title: Intravenous immunoglobulin in paediatric neurology: safety, adherence to guidelines, and long-term outcome.
abstract: OBJECTIVE
  Intravenous immunoglobulin (IVIG) is an expensive therapy used in immunodeficiency and autoimmune disorders. Increasing demands and consequent shortages result in a need for usage to conform to guidelines.
  
  
  METHODS
  We retrospectively evaluated IVIG use for neuroimmunological indications and adherence to existing guidelines in a major Australian paediatric hospital between 2000 and 2014.
  
  
  RESULTS
  One-hundred and ninety-six children (96 male, 100 female; mean age at disease onset 6y 5mo [range 3mo-15y 10mo], mean age at first IVIG dose 7y 2mo [range 3mo-16y 5mo]) received IVIG for neuroimmunological indications during the study period (28.1% had Guillain-Barré syndrome), representing 15.5% of all hospital indications. In total, 1669 IVIG courses were administered (total 57 221g, median 78g/patient, range 12-5748g). The highest median numbers of courses were in chronic inflammatory demyelinating polyneuropathies, opsoclonus-myoclonus ataxia syndrome, suspected immune-mediated epilepsies, and Rasmussen's encephalitis. Adverse reactions occurred in 25.5% of patients, but these were mostly minor. Outcome at follow-up was best in anti-N-methyl-d-aspartate receptor (anti-NMDAR) encephalitis, Guillain-Barré syndrome, and myasthenia gravis, and worst in Rasmussen's encephalitis and epilepsies. The total cost of IVIG was US$2 595 907 (median $3538/patient, range $544-260 766). Of patients receiving IVIG, 45.4% to 57.1% were given the therapy for 'weak' indications or indications 'not listed' in international guidelines. Some entities commonly treated with IVIG in current practice, such as anti-NMDAR encephalitis and transverse myelitis, are not listed in most guidelines.
  
  
  CONCLUSIONS
  Our study demonstrates that IVIG is generally well tolerated but expensive, and discloses discrepancies between guidelines and clinical practice in paediatric neurology, suggesting both the need for greater adherence to current recommendations, and for recommendations to be updated to accommodate emerging indications.
authors.affiliation: Neuroimmunology Group, Institute for Neuroscience and Muscle Research, Kids Research Institute, Children's Hospital at Westmead, University of Sydney, Sydney, NSW, Australia.|Paediatric Neurology Unit, Department of Paediatrics, University of Padua, Padua, Italy.
authors.forename: Margherita
authors.initials: M
authors.lastname: Nosadini
authors.affiliation: Neuroimmunology Group, Institute for Neuroscience and Muscle Research, Kids Research Institute, Children's Hospital at Westmead, University of Sydney, Sydney, NSW, Australia.
authors.forename: Shekeeb S
authors.initials: SS
authors.lastname: Mohammad
authors.affiliation: Paediatric Neurology Unit, Department of Paediatrics, University of Padua, Padua, Italy.
authors.forename: Agnese
authors.initials: A
authors.lastname: Suppiej
authors.affiliation: Paediatric Neurology Unit, Department of Paediatrics, University of Padua, Padua, Italy.
authors.forename: Stefano
authors.initials: S
authors.lastname: Sartori
authors.affiliation: Neuroimmunology Group, Institute for Neuroscience and Muscle Research, Kids Research Institute, Children's Hospital at Westmead, University of Sydney, Sydney, NSW, Australia. russell.dale@health.nsw.gov.au.
authors.forename: Russell C
authors.initials: RC
authors.lastname: Dale
authors.affiliation: Neuroimmunology Group, Institute for Neuroscience and Muscle Research, Kids Research Institute, Children's Hospital at Westmead, University of Sydney, Sydney, NSW, Australia.
mesh_terms: D000293:Adolescent; D001315:Australia; D020274:Autoimmune Diseases of the Nervous System / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D019983:Guideline Adherence / Q000592:standards* / Q000706:statistics & numerical data; D006776:Hospitals, Pediatric / Q000706:statistics & numerical data; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000008:administration & dosage / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D007155:Immunologic Factors / Q000008:administration & dosage / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D009462:Neurology / Q000592:standards* / Q000706:statistics & numerical data; D017063:Outcome Assessment, Health Care / Q000706:statistics & numerical data; D010372:Pediatrics / Q000592:standards* / Q000706:statistics & numerical data; D012189:Retrospective Studies
chemical_list: D016756:Immunoglobulins, Intravenous; D007155:Immunologic Factors