Management and outcomes in AL amyloidosis: a retrospective analysis of an institutional registry of Argentina

POSADAS MARTINEZ, MARIA LOURDES; ET AL

Congreso; XVII INTERNATIONAL SYMPOSIUM ON AMYLOIDOSIS (ISA); 2020

Ma Lourdes Posadas-Martinez+, Maria Adela Aguirre+, Soledad Saez+, Cesar Belziti+, Rosario Luxardo+, Jorge Arbelbide+, Dorotea Fantl+, Gerardo Machnicki*, Mariana Fernandez*, Elsa Nucifora+Background: AL amyloidosis is a monoclonal plasma cell proliferative disorder characterized by tissue deposits leading to organ dysfunction. There is no standard of care approved in Argentina. Disease extension, number of sites, comorbidities and treatment availability may impact treatment outcomes.  Aim: Describe treatment patterns, overall and progression free survival (OS/PFS) of AL amyloidosis in one institution from Argentina. Methods: Retrospectivecohort (2010-2019) of adult AL amyloidosis patients from Italian Hospital of Buenos Aires - Institutional Registry of Amyloidosis (IRA - ClinicalTrials.gov#: NCT01347047). First line treatment was defined as first regimen received, regardless of subsequent changes. Treatment was categorized as ASCT upfront or following induction, CyBorD, Thalidomide- Dexamethasone/Cyclophosphamide, Rituximab+ Cyclophosphamide + Vincristine +Prednisone(R-CVP), Lenalidomide/Dexamethasone, or other. All patients were followed to deathfor all causes or follow-up loss. Hematological best response was defined as complete remission (CR), very good partial remission (VGPR), partial remission (PR) or no response. PFS wasevaluated and survival rates are expressed as the % surviving by Kaplan Meier method. Results:91 AL amyloidosis patients were included. Organ compromise at diagnosis (median of 2 ? CI:1-3) cardiac 72% (65), renal 71% (62), gastrointestinal 37% (34) and neurological 36% (33).Median ECOG of 2 (1-2) and Charlson score 2 (CI 1-3). The median follow-up of this cohortwas 37 months (CI 12-114). Sixty-nine (76%) patients received treatment, 9 (10%) requiredcardiac transplant and 1 kidney, while 13 (14%) underwent ASCT. From those who receivedchemotherapy, regimens used were 76% (51) CyBorD, 11% (7) Thalidomide-Dexamethasone/Cyclophosphamide, 2% (1) rituximab, 2% (1) Lenalidomide/Dexamethasone,2% (1) R-CVP and 9% (6) others; with mean 5 cycles (DS 5). Twenty-six patients receivedsecond line with median 4 cycles (CI 2-6), using Lenalidomide/Dexamethasone in 42% (11) andDaratumumab in 19% (5), and 3 patients received 3rd line with median 6 cycles (CI 1-6).Hematologic best response (intention-to-treat) was: CR 48% (32), VGPR 7% (5), PR 5% (3), noresponse 24% (16).  Response was not evaluable in 16% (11) patients - 2 deaths, 1 discontinuedtreatment by patient decision, 2 insufficient follow-up time and 6 were referred to otherinstitutions. Overall mortality was 42% (n=38, CI 32-53), median survival was 126 months (CI23-170), and OS at one year was 86% (77-90), 5 years 65% (53-74) and 10 years 53% (39-65).43% (29) presented deep responses. The relapse rate was 21% (CI 11-38, n 9). The median PFSrate was 91% (78-97) at 5 and 79% (63-89) at 10 years. Conclusion: Bortezomib basedtreatment was the most used for first line and 48% of patients achieved CR. Only 38% of patientsreceived second line. OS and PFS are similar to other published cohorts.