The BELS questionnaire: A novel screening tool for neurodevelopmental and psychiatric symptoms in pediatric dystrophinopathy.

Introduction/aims: Pediatric patients with dystrophinopathies [Becker and Duchenne muscular dystrophy (BDMD)] are more likely to have neurodevelopmental and neuropsychiatric conditions. This prospective pilot study tested a novel screening questionnaire developed to identify the common behavioral (B), emotional (E), learning (L), and social (S) difficulties in BDMD.

Methods: A total of 45 caregivers of BDMD patients (ages 4-19 years) seen at the Arkansas Children's Hospital Dystrophinopathy Clinic completed the BELS questionnaire during standard clinic visits. To establish external validity, caregivers also completed four well-validated standardized questionnaires assessing overlapping symptoms [Pediatric Symptom Checklist-17 (PSC-17), Colorado Learning Difficulties Questionnaire (CLDQ), the obsessive compulsive disorder (OCD) subscale from the Revised Children's Anxiety and Depression Scale (RCADS), and Barkley Deficits in Executive Functioning Scale (BDEFS)].

Results: Caregivers reported high rates of behavioral (>60%), emotional (50%-70%), learning (50%-70%), and social (30%-50%) concerns, even though relatively few patients had clinical diagnoses related to these symptoms. Passive suicidality (i.e., thoughts of death) were also common (22.2%; 10/45 patients). The BELS questionnaire was moderately to highly correlated with total scores on the four well-validated questionnaires (p < .001), and BELS subscales showed specificity. A BELS score of 21 corresponded with the established PSC-17 cutoff.

Discussion: The BELS questionnaire shows promise as a screening questionnaire to identify neurobehavioral and neuropsychiatric symptoms common in dystrophinopathy. While BELS cannot currently be used as a standalone measure to make clinical diagnoses, identification of symptoms that warrant further follow-up may improve clinical care and quality of life.