nome: Anne Breaks, PhD
- Número de telefone: +447808723798
- E-mail: anne.breaks@nhs.net
Estude backup de contato
- Nome: Heinz Jungbluth, PhD
- Número de telefone: 02071883998
- E-mail: heinz.jungbluth@nhs.net
Locais de estudo
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London, Reino Unido, SE1 7EU
- GSTT Evelina Neurosciences
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Contato:
- Ming Lim, PhD
- Número de telefone: 02071883992
- E-mail: ming.lim3@nhs.net
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
- Filho
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Método de amostragem
População do estudo
Descrição
Inclusion Criteria:
- Parents/guardians of children with a diagnosis of SMA1 who have received any one or more disease modifying therapies[CE7.1][BA7.2][AB7.3]
- Participants need to be able to carry out interview in English In addition to parents, grandparents or other relatives with full parental responsibility will be included
Exclusion Criteria:
- Parents/carers who require an interpreter will not be included within the study for reasons of time and cost and because parents may feel less able to be open and honest when communicating with the researcher through a third party.
- Primary carer who is a foster carer or corporate parent (i.e. a looked after child) as they are not likely to have the same decision-making 'freedoms'.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
Coortes e Intervenções
Grupo / Coorte |
|---|
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Parents/guardians of children with a diagnosis of SMA1
Parents/guardians of children with a diagnosis of SMA1 who have received any one or more disease modifying therapies Participants need to be able to carry out interview in English In addition to parents, grandparents or other relatives with full parental responsibility will be included
|
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Qualitative themes describing caregiver experiences of feeding and communication in children with SMA Type 1.
Prazo: From interview to completion of thematic analysis 2 weeks later
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The primary outcome is the set of emergent qualitative themes describing caregiver experiences of feeding and communication in children with SMA Type 1. Data will be collected via a single semi-structured interview per participant (approximately one hour) conducted post-consent, and analysed using inductive thematic analysis (NVivo) following completion of data collection.
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From interview to completion of thematic analysis 2 weeks later
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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Broader issues
Prazo: From interview to 2 weeks after when thematic analysis is completed
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Social, emotional, and practical challenges related to feeding and communication. Caregiver perceptions of bulbar function following disease-modifying therapies (DMTs). Identification of perceived barriers and support systems impacting feeding and communication, assessed during the same single interview session and analysed thematically. |
From interview to 2 weeks after when thematic analysis is completed
|
Colaboradores e Investigadores
Patrocinador
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
- Doenças do Sistema Nervoso Central
- Doenças do Sistema Nervoso
- Doenças Neuromusculares
- Doenças Genéticas, Congênitas
- Doenças do aparelho digestivo
- Doenças Gastrointestinais
- Doenças Neurodegenerativas
- Doenças Esofágicas
- Doenças Otorrinolaringológicas
- Distúrbios Heredodegenerativos, Sistema Nervoso
- Doenças Faríngeas
- Doenças da Medula Espinhal
- Doença do neurônio motor
- Atrofia Muscular Espinhal
- Doenças e Anormalidades Congênitas, Hereditárias e Neonatais
- Comportamento
- Comportamento verbal
- Distúrbios da Deglutição
- Atrofias Musculares Espinhais da Infância
- Comunicação
- Discurso
- Língua
Outros números de identificação do estudo
- 358595
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Informações sobre medicamentos e dispositivos, documentos de estudo
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