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Thrombotic Microangiopathy in Childhood Steroid-Resistant Nephrotic Syndrome: Case Series
Dor Fisher1, Orly Haskin2,3, Daniel Landau2,3
1Pediatric Nephrology Service, Kaplan Medical Center, Rehovot, Israel, dorfisher@gmail.com.
Introduction:
Thrombotic microangiopathy (TMA) with associated nephrotic syndrome is a unique and rare condition that is infrequently described in pediatrics.
Case Presentations:
Four children with steroid-resistant nephrotic syndrome (SRNS), immune-mediated and of monogenic origin, presented with TMA during late stages of chronic kidney disease. All patients tested negative for Shiga toxin and had normal A Disintegrin-like and Metalloprotease with Thrombospondin type 1 repeats 13 (ADAMTS13) activity. Genetic and functional studies of complement dysregulation were negative. Three of the 4 patients treated with Eculizumab showed good hematologic response but no kidney function recovery. None of the three children who underwent kidney transplantation had recurrent TMA.
Conclusions:
TMA may develop in patients with SRNS, manifesting as an unexplained worsening of kidney function, new-onset hypertension, hemolytic anemia, and thrombocytopenia. Eculizumab was effective in improving hemolytic markers without kidney function recovery. TMA did not recur after kidney transplantation in this entity.
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