Autologous HSCT Promising for Systemic Sclerosis

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Autologous Hematopoietic Stem Cell Transplant Promising for Systemic

Sclerosis

Laurie Barclay, MD

July 16, 2004 ‹ Autologous hematopoietic stem cell transplantation (HSCT) is

promising for systemic sclerosis (SS), according to the results of a study

published in the current issue of the ls of the Rheumatic Diseases.

Randomized trials are under way.

³Several European groups applied myelo- and immunosuppression followed by

autologous [HSCT] as a potential treatment for severe autoimmune diseases

under the auspices of the European Group for Blood and Marrow

Transplantation and the European League Against Rheumatism (EBMT/EULAR),²

write D. Farge, from St. Louis Hospital in Paris, France, and colleagues.

³Striking improvement in the skin score was shown after autologous stem cell

transplantation, with a trend towards stabilisation of lung disease.²

The objective of this study, which includes longer follow-up of original

subjects in EBMT/EULAR trials as well as newly recruited cases, was to

analyze the durability of responses after HSCT for severe SS and to

determine whether the high transplant-related mortality (TRM) rate observed

previously improved with experience. Inclusion criteria were diagnosis of

SS, treatment with HSCT in European phase I to II studies from 1996 through

2002, and follow-up longer than six months. Transplant regimens were defined

by the international consensus statements.

Median age of the 57 patients was 40 years (range, 9.1 - 68.7 years). Skin

scores were improved at six months after HSCT in 37 patients, at 12 months

in 30 patients, at 24 months in 19 patients, and at 36 months in 10 patients

(P < .005). After median follow-up of 22.9 months (range 4.5 - 81.1 months),

92% of 50 observed cases had partial (n = 32) or complete response.

Within a median of 10 months (range, 2.2 - 48.7 months) after HSCT, relapse

occurred in 13 of 32 patients with initial partial response and in three of

14 patients with complete response. Total mortality rate was 23% (13 deaths

in 57 patients, including TRM of 8.7% (5 of 57) and eight deaths related to

progression (14%). At five years, the probability of progression was 48%

(95% confidence interval [CI] 28% - 68%), and the projected survival was 72%

(95% CI, 59% - 75%).

³This EBMT/EULAR report showed that response in two thirds of the patients

after HSCT was durable with an acceptable TRM,² the authors write. ³It

confirmed that autologous HSCT is feasible, with low mortality in carefully

selected patients, and allows impressive initial clinical responses which

had not been previously obtained with any other therapeutic intervention in

severe SS.²

Based on these results, prospective, randomized trials are underway. The

Horten Foundation and the Délégation Régionale à la Recherche Clinique

Assistance Publique-Hôpitaux de Paris partly supported this study.

Ann Rheum Dis. 2004;63:974-981