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Activity and participation in children with neurofibromatosis type 1
Barbara A Johnson1, Xiaoming Sheng2, Amber S Perry1
1Shriners Hospitals for Children Salt Lake City, Salt Lake City, UT, United States.
Insights
A strengthening program improved upper extremity function in children with neurofibromatosis type 1 (NF1) short-term, but long-term participation and function did not differ significantly from controls. NF1 patients showed lower function and social participation.
Area of Science:
- Pediatric Rehabilitation
- Neurodevelopmental Disorders
- Physical Therapy
Background:
- Neurofibromatosis type 1 (NF1) affects children and youth, impacting their activity and participation.
- Limited data exists on the effectiveness of targeted interventions for improving functional outcomes in this population.
Purpose of the Study:
- To evaluate the impact of a strengthening program on activity and participation in children and youth with NF1.
- To compare outcomes between an intervention group and a control group using standardized assessments.
Main Methods:
- A randomized controlled trial involving 36 children and youth (ages 5-18) with NF1.
- Intervention group: 10-week supervised strengthening program + 9-month independent program.
- Assessments: Pediatric Outcomes Data Collection Instrument (PODCI) and Children's Assessment of Participation and Enjoyment (CAPE) at baseline, 12 weeks, and 1 year.
Main Results:
- Significant baseline differences in formal vs. informal participation (CAPE).
- At 12 weeks, intervention group showed improved upper extremity function (PODCI) and decreased happiness, while controls showed decreased function and increased happiness.
- No significant differences in CAPE or PODCI change scores from baseline to 1 year between groups.
Conclusions:
- The strengthening program yielded short-term improvements in upper extremity function but no significant long-term benefits in participation or overall function compared to controls.
- Children with NF1 exhibit lower than normative scores in upper extremity function, sport, physical function, comfort/pain, and happiness.
- Participation patterns indicate a tendency towards family-based activities at home, suggesting potential social isolation from peers and limited engagement in formal physical and skill-based activities.
Abstract:
We describe activity and participation in children and youth with neurofibromatosis type 1 (NF1), and compared an intervention and control group after a strengthening program using the Pediatric Outcomes Data Collection Instrument (PODCI) and the Children's Assessment of Participation and Enjoyment (CAPE). Questionnaires were filled out by parents at baseline, 12-weeks, and 1-year. The intervention group performed a strengthening program twice a week for ten weeks, followed by a 9-month independent program. Thirty-six participants (18 control, 18 intervention) between the ages of 5- and 18-years (mean 10.6 years, SD 4.6 years) were enrolled, and 34 completed the 1-year assessment. There were significant differences between formal and informal participation (p<0.0001) in baseline CAPE scores for the entire cohort. At 12 weeks, PODCI upper extremity function improved in intervention and decreased in controls (p=0.040), while happiness declined in intervention and increased in control (p=0.003). There were no significant differences between control and intervention groups in any of the CAPE or PODCI change scores from baseline to 1-year. Upper extremity function, sport and physical function, comfort/pain and happiness PODCI scores were lower than normative values. The NF1 cohort had low participation in formal active physical and skill-based activities. The companionship and location dimensions suggest participation occurs with family and other relatives in the home or a relative's home and reflects a pattern of social isolation from peers.
