Efficacy and safety of adjuvant immunoadsorption in pemphigus vulgaris and pemphigus foliaceus (IA-Pem Study): a multicentre randomised controlled trial

Publikation: Beitrag in FachzeitschriftForschungsartikelBeigetragenBegutachtung

Beitragende

  • Nina van Beek - (Autor:in)
  • Rüdiger Eming - (Autor:in)
  • Alexander Reuss - (Autor:in)
  • Detlef Zillikens - (Autor:in)
  • Miklós Sárdy - (Autor:in)
  • Claudia Günther - , Klinik und Poliklinik für Dermatologie (Autor:in)
  • Dimitra Kiritsi - (Autor:in)
  • Sandrine Benoit - (Autor:in)
  • Stefan Beissert - , Klinik und Poliklinik für Dermatologie (Autor:in)
  • Regine Gläser - (Autor:in)
  • Orsolya N Horváth - (Autor:in)
  • Christiane Pfeiffer - (Autor:in)
  • Martin Röcken - (Autor:in)
  • Franziska Schauer - (Autor:in)
  • Kerstin Steinbrink - (Autor:in)
  • Carmen Schade-Brittinger - (Autor:in)
  • Michael Hertl - (Autor:in)
  • Enno Schmidt - (Autor:in)

Abstract

BACKGROUND: Pemphigus vulgaris (PV) and pemphigus foliaceus (PF) are potentially life-threatening autoimmune blistering diseases. Treatment is based on long term immunosuppression with high doses of glucocorticosteroids in combination with potentially corticosteroid-sparing agents and/or rituximab. Immunoadsorption (IA) has emerged as fast acting adjuvant treatment option.

OBJECTIVES: To assess the clinical efficacy of IA in addition to best medical treatment.

METHODS: Multicentre (26 centres from Germany and Austria) randomised controlled trial in 72 patients with newly diagnosed, relapsed or chronic active PV or PF (34 females, 38 males, aged 42-72 years) comparing best medical treatment (BMT) (prednisolone 1.0 mg/kg/d plus azathioprine or mycophenolate) to adjuvant IA (BMT+IA). Central 1:1 randomization was done at the coordinating centre for clinical trials (KKS) Marburg. The primary endpoint was analyzed using Kaplan-Meier and Cox regression methods.

RESULTS: The study was ended prematurely due to safety concerns after random allocation of 72 patients to BMT+IA (n=34) or BMT (n=38). The primary endpoint, time to complete remission on therapy, was not significantly different between the groups (p=0.39; HR 1.35 (95%CI: 0.68-2.69). The cumulative dose of prednisolone was significantly lower in the BMT+IA compared to BMT alone (difference -1,214; 95%CI, -2,225 - -70; p=0.03). In a post hoc analysis, patients with more extensive PV/PF showed a tendency towards a shorter time to remission in the BMT+IA group compared to the BMT group (HR=1.87, p=0.17 in patients with baseline PDAI ≥ 15). While more adverse events were observed in patients of the BMT group (29 vs. 25), severe adverse events were more frequent in patients of the BMT+IA group (17 events in 10 patients vs. 11 events in 8 patients).

CONCLUSIONS: In this study, adjuvant IA did not show a shorter time to clinical remission but a corticosteroid-sparing effect. In patients with extensive PV/PF, post hoc analysis suggests that adjuvant IA may possibly lead to earlier remission, but potential adverse events must be carefully weighed against the expected benefits.

Details

OriginalspracheEnglisch
Aufsatznummerljad489
FachzeitschriftBritish Journal of Dermatology
PublikationsstatusElektronische Veröffentlichung vor Drucklegung - 22 Dez. 2023
Peer-Review-StatusJa

Externe IDs

ORCID /0000-0002-4330-1861/work/151982049

Schlagworte