Abstract Perspective: Efficacy of allogeneic hematopoietic cell transplantation for autoimmune diseases


Autoimmune diseases are usually treatable, but rarely curable. Allogeneic blood or bone marrow transplantation (BMT) has been shown in case reports or small series to potentially result in long-term remission (cure?). In this retrospective study we reviewed cases of patients who had both an autoimmune disease and a blood/marrow disease like leukemia for which BMT was needed. We found that the autoimmune disease entered a long-term remission (cure?) in 7/9 patients with lupus,  8/12 with rheumatoid arthritis (RA),  4/4 with systemic sclerosis (SSc),  13/16 with psoriasis,  11/12 with Behcet’s disease (BD),  14/15 with Crohn’s disease (CD),  5/5 with ulcerative colitis (UC),  4/8 with multiple sclerosis (MS),  and  0/3 with type 1 diabetes (T1DM). However, when a more stringent definition of long-term remission was used, it occurred in only 3/3 patients with lupus,  5/7 with RA,  2/2 with SSc,  3/6 with psoriasis,  3/3 with BD,  10/10 with CD,  3/3 with UC,  1/3 with MS,  and  0/2 with T1DM. Prospective clinical studies are needed to determine the likelihood of long-term remission after BMT, using relatively nontoxic BMT protocols. I have written such a study for rheumatoid arthritis and am searching for a funder of the study.

– Study author Jan Storek, MD, PhD, FACP