title: Allogeneic hematopoietic stem cell transplantation for neuromyelitis optica.
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.
authors: Hematology and Bone Marrow Transplantation Unit.
authors: Raffaella
authors: R
authors: Greco
authors: Attilio
authors: A
authors: Bondanza
authors: Luca
authors: L
authors: Vago
authors: Lucia
authors: L
authors: Moiola
authors: Paolo
authors: P
authors: Rossi
authors: Roberto
authors: R
authors: Furlan
authors: Gianvito
authors: G
authors: Martino
authors: Marta
authors: M
authors: Radaelli
authors: Vittorio
authors: V
authors: Martinelli
authors: Maria Rosaria
authors: MR
authors: Carbone
authors: Maria Teresa
authors: MT
authors: Lupo Stanghellini
authors: Andrea
authors: A
authors: Assanelli
authors: Massimo
authors: M
authors: Bernardi
authors: Consuelo
authors: C
authors: Corti
authors: Jacopo
authors: J
authors: Peccatori
authors: Chiara
authors: C
authors: Bonini
authors: Paolo
authors: P
authors: Vezzulli
authors: Andrea
authors: A
authors: Falini
authors: Fabio
authors: F
authors: Ciceri
authors: Giancarlo
authors: G
authors: Comi
mesh_terms: D000328:Adult; D051401:Aquaporin 4 / Q000276:immunology; D001323:Autoantibodies / Q000276:immunology; D001853:Bone Marrow / Q000601:surgery; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D016131:Lymphocyte Subsets / Q000276:immunology; D008297:Male; D009471:Neuromyelitis Optica / Q000276:immunology / Q000628:therapy*; D014184:Transplantation, Homologous
chemical_list: C496030:AQP4 protein, human; D051401:Aquaporin 4; D001323:Autoantibodies