Il trattamento dell’amiloidosi AL si basa su regimi di chemioterapia rivolti contro il clone plasmacellulare che produce il precursore amiloidogenico con l’obiettivo di indurre un miglioramento della funzione degli organi coinvolti e migliorare la sopravvivenza dei pazienti.
La riduzione della FLC è associata ad un miglioramento di diversi parametri della malattia. Il gruppo del Mayo Clinic ha mostrato che il miglioramento delle
condizioni cliniche è strettamente correlato al valore assoluto delle FLC raggiunto dopo la chemioterapia (69).
Il gruppo di studio di Pavia ha dimostrato che la riduzione delle FLC è accompagnata anche ad una riduzione simultanea del NT-proBNP, ad un miglioramento dei sintomi di scompenso cardiaco e a un beneficio in termini di sopravvivenza. (63).
Ulteriori studi (70) hanno dimostrato che la sopravvivenza di pazienti con amiloidosi AL è strettamente legata alla concentrazione delle FLC ed è tanto migliore quanto sono basse le concentrazioni raggiunte dopo la chemioterapia. I pazienti che raggiungono la remissione completa (Kappa/Lambda nei limiti e immunofissazione siero e urine negativa) hanno una sopravvivenza eccellente, si è capito che la prognosi è favorevole anche per chi raggiunge una dFLC < 40 mg/L.
I nuovi criteri di risposta sono riportati in figura 21, ed in questi compare la nuova categoria di “risposta parziale molto buona” in cui vengono inclusi i pazienti con una dFLC al di sotto del limite precedentemente citato, senza che sia raggiunta la risposta completa.
FIGURA 21
La risposta parziale, in cui si ha una riduzione del 50% delle dFLC rallenta l’evoluzione della malattia, ma non è considerato un risultato soddisfacente.
La figura 22 (74) mostra l’esempio di 4 casi di risposta ematologica prima e dopo il trattamento, in cui è possibile osservare in quale modo vengono attribuiti i criteri di risposta alla terapia.
CONCLUSIONI
Il laboratorio di analisi cliniche svolge un ruolo insostituibile nella gestione del paziente con amiloidosi AL, dal momento che risulta essenziale per la diagnosi e per il monitoraggio, nonché nella valutazione della risposta alla terapia. Il dosaggio delle FLC, assieme con l’immunofissazione siero e urine, ha permesso di raggiungere una elevata sensibilità diagnostica nell’individuazione delle componenti monoclonali amiloidogeniche. Tutto ciò ha contribuito alla stratificazione prognostica ed ha permesso di seguire in modo accurato i pazienti nel corso della terapia, consentendo una rapida valutazione della risposta al trattamento e un eventuale cambiamento di strategia terapeutica nel caso in cui si riveli necessario.
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