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The rise of autologous HCT for autoimmune diseases: what is behind it and what does it mean for the future of treatment? An update on behalf of the EBMT Autoimmune Diseases Working Party

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Autoimmune diseases (ADs) are now the fastest growing disease category for autologous hematopoietic cell transplantation (aHCT). Despite the evolution of competing biological and targeted therapies, there are poor prognosis patients… Click to show full abstract

Autoimmune diseases (ADs) are now the fastest growing disease category for autologous hematopoietic cell transplantation (aHCT). Despite the evolution of competing biological and targeted therapies, there are poor prognosis patients in many ADs who respond unsatisfactorily and for whom the risk: benefit ratio of aHCT is acceptable. The European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) has been central to developments, with over 3000 HCT registrations for ADs. Recent data has improved the evidence-base to support aHCT in MS, systemic sclerosis and Crohn’s disease, along with a wide range of rarer disease indications. Currently, aHCT is in a phase of ‘implementation science’, where aHCT is now being integrated into algorithms of various ADs. Center quality and collaborative experience are important and aHCT as a single one-off procedure may have health economic advantages versus ongoing administration of biological therapies. Mechanistic studies have provided an evolving insight into ‘re-booting’ of the immune system through a re-diversification of naïve and regulatory immune effector cells and, along with clinical trials, may help fine tune transplant technique and improved patient selection through early prognostic biomarkers with the ultimate goal of safe delivery of aHCT with maximal benefit.

Keywords: autoimmune; diseases working; ebmt autoimmune; ahct; autoimmune diseases; working party

Journal Title: Expert Review of Clinical Immunology
Year Published: 2019

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