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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
来源:
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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