Treatment patterns and outcomes in light chain amyloidosis: An institutional registry of amyloidosis report in Argentina

POSADAS MARTÍNEZ, MARÍA LOURDES; SAEZ, MARÍA SOLEDAD; FERNANDEZ, MARIANA; BRULC, ERIKA B.; MACHNICKI, GERARDO; AGUIRRE, MARÍA ADELA; SORROCHE, PATRICIA BEATRIZ; NUCIFORA, ELSA MERCEDES

PLOS ONE

PUBLIC LIBRARY SCIENCE

Lugar: San Francisco; Año: 2022

1932-6203

Light chain (AL) amyloidosis is a form of systemic amyloidosis, causing organ dysfunction,mainly affecting the heart and kidney. Patient-tailored and risk-adapted decision making iscritical in AL amyloidosis management. There is limited real-world evidence data fromArgentina and Latin America regarding the treatment approaches for AL amyloidosis. Thisretrospective cohort study aimed to describe the treatment patterns and outcomes in adultpatients (>18 years) diagnosed with AL amyloidosis at the Hospital Italiano in Buenos Aires,Argentina, using a 10-yearfollow-up data (June 1, 2010 to May 31, 2019) from the institutional registry of amyloidosis (IRA). The study population had a mean age of 63 years and54.4% weremale. Heart and kidney were the most frequently affected organs. Of the 90 eligible patients included in the study, 70underwent treatment. Bortezomib-based regimenwas the preferred first-line treatment (75.7% patients). Overall,54.4% of the patients presented a deep response (complete or very good partial response). Median overall survival(OS) was 5years, the 1-year OS and progression free survival rates were 80% (95% confidence interval [CI]: 68?87) and 80% (95%CI 68?87)), respectively. This study provides vitalreal-world evidence for the long-term treatment patterns and survival in a large cohort of ALamyloidosis patients in Argentina.