Best Pract Res Clin Haematol 2001 Dec;14(4):755-76
Gratwohl A, Passweg J, Gerber I, Tyndall A; International Stem Cell Project for Autoimmune Diseases.
Division of Haematology, Department of Internal Medicine, Kantonsspital Basel, Switzerland.
Much progress has been made in the field of haemopoietic stem cell transplants (HSCTs) for severe autoimmune disorders.
Theoretical considerations, animal data and anecdotal evidence suggested some time ago that intensive immunoablation followed by autologous HSCT could restore normal immune reactivity in patients with severe autoimmune disorders.
Based on a concept statement issued in 1995, two European societies, the European League Against Rheumatism (EULAR) and the European Group for Blood and Marrow Transplantation (EBMT) began collecting phase I/II trial data in an international collaborative network.
Sufficient information from more than 350 patients allows a preliminary assessment with level three evidence.
Autologous HSCTs can induce remissions in all disease categories tested so far.
Remissions can be transient or durable.
HSCTs are associated with significant morbidity and mortality.
Treatment-related mortality (TRM) is near 10% at 1 year and is associated with the intensity of the conditioning and the stage of the disease at the time of transplant.
Marked interdisease differences exist.
There are few data available in haematological autoimmune diseases, more in systemic sclerosis (SSc), systemic lupus erythematosus (SLE), juvenile idiopathic arthritis (JIA) and multiple sclerosis (MS).
Patient selection has been recognized as a crucial element from the phase I-II trials.
Patients with advanced disease, severely compromised organ function or irreversible organ damage should not be considered as candidates for HSCT.
Prospective randomized studies should now determine the value of HSCT compared to standard therapy.
Such trials are ongoing for patients with systemic sclerosis (ASTIS trial--Autologous Stem Cell Transplantation International Scleroderma Trial) or are planned for patients with multiple sclerosis (ASTIMS trial--Autologous Stem Cell Transplantation International Multiple Sclerosis Trial) and rheumatoid arthritis (ASTIRA trial--Autologous Stem Cell Transplantation International Rheumatoid Arthritis Trial).
More phase II data are needed for other indications such as SLE and JIA.