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

Hematopoietic Immune Cytopenias

Autologous HSCT has been occasionally proposed for the treatment of refractory hematopoietic immune cytopenia. This approach raises some perplexity since autoimmune pancytopenias are among the most frequent autoimmune events complicating the course of autologous HSCT performed because of a neoplastic disease and even a AD.[39,40,148–153] On the other hand, even though most patients with these diseases are not expected to die, patients with severe, refractory, single or combined cytopenias are at high risk of developing lethal complications, either attributable to the low platelet or white blood cell counts, the low hemoglobin levels or to the complications of previous ineffective treatments.[154,155] As a consequence, autologous HSCT in autoimmune cytopenias can be regarded as an extemporary choice in selected patients rather than the results of a planned therapeutic strategy. According to this assumption, case reports outnumber homogeneous series,[156,157] so that no definite conclusions can be drawn regarding selection criteria and mobilization, harvest manipulation or conditioning regimens, which are notably heterogeneous.[155] This assumption is confirmed by the EBMT registry survey, showing a high TRM and an extremely variable disease response, with refractory disease, long-term remission and relapse being outcomes approximately equally represented.[155] Moreover, the possibility of referring a patient to autologous HSCT should be weighed alongside the development of newer drugs and, therefore, aligned with the evolving concept of refractoriness. At the same time, since newer drugs are not devoid of long-term effects; also their risk–benefit ratio should be compared with that of autologous HSCT in the single patient.


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