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Long-term follow-up of children with chronic relapsing polyneuropathy
Nina Barisić1, Stefano Regis, Leo Pazanin
1Department of Pediatrics, University Medical School Zagreb, Kispatićeva 12, 10000, Croatia.
Insights
This study tracks three children with early-onset chronic inflammatory demyelinating polyneuropathy. Excellent outcomes were observed despite varied clinical courses, with one patient showing low arylsulfatase A activity.
Area of Science:
- Pediatric Neurology
- Neuromuscular Disorders
- Genetic Metabolic Disorders
Background:
- Chronic inflammatory demyelinating polyneuropathy (CIDP) can present in early childhood.
- Understanding long-term outcomes and potential contributing factors is crucial for pediatric neurological care.
Observation:
- Follow-up of three children with early-onset CIDP revealed diverse clinical presentations and relapse patterns.
- One patient exhibited decreased arylsulfatase A activity, a finding also noted in family members.
- Hypertension and cardiac involvement were observed in two patients during the initial phase.
Findings:
- All three patients achieved excellent clinical outcomes.
- Clinical course, therapeutic response, and electrophysiologic findings varied significantly, particularly in the patient with low arylsulfatase A activity.
- The presence of low arylsulfatase A activity may influence the disease's trajectory in pediatric CIDP.
Implications:
- Early-onset CIDP can have variable courses, even with genetic factors like reduced arylsulfatase A activity.
- Further research is needed to elucidate the specific role of arylsulfatase A in pediatric CIDP.
- Comprehensive long-term monitoring is essential for managing pediatric neuromuscular inflammatory conditions.
Abstract:
Long-term follow-up of three children with early-onset chronic inflammatory demyelinating polyneuropathy is presented. A 3-year-old male (Patient 1) manifested initially progressive muscle weakness during 6 months with spontaneous regression, followed by two severe relapses at 5 and 6 years of age. Decreased arylsulfatase A activity was present in Patient 1 (17.6) and his family members (24.1-40 nmol/mg/hour). Arterial hypertension up to 20/12 kPa was present in two patients in the initial phase associated with muscle stiffness, occasional meningism, and left ventricular hypertrophy in one of them (Patient 3). Subsequently, they both developed two mild relapses at 3.5 and 6 years of age. Clinical outcome was excellent in all three cases, although clinical course, therapy response, and electrophysiologic outcome was quite different in the only patient with low arylsulfatase A activity. The significance of this difference is discussed.