Puyade, Mathieu, Patel, Amit, Lim, Yeong Jer, Blank, Norbert, Badoglio, Manuela, Gualandi, Francesca, Ma, David D, Maximova, Natalia, Greco, Raffaella, Alexander, Tobias et al (show 1 more authors)
(2021)
Autologous Hematopoietic Stem Cell Transplantation for Behcet's Disease: A Retrospective Survey of Patients Treated in Europe, on Behalf of the Autoimmune Diseases Working Party of the European Society for Blood and Marrow Transplantation.
, Switzerland.
Abstract
<h4>Background</h4>Behçet's Disease (BD) is an autoimmune disease mostly presenting with recurrent oral and genital aphthosis, and uveitis. Patients are rarely refractory to immunosuppressive treatments. Autologous hematopoietic stem cell transplantation (aHSCT) is a standard of care in other autoimmune diseases. Some patients with BD have been treated with aHSCT based on compassionate use.<h4>Objectives</h4>Evaluate the outcome of aHSCT in adult patients with BD treated in member centers of the European Society for Blood and Marrow Transplantation (EBMT).<h4>Methods</h4>Adults who received aHSCT primarily for BD were identified retrospectively in the EBMT registry and/or in published literature. Data were extracted from either medical records of the patient or from publications.<h4>Results</h4>Eight out of 9 cases reported to the registry and extracted data of 2 further patients from literature were analyzed. Four were female, median age at onset of BD was 24y (range 9-50). Median age at aHSCT was 32y (27-51). Patients had received median 4 (2-11) previous lines of therapy (89% corticosteroids, 50% methotrexate, anti-TNFα therapy or cyclophosphamide). All patients had active disease before mobilization. Conditioning regimen was heterogeneous. Median follow-up was 48 months (range 6-240). No treatment-related mortality was reported. This procedure induced complete remission (CR) in 80%, partial remission in 10% and lack of response in 10% of the patients. Relapse rate was 30% (2 relapses in patients in CR and 1 relapse in the patient in PR) with panuveitis (n=1), aphthosis (n=2) and arthralgia (n=1). Six patients were in CR. No late complications were reported.<h4>Conclusion</h4>aHSCT has an acceptable safety profile and represents a feasible and relatively effective procedure in severe and conventional treatment-resistant cases of BD and has the potential to stabilize BD in patients with life-threatening involvements.
Item Type: | Conference or Workshop Item (Unspecified) |
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Uncontrolled Keywords: | Behç, et’, s disease, autologous stem cell transplantation, efficacy, toxicity, immune reset |
Divisions: | Faculty of Health and Life Sciences Faculty of Health and Life Sciences > Institute of Systems, Molecular and Integrative Biology |
Depositing User: | Symplectic Admin |
Date Deposited: | 12 Jul 2021 09:44 |
Last Modified: | 18 Jan 2023 21:36 |
DOI: | 10.3389/fimmu.2021.638709 |
Open Access URL: | http://doi.org/10.3389/fimmu.2021.638709 |
Related URLs: | |
URI: | https://livrepository.liverpool.ac.uk/id/eprint/3129738 |