title: Rituximab for IgG4-related disease: a prospective, open-label trial.
abstract: OBJECTIVE
  To evaluate the efficacy of rituximab (RTX) in IgG4-related disease (IgG4-RD) in an open-label pilot trial.
  
  
  METHODS
  We treated 30 IgG4-RD patients with two doses of RTX (1000 mg each). The participants were either treated with RTX alone (n = 26; 87%) or required to discontinue baseline glucocorticoids (GC) within 2 months (n = 4; 13%). Disease activity was measured by the IgG4-RD Responder Index (IgG4-RD RI) and physician's global assessment (PGA). Disease response was defined as the improvement of the IgG4-RD RI by two points. The primary outcome, measured at 6 months, was defined as: (1) decline of the IgG4-RD RI ≥2 points compared with baseline; (2) no disease flares before month 6; and (3) no GC use between months 2 and 6. Complete remission was defined as an IgG4-RD RI score of 0 with no GC use.
  
  
  RESULTS
  Disease responses occurred in 97% of participants. The baseline IgG4-RD RI and PGA values, 11±7 and 63±22 mm, respectively, declined to 1±2 and 11±16 mm at 6 months (both p<0.00001). The primary outcome was achieved by 23 participants (77%). Fourteen (47%) were in complete remission at 6 months, and 12 (40%) remained in complete remission at 12  months. Among the 19 with elevated baseline serum IgG4, IgG4 concentrations declined from a mean of 911 mg/dL (range 138-4780 mg/dL) to 422 mg/dL (range 56-2410 mg/dL) at month 6 (p<0.05). However, only 8 (42%) of the 19 achieved normal values.
  
  
  CONCLUSIONS
  RTX appears to be an effective treatment for IgG4-RD, even without concomitant GC therapy.
  
  
  BACKGROUND
  ClinicalTrials.gov identifier: NCT01584388.
authors: Rheumatology Unit, Division of Rheumatology, Allergy, and Immunology, Department of Medicine, Massachusetts General Hospital and Harvard Medical School, Boston, Massachusetts, USA.
authors: Mollie N
authors: MN
authors: Carruthers
authors: Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, Minnesota, USA.
authors: Mark D
authors: MD
authors: Topazian
authors: Division of Rheumatology, Department of Medicine, Emory University School of Medicine, Atlanta, Georgia, USA.
authors: Arezou
authors: A
authors: Khosroshahi
authors: Department of Pathology, Massachusetts General Hospital, Boston, Massachusetts, USA.
authors: Thomas E
authors: TE
authors: Witzig
authors: Rheumatology Unit, Division of Rheumatology, Allergy, and Immunology, Department of Medicine, Massachusetts General Hospital and Harvard Medical School, Boston, Massachusetts, USA.
authors: Zachary S
authors: ZS
authors: Wallace
authors: Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, Minnesota, USA.
authors: Philip A
authors: PA
authors: Hart
authors: Department of Pathology, Mayo Clinic, Rochester, Minnesota, USA.
authors: Vikram
authors: V
authors: Deshpande
authors: Division of Hematology, Mayo Clinic, Rochester, Minnesota, USA.
authors: Thomas C
authors: TC
authors: Smyrk
authors: Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, Minnesota, USA.
authors: Suresh
authors: S
authors: Chari
authors: Rheumatology Unit, Division of Rheumatology, Allergy, and Immunology, Department of Medicine, Massachusetts General Hospital and Harvard Medical School, Boston, Massachusetts, USA.
authors: John H
authors: JH
authors: Stone
mesh_terms: D000368:Aged; D058846:Antibodies, Monoclonal, Murine-Derived / Q000627:therapeutic use*; D018501:Antirheumatic Agents / Q000627:therapeutic use*; D001327:Autoimmune Diseases / Q000188:drug therapy* / Q000276:immunology; D015331:Cohort Studies; D004359:Drug Therapy, Combination; D005260:Female; D005938:Glucocorticoids / Q000627:therapeutic use; D006801:Humans; D007074:Immunoglobulin G / Q000276:immunology*; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D011446:Prospective Studies; D012074:Remission Induction; D000069283:Rituximab; D012720:Severity of Illness Index; D016896:Treatment Outcome
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D018501:Antirheumatic Agents; D005938:Glucocorticoids; D007074:Immunoglobulin G; D000069283:Rituximab
keywords: B cells; Inflammation; Treatment