A pre-post single-arm pilot trial of a digital multicomponent intervention for self-efficacy enhancement among online support groups of primary caregivers of children with neuro-muscular disorders: SEE-NMD protocol
- 1Brain and Mind Institute, Aga Khan University, Karachi, Sindh, Pakistan
- 2Department of Community Health Sciences, Aga Khan University, Karachi, Sindh, Pakistan
- 3Department of Medicine, Section of Neurology, Aga Khan University, Karachi, Sindh, Pakistan
Abstract
Introduction: Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD) are progressive genetic neuromuscular disorders that impose substantial caregiving demands on families. In Pakistan, where disease-modifying therapies remain largely inaccessible, care is predominantly supportive, frequently resulting in psychological distress and diminished caregiver confidence. An individual's belief in their capacity to execute required behaviours, self-efficacy, is a recognised determinant of caregiving quality, yet evidence on caregiver self-efficacy in SMA and DMD within low-resource settings remains sparse. This protocol describes the methods for an exploratory, single-arm, pre-post trial. The trial aims to generate preliminary evidence on changes in self-efficacy following a multicomponent, digitally delivered intervention and its implementation feasibility among primary caregivers of children with SMA and DMD in Pakistan. Methods and analysis: A single-arm, pre-post study design will recruit primary caregivers of children with SMA and DMD enrolled in the Treat-NMD Registry of Pakistan, with 40 caregivers per condition (80 total) analyzed as separate disease-specific cohorts. An eight-week intervention will be delivered via online support groups, comprising educational video clips, expert-led live sessions, pictorial guides, and progressively tapering audio reminders. The primary outcome, self-efficacy, will be measured using a culturally adapted, content-validated Urdu version of the DMD Caregiver Self-Efficacy Scale (DMD-CSES), assessed at baseline (T0) and eight weeks post-intervention (T1). Pre- and post-intervention scores will be compared using a paired t-test or Wilcoxon signed-rank test, depending on the data distribution, with analyses conducted in STATA Version 17. Ethics and dissemination: Ethical approval has been granted by the Ethics Review Committee of Aga Khan University (2025-11875-37040). Verbal informed consent will be obtained from all participants, and confidentiality will be maintained throughout the trial. The findings will be disseminated via peer-reviewed publications, conference presentations, and shared with the Treat-NMD registry network. Trial registration name and number: NCT07356063, Date of registration: 11/January/2026.
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BibTeX
@article{aziz2026prepost,
title = {{A pre-post single-arm pilot trial of a digital multicomponent intervention for self-efficacy enhancement among online support groups of primary caregivers of children with neuro-muscular disorders: SEE-NMD protocol}},
author = {Nashit Irfan Aziz and Wardah Khalid and Sara Khan},
journal = {PLOS One},
year = {2026},
month = nov,
doi = {10.1371/journal.pone.0349543},
url = {https://doi.org/10.1371/journal.pone.0349543},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - A pre-post single-arm pilot trial of a digital multicomponent intervention for self-efficacy enhancement among online support groups of primary caregivers of children with neuro-muscular disorders: SEE-NMD protocol
AU - Nashit Irfan Aziz
AU - Wardah Khalid
AU - Sara Khan
PY - 2026
DA - 2026/11/06
JO - PLOS One
DO - 10.1371/journal.pone.0349543
UR - https://doi.org/10.1371/journal.pone.0349543
AB - Introduction: Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD) are progressive genetic neuromuscular disorders that impose substantial caregiving demands on families. In Pakistan, where disease-modifying therapies remain largely inaccessible, care is predominantly supportive, frequently resulting in psychological distress and diminished caregiver confidence. An individual's belief in their capacity to execute required behaviours, self-efficacy, is a recognised determinant of caregiving quality, yet evidence on caregiver self-efficacy in SMA and DMD within low-resource settings remains sparse. This protocol describes the methods for an exploratory, single-arm, pre-post trial. The trial aims to generate preliminary evidence on changes in self-efficacy following a multicomponent, digitally delivered intervention and its implementation feasibility among primary caregivers of children with SMA and DMD in Pakistan. Methods and analysis: A single-arm, pre-post study design will recruit primary caregivers of children with SMA and DMD enrolled in the Treat-NMD Registry of Pakistan, with 40 caregivers per condition (80 total) analyzed as separate disease-specific cohorts. An eight-week intervention will be delivered via online support groups, comprising educational video clips, expert-led live sessions, pictorial guides, and progressively tapering audio reminders. The primary outcome, self-efficacy, will be measured using a culturally adapted, content-validated Urdu version of the DMD Caregiver Self-Efficacy Scale (DMD-CSES), assessed at baseline (T0) and eight weeks post-intervention (T1). Pre- and post-intervention scores will be compared using a paired t-test or Wilcoxon signed-rank test, depending on the data distribution, with analyses conducted in STATA Version 17. Ethics and dissemination: Ethical approval has been granted by the Ethics Review Committee of Aga Khan University (2025-11875-37040). Verbal informed consent will be obtained from all participants, and confidentiality will be maintained throughout the trial. The findings will be disseminated via peer-reviewed publications, conference presentations, and shared with the Treat-NMD registry network. Trial registration name and number: NCT07356063, Date of registration: 11/January/2026.
SN - 1932-6203
ER -