Outcomes in Children and Adults With Antiglomerular Basement Membrane Disease

抗肾小球基底膜病患儿及成人的预后

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Abstract

INTRODUCTION: Antiglomerular basement membrane (anti-GBM) disease is a rare, small-vessel vasculitis because of antibodies targeting the glomerular and alveolar capillaries, leading to rapidly progressive glomerulonephritis and/or pulmonary hemorrhage. Data on children with anti-GBM are scarce. METHODS: We collected clinical and biochemical data from European pediatric and adult patients diagnosed with anti-GBM disease between 2020 and 2024. RESULTS: A total of 72 patients (35% children) with anti-GBM disease and with a median follow-up of 18 months were analyzed. Pediatric cases were more often female and had higher estimated glomerular filtration rate (eGFR) at the time of diagnosis (each P < 0.01), whereas the percentage of patients requiring dialysis, presence of pulmonary hemorrhage, and immunological findings did not statistically differ between groups. Treatment consisted mainly of daily plasma exchanges (PEXs)and corticosteroids at higher weight-based doses in children (P < 0.0001), in combination with cyclophosphamide (CYC) or, preferably in children (P < 0.05), with rituximab (RTX) and mycophenolate mofetil (MMF). Final eGFR was higher in children than in adults (P < 0.0001), although the frequency of kidney failure did not significantly differ between children (24%) and adults (38%). Adult patients and patients who required dialysis at the time of diagnosis had a 16-fold and 11-fold increased risk of chronic kidney disease (CKD) stage 3 or higher, respectively. CONCLUSION: Our study indicates that girls predominate among children with anti-GBM disease and that children have a better outcome in terms of eGFR than adults, which is at least partly because of better eGFR values at diagnosis. The need for dialysis is a strong predictor of outcome, regardless of age.

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