Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy

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 on the effectiveness of a multicomponent, digitally delivered intervention and its implementation feasibility among primary caregivers of children with SMA and DMD in Pakistan.

• Multicomponent Intervention (Behavioral): Multiple support groups will be created for SMA and DMD patients, separately. Within SMA and DMD, further subgroups will be created on the basis of caregivers educational level and socioeconomic background. Each support group will include up to 10-14 patients' caregivers, 1 neurologist, 1 pediatric neurologist, 1 physiotherapist, 1 nutritionist, 1 speech therapist, 1 occupational therapist, 1 epidemiologist and 1 Research Associate. Sessions with each group will integrate peer support, coaching to address the multifaceted needs of patients, and time for open questions and answer sessions with the group. The sessions with each group will be 30-60 minutes in duration. In between these live sessions, short video clips, pictorial guides, audios will be sent to the participants to re-enforce what they could learn to improve care of their child and potentially increase their self-efficacy levels.

Primary outcomes

• Change in Self-Efficacy Scores [Baseline and 8 weeks]: Change from Baseline in the Self-Efficacy Scores at 8 weeks. Care giver self efficacy tool is a 20-item self-report questionnaire adapted from the original validated scale developed for caregivers of individuals with DMD (DMD-CSES). The scale assesses caregiver self-efficacy across multiple domains of caregiving. Each item is rated on a 5-point Likert scale ranging from 1 (Strongly disagree) to 5 (Strongly agree). Total scores range from 19 to 95, calculated by summing responses across all items. Higher scores indicate greater caregiver self-efficacy (better outcome).

Sex: All, ages: any to 18 Years, accepts healthy volunteers

Inclusion Criteria:

* Primary caregiver of a child diagnosed with SMA or DMD.
* Enrolled in the Treat-NMD registry- Pakistan.
* Access to a smart phone with internet connectivity.

Exclusion Criteria:

* Caregivers who are currently participating in another support group intervention.
* Caregivers who are unable to understand the language of communication (Urdu)

• Nashit Irfan Aziz, MBBS, MSc, Principal investigator, Aga Khan University
• Sara Khan, MBBS, MD, Principal investigator, Aga Khan University
• Wardah Khalid, MBBS, MSc, PhD, Principal investigator, Aga Khan University

NIH grants: 2025