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Published on: August 9, 2022
Blood stem cells as therapy for severe lupus
Gabor G Illei1, Steven Z Pavletic
1National Institute of Dental and Craniofacial Research, Sjögren's Syndrome Clinic, 9000 Rockville Pike, Bethesda, MD 20892-1190, USA. illeig@mail.nih.gov
High-dose immunosuppression followed by autologous haematopoietic stem cell therapy has the promise of long-term response in systemic lupus erythematosus (SLE), but is associated with an increased risk of serious short?term complications. Haematopoietic stem cell transplantation induced major clinical responses in approximately 65% of patients with SLE who failed standard therapies. In some of these patients such responses are durable for at least several years, but the curative potential of this procedure in severe SLE is still unknown. Procedure-related mortality in earlier studies was up to 12%, but is < 5% in more recent experiences from larger single-centre studies. Allogeneic haematopoietic stem cell therapy from HLA-matched donors holds a strong promise for cure, and newer, much safer transplantation regimens are now available. To accomplish the best therapeutic and scientific results, it is necessary to treat all patients in carefully planned innovative protocols conducted by specialised teams of lupus specialists and transplanters. All immunoablative protocols should incorporate studies of immune reconstitution to understand the mechanisms of cure or failure.
High-dose immunosuppression followed by autologous haematopoietic stem cell therapy has the promise of long-term response in systemic lupus erythematosus (SLE), but is associated with an increased risk of serious short?term complications. Haematopoietic stem cell transplantation induced major clinical responses in approximately 65% of patients with SLE who failed standard therapies. In some of these patients such responses are durable for at least several years, but the curative potential of this procedure in severe SLE is still unknown. Procedure-related mortality in earlier studies was up to 12%, but is < 5% in more recent experiences from larger single-centre studies. Allogeneic haematopoietic stem cell therapy from HLA-matched donors holds a strong promise for cure, and newer, much safer transplantation regimens are now available. To accomplish the best therapeutic and scientific results, it is necessary to treat all patients in carefully planned innovative protocols conducted by specialised teams of lupus specialists and transplanters. All immunoablative protocols should incorporate studies of immune reconstitution to understand the mechanisms of cure or failure.
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