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Kidney Week

Abstract: TH-PO981

A Case of Pediatric Dense Deposit Disease (DDD) in Remission for 7 Years on Eculizumab

Session Information

Category: Trainee Case Report

  • 1700 Pediatric Nephrology

Authors

  • Vuong, Kimmy Thien, UCSD Rady Childrens Hospital, San Diego, California, United States
  • Shayan, Katayoon, UCSD Rady Childrens Hospital, San Diego, California, United States
  • Ingulli, Elizabeth G., UCSD Rady Childrens Hospital, San Diego, California, United States
Introduction

DDD affects only 2-3 people per million. Slowing the disease course is critical because 50% of affected children progress to ESRD within 10 years, ultimately requiring dialysis or renal transplant. Recurrence post-transplant leads to at least 50% of graft loss. Current guidelines suggest Eculizumab as a potential treatment for refractory cases defined by persistent proteinuria, hematuria, alternative complement pathway activation or elevated C3 nephritic factor (C3NEF).

Case Description

A 5 year old previously healthy male presented with at least 2 months of asymptomatic microscopic hematuria without associated illness or relevant family history. He had normal BP, a normal exam, mild proteinuria (UPr/UCr 0.43), hematuria (URBCs >25), hypocomplementemia (C3 <20 mg/dL, C4 23 mg/dL), normal renal function and albumin. Renal biopsy had membranoproliferative glomerulonephritis with isolated intramembranous deposits. Complement genetic analysis was negative. Functional studies showed alternative pathway complement activation and the presence of C3NEF.

Discussion

He received steroids and Cellcept 700 mg/m2/d but continued to have complement activation (based on functional studies), microhematuria and worsening proteinuria (UPC peak 1.8). He was weaned off steroids and Cellcept (after 7 months) then received Eculizumab without significant adverse effects or complications. A follow-up renal biopsy improved and his proteinuria, hematuria. Functional studies suggest ongoing complement activation and lapses in therapy increase proteinuria. Thus, eculizumab has been continued for 7 years. To date, he may have one of the longest DDD remissions on Eculizumab monotherapy. Further studies including a randomized controlled trial of Eculizumab monotherapy could define parameters for starting and discontinuing treatment.