Cardiac Involvement and Treatment-Related Mortality After Non-Myeloablative Haemopoietic Stem-Cell Transplantation With Unselected Autologous Peripheral Blood for Patients With Systemic Sclerosis: A Retrospective Analysis

Study Questions:

Should cardiac screening be used to risk stratify patients with systemic sclerosis being considered for hemopoietic stem-cell transplantation (HSCT)?


This was a retrospective analysis of 90 patients with systemic sclerosis and interstitial lung disease treated with HSCT and followed for 5 years.


Six percent of patients died from treatment-related causes. Four treatment-related deaths occurred because of cardiovascular complications (one constrictive pericarditis, two right heart failures without underlying infection, and one heart failure during mobilization), and one death was secondary to sepsis without documented underlying heart disease. Kaplan-Meier analysis showed survival was 78% at 5 years (after eight relapse-related deaths), and relapse-free survival was 70% at 5 years. Compared with baseline, improvements were noted in Rodnan skin scores at 1 year (58 patients; p < 0.0001), 2 years (42 patients; p < 0.0001), and 3 years (27 patients; p < 0.0001) and forced vital capacity at 1 year (58 patients; p = 0.009), 2 years (40 patients; p = 0.02), and 3 years (28 patients; p = 0.004), but total lung capacity and diffusion capacity of carbon monoxide (DLCO) were not improved significantly after HSCT. Overall mean DLCO was improved in patients with normal baseline echocardiograms (p = 0.005) or electrocardiograms (p = 0.05).


The authors concluded that autologous HSCT with a non-myeloablative regimen of cyclophosphamide and rabbit anti-thymocyte globulin (rATG) with a nonselected autograft results in sustained improvement in skin thickness and forced vital capacity. DLCO is affected by baseline cardiac function. Guidelines for cardiac screening of patients with systemic sclerosis to assess treatment-related risk from pulmonary artery hypertension, primary cardiac involvement, or pericardial disease should be reconsidered and updated.


Cardiac screening, including echocardiography, magnetic resonance imaging, and right heart catheterization with fluid challenge may be useful in predicting which patients with systemic sclerosis are likely to receive most benefit from HSCT. Additional prospective studies would help to confirm which SS patients need to be studied and which tests are most useful.

Clinical Topics: Heart Failure and Cardiomyopathies, Pericardial Disease, Prevention, Acute Heart Failure, Hypertension

Keywords: Kaplan-Meier Estimate, Pericarditis, Constrictive, Sepsis, Cardiac Catheterization, Pulmonary Artery, Cell Transplantation, Lung Diseases, Interstitial, Cyclophosphamide, Autografts, Heart Failure, Scleroderma, Systemic, Hypertension

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