Pediatric Nephrology

, Volume 34, Issue 3, pp 517–527 | Cite as

A pediatric neurologic assessment score may drive the eculizumab-based treatment of Escherichia coli-related hemolytic uremic syndrome with neurological involvement

  • Paolo Giordano
  • Giuseppe Stefano Netti
  • Luisa Santangelo
  • Giuseppe Castellano
  • Vincenza Carbone
  • Diletta Domenica Torres
  • Marida Martino
  • Michela Sesta
  • Franca Di Cuonzo
  • Maria Chiara Resta
  • Alberto Gaeta
  • Leonardo Milella
  • Maria Chironna
  • Cinzia Germinario
  • Gaia Scavia
  • Loreto Gesualdo
  • Mario GiordanoEmail author
Original Article



Thrombotic microangiopathy (TMA) is a clinical syndrome encompassing a large group of rare but severe disorders including thrombotic thrombocytopenic purpura (TTP) and both typical and atypical forms of hemolytic uremic syndrome (HUS). The key role of the complement system is well known in TTP and atypical HUS, but recent reports describe its involvement in the pathogenesis of HUS secondary to gastrointestinal infections due to Shiga toxin-producing Escherichia coli (STEC).


TMA mainly affects the kidney, but extra-renal complications are frequently described. The involvement of the central nervous system (CNS) represents often a life-threatening condition and it can result in serious long-term disability in HUS patients who overcome the acute phase of illness. In the present study, we retrospectively analyzed a pediatric cohort of a single tertiary pediatric hospital in Southern Italy, in which this complication occurred in 12/54 children (22% of cases), of whom five with severe neurological involvement had been successfully treated with eculizumab.


The great clinical variability of brain injury in our cohort has led us to retrospectively build a “neurological score” useful to assess the clinical severity of neurologic involvement. Subjects with higher neurologic score due to the most severe CNS involvement resulted in the group of patients early treated with eculizumab, obtaining a good clinical response (four out five patients). In conclusion, the early treatment with eculizumab in children with severe neurological involvement during STEC-HUS was associated with complete regression of both acute kidney injury (AKI) and neurological lesions observed at magnetic resonance imaging (MRI).


A “neurological score” may be a useful tool to drive the early treatment of CNS complications in STEC-HUS with eculizumab, although future perspective controlled studies are urgently needed to validate this therapeutic approach.


Hemolytic uremic syndrome Neurological involvement Eculizumab Magnetic resonance imaging 


Compliance with ethical standards

Informed consent

We carried out a retrospective study based on registry data and no ethics approval was required. Nevertheless, all the children’s parents signed a written informed consent to collect their clinical data at time of hospital access.

Conflict of interest

The authors declare that they have no conflict of interest.

Supplementary material

467_2018_4112_MOESM1_ESM.docx (25 kb)
ESM 1 (DOCX 24 kb)


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Copyright information

© IPNA 2018

Authors and Affiliations

  • Paolo Giordano
    • 1
  • Giuseppe Stefano Netti
    • 2
  • Luisa Santangelo
    • 1
  • Giuseppe Castellano
    • 3
  • Vincenza Carbone
    • 1
  • Diletta Domenica Torres
    • 1
  • Marida Martino
    • 1
  • Michela Sesta
    • 4
  • Franca Di Cuonzo
    • 5
  • Maria Chiara Resta
    • 5
  • Alberto Gaeta
    • 6
  • Leonardo Milella
    • 7
  • Maria Chironna
    • 8
  • Cinzia Germinario
    • 8
  • Gaia Scavia
    • 9
  • Loreto Gesualdo
    • 3
  • Mario Giordano
    • 1
    Email author
  1. 1.Pediatric Nephrology and Dialysis UnitPediatric Hospital “Giovanni XXIII”BariItaly
  2. 2.Clinical Pathology Unit and Center for Molecular Medicine, Department of Medical and Surgical SciencesUniversity of FoggiaFoggiaItaly
  3. 3.Nephrology, Dialysis and Transplantation Unit, Department of Emergency and Organ TransplantationUniversity of Bari “Aldo Moro”BariItaly
  4. 4.Pediatric Neurology UnitPediatric Hospital “Giovanni XXIII”BariItaly
  5. 5.Department of NeuroradiologyUniversity of Bari “Aldo Moro”BariItaly
  6. 6.Pediatric Radiology UnitPediatric Hospital “Giovanni XXIII”BariItaly
  7. 7.Intensive Care UnitPediatric Hospital “Giovanni XXIII”BariItaly
  8. 8.Department of Biomedical Science and Human OncologyUniversity of Bari “Aldo Moro”BariItaly
  9. 9.Food Safety, Nutrition and Veterinary Public Health DepartmentNational Institute of HealthRomeItaly

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