AUTHOR=Greco Raffaella , Labopin Myriam , Badoglio Manuela , Veys Paul , Furtado Silva Juliana M. , Abinun Mario , Gualandi Francesca , Bornhauser Martin , Ciceri Fabio , Saccardi Riccardo , Lankester Arjan , Alexander Tobias , Gennery Andrew R. , Bader Peter , Farge Dominique , Snowden John A. 

TITLE=Allogeneic HSCT for Autoimmune Diseases: A Retrospective Study From the EBMT ADWP, IEWP, and PDWP Working Parties

JOURNAL=Frontiers in Immunology

VOLUME=10

YEAR=2019

URL=https://www.frontiersin.org/journals/immunology/articles/10.3389/fimmu.2019.01570

DOI=10.3389/fimmu.2019.01570

ISSN=1664-3224

ABSTRACT=<p><bold>Background:</bold> This retrospective study assessed the use and long-term outcome of allogeneic hematopoietic stem cell transplantation (HSCT) in patients with severe autoimmune diseases (ADs), reported to the European Society for Blood and Marrow Transplantation (EBMT) registry.</p><p><bold>Methods:</bold> Between 1997 and 2014, 128 patients received allogeneic HSCT for various hematological (<italic>n</italic> = 49) and non-hematological (<italic>n</italic> = 79) refractory ADs. The median age was 12.7 years (0.2–62.2). Donors were syngeneic for seven, matched related for 46, unrelated for 51, haploidentical for 15, and cord blood for nine patients.</p><p><bold>Results:</bold> The incidence of grades II-IV acute graft-vs.-host disease (GvHD) was 20.8% at 100 days. Cumulative incidence of chronic GvHD was 27.8% at 5-years. Non-relapse mortality (NRM) was 12.7% at 100-days. Overall survival (OS) and Progression-Free Survival (PFS) were 70.2 and 59.4% at 5-years, respectively. By multivariate analysis, age &lt;18 years, males, and more recent year of transplant were found to be significantly associated with improved PFS. Reduced conditioning intensity was associated with a lower NRM. On a subgroup of 64 patients with detailed information a complete clinical response was obtained in 67% of patients at 1-year.</p><p><bold>Conclusions:</bold> This large EBMT survey suggests the potential of allogeneic HSCT to induce long-term disease control in a large proportion of refractory ADs, with acceptable toxicities and NRM, especially in younger patients.</p>