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Clinical Trial
. 2019 Jul 4:10:1570.
doi: 10.3389/fimmu.2019.01570. eCollection 2019.

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

Raffaella Greco  1 , Myriam Labopin  2 , Manuela Badoglio  2 , Paul Veys  3 , Juliana M Furtado Silva  3 , Mario Abinun  4 , Francesca Gualandi  5 , Martin Bornhauser  6 , Fabio Ciceri  1 , Riccardo Saccardi  7 , Arjan Lankester  8 , Tobias Alexander  9 , Andrew R Gennery  4 , Peter Bader  10 , Dominique Farge  11   12   13   14 , John A Snowden  15
Affiliations
Clinical Trial

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

Raffaella Greco et al. Front Immunol. .

Abstract

Background: 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. Methods: Between 1997 and 2014, 128 patients received allogeneic HSCT for various hematological (n = 49) and non-hematological (n = 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. Results: 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 <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. Conclusions: 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.

Keywords: allogeneic hematopoietic stem cell transplantation; autoimmune diseases; hematological autoimmune diseases; long-term outcome; non-hematological autoimmune diseases.

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Figures

Figure 1
Figure 1
Relapse incidence (RI) and progression-free survival (PFS). RI and PFS in overall population (n = 128) are shown in (A,B) respectively.
Figure 2
Figure 2
OS and cumulative incidence of NRM. OS and cumulative incidence of NRM in overall population (n = 128) are shown in (A,B), respectively.
See this image and copyright information in PMC

Comment in

References

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