Autologous Hematopoietic Stem Cell Transplantation for Behçet’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

Abstract

BackgroundBehçet’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.ObjectivesEvaluate the outcome of aHSCT in adult patients with BD treated in member centers of the European Society for Blood and Marrow Transplantation (EBMT).MethodsAdults 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.ResultsEight 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.ConclusionaHSCT 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.

Indications for haematopoietic cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2022

“…Evidence is continually evolving for auto-HCT, predominantly in MS [208][209][210][211][212], and SSc [213][214][215][216][217], for which auto-HCT can be regarded as a standard of care. There is evidence to support treatment of carefully selected patients with Crohn's disease [218,219], systemic lupus erythematosus [220,221], neuromyelitis optica [222,223], chronic inflammatory demyelinating polyradiculoneuropathy [224], myasthenia gravis [225], stiff person syndrome [226,227], systemic vasculitis (ANCA positive [228], Takayasu [229], Behcȩt's disease [230]) and refractory coeliac disease [231]. Altogether, available data support considering auto-HCT as an efficacious, one-off intensive therapeutic procedure for severe and refractory AD.…”

Hematopoietic stem cell transplantation and cellular therapies for autoimmune diseases: overview and future considerations from the Autoimmune Diseases Working Party (ADWP) of the European Society for Blood and Marrow Transplantation (EBMT)

“…Based on these data, SSc is recommended as ‘standard indication’ for autologous HSCT and endorsed by updated recommendations of the European League Against Rheumatism [ 71 ]. Rarer indications of HSCT for rheumatic diseases with positive results from retrospective EBMT studies include ANCA-associated vasculitis [ 72 ], Takayasu arteritis [ 73 ] and Behcet’s disease [ 74 ].…”

Autologous stem-cell transplantation in systemic sclerosis-associated interstitial lung disease: early action in selected patients rather than escalation therapy for all

“…21 In addition, recent reports on experiences with SCT in systemic lupus erythematosus, Behcet’s disease and vasculitis illustrate the need for this treatment in refractory cases of other rare autoimmune conditions. 22,23…”

“…21 In addition, recent reports on experiences with SCT in systemic lupus erythematosus, Behcet's disease and vasculitis illustrate the need for this treatment in refractory cases of other rare autoimmune conditions. 22,23 SCT is thought to reset the immune system through elimination of autoreactive immune cells and regeneration of a new, rebalanced immune system. The exact mechanisms driving this reset are, however, not completely known.…”