scholarly journals Utility of Doppler Myocardial Imaging, Cardiac Biomarkers, and Clonal Immunoglobulin Genes to Assess Left Ventricular Performance and Stratify Risk Following Peripheral Blood Stem Cell Transplantation in Patients with Systemic Light Chain Amyloidosis (AL)

2011 ◽  
Vol 24 (4) ◽  
pp. 444-454.e2 ◽  
Author(s):  
Diego Bellavia ◽  
Roshini S. Abraham ◽  
Patricia A. Pellikka ◽  
Angela Dispenzieri ◽  
John C. Burnett ◽  
...  

Blood ◽  
2004 ◽  
Vol 104 (6) ◽  
pp. 1881-1887 ◽  
Author(s):  
Angela Dispenzieri ◽  
Morie A. Gertz ◽  
Robert A. Kyle ◽  
Martha Q. Lacy ◽  
Mary F. Burritt ◽  
...  

Abstract Primary systemic amyloidosis (AL) is a fatal plasma cell disorder. Pilot data suggest survival is better in patients undergoing peripheral blood stem cell transplantation (PBSCT), but the selection process makes the apparent benefit suspect. We have reported that circulating cardiac biomarkers are the best predictors of survival outside of the transplantation setting. We now test whether cardiac troponins (cTnT and cTnI) and N-terminal pro-brain natriuretic peptide (NT-proBNP) are prognostic in transplant recipients. In 98 patients with AL undergoing PBSCT, serum cardiac biomarkers were measured (cTnT, 98 patients; cTnI, 65 patients; and NT-proBNP, 63 patients). Elevated levels of cTnT, cTnI, and NT-proBNP were present in 14%, 43%, and 48% of patients, respectively. At 20 months median follow-up, median survival has not been reached for patients with values below the thresholds; in patients with values above the thresholds, median survival is 26.1 months, 66.1 months, and 66.1 months, respectively. Our previously reported risk systems incorporating these markers were also prognostic, notably the cTnT/NT-proBNP staging. Using this system, 49%, 38%, and 13% of patients were in stage I, stage II, and stage III, respectively. Determining levels of circulating biomarkers may be the most powerful tool for staging patients with AL undergoing PBSCT.



Blood ◽  
2004 ◽  
Vol 103 (10) ◽  
pp. 3960-3963 ◽  
Author(s):  
Angela Dispenzieri ◽  
Robert A. Kyle ◽  
Martha Q. Lacy ◽  
Terry M. Therneau ◽  
Dirk R. Larson ◽  
...  

Abstract Primary systemic amyloidosis (AL) is a plasma cell dyscrasia resulting in multisystem failure and death. High-dose chemotherapy with peripheral blood stem cell transplantation (PBSCT) has been associated with higher response rates and seemingly higher overall survival than standard chemotherapy. Selection bias, however, confounds interpretation of these results. We performed a case-match-control study comparing overall survival of 63 AL patients undergoing transplantation with 63 patients not undergoing transplantation. Matching criteria included age, sex, time to presentation, left ventricular ejection fraction, serum creatinine, septal thickness, nerve involvement, 24-hour urine protein, and serum alkaline phosphatase. According to design, there was no difference between the groups with respect to sex (57% males), age (median, 53 years), left ventricular ejection fraction (65%), number of patients with peripheral nerve involvement (17%), cardiac interventricular septal wall thickness (12 mm), serum creatinine (1.1 mg/dL [97.24 μmol/L]), and bone marrow plasmacytosis (8%). Sixty-six patients have died (16 cases and 50 controls). For PBSCT and control groups, respectively, the 1-, 2-, and 4-year overall survival rates are 89% and 71%; 81% and 55%; and 71% and 41%. Outside a randomized clinical trial, these results present the strongest data supporting the role of PBSCT in selected patients with AL.









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