Változások a könnyűlánc-amyloidosis kezelésében – egy nagy budapesti centrum adatai

Le résumé fourni par la source

Introduction: Primary or light chain amyloidosis is caused by monoclonal immunoglobulin fragments that accumulate in the organs, and the resulting amyloid deposits cause organ dysfunction. Objective and method: During our research, we examined the clinical and survival data of patients diagnosed with light chain amyloidosis between 2010 and 2024, at the Department of Internal Medicine and Hematology of Semmelweis University. We paid special attention to exploring what factors may affect the prognosis, therefore we reviewed the connection between survival and the date of diagnosis, the age of the patient, the cytogenetics, and different treatment schemes. Results: In the examined period, 56 patients were diagnosed with light chain amyloidosis. Based on the growing number of cases, we can see an increasing tendency in the recognition of the illness, however, the diagnosis was still mostly made in the advanced stages. The median overall survival of the group was 44 months, and it showed no significant improvement between the examined eras. The prognosis was considerably influenced by the age of the patients and the stage of the disease at the time of the diagnosis. The treatment of light chain amyloidosis went through considerable change in the past 15 years. The newer therapeutic agents – such as daratumumab and venetoclax – induce complete hematologic remission more often and offer a higher rate of organ response compared to previous treatment options. Conclusion: Due to the more favorable response rates to newer agents, in the future we can expect to see improvements in survival as well, although our study was not yet able to demonstrate this. Early stage remains the most important prognostic marker, which highlights the importance of early recognition of this condition. Orv Hetil. 2024; 165(47): 1860–1870.

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