Bone marrow transplantation

Some severe auto-immune diseases, including rheumatoid arthritis, require i mmunosuppression to save life or vital organ function. One major limiting f actor in such immunosuppression is the unwanted and dangerous haematoablati on occurring at the same time. It is now possible to give supralethal doses of haematoimmunoablative drugs, and to 'rescue' the patient with haematopo ietic stem cell transplantation. There are case reports of patients with rh eumatoid arthritis who have received bone marrow transplantation, now calle d haematopoietic stem cell transplantation (HSCT), for a conventional indic ation such as aplastic anaemia or malignancy, in whom a long lasting improv ement of the arthritis was observed. However, not all cases have responded, and there are still many open questions concerning optimal treatment regim ens. In addition, some animal model research has supported the concept of H SCT as a permanent treatment of auto-immune disease including arthritis. In the past three years, HSCT has been applied to treat auto-immune disease a lone in around 180 patients, 20 of whom having had rheumatoid arthritis and 13 juvenile arthritis. Early results are encouraging, but many issues requ ire further clarification through co-ordinated clinical trials. The evoluti on of this project, details of the early results, the remaining open questi ons and possible strategies to resolve these are the subject of this chapte r.