PDF herunterladen
CC BY-NC-ND 4.0 · AJP Rep 2021; 11(02): e95-e98
DOI: 10.1055/s-0041-1731057
Case Report

Neonatal Atypical Hemolytic Uremic Syndrome in the Eculizumab Era

Sara Madureira Gomes
1   Department of Pediatrics, Centro Hospitalar Universitário de São João, Porto, Portugal
,
Rita Pissarra Teixeira
1   Department of Pediatrics, Centro Hospitalar Universitário de São João, Porto, Portugal
,
Gustavo Rocha
2   Department of Neonatology, Centro Hospitalar Universitário de São João, Porto, Portugal
,
Paulo Soares
2   Department of Neonatology, Centro Hospitalar Universitário de São João, Porto, Portugal
,
Hercilia Guimaraes
3   Department of Pediatrics, Faculty of Medicine, University of Porto, Porto, Portugal
,
Paulo Santos
4   Department of Pediatric Cardiology, Centro Hospitalar Universitário de São João, Porto, Portugal
,
Joana Jardim
5   Pediatric Nephrology Unit, Department of Pediatrics, Centro Hospitalar Universitário de São João, Porto, Portugal
,
João Luís Barreira
5   Pediatric Nephrology Unit, Department of Pediatrics, Centro Hospitalar Universitário de São João, Porto, Portugal
,
Helena Pinto
5   Pediatric Nephrology Unit, Department of Pediatrics, Centro Hospitalar Universitário de São João, Porto, Portugal
› Institutsangaben
› Weitere Informationen
Auch verfügbar auf
   Lizenzen und Reprints

Abstract

The atypical hemolytic uremic syndrome (aHUS) in the newborn is a rare disease, with high morbidity. Eculizumab, considered a first-line drug in older children, is not approved in neonates and in children weighing less than 5 kg. We present a 5-day-old female newborn, born at 36 weeks' twin gestation, by emergency cesarean section due to cord prolapse, with birth weight of 2,035 g and Apgar score of 7/7/7, who develops microangiopathic hemolytic anemia, thrombocytopenia, and progressive acute renal failure. In day 5, after diagnosis of aHUS, a daily infusion of fresh frozen plasma begins, with improvement of thrombocytopenia and very slight improvement in renal function. The etiologic study (congenital infection, Shiga toxin, ADAMTS13 activity, directed metabolic study) was normal. C3c was slightly decreased. On day 16 for maintenance of anemia and severe renal failure, she started 300 mg/dose eculizumab. Anemia resolves in 10 weeks and creatinine has normal values after 13 weeks of treatment. The genetic study was normal. In this case, eculizumab is effective in controlling microangiopathy and in the recovery of renal function. Diagnosis of neonatal aHUS can be challenging because of phenotypic heterogeneity and potential overlap with other manifestations that may confound it, such as perinatal asphyxia or sepsis/disseminated intravascular coagulation.

Keywords

atypical hemolytic uremic syndrome - thrombotic microangiopathy - neonate - eculizumab - perinatal asphyxia


Publikationsverlauf

Eingereicht: 15. Februar 2021

Angenommen: 25. März 2021

Artikel online veröffentlicht:
23. Juni 2021

© 2021. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/)

Thieme Medical Publishers, Inc.
333 Seventh Avenue, 18th Floor, New York, NY 10001, USA

 

  • References

  • 1 Walsh PR, Johnson S. Treatment and management of children with haemolytic uraemic syndrome. Arch Dis Child 2018; 103 (03) 285-291
    CrossrefPubMedSuche in Google Scholar
  • 2 Azevedo A, Faria B, Teixeira C. et al. Portuguese consensus document statement in diagnostic and management of atypical hemolytic uremic syndrome. Port J Nephrol Hypert 2018; 32 (03) 1-22
    PubMedSuche in Google Scholar
  • 3 Dixon BP, Gruppo RA. Atypical hemolytic uremic syndrome. Pediatr Clin North Am 2018; 65 (03) 509-525
    CrossrefPubMedSuche in Google Scholar
  • 4 Anastaze Stelle K, Gonzalez E, Wilhelm-Bals A, Michelet PR, Korff CM, Parvex P. Efficacité du traitement d'un syndrome hémolytique et urémique atypique néonatal par anticorps monoclonal C5. Arch Pediatr 2016; 23 (03) 283-286
    CrossrefPubMedSuche in Google Scholar
  • 5 Çakar N, Ozcakar ZB, Ozaltin F. et al. Atypical hemolytic uremic syndrome in children aged <2 years. Nephron 2018; 139 (03) 211-218
    CrossrefPubMedSuche in Google Scholar
  • 6 Greenbaum LA, Fila M, Ardissino G. et al. Eculizumab is a safe and effective treatment in pediatric patients with atypical hemolytic uremic syndrome. Kidney Int 2016; 89 (03) 701-711
    CrossrefPubMedSuche in Google Scholar
  • 7 Loirat C, Fakhouri F, Ariceta G. et al; HUS International. An international consensus approach to the management of atypical hemolytic uremic syndrome in children. Pediatr Nephrol 2016; 31 (01) 15-39
    CrossrefPubMedSuche in Google Scholar
  • 8 Michaux K, Bacchetta J, Javouhey E, Cochat P, Frémaux-Bacchi V, Sellier-Leclerc AL. Eculizumab in neonatal hemolytic uremic syndrome with homozygous factor H deficiency. Pediatr Nephrol 2014; 29 (12) 2415-2419
    CrossrefPubMedSuche in Google Scholar
  • 9 Szarvas N, Szilágyi Á, Tasic V. et al. First-line therapy in atypical hemolytic uremic syndrome: consideration on infants with a poor prognosis. Ital J Pediatr 2014; 40: 101
    CrossrefPubMedSuche in Google Scholar
  • 10 Alonso-Triana YM, Perrino MJ. Eculizumab for atypical hemolytic uremic syndrome in newborn: a case report [in Spanish]. Farm Hosp 2015; 39 (04) 217-218
    PubMedSuche in Google Scholar
  • 11 Ariceta G, Arrizabalaga B, Aguirre M, Morteruel E, Lopez-Trascasa M. Eculizumab in the treatment of atypical hemolytic uremic syndrome in infants. Am J Kidney Dis 2012; 59 (05) 707-710
    CrossrefPubMedSuche in Google Scholar
  • 12 Biran V, Fau S, Jamal T. et al. Perinatal asphyxia may present with features of neonatal atypical hemolytic uremic syndrome. Pediatr Nephrol 2007; 22 (12) 2129-2132
    CrossrefPubMedSuche in Google Scholar
  • 13 Fallahpour M, Hafizi A, Fouladgar A, Rajabian B. Neonatal atypical hemolytic uremic syndrome may cause prenatal asphyxia. Arch Iran Med 2012; 15 (11) 729-730
    PubMedSuche in Google Scholar
  • 14 Sharma S, Pradhan M, Meyers KEC, Le Palma K, Laskin BL. Neonatal atypical hemolytic uremic syndrome from a factor H mutation treated with eculizumab. Clin Nephrol 2015; 84 (03) 181-185
    CrossrefPubMedSuche in Google Scholar
  • 15 Besbas N, Gulhan B, Karpman D. et al. Neonatal onset atypical hemolytic uremic syndrome successfully treated with eculizumab. Pediatr Nephrol 2013; 28 (01) 155-158
    CrossrefPubMedSuche in Google Scholar
  • 16 Ito S, Hidaka Y, Inoue N. et al. Safety and effectiveness of eculizumab for pediatric patients with atypical hemolytic-uremic syndrome in Japan: interim analysis of post-marketing surveillance. Clin Exp Nephrol 2019; 23 (01) 112-121
    CrossrefPubMedSuche in Google Scholar
  • 17 Lebel E, Trahtemberg U, Block C, Zelig O, Elinav H. Post-eculizumab meningococcaemia in vaccinated patients. Clin Microbiol Infect 2018; 24 (01) 89-90
    CrossrefPubMedSuche in Google Scholar
  • 18 Tiwari L, Vashissht N, Kaur P, Gupta N, Saikia B, Puliyel J. 568 Neonatal hemolytic uremic syndrome. Arch Dis Child 2012; 97 (Suppl. 02) A1-A539
    CrossrefPubMedSuche in Google Scholar
  • 19 Wijnsma KL, Duineveld C, Wetzels JFM, van de Kar NCAJ. Eculizumab in atypical hemolytic uremic syndrome: strategies toward restrictive use. Pediatr Nephrol 2019; 34 (11) 2261-2277
    CrossrefPubMedSuche in Google Scholar
  • 20 Ariceta G. Optimal duration of treatment with eculizumab in atypical hemolytic uremic syndrome (aHUS)-a question to be addressed in a scientific way. Pediatr Nephrol 2019; 34 (05) 943-949
    CrossrefPubMedSuche in Google Scholar
  • 21 Fakhouri F, Loirat C. Anticomplement treatment in atypical and typical hemolytic uremic syndrome. Semin Hematol 2018; 55 (03) 150-158
    CrossrefPubMedSuche in Google Scholar
  • 22 Goodship TH, Cook HT, Fakhouri F. et al; Conference Participants. Atypical hemolytic uremic syndrome and C3 glomerulopathy: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference. Kidney Int 2017; 91 (03) 539-551
    CrossrefPubMedSuche in Google Scholar