This real-world study characterized the burden of caring for pediatric patients in the USA who had neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN), a condition with multiple comorbidities that require specialized healthcare.

Caregivers were considerably burdened as a result of providing care for their pediatric patient with NF1-PN, experiencing one-fifth of overall loss in their ability to fully attend work as required along with diminished effectiveness while working (i.e., taken together, overall loss in work productivity) and impairment in activities of daily living.

Our findings highlight that both the patient with NF1-PN and their caregiver experience a substantial burden from the disease, underscoring an unmet need for better management of NF1-PN.