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Autologous stem cell transplantation in Behcet ' s disease: a retrospective studyPuyade, M.; Patel, Amit; Lim, Y. L.; Blank, N.; Badoglio, M.; Gualandi, F.; Ma, D.; Maximova, N.; Alexander, T.; Snowden, J.; et al. (2020)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.