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Severe, persistent neurotoxicity after transplant‐associated thrombotic microangiopathy in a pediatric patient despite treatment with eculizumab
Author(s) -
Schoettler Michelle,
Duncan Christine,
Lehmann Leslie
Publication year - 2019
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.13381
Subject(s) - medicine , eculizumab , thrombotic microangiopathy , sequela , posterior reversible encephalopathy syndrome , pediatrics , kidney disease , surgery , disease , complement system , magnetic resonance imaging , antibody , immunology , radiology
Abstract Background TA‐TMA is a described complication of aHCT in children with neuroblastoma. Outcomes are poor with mortality rates approaching 60%. Described late effects in survivors include chronic kidney disease and persistent pulmonary hypertension. Case We report a case of a 2‐year‐old with neuroblastoma who developed severe TA‐TMA 35 days after high dose chemotherapy and autologous stem cell rescue. He presented with respiratory failure, pericardial and pleural effusions, hemolysis, hypertension, and mild altered mental status. He was mechanically ventilated for 3 weeks and after sedation was lifted, he was minimally responsive. He was treated with eculizumab with resolution of hemolysis, kidney injury and polyserositis. Initially he was more responsive; however, after almost a year of intensive therapy he remained nonverbal and had persistent irritability and behavioral changes. He had an extensive negative evaluation. On day +345, he presented with severe, refractory epilepsy. Three years after TA‐TMA, he continues to have severe neurologic disabilities. Conclusions To our knowledge, persistent neurologic toxicity has not been reported in TA‐TMA. However, deficits and seizures are reported in other TMAs, particularly in children with atypical HUS who present with significant neurologic changes at diagnosis. Our patient's persistent neurologic disability despite eculizumab response in all other involved organs may reflect irreversible damage. This case describes a new long‐term sequela of TA‐TMA and highlights the need for further studies to understand both acute and long‐term neurologic complications of this disease.