@article{GoebelerBataCsoergőSimoneetal.2022,
  author    = {Goebeler, M. and Bata-Cs{\"o}rgő, Z. and Simone, C. de and Didona, B. and Remenyik, E. and Reznichenko, N. and Stoevesandt, J. and Ward, E. S. and Parys, W. and Haard, H. de and Dupuy, P. and Verheesen, P. and Schmidt, E. and Joly, P.},
  title     = {Treatment of pemphigus vulgaris and foliaceus with efgartigimod, a neonatal Fc receptor inhibitor: a phase II multicentre, open-label feasibility trial},
  series = {British Journal of Dermatology},
  volume    = {186},
  journal   = {British Journal of Dermatology},
  number    = {3},
  doi       = {10.1111/bjd.20782},
  url       = {http://nbn-resolving.de/urn:nbn:de:bvb:20-opus-258328},
  pages     = {429-439},
  year      = {2022},
  abstract  = {Background Pemphigus vulgaris and pemphigus foliaceus are potentially life-threatening autoimmune disorders triggered by IgG autoantibodies against mucosal and epidermal desmogleins. There is an unmet need for fast-acting drugs that enable patients to achieve early sustained remission with reduced corticosteroid reliance. Objectives To investigate efgartigimod, an engineered Fc fragment that inhibits the activity of the neonatal Fc receptor, thereby reducing serum IgG levels, for treating pemphigus. Methods Thirty-four patients with mild-to-moderate pemphigus vulgaris or foliaceus were enrolled in an open-label phase II adaptive trial. In sequential cohorts, efgartigimod was dosed at 10 or 25 mg kg\(^{-1}\) intravenously with various dosing frequencies, as monotherapy or as add-on therapy to low-dose oral prednisone. Safety endpoints comprised the primary outcome. The study is registered at ClinicalTrials.gov (identifier NCT03334058). Results Adverse events were mostly mild and were reported by 16 of 19 (84\%) patients receiving efgartigimod 10 mg kg\(^{-1}\) and 13 of 15 (87\%) patients receiving 25 mg kg-1, with similar adverse event profiles between dose groups. A major decrease in serum total IgG and anti-desmoglein autoantibodies was observed and correlated with improved Pemphigus Disease Area Index scores. Efgartigimod, as monotherapy or combined with prednisone, demonstrated early disease control in 28 of 31 (90\%) patients after a median of 17 days. Optimized, prolonged treatment with efgartigimod in combination with a median dose of prednisone 0·26 mg \(^{-1}\) per day (range 0·06-0·48) led to complete clinical remission in 14 of 22 (64\%) patients within 2-41 weeks. Conclusions Efgartigimod was well tolerated and exhibited an early effect on disease activity and outcome parameters, providing support for further evaluation as a therapy for pemphigus.},
  language  = {en}
}
@article{MuellerBroeckerKlinkeretal.2012,
  author    = {M{\"u}ller, P.A. and Br{\"o}cker, E.B. and Klinker, E. and Stoevesandt, J. and Benoit, S.},
  title     = {Adjuvant treatment of recalcitrant Bullous pemphigoid with immunoadsorption},
  series = {Dermatology},
  volume    = {224},
  journal   = {Dermatology},
  number    = {3},
  issn      = {1018-8665},
  doi       = {10.1159/000339071},
  url       = {http://nbn-resolving.de/urn:nbn:de:bvb:20-opus-196620},
  pages     = {224 -- 227},
  year      = {2012},
  abstract  = {Elimination of pathogenic autoantibodies by immunoadsorption (IA) has been described as an effective adjuvant treatment in severe bullous autoimmune diseases, especially in pemphigus. There is much less experience in the treatment of bullous pemphigoid (BP). BP was diagnosed in a 62-year-old Caucasian woman presenting a pruritic rash with multiple tense blisters. Standard treatments with topical and oral corticosteroids, steroid-sparing agents including dapsone, azathioprine, mycophenolate mofetil (MMF) and intravenous immunoglobulins were ineffective or had to be discontinued due to adverse events. An immediate clinical response could be achieved by two treatment cycles of adjuvant protein A immunoadsorption (PA-IA) in addition to continued treatment with MMF (2 g/day) and prednisolone (1 mg/kg/day). Tolerance was excellent. Clinical improvement remained stable after discontinuation of IA and went along with sustained reduction of circulating autoantibodies. Our data demonstrate that PA-IA might be a safe and effective adjuvant treatment in severe and recalcitrant BP.},
  language  = {en}
}