Autologous Hematopoietic Stem Cell Transplantation in Autoimmune Diseases

Claudio Annaloro; Francesco Onida; Giorgio Lambertenghi Deliliers


Expert Rev Hematol. 2009;2(6):699-715. 

In This Article

Rheumatoid Arthritis

Rheumatoid arthritis is an AD characterized by diffuse joint involvement and less commonly visceral compromise. Conventional treatment has long relied on anti-inflammatory and immunosuppressive drugs but has been recently surpassed by the introduction of multiple effective biological agents. RA is a disabling AD but does not reduce life expectancy; therefore, the referral of RA patients to autologous HSCT seems at glance somewhat unwise. On the other hand, it has been suggested that subsets at highly adverse prognosis can be recognized also among refractory RA patients.[121] Since at least some of these patients could be responsive to autologous HSCT, in these patients the procedure seems to be cost effective. Some effort has been paid to the effort of recognizing both adverse prognosis patients among refractory RA ones and, among these, HSCT responsive patients. Anti-cyclic citrullinated protein autoantobodies have been proposed as markers of refractory RA;[122] according to Teng et al.,[122] only patients with low-avidity antibodies and a decreasing titer after HSCT have a chance of responding to autografting. It has also been speculated that RA patients with some degree of hematopoietic impairment could be the best suited candidates to autografting.[25]

The interest for autologous HSCT in RA has been raised in a recent historical phase where the problem of refractoriness was felt as primary and the potential of newer biological agents had not yet been fully disclosed. This led to the appearance of some monocentric series, where autologous HSCT was mostly performed according to common AD strategies, including CD34 selection and immunoablative, rather than myeloablative, conditioning.[123] The published experiences showed that TRM was a rare event.[9,123,124] On the other hand, response rate was not that encouraging and long-term response was uncommon[57,58,125] or at least still to be established,[126,127] although the available results were differently evaluated.[124] It should be considered that different strategies have been pursued concerning the choice of conditioning regimen and the policies of T-cell depletion. Perplexity is reinforced by the results of the EBMT survey.[58,123] Better definition of inclusion criteria was frequently claimed as the way to optimize risk–benefit ratio.[128] For this last purpose, the EBMT Autologous Stem cell Transplantation International Rheumatoid Arthritis (ASTIRA) Phase III trials has been designed.[129]

Over the last few years, newer biological drugs have proved to have a high therapeutic potential so that the development of rationale combinations cannot infrequently overcome the problem of resistance.[130] The concurrence of the availability of new effective drugs and of unsatisfying results with HSCT, forces a rethinking of autologous HSCT in RA, which could probably be offered to a small minority of properly selected patients.[10]


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