Autologous haematopoietic stem cell transplants for autoimmune disease – feasibility and transplant-related mortality

Abstract

Summary:This ongoing multicentre prospective phase I/II trial enrolled 74 consecutive patients from 22 centres worldwide with severe autoimmune disease, 35 with rheumatological disorders, 31 with neurological, five with haematological and three with vasculitides. They were treated with autologous peripheral blood or bone marrow transplants according to predetermined criteria. Two patients died after mobilisation before transplant. Seventy-two patients were given 73 transplants, seven bone marrow, and 66 mobilised peripheral blood stem cell transplants. The graft was manipulated to remove T and/or B cells in 43 cases. All 73 transplants engrafted. Five patients died of transplant-related complications: two from bleeding, three from infections. Two patients died of progressive disease. The transplant-related mortality at 1 year of 9% (1-17%; 95% CI) is comparable to the transplant-related mortality of 6% (3-9%; 95% CI) in patients transplanted during the same period in Europe for non-Hodgkin's lymphoma in sensitive relapse (P = 0.39). Sixty patients are evaluable for response, 40 patients (65%) showed some improvement in their disease. Haematopoietic stem cell transplants are feasible for patients with severe refractory autoimmune disease. Transplant-related mortality is comparable to results in patients with non-Hodgkin's lymphoma in responsive relapse. Two-thirds of the patients show at least some response. These preliminary data are promising. Although associated with considerable risk, randomised trials comparing autologous stem cell transplants to conventional therapy are warranted.

Resolution of psoriasis following allogeneic bone marrow transplantation for chronic myelogenous leukemia: Case report and review of the literature

“…Recently, autologous hematopoietic stem cell transplantation for autoimmune diseases has been reported [19,20]. However, the data are preliminary, and transplant-related mortality is comparable to patients transplanted for non-Hodgkin's lymphoma in sensitive relapse [20].…”

“…However, the data are preliminary, and transplant-related mortality is comparable to patients transplanted for non-Hodgkin's lymphoma in sensitive relapse [20]. The recurrence of co-existing psoriasis has also been reported in 3 patients who received autologous BMT or stem cell transplantation for their hematologic malignancy [21].…”

Resolution of psoriasis after allogeneic bone marrow transplantation for chronic myelogenous leukemia: late complications of therapy

“…3 However, even autologous BMT is associated with substan-tial risk of early toxicity, with a reported TRM of 9% when used to treat patients with autoimmune diseases. 9 This TRM approaches that recently observed with some low intensity or non-myeloablative conditioning regimens used for matched related donor transplantation, 10 suggesting that the latter may be a reasonable option for some patients, especially those whose autoimmune disease may be genetically determined.…”

Pseudoautologous blood stem cell transplantation for refractory chronic graft-versus-host disease

“…[1][2][3] Chemotherapy at immunoablative doses is intended to eliminate autoreactive lymphocyte clones or to interrupt autoimmune regulatory circuits. Reinfusion of stem cells for hematopoietic rescue is also intended to reconstitute a new immune system that may be 'naive' and immunologically tolerant towards putative autoantigens.…”

“…Acitretin and photopheresis were restarted to maintain the response. During the next 3 1 2 months, although she was noncompliant in taking cyclosporine, her symptoms were stable and after the addition of azathioprine, the prednisone dose was tapered to less than 10 mg/day. The skin was much softer, without erythema and she could perform nearly full extension and flexion of her ankles.…”