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

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.

The role of registries in improving health and bridging healthcare, research, education, innovation and development: a research department perspective

“…The network has published articles, won awards at conferences, taught courses and/ or seminars in Latin American countries, presented work at congresses and symposia, developed clinical practice guidelines on the diagnosis and treatment of amyloidosis in our region and published related articles on different aspects of amyloidosis, including documentation of the existence and magnitude of the disease, [34][35][36] the characteristics of the disease, [37][38][39] the understanding of the patterns of diagnosis and treatment, 40,41 and the development of strategies that seek to resolve or mitigate these problems and/or monitor their evolution. [42][43][44][45][46][47] The To address current gaps in knowledge, innovation and development are being conducted on diagnostic methods to identify the amyloid protein with local resources.…”

Free Light Chains κ and λ as New Biomarkers of Selected Diseases

“…Moreover, it was suggested that the majority of patients present the λ type of monoclonal light chain [22,23]. Literature data have indicated that except for kidneys, the heart is the most frequently affected organ by light chain amyloidosis [24]. In addition, in patients with primary systemic immunoglobulin light chain amyloidosis, the association between cardiac biomarkers, echocardiographic parameters and FLCs was investigated.…”