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

PLoS One. 2022 Oct 27;17(10):e0274578. doi: 10.1371/journal.pone.0274578. eCollection 2022.

Abstract

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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Amyloidosis* / diagnosis
  • Amyloidosis* / therapy
  • Argentina / epidemiology
  • Bortezomib / therapeutic use
  • Humans
  • Immunoglobulin Light-chain Amyloidosis* / diagnosis
  • Immunoglobulin Light-chain Amyloidosis* / therapy
  • Middle Aged
  • Registries
  • Retrospective Studies

Substances

  • Bortezomib

Grant support

We received financial support for the study and the article by Jansen LATAM (https://www.janssen.com/latinoamerica). The authors, that received partial funding for this study, were Maria Lourdes Posadas-Martinez, Maria Adela Aguirre, Erika Brulc, Elsa Nucifora, Soledad Saez and Patricia Sorroche. Both Gerardo Machnicki and Mariana Fernandez, are employees of Janssen-Cilag Farmacêutica, Argentina and Spain, respectively. Both authors participated in the conception, preparation and decision to publish the manuscript. The remaining authors design the research question, methods, data collection and data analyses independently.