Results of HSCT for Autoimmune Diseases
Data on the effect of HSCT on patients suffering from autoimmune diseases are obtained from retrospective case series, the EBMT/European League Against Rheumatism (EULAR) database, and from published phase I/II trials.
Up until March 2008, the EBMT/EULAR database included results from nearly 1,000 patients who received an HSCT for the indication autoimmune disease registered. Among these patients, more than 900 received an autologous HSCT and around 50 an allogeneic HSCT. Indication for autologous HSCT have been multiple sclerosis (MS) in 353 patients, SSc in 176, systemic lupus erythematosus (SLE) in 85, rheumatoid arthritis in 86, and other autoimmune diseases in fewer patients. (See Table 1, below left.) Most often, peripheral blood stem cells have been used as a stem-cell source but, in 65 cases, bone marrow was used. In more than one-third of the patients, in vitro CD34 positive selection had been performed.
The results of the phase I/II trials and registered case reports have been published.2 In essence, two publications have shown that around 50% of SLE patients achieved remission at some stage post-transplant,4,5 and, in MS and SSc, durable remission occurred in one-third. (See Figure 3, p. 22.) Transplant-related mortality was highest in the multicenter SLE series, but in general, that is now less, due to more stringent patient selection.