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 :

» Research Highlights: Stem Cell Research

James White was in his 60's when he was diagnosed with , which progressed to. The traditional treatment for patients like White is standard , which brings a median survival time of about a year. But Duke oncologist David A. Rizzieri, MD, offered...

» Researchers Identify Core Genetic Switch As A Viable Target For New Scleroderma Treatments

Scleroderma is a rare, autoimmune disease. Often fatal, it causes the fibrosis or thickening of the connective tissue which support the skin and other vital organs within the body, through the overproduction of . The disease currently lacks a cure...

» L-selectin and Skin Damage in Systemic Sclerosis

L-selectin ligands are induced on the endothelium of inflammatory sites. L-selectin expression on neutrophils and monocytes may mediate the primary adhesion of these cells at sites of inflammation by mediating the leukocyte-leukocyte interactions...

» An Approach to the Treatment of Scleroderma

Systemic sclerosis is unique among the rheumatic diseases because it presents the challenge of managing a chronic multisystem autoimmune disease with a widespread obliterative vasculopathy of small arteries that is associated with varying degrees of...

» Glutamine: Explained

Glutamine is the most abundant (building block of protein) in the body. The body can make enough glutamine for its regular needs, but extreme stress (the kind you would experience after very heavy exercise or an injury), your body may need more...

» Tyrosine Kinase Inhibitors in the Treatment of Systemic Sclerosis: The Difficulty in Interpreting Proof-of-Concept Studies

Jessica Gordon and Robert SpieraReceived 1 June 2011; Revised 2 August 2011; Accepted 2 August 2011Systemic sclerosis (SSc) is a heterogeneous, multisystem disorder characterized by vasculopathy, fibrosis, and autoimmunity in the context of both...