Eculizumab in paediatric atypical haemolytic uraemic syndrome: Lessons learned from a single‐centre experience in the United Arab Emirates

Abstract

Aim: Atypical haemolytic uraemic syndrome (aHUS) is a debilitating condition that can cause significant morbidity and mortality in children if not adequately and promptly treated. This report shares real-world data on the use of eculizumab in children with aHUS. Methods: We report our experience with the use of eculizumab in 14 children with aHUS. Results: The median age at aHUS diagnosis was 12 months (range: 2-108 months), with six (42.9%) patients presenting in infancy and six (42.9%) being males. Eculizumab therapy was associated with haematological and thrombotic microangiopathy responses in 14 (100%) and 13 (92.9%) patients after a median of 9 days (range: 7-12 days) and 9.5 days (range: 7-14 days), respectively. None of the six patients who were previously treated with plasma therapy required any further infusions. Of the six patients who previously required dialysis, only one patient continued to do so and eventually received a renal transplant. The median time to ≥25% decrease in serum creatinine level in the remaining patients was 16 days (range: 14-21 days), and estimated glomerular filtration rate increased from a median of 17-101 mL/min/1.73 m 2 . The safety profile was similar to that reported in the literature, and 10 patients continue to receive therapy, with 3 being on the drug for 4 or more years. Conclusion: Our study adds to the growing body of evidence highlighting the efficacy and safety of eculizumab for the management of children with aHUS.

Clinical features and management of atypical hemolytic uremic syndrome patient with DGKE gene variants: a case report

“…The finding that podocyte dysfunction with nephrotic-range proteinuria is a complication of this form of DGKE -aHUS predisposes patients to the development of TMA. Some patients with combined variants in the DGKE gene and the complement system have been identified, which possibly explains the spectrum of clinical manifestations ( 9 , 11 , 25 ). In addition, the loss of various regulators through the leaked kidney and hepatic synthesis of procoagulation factors results in an imbalance of coagulation regulators, which also contribute to the development of TMA, consistent with the pathogenesis of nephrotic syndrome with hypercoagulability.…”

Comparative efficacy of the original and biosimilar eculizumab in the treatment of obstetric atypical hemolytic-uremic syndrome

“…В литературе имеются многочисленные доказательства, подтверждающие эффективность экулизумаба при лечении аГУС. Так, в результате терапии этим препаратов исчезновение потребности в диализе было отмечено у 80-95% пациентов, а улучшение/восстановление функции почек -у 48-95% [2,[7][8][9][10][11][12][13][14][15]. По данным литературы, среди пациенток с акушерским аГУС, получавших лечение экулизумабом, ремиссия заболевания с восстановлением/улучшением функции почек наблюдалась в 88% случаев [16].…”

Long-term outcomes and response to treatment in diacylglycerol kinase epsilon nephropathy

“…28 Some clinical data for patients NCL31, NCL32, and NCL35 have previously been published. 29 The incidence rates for complement-mediated aHUS and DGKE aHUS were calculated using the estimated population of England according to the Office for National Statistics. 30 The calculation of the incidence of complement-mediated aHUS included patients referred to the NRCTC with TMA in which the following conditions were excluded using genetic and serological analysis or on the basis of clinical context: thrombotic thrombocytopenic purpura, shiga toxin-producing E coli HUS, disseminated intravascular coagulation, pneumococcal HUS, HIV, drug-induced TMA, severe hypertensionassociated TMA, cobalamin C deficiency-associated TMA, malignant neoplasm-associated TMA, bone marrow transplantationassociated TMA, de novo TMA after solid organ transplantation, glomerular disease-associated TMA, and autoimmune diseaseassociated TMA.…”