The real-world data on patients with cardiac stage IIIb AL amyloidosis

Background Morbidity and mortality of patients with immunoglobulin light chain (AL) amyloidosis are strongly associated with the severity of cardiac involvement, especial in patients with cardiac stage IIIb, but the real-world data on these patients is still limited. Patients and Methods A retrospective analysis was conducted on 77 patients diagnosed with cardiac stage IIIb AL amyloidosis at our center. We analyzed the clinical characteristics, treatment and outcome of the patients. Results The median age of patients was 57 years and 49.4% were male. Median serum N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP) and cardiac troponin T (cTnT) were 13384 ng/L and 0.166 ug/L, and 42(54.5%) patients had heart failure at diagnosis. Fifty-seven (74.0%) patients received anti-plasma cell treatment, and the main treatment options include bortezomib or thalidomide combined with dexamethasone. The hematologic overall response rate was 70% (28/40), and at 6-month landmark analysis, patients with hematologic responses had a higher survival rate. Cardiac and renal responses were achieved in 14(37.8%) and 13(32.5%) patients, respectively. After a median follow-up of 10 months (range 1-115 months), median overall survival (OS) was 18 months, and the estimated survival rates at 3, 6, and 12 months were 79.9%, 75.6%, and 54.5%, respectively. In Cox regression models, age, hypotension and cTnT were independently predictive of mortality after adjusting for heart failure. Conclusion The hematologic, cardiac and renal responses were relative lower in patients with cardiac stage IIIb AL amyloidosis. The overall prognosis of patients was poor, and age, hypotension, and cTnT can be used to predict mortality. Micro-abstract The clinical characteristics, treatment modalities and outcomes of Chinese patients with cardiac stage IIIb AL amyloidosis have not been fully investigated. Through consulting the electronic medical record system and telephone follow-up, we documented hematologic and organ responses after treatment in 77 patients diagnosed at our center. The unsatisfactory prognosis of these patients suggests that a rapid-acting and safe therapy is needed for these patients.