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

Autor: Posadas-Martinez ML; Internal Medicine Department, Internal Medicine Research Unit, CONICET, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina., Aguirre MA; Internal Medicine Department, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina., Brulc E; Internal Medicine Department, Hematology Service, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina., Saez MS; Internal Medicine Department, Biochemestry Service, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina., Sorroche P; Internal Medicine Department, Biochemestry Service, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina., Machnicki G; Janssen-Cilag Farmacêutica Ltda, Buenos Aires, Argentina., Fernandez M; Janssen-Cilag S A, Madrid, Spain., Nucifora EM; Hematology Service, Internal Medicine Department, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina.
Jazyk: angličtina
Zdroj: PloS one [PLoS One] 2022 Oct 27; Vol. 17 (10), pp. e0274578. Date of Electronic Publication: 2022 Oct 27 (Print Publication: 2022).
DOI: 10.1371/journal.pone.0274578
Abstrakt: 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 is critical in AL amyloidosis management. There is limited real-world evidence data from Argentina and Latin America regarding the treatment approaches for AL amyloidosis. This retrospective cohort study aimed to describe the treatment patterns and outcomes in adult patients (>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 and 54.4% weremale. Heart and kidney were the most frequently affected organs. Of the 90 eligible patients included in the study, 70underwent treatment. Bortezomib-based regimen was 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 vital real-world evidence for the long-term treatment patterns and survival in a large cohort of AL amyloidosis patients in Argentina.
Competing Interests: I have read the journal’s policy and the authors of this manuscript have the following competing interests: Dr. Gerardo Machnicki is an employee of Janssen-Cilag Farmacêutica Ltda (Argentina) and Dr. Mariana Fernandez is an employee of Janssen-Cilag S A (Spain). All the other authors are employees of Hospital Italiano de Buenos Aires (Argentina). The authors received financial support for this research from a grant Jansen LATAM, we report no other conflict of interest. The authors-maintained independence from design through data collection analysis and writing the manuscript. This does not alter our adherence to PLOS ONE policies on sharing data and materials. There are no patents, products in development or marketed products associated with this research to declare.
Databáze: MEDLINE
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