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

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.

Non-Responsive Coeliac Disease: A Comprehensive Review from the NHS England National Centre for Refractory Coeliac Disease

“…Immunoablative chemotherapy, followed by autologous haemopoietic stem cell transplantation (aHSCT), now indicated in a range of autoimmune and inflammatory diseases [86][87][88], has been used rarely to treat RCD2, mainly in a single center, with limited evidence that it may prevent progression to EATL. Results of a step-up program, incorporating the use of aHSCT in patients who do not achieve histological responses to cladribine, have been published [89][90][91].…”

Coeliac disease and noncoeliac wheat or gluten sensitivity

“…Cladribine may potentially be used in RCD 2, with it being demonstrated to induce clinical and histological improvement in some patients, but not prevent EATL development [91]. Numerous medical therapies have been trialled in RCD 2, including immunosuppressants [88], ciclosporin [92] and autologous stem cell transplant [93].…”

Nursing interventions in autologous stem cell transplantation for autoimmune diseases

“…Autologous haematopoietic stem cell transplantation (AHSCT) has been used as treatment for severe autoimmune diseases such as multiple sclerosis, systemic sclerosis, idiopathic juvenile arthritis, inflammatory bowel disease and type 1 diabetes (Farge et al., 2010; Snowden et al., 2017). Since 1995, approximately 3,000 transplants for autoimmune diseases have been registered worldwide (Snowden, Badoglio, & Alexander, 2019). Data from the European Society for Blood and Marrow Transplantation (EBMT) indicate improvements, over the past decades, in progression‐free survival, disease relapse and progression and non‐relapse mortality after AHSCT for autoimmune diseases (Snowden et al., 2017), suggesting that centres have earned experience in the management of such patients.…”