title: Autologous hematopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: comparison with secondary progressive multiple sclerosis.
abstract: The main objective of our work is to describe the long-term results of myeloablative autologous hematopoietic stem cell transplant (AHSCT) in multiple sclerosis patients. Patients that failed to conventional therapies for multiple sclerosis (MS) underwent an approved protocol for AHSCT, which consisted of peripheral blood stem cell mobilization with cyclophosphamide and granulocyte colony-stimulating factor (G-CSF), followed by a conditioning regimen of BCNU, Etoposide, Ara-C, Melphalan IV, plus Rabbit Thymoglobulin. Thirty-eight MS patients have been transplanted since 1999. Thirty-one patients have been followed for more than 2 years (mean 8.4 years). There were 22 relapsing-remitting multiple sclerosis (RRMS) patients and 9 secondary progressive multiple sclerosis (SPMS) patients. No death related to AHSCT. A total of 10 patients (32.3%) had at least one relapse during post-AHSCT evolution, 6 patients in the RRMS group (27.2%) and 4 in the SPMS group (44.4%). After AHSCT, 7 patients (22.6%) experienced progression of disability, all within SP form. By contrast, no patients with RRMS experienced worsening of disability after a median follow-up of 5.4 years, 60% of them showed a sustained reduction in disability (SRD), defined as the improvement of 1.0 point in the expanded disability status scale (EDSS) sustains for 6 months (0.5 in cases of EDSS ≥ 5.5). The only clinical variable that predicted a poor response to AHSCT was a high EDSS in the year before transplant. AHSCT using the BEAM-ATG scheme is safe and efficacious to control the aggressive forms of RRMS.
authors.affiliation: Neuroimmunology Unit, Hospital Universitari I Politècnic La Fe, Avd Abril Martorell, 46026, Valencia, Spain. Casanova.bonaventura@gmail.com.
authors.forename: Bonaventura
authors.initials: B
authors.lastname: Casanova
authors.affiliation: Hematology Service, Hospital Universitari I Politècnic La Fe, Valencia, Spain.
authors.forename: Isidro
authors.initials: I
authors.lastname: Jarque
authors.affiliation: Neuroimmunology Unit, Hospital Clínic Universitari de València, Valencia, Spain.
authors.forename: Francisco
authors.initials: F
authors.lastname: Gascón
authors.affiliation: Hematology Service, Hospital Clínic Universitari, Valencia, Spain.
authors.forename: Juan Carlos
authors.initials: JC
authors.lastname: Hernández-Boluda
authors.affiliation: Neuroimmunology Unit, Hospital Universitari I Politècnic La Fe, Avd Abril Martorell, 46026, Valencia, Spain.
authors.forename: Francisco
authors.initials: F
authors.lastname: Pérez-Miralles
authors.affiliation: Hematology Service, Hospital Universitari Dr. Peset and Universidad Católica "San Vicente Mártir", Valencia, Spain.
authors.forename: Javier
authors.initials: J
authors.lastname: de la Rubia
authors.affiliation: Neuroimmunology Unit, Hospital Universitari I Politècnic La Fe, Avd Abril Martorell, 46026, Valencia, Spain.
authors.forename: Carmen
authors.initials: C
authors.lastname: Alcalá
authors.affiliation: Hematology Service, Hospital Universitari I Politècnic La Fe, Valencia, Spain.
authors.forename: Jaime
authors.initials: J
authors.lastname: Sanz
authors.affiliation: Neurology Service, Hospital General de Elda, Elda, Spain.
authors.forename: Javier
authors.initials: J
authors.lastname: Mallada
authors.affiliation: Neurology Service, Hospital General Universitari de València, Valencia, Spain.
authors.forename: Angeles
authors.initials: A
authors.lastname: Cervelló
authors.affiliation: Neurology Unit, Hospital de Sagunt, Valencia, Spain.
authors.forename: Arantxa
authors.initials: A
authors.lastname: Navarré
authors.affiliation: Neurology Service, Hospital General Universitari de València, Valencia, Spain.
authors.forename: María
authors.initials: M
authors.lastname: Carcelén-Gadea
authors.affiliation: Neuroimmunology Unit, Hospital Universitari I Politècnic La Fe, Avd Abril Martorell, 46026, Valencia, Spain.
authors.forename: Isabel
authors.initials: I
authors.lastname: Boscá
authors.affiliation: Neuroimmunology Unit, Hospital Universitari I Politècnic La Fe, Avd Abril Martorell, 46026, Valencia, Spain.
authors.forename: Sara
authors.initials: S
authors.lastname: Gil-Perotin
authors.affiliation: Neuroimmunology Unit, Hospital Clínic Universitari de València, Valencia, Spain.
authors.forename: Carlos
authors.initials: C
authors.lastname: Solano
authors.affiliation: Hematology Service, Hospital Universitari I Politècnic La Fe, Valencia, Spain.
authors.forename: Miguel Angel
authors.initials: MA
authors.lastname: Sanz
authors.affiliation: Neuroimmunology Unit, Hospital Clínic Universitari de València, Valencia, Spain.
authors.forename: Francisco
authors.initials: F
authors.lastname: Coret
mesh_terms: D000328:Adult; D000818:Animals; D003561:Cytarabine / Q000627:therapeutic use; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D008297:Male; D008875:Middle Aged; D020528:Multiple Sclerosis, Chronic Progressive / Q000628:therapy*; D020529:Multiple Sclerosis, Relapsing-Remitting / Q000628:therapy*; D011817:Rabbits; D014182:Transplantation, Autologous / Q000379:methods; D016896:Treatment Outcome
chemical_list: D003561:Cytarabine
keywords: Autologous hematopoietic stem cell transplantation; Immunosupression; Immunotherapy; Multiple sclerosis; Neurodegeneration; Secondary progressive multiple sclerosis