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

被引:4
作者
Posadas-Martinez, Maria Lourdes [1 ]
Adela Aguirre, Maria [2 ]
Brulc, Erika [3 ]
Soledad Saez, Maria [4 ]
Sorroche, Patricia [4 ]
Machnicki, Gerardo [5 ]
Fernandez, Mariana [6 ]
Mercedes Nucifora, Elsa [3 ]
机构
[1] Hosp Italiano Buenos Aires, Internal Med Dept, Internal Med Res Unit, CONICET, Buenos Aires, DF, Argentina
[2] Hosp Italiano Buenos Aires, Internal Med Dept, Buenos Aires, DF, Argentina
[3] Hosp Italiano Buenos Aires, Internal Med Dept, Hematol Serv, Buenos Aires, DF, Argentina
[4] Hosp Italiano Buenos Aires, Internal Med Dept, Biochemestry Serv, Buenos Aires, DF, Argentina
[5] Janssen Cilag Farmaceut Ltda, Buenos Aires, DF, Argentina
[6] Janssen Cilag SA, Madrid, Spain
关键词
AL AMYLOIDOSIS; RANDOMIZED-TRIAL; MELPHALAN; DEXAMETHASONE; BORTEZOMIB; DIAGNOSIS; CRITERIA; EPIDEMIOLOGY; MANAGEMENT;
D O I
10.1371/journal.pone.0274578
中图分类号
O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];
学科分类号
07 ; 0710 ; 09 ;
摘要
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.
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页数:14
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