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

• Published in: Developmental medicine and child neurology Vol. 65; no. 9; pp. 1256 - 1263
• Main Authors: Lee, Vanessa Wan Mun, Khoo, Teik Beng, Teh, Chee Ming, Heng, Hock Sin, Li, Limin, Yusof, Yusma Lyana Md, Yahaya, Nor Azni, Dharshini, Sangita, Wong, Sau Wei, Nickson, Tai, Mohamed, Ahmad Rithauddin, Fong, Choong Yi, Tan, Khian Aun, Murugesu, Sumitha
• Format: Journal Article
• Language: English
• Published: England 01-09-2023
• Subjects: Brain Diseases; Child; Humans; Immunoglobulins, Intravenous - therapeutic use; Leukoencephalitis, Acute Hemorrhagic - therapy; Malaysia; Male; Methylprednisolone; Retrospective Studies; Malaysia
• ISSN: 0012-1622; 1469-8749
• DOI: 10.1111/dmcn.15536

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.
Acute necrotizing encephalopathy in childhood (ANEC) is a rare clinico‐radiological syndrome with high mortality. Whilst there is no clear consensus for treatment, key therapeutic strategy is the early initiation of first‐line immunotherapy. More recently, early tocilizumab usage appear to have resulted in improved survival and recovery in a handful of severe cases. This case series compared clinical variables and various combinations of immunotherapy received with outcomes of patients with severe ANEC.
This original article is commented on by Khamis et al. on pages 1139–1140 of this issue.