Autori: Luisa Santangelo (1), Paolo. Giordano (1), Giuseppe Stefano Netti (2), Giuseppe Castellano (3), Vincenza Carbone (1), Diletta Torres (1), Michele Sesta (4), Pasquale Conti (4), Delio Gagliardi (4), Alberto Gaeta (5), Franca Di Cuonzo (6), Maria Chiara Resta (6), Leonardo Milella (7), Gaia Scavia (8), Loreto Gesualdo (3), Mario Giordano (1).
Affiliazioni: (1) Pediatric Nephrology and Dialysis Unit, Pediatric Hospital “Giovanni XXIII”, Bari (Italy); (2) Clinical Pathology Unit and Center for Molecular Medicine, Department of Medical and Surgical Sciences, University of Foggia, Foggia (Italy); (3) Nephrology, Dialysis and Transplantation Unit, Department of Emergency and Organ Transplantation, University of Bari “Aldo Moro”, Bari (Italy); (4) Pediatric Neurology Unit, Pediatric Hospital “Giovanni XXIII”, Bari (Italy); (5) Radiology Unit, Pediatric Hospital “Giovanni XXIII”, Bari (Italy); (6) Department of Neuroradiology, University of Bari “Aldo Moro”, Bari (Italy); (7) Intensive Care Unit, Pediatric Hospital “Giovanni XXIII”, Bari (Italy); (8) Food Safety, Nutrition and Veterinary Public Health Department, National Institute of Health, Rome (Italy).
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 analyze a pediatric cohort of a single tertiary pediatric hospital in Southern Italy, in which this complication occurred in 12/54 children (22% of cases). The great clinical variability of brain injury has led us to build a “neurological score” useful to assess the clinical severity of neurologic. Subjects with the most severe CNS involvement during STEC-HUS having a high neurological score were treated with Eculizumab with good clinical response (4 out 5 patients). In conclusion, the treatment with Eculizumab in patients’ with severe neurological involvement during STEC-HUS was associated with complete regression of both acute renal failure and neurological symptoms resulting in good patients outcome.