Factors associated with outcomes of severe acute necrotizing encephalopathy: A multicentre experience in Malaysia

• Published in: Developmental Medicine and Child Neurology
• Main Author: Lee V.W.M.; Khoo T.B.; Teh C.M.; Heng H.S.; Li L.; Yusof Y.L.M.; Yahaya N.A.; Dharshini S.; Wong S.W.; Nickson T.; Mohamed A.R.; Fong C.Y.; Tan K.A.; Murugesu S.
• Format: Article
• Language: English
• Published: John Wiley and Sons Inc 2023
• Online Access: https://www.scopus.com/inward/record.uri?eid=2-s2.0-85147450139&doi=10.1111%2fdmcn.15536&partnerID=40&md5=71aa0299adcf2cf78c50569519910a9f

This case series compared clinical variables and various combinations of immunotherapy received with outcomes of patients with severe acute necrotizing encephalopathy (ANE). We performed a retrospective review of clinical variables, immunotherapy received, and outcomes (based on the modified Rankin Scale) in Malaysia between February 2019 and January 2020. Twenty-seven children (12 male), aged 7 months to 14 years (mean 4 years) at diagnosis were included. Of these, 23 had an ANE severity score of 5 to 9 out of 9 (high risk). Eleven patients received tocilizumab (four in combination with methylprednisolone [MTP], seven with MTP + intravenous immunoglobulin [IVIG]) and 16 did not (two received MTP alone, 14 received MTP + IVIG). Nine died. Among the survivors, six had good outcomes (modified Rankin Score 0–2) at 6 months follow-up. All patients who received tocilizumab in combination with MTP + IVIG survived. Twenty children received first immunotherapy within 48 hours of admission. No significant association was found between the timing of first immunotherapy with outcomes. Those with brainstem dysfunction (p = 0.016) were observed to have poorer outcomes. This study showed a trend towards better survival when those with severe ANE were treated with tocilizumab in combination with MTP + IVIG. However, larger studies will be needed to determine the effect of this regime on the long-term outcomes. © 2023 Mac Keith Press.