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Bone Marrow Transplantation (2008) 41, S96S99 & 2008 Nature Publishing Group All rights reserved 0268-3369/08 $30.00
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ORIGINAL ARTICLE
Haematopoietic SCT in autoimmune diseases in children: rationale and new perspectives
M Rabusin, M Andolina, N Maximova, on behalf of the EBMT Paediatric and Autoimmune Diseases Working Parties
Bone Marrow Transplantation Unit, Department of Paediatrics, Institute of Maternal and Child Health Burlo Garofolo, Trieste, Italy
The possible role of haematopoietic SCT (HSCT) for the treatment of severe autoimmune diseases was originally supported by animal experiments and remission of concomitant autoimmune diseases in patients undergoing transplantation for haematological disorders. Since 1996, over 100 procedures were performed in children with different severe autoimmune diseases such as juvenile idiopathic arthritis, systemic lupus erythematosus, systemic sclerosis, immune cytopaenias and Crohns disease. This review tries to summarize the published data on efcacy and toxicity of HSCT in this group ofpatients. Bone Marrow Transplantation (2008) 41, S96S99; doi:http://dx.doi.org/10.1038/bmt.2008.64
Web End =10.1038/bmt.2008.64 Keywords: haematopoietic SCT; autoimmune disease; children
Introduction Autoimmune diseases (ADs) affect around 35% of the general population.1,2 The majority of ADs are controlled by conventional therapies acting on the immune system. Such treatments are rarely curative and they may contribute to long-term morbidity and mortality. Moreover, there is a hard core of refractory/relapsing treatment-resistant forms of ADs for which the term malignant ADs has been appropriately proposed.3,4
Studies from animal models showed that both allo-SCT and auto-SCT were able to cure some spontaneous and induced ADs.57 In human beings, it has been documented that the transfer of a broad range of ADs from donor to recipient may occur, including diabetes mellitus, thyroiditis, psoriasis, myasthaenia gravis, coeliac disease, ulcerative colitis and immune thrombocytopaenic purpura.8 A few
anecdotal reports showed that allogeneic haematopoietic SCT (allo-HSCT) and autologous haematopoietic SCT (auto-HSCT) performed for a coexisting haematological condition either succeeded in curing patients with rheuma-
toid arthritis and other autoimmune disorders, or resulted in long-lasting remission.911
Allogeneic transplant is believed to be effective by reducing self-reactive lymphocytes during the conditioning regimen, hence eliminating residual immune cells by a graft-vs-autoimmune effect of the healthy donor-derived immune system. On the other hand, the efcacy of auto-HSCT may result from a similar ablation of self-reactive lymphocytes during conditioning, followed by the induction of self-tolerance thanks...