Haematological stem cell transplantation in the treatment of severe autoimmune diseases: first experiences from an international project.
The benefit:risk ratio of HSCT in autoimmune disease appears to justify the initiation of prospective controlled comparative studies. The comparator arm is open, one possibility being mobilized (Cy 2 or 4 g/m2 + G-CSF), but not transplanted. Inclusion and exclusion criteria for different disease categories need to be standardized, as do outcome measurements. In Europe, the EBMT has established a new working party for autoimmune disease with representatives from all involved groups, including the USA. A similar parallel group is being established in North America. The aim will be consensus and standardization of disease-specific aspects. Standardization of immune reconstitution parameters could prove critical in the understanding of autoimmune mechanisms, with early guidelines being developed and available to interested groups. Data collection is critical, with advanced discussions on common registration forms between the EBMT and the American Bone Marrow Transplantation Registry (ABMTR) already taking place. Common detailed disease-specific clinical data forms are now a top priority, so that data from the two major databases may be compared. All patients fulfilling entry criteria should be registered and followed long term, including those not able to be treated for non-medical reasons, such as insurance. This will provide a prospective 'conventional treatment' control group. Regular and flexible liaison between the data managers of both groups will be encouraged, as with regulatory authorities such as the Food and Drug Administration. The following such meeting was in Basel, 8-10 October 1998.
Tyndall
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Department of Rheumatology, University Hospital Felix Platter Spital, Basel, Switzerland.
D000328:Adult; D001327:Autoimmune Diseases / Q000628:therapy*; D015897:Comorbidity; D018380:Hematopoietic Stem Cell Transplantation / Q000706:statistics & numerical data; D006801:Humans; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous / Q000706:statistics & numerical data; D016896:Treatment Outcome