Treatment of severe acute necrotizing encephalopathy of childhood with interleukin-6 receptor blockade in the first 24 h as add-on immunotherapy shows favorable long-term outcome at 2 years.

Acute necrotizing encephalopathy of childhood Encephalitis Immunotherapy Interleukin 6 Long-term outcome Neurorehabilitation Tocilizumab

Journal

Brain & development
ISSN: 1872-7131
Titre abrégé: Brain Dev
Pays: Netherlands
ID NLM: 7909235

Informations de publication

Date de publication:
Aug 2023
Historique:
received: 12 10 2022
revised: 28 01 2023
accepted: 06 03 2023
medline: 15 6 2023
pubmed: 27 3 2023
entrez: 26 3 2023
Statut: ppublish

Résumé

Acute necrotizing encephalopathy (ANE) of childhood is a rare and devastating infection-associated acute encephalopathy. While there are no consensus treatments for ANE, recent case reports suggest a beneficial role for the use of tocilizumab, a recombinant humanized monoclonal antibody against the interleukin-6 (IL-6) receptor. The correlation of the timing of add-on tocilizumab in relation to long-term outcome has not been reported. We report on the timing of administration of tocilizumab in two patients classified as high-risk using the ANE severity score (ANE-SS) with respect to the long-term outcome at 2 years. Case 1 was a 19-month-old previously well boy who presented to a tertiary children's hospital with seizures, evolving status dystonicus and shock. Case 2 was a three-year-old boy who presented to a peripheral hospital with fever, sepsis and encephalopathy. The patients were transferred to the tertiary intensive care unit and MRI confirmed ANE with extensive brainstem involvement. Case 1 received intravenous immunoglobulin (IVIg), methylprednisolone and tocilizumab at 21, 39 and 53 h respectively. His modified Rankin scale (mRS) at discharge and two years was unchanged at 5. The functional independence measure - for children (WeeFIM) at two years was very low (19/126). Case 2 received dexamethasone at 1 h, methylprednisolone at 21 h and IVIg and tocilizumab at 22 h. The mRS at discharge and two years was 4 and 3 respectively. The WeeFIM score at two years showed substantial improvement (96/126). The very early use of interleukin-6 blockade as 'add-on' immunotherapy in the first 24 h demonstrates potential for improving the long-term outcome in patients classified as high-risk using the ANE-SS.

Sections du résumé

BACKGROUND BACKGROUND
Acute necrotizing encephalopathy (ANE) of childhood is a rare and devastating infection-associated acute encephalopathy. While there are no consensus treatments for ANE, recent case reports suggest a beneficial role for the use of tocilizumab, a recombinant humanized monoclonal antibody against the interleukin-6 (IL-6) receptor. The correlation of the timing of add-on tocilizumab in relation to long-term outcome has not been reported.
METHODS METHODS
We report on the timing of administration of tocilizumab in two patients classified as high-risk using the ANE severity score (ANE-SS) with respect to the long-term outcome at 2 years.
RESULTS RESULTS
Case 1 was a 19-month-old previously well boy who presented to a tertiary children's hospital with seizures, evolving status dystonicus and shock. Case 2 was a three-year-old boy who presented to a peripheral hospital with fever, sepsis and encephalopathy. The patients were transferred to the tertiary intensive care unit and MRI confirmed ANE with extensive brainstem involvement. Case 1 received intravenous immunoglobulin (IVIg), methylprednisolone and tocilizumab at 21, 39 and 53 h respectively. His modified Rankin scale (mRS) at discharge and two years was unchanged at 5. The functional independence measure - for children (WeeFIM) at two years was very low (19/126). Case 2 received dexamethasone at 1 h, methylprednisolone at 21 h and IVIg and tocilizumab at 22 h. The mRS at discharge and two years was 4 and 3 respectively. The WeeFIM score at two years showed substantial improvement (96/126).
CONCLUSION CONCLUSIONS
The very early use of interleukin-6 blockade as 'add-on' immunotherapy in the first 24 h demonstrates potential for improving the long-term outcome in patients classified as high-risk using the ANE-SS.

Identifiants

pubmed: 36967317
pii: S0387-7604(23)00045-1
doi: 10.1016/j.braindev.2023.03.002
pii:
doi:

Substances chimiques

Interleukin-6 0
Immunoglobulins, Intravenous 0
Methylprednisolone X4W7ZR7023
Receptors, Interleukin-6 0

Types de publication

Case Reports

Langues

eng

Sous-ensembles de citation

IM

Pagination

401-407

Informations de copyright

Copyright © 2023 The Japanese Society of Child Neurology. Published by Elsevier B.V. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of Competing Interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: [Dr Michelle Farrar is the recipient of a National Health and Medical Research Council of Australia Investigator grant (APP1194940), but we have no conflicts of interest to disclose.]

Auteurs

Patrick H Hosie (PH)

Department of Neurology, Sydney Children's Hospital, Randwick, Australia.

Carylyn Lim (C)

Rehab2Kids, Sydney Children's Hospital, Randwick, Australia.

Timothy R D Scott (TRD)

Rehab2Kids, Sydney Children's Hospital, Randwick, Australia; Discipline of Pediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Australia; Graduate School of Biomedical Engineering, UNSW Sydney, Australia.

Michael Cardamone (M)

Department of Neurology, Sydney Children's Hospital, Randwick, Australia; Discipline of Pediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Australia.

Michelle A Farrar (MA)

Department of Neurology, Sydney Children's Hospital, Randwick, Australia; Discipline of Pediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Australia.

Catherine Frith (C)

Department of Immunology and Infectious Diseases, Sydney Children's Hospital, Randwick, Australia.

Peter I Andrews (PI)

Department of Neurology, Sydney Children's Hospital, Randwick, Australia.

Jason Pinner (J)

Centre for Clinical Genetics, Sydney Children's Hospital, Randwick, Australia.

Sekhar Pillai (S)

Department of Neurology, Sydney Children's Hospital, Randwick, Australia; Discipline of Pediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Australia. Electronic address: sekhar.pillai@health.nsw.gov.au.

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Classifications MeSH