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Survival increased in systemic sclerosis patients by haematopoieticstem cell transplantation by qrt etget





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Survival increased in systemic sclerosis patients by haematopoieticstem cell transplantation by
Article Posted: 10/07/2013
Article Views: 95
Articles Written: 2163
Word Count: 431
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Survival increased in systemic sclerosis patients by haematopoieticstem cell transplantation


 
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Initial results from an international, investigator-initiated, openlabel phase III trial were presented at EULAR 2012, the AnnualCongress of the European League Against Rheumatism. Data indicatethat haematopoietic stem cell transplantation (HSCT) results in better long term survival thanconventional treatment for patients with poor prognosis earlydiffuse cutaneous systemic sclerosis. The ASTIS (Autologous Stem Cell Transplantation InternationalScleroderma) trial enrolled more than 150 patients between 2001 and2009, and randomised patients to the HSCT arm or to intravenouspulse cyclophosphamide treatment. As of May 1, 2012, significantlymore deaths have occurred in the conventional treatment group.

Halfof the deaths in the HSCT group occurred early and were deemedtreatment-related according to an independent data monitoringcommittee. In the conventional treatment group in contrast, none ofthe deaths were deemed to be treatment-related; but more deathsoccurred later and most were related to progressive disease. "Systemic sclerosis is a debilitating disease that can lead toheart, lung or kidney failure and premature death, especially inpatients who have the diffuse cutaneous form of the condition,where skin thickening is more generalised and involvement of vitalorgans more common. The ASTIS study shows that such patients maybenefit from early intensive immunosuppressive treatment," saidProfessor Jaap van Laar from Newcastle University, ProfessorDominique Farge, Assistance Publique Hopitaux de Paris (Sponsor inFrance, Paris 7 University) and Professor Alan Tyndall from BaselUniversity, on behalf of their colleagues from the EBMT EULARScleroderma Study Group.

"These initial results are veryencouraging and will help identify patients who benefit from stemcell transplantation." The ASTIS trial was a unique collaborative project of 27multidisciplinary teams from 10 countries conducted under theauspices of two leading organisations in the respective fields, theEuropean Group for Blood and Marrow Transplantation and theEuropean League Against Rheumatism. The primary endpoint of thetrial was event-free survival, defined as survival until death ordevelopment of major organ failure. Systemic sclerosis is a rare but severe autoimmune systemicconnective tissue disease*. Increased fibroblast activity resultsin abnormal growth of connective tissue which causes vasculardamage and fibrosis of the skin, gastrointestinal (GI) tract andother internal organs**. Characteristics of systemic sclerosisinclude vasomotor disturbances; fibrosis; subsequent atrophy of theskin, subcutaneous tissue, muscles, and internal organs andimmunologic disturbances*.

Systemic sclerosis is estimated to occurin 2.3-10 people per one million*. Diffuse cutaneous systemicsclerosis cases make up 30% of all systemic sclerosis cases andinvolve the upper arms, thighs and trunk**. Lung fibrosis andpulmonary hypertension are important causes of mortality in these patients and there isno curative treatment available so far*. Additional References Citations.

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