Autologous Stem Cell Transplants Reverse Systemic Sclerosis

February 4, 2013

Patients treated with autologous hemopoietic stem cells for systemic sclerosis had a 78% five-year survival rate, relapse-free among the large majority, in a retrospective mutlicenter trial: Also, bad news about vigilance for future drug contamination threats.

Systemic Sclerosis

 

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 analysisThe Lancet, published online Jan. 28, 2013. Full text $31.50

Autologous hemopoietic stem-cell transplantation (HSCT) for systemic sclerosis gave sustained improvement in skin thickness and forced vital capacity with acceptable treatment-related mortality (5.5%). In this multinational multicenter retrospective analysis of 90 patients, five-year survival was 78%, and five-year relapse-free survival was 70%.  Five (5.5%) died from treatment, four from cardiovascular complications. Guidelines should exclude patients with pulmonary artery hypertension, primary cardiac involvement, or pericardial disease, who cannot tolerate HSCT, say the authors. They developed a standard screening procedure: echocardiogram with tricuspid annular plane systolic excursion, right heart catheterization, and cardiac MRI with gadolinium.
 

Comment: Improving safety in autologous HSCT for systemic sclerosisThe Lancet, published online Jan. 28, 2013. Full text $31.50
 

A comment concludes that autologous HSCT and anti-thymocyte globulin with cyclophosphamide had a long-term overall and event-free survival advantage over cyclophosphamide alone. In earlier studies, treatment-related mortality was a discouraging 10%. In this study, it was an acceptable 5.5%. This can be weighed favorably against a five-year relapse-free survival of 70% – and the promise of reversing systemic sclerosis. The high treatment-related mortality of autologous HSCT was due to cardiac and pulmonary causes. Patient selection with an extended cardiopulmonary workup including right heart catheterization with fluid challenge and MRI, will reduce it.
 

Drug Supply Issues

Viewpoint: Responding to the Outbreak of Invasive Fungal Infections: The Value of Public Health to AmericansJAMA, online first, Jan. 30, 2013.  Free full text. 

The health departments that tracked down contaminated methylprednisolone are being cut. A viewpoint says that the prompt, effective response came from a coordinated effort among the Centers for Disease Control and Prevention (CDC), local health workers such as the Tennessee Department of Health, an alert clinician (who realized the significance of an unusual meningitis), industry, academia, the media, professional organizations, consumer groups, and frequent communications among them. Those state and local health departments have lost more than 45,000 jobs since 2008, leaving their work to the CDC. Funding cuts have prevented broad adoption of best technologies, from information sciences to culture-free surveillance.