PhD Scientific Days 2025

Poster Session III. - X: Conservative Medicine

Changes in the Treatment Outcomes of Light Chain Amyloidosis – Findings of a Large Center in Budapest

Name of the presenter

Szél Fruzsina

Institute/workplace of the presenter

Semmelweis Egyetem

Authors

Szél Fruzsina¹, Dr. Varga Gergely², Dr. Masszi Tamás²

1: Semmelweis University
2: Department of Internal Medicine and Hematology, Semmelweis University

Text of the abstract

Introduction
Light chain (AL) amyloidosis is a rare plasma cell disorder that causes the deposition of misfolded monoclonal immunoglobulin light chains in various organs, leading to progressive organ dysfunction and high morbidity. Timely diagnosis and advances in treatment are critical to improving patient outcomes.

Aims
This study aimed to evaluate changes in clinical outcomes and treatment responses in patients diagnosed with light chain amyloidosis over a 15-year period at a major Hungarian center. Specifically, we assessed how factors such as time of diagnosis, patient age, cytogenetics, and treatment modalities influenced prognosis and survival.

Method
We retrospectively analyzed clinical and survival data of 56 patients diagnosed with light chain amyloidosis between 2010 and 2024 at the Department of Internal Medicine and Hematology, Semmelweis University. We examined correlations between survival and various prognostic indicators, including treatment regimens and disease stage at diagnosis.

Results
The number of diagnosed cases increased over time, indicating improved disease recognition, although most diagnoses were still made at advanced stages. Median overall survival was 44 months, with no significant improvement observed across the study period. Patient age and disease stage at diagnosis significantly influenced prognosis. A notable shift in treatment occurred, with newer agents such as daratumumab and venetoclax leading to higher rates of complete hematologic and organ responses compared to earlier therapies.

Conclusion
Although survival improvements have yet to be demonstrated, the introduction of novel agents has led to better treatment responses, suggesting potential future gains in patient outcomes. Early-stage diagnosis remains the most significant prognostic factor, underscoring the need for heightened clinical awareness and early detection strategies.

Funding
This work was supported by the Semmelweis University Researchers’ Scholarship Program (EKÖP).