English 
Italian Identificazione di danno renale reversibile e di precoce risposta alla chemioterapia in pazienti con amiloidosi AL
AUTORI
1Centro per lo Studio e la Cura delle Amiloidosi Sistemiche, Laboratori Sperimentali di Ricerca di Biotecnologie e Servizio di Analisi Chimico-Cliniche, Fondazione IRCCS Policlinico San Matteo, Dipartimento di Medicina Molecolare, Università di Pavia
2Centro per l’Amiloidosi, Dipartimento di Medicina Interna V, Divisione di Ematologia/Oncologia/Reumatologia, Università di Heidelberg, Germania
3Laboratorio di Analisi Chimico-Cliniche, Fondazione IRCCS Policlinico San Matteo, Pavia
4Direzione Scientifica, Fondazione IRCCS Policlinico San Matteo, Pavia
Questo lavoro è stato in parte presentato al 46° Congresso Nazionale SIBioC, 13-15 Ottobre 2014, Roma, sotto forma di poster, ricevendo, nella persona del suo primo autore (P. Milani), il premio SIBioC destinato ai 4 migliori poster presentati.
ABSTRACT
Identification of reversible renal damage and early response to chemotherapy in AL amyloidosis
The kidney is involved in 70% of patients with immunoglobulin light-chain (AL) amyloidosis, but little is known on progression or reversibility of renal involvement. Furthermore, criteria for renal response have never been validated. We designed a staging system for renal damage and identified criteria for renal response and progression in a population of 732 newly diagnosed patients with AL amyloidosis. The population was composed of 461 patients from Pavia (testing cohort) and 271 subjects from Heidelberg (validation cohort). Baseline proteinuria >5 g/24 h and estimated glomerular filtration rate (eGFR) <50 mL/min/1.73 m2 were independently associated with poorer renal survival and discriminated between 3 stages (with none, one or two markers above the cut-off) with significant different renal survival. At 6-month follow-up, a ≥25% eGFR decrease predicted poor renal survival in both cohorts and was adopted as criterion for renal progression. A decrease in proteinuria ≥30% or below the cut-off of 0.5 g/24 h in absence of renal progression were the criteria for renal response, being associated with longer renal survival in the testing and validation cohorts. These endpoints can be used as validated response criteria in renal AL amyloidosis, allowing early assessment of treatment efficacy.