Hematopoietic stem cell transplantation for curing children with severe autoimmune diseases: Is this a valid option?

Paediatric Oncology and Haematology Unit Lalla Seràgnoli, Department of Paediatrics, University of Bologna Sant'Orsola-Malpighi Hospital, Bologna, Italy.
Pediatric Transplantation (Impact Factor: 1.44). 04/2012; 16(5):413-25. DOI: 10.1111/j.1399-3046.2012.01691.x
Source: PubMed


The cure of children with severe AD, especially patients with severe, progressive, and therapy-resistant autoimmunity, represents a challenge for current medical practice. The idea of HSCT as a promising therapeutic opportunity was borne accidentally from finding patients who, after undergoing HSCT for a hematological indication, were cured of a concomitant AD. Thus, over the last two decades, HSCT has been extensively investigated, and it has become an appealing therapy for rheumatological (juvenile rheumatoid arthritis, systemic lupus erythematosus, systemic sclerosis) and hematological diseases (immune cytopenias). Recently, interesting results have been also described in type 1 diabetes mellitus and Crohn's disease. Although the use of HSCT has been steadily rising in the last few years, many questions are still open, especially after the discoveries of many new biological agents. Given the low incidence of ADs in children, most of the data about the use of the HSCT for these diseases are taken from a mixed cohort of adults and children. The aim of this review is to summarize the published studies and to try to answer the question as to whether this procedure can be considered a promising approach.

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