Hematopoietic stem cell transplantation for systemic lupus erythematosus, the antiphospholipid syndrome and bullous skin diseases.
Systemic lupus erythematosus (SLE) is considered the paradigm of autoimmune diseases (AD), and the murine models are known to be curable by means of allogeneic hematopoietic stem cell transplantation (HSCT). However autologous transplantations were predominantly utilized in the clinic, starting from 1996, and by now well over 150 very severe patients have been transplanted worldwide. Transplant-related mortality (TRM) in 153 cases was 7%, with a wide center effect (from 0-2% to 13%). The disease arresting effect was dramatic even in patients on dialysis, although ASCT should not be considered a last resource, salvage therapy, but a disease- modifying intervention to be utilized in the early stages of patently aggressive disease. The autoimmune biological parameters are consistently modified, although some degree of ANA-positivity generally persists. Similar encouraging results have been obtained in the primary antiphospholipid syndrome (APS) and in bullous disorders of the skin.
Marmont
Alberto M
AM
Divisione di Ematologia e Trapianto di Cellule Staminali, Azienda Ospedaliera-Universitaria S.Martino, Genoa, Italy. alberto.marmont@hsanmartino.it
Burt
Richard K
RK
D000328:Adult; D016736:Antiphospholipid Syndrome / Q000276:immunology / Q000628:therapy; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008180:Lupus Erythematosus, Systemic / Q000276:immunology / Q000628:therapy*; D012872:Skin Diseases, Vesiculobullous / Q000276:immunology / Q000628:therapy; D055815:Young Adult