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A prospective 10 year follow up study of patients with neurofibromatosis type 1
M H Cnossen1, A de Goede-Bolder, K M van den Broek
1Department of Paediatrics, University Hospital Sophia/Dijkzigt, Rotterdam, Netherlands. cnossen@alkg.azr.nl
Insights
This study found that 41.3% of children with neurofibromatosis type 1 (NF1) developed complications, with behavioral issues linked to their presence. Regular check-ups are recommended for all children with NF1.
Area of Science:
- Pediatric Neurology
- Genetics
- Clinical Medicine
Background:
- Neurofibromatosis type 1 (NF1) is a genetic disorder with varied clinical manifestations.
- Understanding the prevalence and incidence of NF1 complications in children is crucial for timely intervention.
Purpose of the Study:
- To determine the prevalence and incidence of symptoms and complications in children with NF1.
- To identify potential risk factors associated with the development of NF1 complications.
Main Methods:
- A 10-year prospective, multidisciplinary follow-up study.
- Inclusion of 150 children diagnosed with NF1 based on NIH criteria.
- Data collection on symptoms, complications, and follow-up duration.
Main Results:
- Complications were present in 41.3% (62/150) of children.
- The incidence of complications was 2.4 per 100 person-years in children initially without complications.
- Behavioral problems were associated with the presence of complications.
Conclusions:
- This study represents the largest single-center case series of NF1-affected children followed to age 18.
- Regular clinical examinations are essential for all children with NF1, irrespective of initial presentation.
- Early identification and management of complications can improve outcomes for children with NF1.
Objective:
To establish the prevalence and incidence of symptoms and complications in children with neurofibromatosis type 1 (NF1) and to assess possible risk factors for the development of complications.
Design:
A 10 year prospective multidisciplinary follow up study.
Patients:
One hundred and fifty children diagnosed with NF1 according to criteria set by the National Institutes of Health.
Results:
In 62 of 150 children (41.3%) complications were present, including 42 (28.0%) children with one complication, 18 (12.0%) with two complications, and two (1.3%) with three complications (mean (SD) duration of follow up 4.9 (3.8) years). Ninety five of the 150 children presented without complications (follow up, 340.8 person-years). The incidence of complications was 2.4/100 person-years in this group. An association was found between behavioural problems and the presence of complications.
Conclusion:
This is the largest single centre case series of NF1 affected children followed until 18 years of age. Children with NF1, including those initially presenting without complications, should have regular clinical examinations.