Autologous stem cell transplantation in Behcet ' s disease: a retrospective study
Puyade, M. Patel, Amit Lim, Y. L. Blank, N. Badoglio, M. Gualandi, F. Ma, D. Maximova, N. Alexander, T. Snowden, J.
Puyade, M.
Patel, Amit
Lim, Y. L.
Blank, N.
Badoglio, M.
Gualandi, F.
Ma, D.
Maximova, N.
Alexander, T.
Snowden, J.
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Abstract
Background: Behcet´s disease (BD) is a rare autoimmune
disease mostly presenting with recurrent oral and genital
aphthous ulcers, and uveitis. Other common symptoms
include gastrointestinal, vascular, neurological and articular
manifestations1. Treatment is based on chronic immunosuppression
with conventional disease modifying or targeted
biologic drugs2, although some refractory patients
have been treated by autologous hematopoietic stem cell
transplantation (AHSCT)3. This study aims to evaluate the
outcome of AHSCT in adult patients with BD.
Methods: Adults who received AHSCT primarily for
BD (according to International Classification Criteria) were
identified retrospectively within the EBMT registry. Treating
physicians were surveyed to produce a retrospective
evaluation of outcomes. Complete Remission (CR) was
defined as no evidence of disease activity and Partial
Remission (PR) was defined as any documented clinical
and/or laboratory response in patients that is less than CR.
Results: We retrospectively collected the data from 8 out
of 9 cases reported to the EBMT registry and extracted data
of 2 further patients from published literature4. Four were
female, median age at onset of BD was 24 years (range 9-
50). Median age at AHSCT was 32 years (27-51). Patients
had received median 4 (2-12) prior lines of therapy: 89 % of
the patients were treated with corticosteroids, and 50 %
received either methotrexate, antiTNFa or cyclophosphamide.
All had active disease before mobilization, which
was performed with cyclophosphamide and G-CSF in 9
patients and G-CSF alone in 1 patient. Conditioning regimen
was Melphalan 200mg/m² in 5 patients, BEAM in 3
patients, and Cyclophosphamide 200mg/kg plus
antithymocyte-globulin (ATG) in 2 patients. Median
follow-up was 48 months (range 6-120 months). No TRM
was reported, three patients had infectious complications
and a single patient had paroxysmal atrial fibrillation, lineassociated
deep venous thrombosis and depression. At
6 months, 6 patients were in PR with corticosteroid maintenance
and 3 in CR without any further treatment. There was one relapse with pan-uveitis. One patient failed to
respond to AHSCT and proceeded to rescue with allogeneic
HSCT, but died five years post-transplant from chronic
GvHD and CMV infection. Otherwise, no late complications
in patients treated with AHSCT were reported.
Conclusions: AHSCT is feasible and safe in multirefractory
patients with BD. Although treatment-free CR
was achieved in only 3 of 10 patients analyzed, AHSCT has
the potential to stabilize BD in patients who fail to respond
to conventional therapies. Further evidence, ideally from
prospective studies, are warranted to determine whether
AHSCT should be considered a standard of care in
treatment-resistant BD.
Affiliation
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Date
2020
Publisher
Collections
Keywords
Type
Meetings and Proceedings
Citation
Puyade M, Patel A, Lim YL, Blank N, Badoglio M, Gualandi F, et al. Autologous stem cell transplantation in Behcet ' s disease: a retrospective study. Bone Marrow Transplantation. 2020;55(SUPPL 1):224-5.