Hematopoietic stem cell transplantation in autoimmune diseases: update from EBMT Autoimmune Diseases Working Party with special reference to Poland

Abstract

Hematopoietic stem cell transplantation (HSCT) is now evolving into a standard treatment in some autoimmune diseases (AD) alongside other modern therapy. The main indications are multiple sclerosis and systemic sclerosis for which HSCT has become an integral and standard-of-care part of treatment algorithms. From 1994 to the beginning of 2021, data from the EBMT Registry indicates that 3,442 patients (60% females, 40% males; 91% adults, 9% pediatric) received 3,514 transplant procedures for autoimmune diseases, with over 90% receiving autologous transplant. Autoimmune diseases are currently the fastest growing indication for autologous HSCT in European Society for Blood and Marrow Transplantation (EBMT), whilst allogeneic HSCT for ADs is mainly restricted to pediatrics, especially diseases with a genetic component. Patient selection plays a key role in providing the best risk/benefit ratio of the procedure. Intensity of conditioning regimen and center experience are also important. Ultimately, the future of HSCT for ADs depends on the standard of care therapy, which influences uptake within national/international disease specialist communities. Further studies are necessary in order to establish relative benefit over current/future standard of care therapy, to establish the best HSCT regimen for each disease, to define mechanisms, develop clinical biomarkers to help select and monitor patients, and to define health economic benefits and public health delivery. We present a current perspective summarizing activity across EBMT, including centers in Poland.