Background Evidence regarding haematopoietic stem cell transplantation (HSCT) in refractory Behçet's Syndrome (BS) is limited. A systematic review in 2014 identified 20 patients with BS who had undergone HSCT, from which 9 patients underwent HSCT with refractory BS as the primary indication. Of these 20 patients: 15 achieved complete remission, 1 patient achieved partial remission, 2 patients relapsed, 1 patient died and 1 had no outcome reported. Nine patients had reported complications, 5 patients had no complications and 6 patients were missing data. All data was sourced from single case studies (1).

Objectives Outline the indications, outcomes and complications of HSCT in a case series of 4 patients with BS managed at a single centre of excellence.

Methods Case notes were reviewed for 4 patients from the Liverpool Behçet's Centre of Excellence (LBCE) who have undergone HSCT.

Results The primary indication for 3 of the 4 patients who have undergone autologous HSCT at LBCE was refractory BS. Patient 1 underwent HSCT for multiple myeloma, but suffered from severe BS with neurological and venous involvement and is now in partial remission with occasional mild muco-cutaneous ulceration, not requiring systemic immunosuppression. Patient 2 underwent HSCT for severe refractory neuro-Behçet's. This patient is now in complete remission after commencing azathioprine for oral ulceration. The indications for HSCT in patients 3 and 4 were severe muco-cutaneous ulceration and dermatological involvement refractory to numerous biologic agents with high ongoing steroid dependency. Patient 3 also had previous thrombophlebitis and patient 4 had previous gastro-intestinal ulceration. They are both currently in complete remission but follow up time is limited. Patient 3 had a post-HSCT complication of pneumonia and mucositis which resolved without persistent morbidity, patient 4 had an upper limb deep vein thrombosis (DVT) as an inpatient.

Conclusions HSCT is an effective treatment modality for severe refractory BS and can result in medication free remission. However, the associated risks should be considered and alternative treatment options deliberated or exhausted before opting for this treatment modality.

References

1. Soysal T, Salihoğlu A, Esatoğlu SN, Gültürk E, Eşkazan AE, Hatemi G, et al. Bone marrow transplantation for Behçet's disease: a case report and systematic review of the literature. Rheumatology. 2014; 53(6):1136–41.

References

Disclosure of Interest None declared