Table 3 Challenges faced by caregivers and families
Stage of life | Challenges for caregivers and families | |
|---|---|---|
Across PROS | Specific challenges for M-CM | |
Birth to toddler | Primary caregiver (usually mother) needs to work full time on childcare, monitoring medical issues, arranging appointments, and research Caregiver becomes “case manager” balancing HCP communication, research, symptom management, health insurance claims | Brain overgrowth occurs most dramatically during the first few years, leading to many health challenges and the need for frequent monitoring and possibly surgeries Children may have low muscle tone, frequent infections, and delayed milestones |
Elementary school | Caregiver has to work with school, explain issues, navigate missed schooldays | Severely affected patients require constant care and assistance with feeding, toileting, dressing, etc Need to prepare for and manage seizures Caregivers may seek out community-based resources and mobility devices (eg, adaptive strollers) |
Puberty/teenager | Patient becomes more involved but still dependent on caregiver for research and advocacy group involvement | Family is often still involved in care—especially in severe cases Behavior issues may arise, putting additional stress on caregivers |
Adulthood | Patient manages care but caregivers may stay involved for emergencies | Challenges with guardianship and consent after age 18 Patients still reliant on help from family with navigating medical care and health issues |