Hematopoietic stem cell transplantation

Since the start of the international stem cell programme for autoimmune diseases in 1997, 3855 stem cell transplants for autoimmune disease have been registered at the European Group for Blood and Bone Marrow Transplantation (EBMT Registry May 2022) including 787 transplants in systemic sclerosis (SSc), with most patients having received an autologous hematopoietic stem cell transplant (HSCT). The majority of SSc patients had a partial or complete response. In many transplanted SSc patients, morphological improvement such as reduction of skin collagen, normalization of microvasculature, and serum protein profiles was documented, beyond any predicted known effects of intense immunosuppression alone. Three prospective, randomized trials have shown positive outcomes on skin thickening, functional ability, quality of life, and long-term survival despite an early risk of transplant-related mortality and morbidity relating to regimen intensity, age of patient, and comorbidities.