Long term follow up of patients with severe Systemic Sclerosis (SSc) and Rheumatoid Arthritis (RA) who have undergone Haematopoietic Stem Cell Transplantation (HSCT).

Autologous stem cell transplantation (HSCT) is a procedure that is usually used to give high doses of chemotherapy to patients with blood cancers followed by stem cell infusion so that a new blood and immune system can regrow. Over the last 15 years, HSCT has become much safer. At the same time, numerous lines of evidence converged to suggest that a small number of patients with very severe immune conditions could also be treated by HSCT. The evidence came from patients who had chemotherapy for co-existant blood tumours and had an auto-immune disease (AID). Often these patients had prolonged remissions of their AID. In addition, there are animal models of HSCT in AID showing prolonged remissions with HSCT. AID patients suffer from an immune attack on their own body causing tightening of the skin (Systemic Sclerosis), spinal cord and brain problems (Multiple Sclerosis) and multiple other organs in the body (Systemic Lupus Erythromatosis, Crohn's disease and vasculitis). In some cases, people with auto-immune conditions can die from their condition, particularly when the disease has failed multiple treatments. It is possible to select the very sickest AID patients for more intensive therapy with HSCT and provide them with a lasting remission. The procedure requires a two step process. The first step requires chemotherapy with one dose of a drug called cyclophosphamide (2grams) intravenously along with GCSF 10ug/kg subcutaneously x 7 days (a hormone to stimulate the bone marrow) to help stem cells to be collected via a vein in your arm. Within 2 months, patients come into hospital for the second step. Here patients will have high doses of intravenous chemotherapy (cyclophosphamide 200mg/kg over 4 days along with Anti-thymocyte globulin 10mg/kg x 4 days) that also intensely suppresses the immune system. After these 4 days the patient's own stem cells are transfused so they can re-grow a new immune system and protect the patients from the toxic effects of the chemotherapy. It takes about 14 days for the new stem cells to grow and then we will follow patients carefully over the next 10 years (initially every 3-6 months but after 1 year just annually) to see if the immunosuppression controls your auto-immune disease and that there are no long term sequelae.