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1.
Hum Pathol ; 62: 160-169, 2017 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-28087478

RESUMO

Lysinuric protein intolerance (LPI) is a rare autosomal recessive disease caused by mutations in the SLC7A7 gene encoding the light subunit of a cationic amino acid transporter. Symptoms mimic primary urea cycle defects but dysimmune symptoms are also described. Renal involvement in LPI was first described in the 1980s. In 2007, it appeared that it could concern as much as 75% of LPI patients and could lead to end-stage renal disease. The most common feature is proximal tubular dysfunction and nephrocalcinosis but glomerular lesions are also reported. However, very little is known regarding histological lesions associated with LPI. We gathered every kidney biopsy of LPI-proven patients in our highly specialized pediatric and adult institution. Clinical, biological, and histological information was analyzed. Five LPI patients underwent kidney biopsy in our institution between 1986 and 2015. Clinically, 4/5 presented with proximal tubular dysfunction and 3/5 with nephrotic range proteinuria. Histology showed unspecific tubulointerstitial lesions and nephrocalcinosis in 3/5 biopsies and marked peritubular capillaritis in one child. Glomerular lesions were heterogeneous: lupus-like-full house membranoproliferative glomerulonephritis (MPGN) in one child evolved towards monotypic IgG1κ MPGN sensitive to immunomodulators. One patient presented with glomerular non-AA non-AL amyloidosis. Renal biopsy is particularly relevant in LPI presenting with glomerular symptoms for which variable histological lesions can be responsible, implying specific treatment and follow-up.


Assuntos
Erros Inatos do Metabolismo dos Aminoácidos/patologia , Rim/patologia , Adolescente , Adulto , Erros Inatos do Metabolismo dos Aminoácidos/complicações , Erros Inatos do Metabolismo dos Aminoácidos/genética , Erros Inatos do Metabolismo dos Aminoácidos/terapia , Sistema y+L de Transporte de Aminoácidos , Amiloidose/etiologia , Amiloidose/patologia , Biópsia , Criança , Progressão da Doença , Feminino , Imunofluorescência , Cadeias Leves da Proteína-1 Reguladora de Fusão/genética , Predisposição Genética para Doença , Glomerulonefrite Membranoproliferativa/etiologia , Glomerulonefrite Membranoproliferativa/patologia , Humanos , Lactente , Masculino , Mutação , Nefrocalcinose/etiologia , Nefrocalcinose/patologia , Síndrome Nefrótica/etiologia , Síndrome Nefrótica/patologia , Paris , Fenótipo , Proteinúria/etiologia , Proteinúria/patologia , Insuficiência Renal Crônica/etiologia , Insuficiência Renal Crônica/patologia , Fatores de Tempo
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