Successful treatment of neonatal atypical hemolytic uremic syndrome with C5 monoclonal antibody

• Published in: Archives de pédiatrie : organe officiel de la Société française de pédiatrie 2016-03, Vol.23 (3), p.283-286
• Main Authors: Anastaze Stelle, K., Gonzalez, E., Wilhelm-Bals, A., Michelet, P.-R., Korff, C.M., Parvex, P.
• Format: Article
• Language: English
• Subjects: Antibodies, Monoclonal, Humanized - therapeutic use; Atypical Hemolytic Uremic Syndrome - drug therapy; Female; Humans; Infant, Newborn; Remission Induction
• ISSN: 0929-693X; 1769-664X
• DOI: 10.1016/j.arcped.2015.11.020

Hemolytic uremic syndrome (HUS) is rare in neonates. We report the case of atypical HUS (aHUS) revealed by neonatal seizures. This 18-day-old baby presented with repeated clonus of the left arm and eye deviation. Four days earlier, she had suffered from gastroenteritis (non-bloody diarrhea and vomiting without fever). Her work-up revealed hemolytic anemia (120g/L), thrombocytopenia (78g/L), and impaired renal function (serum creatinine=102μmol/L) compatible with the diagnosis of HUS. Levels of C3 and C4 in the serum were normal. Shiga-toxin in the stools as well as the IgM and IgG against Escherichia coli O157 were negative. ADAMTS 13 deficiency, inborn error of the cobalamin pathway, deficiency in the H and I protein, and factor H antibodies were excluded and we concluded in aHUS. Genetic screening of the alternative complement pathway was normal. Cerebral magnetic resonance imaging performed after 24h and 1week showed restricted diffusion areas with periventricular white matter ischemic-hemorrhagic lesions. Extensive infectious work-up was negative. Upon admission the baby received antiepileptic drugs and 2days later C5 monoclonal antibody (eculizumab) and two transfusions of packed erythrocytes because the hemoglobin value had dropped to 55g/L. The platelet value was minimal at 30g/L. Renal function normalized in 48h without dialysis and neurological examination was normal in 1week. She was discharged from the hospital at day 10 with eculizumab perfusions (300mg) planned every 3weeks. After 24months, she was relapse-free and seizure-free, with a normal neurological examination. Le syndrome hémolytique et urémique (SHU) est rare en période néonatale. Nous rapportons le cas d’un nouveau-né de 18jours ayant présenté un SHU atypique (aSHU) diagnostiqué dans un contexte de convulsions néonatales. Ce nouveau-né de 18jours, en bonne santé, a été hospitalisé à la suite d’épisodes répétés de clonies du bras gauche associées à une déviation du regard de l’œil droit vers la gauche. Quatre jours avant, elle avait présenté une gastro-entérite avec diarrhée non sanglante et vomissements. Le bilan a mis en évidence une anémie hémolytique, une thrombopénie et une insuffisance rénale compatibles avec un SHU. Les taux de compléments C3 et C4 étaient normaux. La recherche de shiga-toxines s’est avérée négative, excluant le diagnostic d’un SHU typique. L’absence de déficit en ADAMTS 13 et d’une anomalie du métabolisme des cobalamines a fait poser le diagnostic de SHU atypi