HSCT Benefits In Early Diffuse Cutaneous Systemic Sclerosis PDF Print E-mail
Sunday, 01 July 2012 22:09
Initial results from an international, investigator-initiated, open label phase 3 trial, presented at EULAR 2012, indicate that haematopoietic stem cell transplantation (HSCT) results in better long-term survival than conventional treatment for patients with poor-prognosis, early diffuse cutaneous systemic sclerosis.

The Autologous Stem Cell Transplantation International Scleroderma (ASTIS) trial enrolled more than 150 patients between 2001 and 2009, and randomised participants to HSCT or IV pulse cyclophosphamide treatment. As of 1 May 2012, significantly more deaths had occurred in the conventional treatment group. Half of the deaths in the HSCT group occurred early and were deemed treatment-related, according to an independent data monitoring committee. In the conventional treatment group in contrast, none of the deaths were deemed to be treatment-related, but more deaths occurred later and most were related to progressive disease.

Speaking on behalf of European Bone Marrow Transplantation (EBMT) EULAR Scleroderma Study Group, Prof Jaap van Laar from Newcastle University, Prof Dominique Farge from Assistance Publique Hôpitaux de Paris and Prof Alan Tyndall from Basel University, explained that initial results from the trial have been very encouraging and help to identify patients who benefit from stem cell transplantation. They added that systemic sclerosis can lead to heart, lung or kidney failure and premature death, especially in patients who have the diffuse cutaneous form of the condition, where skin thickening is more generalised and involvement of vital organs more common. The ASTIS study shows that such patients may benefit from early intensive immunosuppressive treatment, they said.

The ASTIS trial was a unique collaborative project of 27 multidisciplinary teams from 10 countries conducted under the auspices of two leading organisations in the respective fields – EBMT and EULAR. The primary endpoint of the trial was event-free survival, defined as survival until death or development of major organ failure.

Systemic sclerosis is estimated to occur in 2.3 to 10 per million population. Systemic sclerosis is therefore a rare but severe autoimmune systemic connective tissue disease. Increased fibroblast activity results in abnormal growth of connective tissue which causes vascular damage and fibrosis of the skin, GI tract and other internal organs. Characteristics of systemic sclerosis include vasomotor disturbances and fibrosis with subsequent atrophy of the skin, subcutaneous tissue, muscle and internal organs, along with immunologic disturbances. Diffuse cutaneous systemic sclerosis cases make up 30 per cent of all systemic sclerosis cases and involve the upper arms, thighs and trunk. Lung fibrosis and pulmonary hypertension are important causes of mortality in these patients and there is no curative treatment available so far.

Source: Medical Independent (2012), "HSCT benefits in early diffuse cutaneous systemic sclerosis"; Original article can be found here.

 
More articles :

» Free the Free Radicals

We’re all used to hearing that everything we once thought was good for us is not. But even within that framework, the latest science about , free radicals and exercise is telling. As many of us have heard, free radicals are molecules created by...

» Researchers Identify Serum Biomarkers to Diagnose SLE and Differentiate Disease Severity and Activity

Scientists report on the development of candidate blood biomarkers for diagnosing (SLE) and (SSc). The team at Lund University in Sweden used recombinant antibody microarrays to identify immunoregulatory protein expression profiles that...

» 1st Systemic Sclerosis World Congress

The world's very first Systemic Sclerosis World Congress is to be held in Florence, Italy, next week from February 11-13th 2010. Carded primarily as an interdisciplinary conference for cardiologists, dermatologists, nephrologists, pulmonologists and...

» Women At Higher Risk For Autoimmune Diseases

In a recent article on , it was reiterated that women are at a significantly higher risk of contracting an autoimmune disease than men, according to an expert in the field. Dr Vivian Pinn, director of the National Institutes of Health recently...

» Cancer Drug May Also Work for Scleroderma

A drug used to treat cancer may also be effective in diseases that cause scarring of the internal organs or skin, such as pulmonary fibrosis or Scleroderma.The drug, with the generic name bortezomib, stopped the production of fibrotic proteins in...

» PDE-5 Inhibitors in Scleroderma Raynaud Phenomenon and Digital Ulcers

Systemic sclerosis- (SSc-) related vasculopathy, as manifested by Raynaud’s Phenomenon (RP) and digital ulcers (DUs), is associated with significant impairment of the quality of life and morbidity. The current vasoactive approach for SSc-RP,...