title: NEDA status in highly active MS can be more easily obtained with autologous hematopoietic stem cell transplantation than other drugs.
abstract: The no evidence of disease activity (NEDA) composite measure has emerged as one attractive new target of therapies in relapsing-remitting multiple sclerosis (RRMS), consisting of the following features: (1) no relapses, (2) no disability progression, and (3) no magnetic resonance imaging (MRI) activity (new or enlarging T2 lesions or Gd-enhancing lesions). Achievement of NEDA status in patients receiving a disease-modifying therapy (DMT) seems to be an ambitious but ideal goal for therapies in RRMS. Recently, published post hoc analyses of clinical trials reported percentages of RRMS patients maintaining the NEDA status after 2 years of therapy ranging between 13% and 46%. Long-term assessment of NEDA patients in real-life settings showed very low probability to be NEDA in the long run. Against this scenario, immunoablative therapy followed by autologous hematopoietic stem cell transplantation (aHSCT) demonstrated the potential to maintain a much higher proportion of NEDA patients at 2 years (ranging from 78% to 83%) and also at 5 years (ranging from 60% to 68%). This is even more relevant when considering that MS patients who underwent aHSCT are much more active than patients usually enrolled in clinical trials. The emerging evidence of the efficacy of this therapeutic approach in early aggressive and treatment-resistant RRMS calls for the organization of a randomized comparative trial to fully evaluate the risk-benefit profile of aHSCT in patients with highly active MS not responding to DMTs.
authors.affiliation: Department of Health Sciences (DISSAL), University of Genoa, Genova, Italy.
authors.forename: Maria Pia
authors.initials: MP
authors.lastname: Sormani
authors.affiliation: Division of Brain Sciences, Imperial College London, London, UK.
authors.forename: Paolo A
authors.initials: PA
authors.lastname: Muraro
authors.affiliation: Haematology Department, Careggi University Hospital, Firenze, Italy.
authors.forename: Riccardo
authors.initials: R
authors.lastname: Saccardi
authors.affiliation: Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genova and IRCCS AOU San Martino-IST, Genova, Italy.
authors.forename: Gianluigi
authors.initials: G
authors.lastname: Mancardi
mesh_terms: D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D020529:Multiple Sclerosis, Relapsing-Remitting / Q000628:therapy*; D019172:Transplantation Conditioning / Q000379:methods; D014182:Transplantation, Autologous / Q000379:methods; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents
keywords: No evidence of disease activity; disease-modifying therapies; hematopoietic stem cell transplantation; multiple sclerosis