Autologous hematopoietic stem cell transplantation for Behcet's disease: a retrospective survey of patients treated in Europe, on the behalf of the autoimmune diseases working party of the European society for blood and bone marrow transplantation
Puyade, M. Amit, Patel Lim, Y. J. Blank, N. Badoglio, M. Gualandi, F. Ma, D. D. Greco, A. Maximova, N. Alexander, T.
Puyade, M.
Amit, Patel
Lim, Y. J.
Blank, N.
Badoglio, M.
Gualandi, F.
Ma, D. D.
Greco, A.
Maximova, N.
Alexander, T.
Citations
Altmetric:
Abstract
Background/Purpose: Behçet's Disease (BD) is a rare autoimmune disease mostly presenting with recurrent oral and genital aphtosis, ulcers, and uveitis. Rare patients are refractory to conventional treatments. Autologous hematological 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.
Methods: Evaluate the outcome of AHSCT in adult patients with BD treated in member centers of the European Society for Blood and Marrow Transplantation (EBMT). 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.
Results: 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) prior lines of therapy (89% corticosteroids, 50% methotrexate, anti-TNFa or cyclophosphamide). All patients had active disease before mobilization. Conditioning regimen was heterogeneous. Median follow-up was 48 months (range 6-240 months). No treatment-related mortality was reported. At last follow-up, one patient did not respond, 3 patients relapsed with pan-uveitis (n=1), aphtosis (n=2) and arthralgia (n=1). Six patients were in CR without any further treatment. No late complications were reported.
Conclusion: AHSCT is feasible and safe in multi-refractory patients with BD and has the potential to stabilize BD in patients with life-threatening involvements.
Description
Date
2020
Publisher
Collections
Keywords
Type
Meetings and Proceedings
Citation
Puyade M, Amit P, Lim YJ, Blank N, Badoglio M, Gualandi F, et al. Autologous hematopoietic stem cell transplantation for Behcet's disease: a retrospective survey of patients treated in Europe, on the behalf of the autoimmune diseases working party of the European society for blood and bone marrow transplantation. Arthritis & Rheumatology. 2020;72.