Assessment of psychosocial adjustment and reduced initiative in children with myotonic dystrophy type 1: a pilot study on the reliability and clinical utility of a short parent-report questionnaire.

Researchers tested a questionnaire to measure social and emotional adjustment in children with a muscle condition called myotonic dystrophy type 1. They found the questionnaire works well and shows that these children often struggle with social activities and taking initiative (starting things on their own). The initiative problems seem to be unique to this condition and aren't explained by other symptoms like ADHD or autism traits.

This pilot study evaluated the reliability and clinical utility of the Personal Adjustment and Role Skills Scale III (PARS-III) questionnaire for assessing psychosocial adjustment in 36 children with myotonic dystrophy type 1 (DM1). The PARS-III demonstrated adequate internal consistency and showed correlations with parent-reported autism spectrum disorder symptoms, ADHD symptoms, and physical disease burden. Parents reported the most significant problems in reduced social activity and reduced initiative, with initiative deficits being particularly pronounced compared to children with Duchenne muscular dystrophy. Notably, reduced initiative was not associated with ADHD, autism symptoms, or physical disease burden, suggesting it may be a distinct brain-related comorbidity in pediatric DM1.

The PARS-III questionnaire may be useful for screening psychosocial adjustment in pediatric DM1, particularly for identifying initiative deficits. Reduced initiative appears to be a distinct feature requiring specific clinical attention and further research to develop targeted interventions.

Small pilot study with only 36 participants from a single registry. Limited comparison group (only Duchenne muscular dystrophy). Cross-sectional design prevents causal inferences. Relies solely on parent-report measures without direct child assessment or multi-informant validation.

We describe the reliability and clinical utility of the personal adjustment and role skills scale III (PARS-III) for screening of psychosocial adjustment in children with myotonic dystrophy type 1 (DM1). Data of 36 pediatric DM1 patients were included from the Dutch MYODRAFT patient registry. Reliability was assessed using Cronbach's alpha. Associations between PARS-III scores and estimates of brain-related comorbidity and parent-reported physical disease burden were explored as possible factors affecting psychosocial adjustment.

PARS-III data of children with DM1 in this study were compared to PARS-III data from children with Duchenne muscular dystrophy in order to describe specificity to the pediatric DM1 population. The PARS-III showed adequate internal consistency. PARS-III total scores correlated with parent-reported symptoms of autism spectrum disorder and attention deficit/hyperactivity disorder and parent-reported physical disease burden. No statistically significant associations were found with intelligence.

Parents reported most problems in reduced social activity and reduced initiative. Reduced initiative was not associated with attention deficit/hyperactivity disorder, autism spectrum disorder or reported physical disease burden. Parents of children with DM1 reported more problems in initiative compared to parents of children with Duchenne muscular dystrophy. We conclude that the PARS-III is a reliable instrument for screening psychosocial adjustment in general and deficits in initiative in particular.

More research is needed on this clinically relevant symptom as a possible brain-related comorbidity of children with DM1.