title: Placebo-controlled trial of rituximab in IgM anti-myelin-associated glycoprotein neuropathy.
abstract: OBJECTIVE
  To determine whether rituximab 375 mg/m(2) was efficacious in patients with immunoglobulin M (IgM) anti-myelin-associated glycoprotein antibody demyelinating neuropathy (IgM anti-MAG demyelinating neuropathy).
  
  
  METHODS
  Fifty-four patients with IgM anti-MAG demyelinating neuropathy were enrolled in this randomized, double-blind, placebo-controlled trial. The inclusion criteria were inflammatory neuropathy cause and treatment (INCAT) sensory score (ISS) ≥4 and visual analog pain scale >4 or ataxia score ≥2. The primary outcome was mean change in ISS at 12 months.
  
  
  RESULTS
  Twenty-six patients were randomized to a group receiving 4 weekly infusions of 375 mg/m(2) rituximab, and 28 patients to placebo. Intention-to-treat analysis, with imputation of missing ISS values by the last observation carried forward method, showed a lack of mean change in ISS at 12 months, 1.0 ± 2.7 in the rituximab group, and 1.0 ± 2.8 in the placebo group. However, changes were observed, in per protocol analysis at 12 months, for the number of patients with an improvement of at least 2 points in the INCAT disability scale (p = 0.027), the self-evaluation scale (p = 0.016), and 2 subscores of the Short Form-36 questionnaire.
  
  
  CONCLUSIONS
  Although primary outcome measures provide no evidence to support the use of rituximab in IgM anti-MAG demyelinating neuropathy, there were improvements in several secondary outcomes in per protocol analysis.
  
  
  METHODS
  This study provides Class I evidence that rituximab is ineffective in improving ISS in patients with IgM anti-MAG demyelinating neuropathy.
authors.affiliation: Department of Neurology, University Hospital Pitié-Salpêtrière, Paris, France. jean-marc.leger@psl.aphp.fr
authors.forename: Jean-Marc
authors.initials: JM
authors.lastname: Léger
authors.forename: Karine
authors.initials: K
authors.lastname: Viala
authors.forename: Guillaume
authors.initials: G
authors.lastname: Nicolas
authors.forename: Alain
authors.initials: A
authors.lastname: Créange
authors.forename: Jean-Michel
authors.initials: JM
authors.lastname: Vallat
authors.forename: Jean
authors.initials: J
authors.lastname: Pouget
authors.forename: Pierre
authors.initials: P
authors.lastname: Clavelou
authors.forename: Christophe
authors.initials: C
authors.lastname: Vial
authors.forename: Andreas
authors.initials: A
authors.lastname: Steck
authors.forename: Lucile
authors.initials: L
authors.lastname: Musset
authors.forename: Benoit
authors.initials: B
authors.lastname: Marin
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D058846:Antibodies, Monoclonal, Murine-Derived / Q000627:therapeutic use*; D003711:Demyelinating Diseases / Q000188:drug therapy* / Q000276:immunology / Q000473:pathology; D004311:Double-Blind Method; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007075:Immunoglobulin M / Q000096:biosynthesis*; D008297:Male; D008875:Middle Aged; D018992:Myelin-Associated Glycoprotein / Q000276:immunology*; D011115:Polyneuropathies / Q000188:drug therapy / Q000276:immunology / Q000473:pathology; D000069283:Rituximab; D055815:Young Adult
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D007075:Immunoglobulin M; D018992:Myelin-Associated Glycoprotein; D000069283:Rituximab