Abstract

Objective To perform a systematic literature review to summarise the evidence, provide recommendations for practice, and suggest future research regarding the use of intravenous immunoglobulins (IVIGs) in the treatment of paediatric neurological and neurodevelopmental disorders. Methods A MEDLINE search was performed for IVIG treatment in paediatric neuroinflammatory, neurodevelopmental and neurodegenerative conditions between September 2003–2013. Results From the 221 citations identified, 30 studies were included (3 randomised control trials, 3 cohort studies and 24 case series). Due to the heterogeneous outcome measures, meta-analysis was not possible within individual neurological disorders, and therefore findings were combined and evidence graded. Level 1: None Level 2: Guillain-Barre Syndrome: Reduces time to recovery (n=117), but not maximal disability (n=76), and is less effective than plasma exchange at reducing ventilation duration (n=41). Acute Encephalitis Syndrome with Myocarditis: Improves mortality and cardiac function (n=83). Rasmussen's: As effective as tacrolimus in preventing disease progression and treatment-resistant seizures (n=16). Level 3: None Level 4: NMDAR-Ab encephalitis: Good outcome at 2 years in conjunction with additional immunotherapy (n=298). Chronic Inflammatory Demyelinating Polyradiculoneuropathy: As effective as corticosteroids at improving neurological function (n=74). Acute Disseminated Encephalomyelitis: As effective as corticosteroids at inducing complete recovery (n=44). Narcolepsy: Reduces cataplexy symptoms (n=8). Tourette's Syndrome with caudate nucleus antibodies: Temporary reduction in tics (n=7). Limbic encephalitis, Landau-Kleffner Syndrome, Refractory Seizure Disorders: No improvement in outcome (n=4, n=10, n=12). Early IVIG therapy may improve efficacy in Rasmussen's, NMDAR-Ab encephalitis and Narcolepsy. No evidence available: Autism, Neurodegeneration. Conclusion In the last 10 years, there is growing evidence for the efficacy of IVIG in a handful of neurological conditions. Nevertheless, well designed, prospective, multi-centre studies with standardized outcome measures are now required to evaluate the efficacy and cost effectiveness of this expensive and resource limited therapeutic agent.

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