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Allogeneic hematopoietic stem cell transplantation for neuromyelitis optica |
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| Authors: | Raffaella Greco MD Attilio Bondanza MD PhD Luca Vago MD PhD Lucia Moiola MD Paolo Rossi MD Roberto Furlan MD PhD Gianvito Martino MD Marta Radaelli MD Vittorio Martinelli MD Maria Rosaria Carbone MSc Maria Teresa Lupo Stanghellini MD Andrea Assanelli MD Massimo Bernardi MD Consuelo Corti MD Jacopo Peccatori MD Chiara Bonini MD Paolo Vezzulli MD Andrea Falini MD PhD Fabio Ciceri MD Giancarlo Comi MD |
| Institution: | 1. Hematology and Bone Marrow Transplantation Unit;2. Experimental Hematology Unit;3. Neurology Department, San Raffaele Scientific Institute, Milan, Italy;4. Neurology Department, Hildebrand Hospital, Brissago, Switzerland;5. Neuroradiology Unit, San Raffaele Scientific Institute, Milan, Italy;6. Vita‐Salute San Raffaele University, Milan, Italy |
| Abstract: | Neuromyelitis optica is a rare neurological autoimmune disorder characterized by a poor prognosis. Immunosuppression can halt disease progression, but some patients are refractory to multiple treatments, experiencing frequent relapses with accumulating disability. Here we report on durable clinical remissions after allogeneic hematopoietic stem cell transplantation in 2 patients suffering from severe forms of the disease. Immunological data evidenced disappearance of the pathogenic antibodies and regeneration of a naive immune system of donor origin. These findings correlated with evident clinical and radiological improvement in both patients, warranting extended clinical trials to investigate this promising therapeutic option. ANN NEUROL 2014;75:447–453 |
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