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Safety and efficacy of low-dose intravenous arsenic trioxide in systemic lupus erythematosus: an open-label phase IIa trial (Lupsenic)

  • Hamidou, Mohamed1
  • Néel, Antoine1
  • Poupon, Joel2
  • Amoura, Zahir3
  • Ebbo, Mikael4
  • Sibilia, Jean5
  • Viallard, Jean-Francois6
  • Gaborit, Benjamin1
  • Volteau, Christelle7
  • Hardouin, Jean Benoit8
  • Hachulla, Eric9
  • Rieger, François10
  • 1 Department of Internal Medicine, CHU Nantes, Nantes Université, Nantes, France , Nantes (France)
  • 2 AP-HP, Lariboisière Hospital, University Paris VII, Paris, France , Paris (France)
  • 3 Hôpital Pitié-Salpétrière, Paris, France , Paris (France)
  • 4 Aix Marseille Univ, APHM, CNRS, INSERM, CIML, Hôpital de la Timone, Marseille, France , Marseille (France)
  • 5 University of Strasbourg, Strasbourg, France , Strasbourg (France)
  • 6 Haut-Lévêque University Hospital, Bordeaux, France , Bordeaux (France)
  • 7 Université de Nantes, Nantes, France , Nantes (France)
  • 8 INSERM UMR 1246-SPHERE, Université de Nantes, Nantes, France , Nantes (France)
  • 9 University of Lille, Lille, France , Lille (France)
  • 10 MEDSENIC, SAS, a company with CNRS participation, Strasbourg, France , Strasbourg (France)
Published Article
Arthritis Research & Therapy
Springer Science and Business Media LLC
Publication Date
Mar 03, 2021
DOI: 10.1186/s13075-021-02454-6
Springer Nature


BackgroundLupus animal model has shown that arsenic trioxide (ATO), a treatment of acute promyelocytic leukaemia, could be effective in SLE. This is the first clinical study to determine the safety and efficacy of a short course of intravenous ATO in patients with active SLE.MethodsThis phase IIa, open-label, dose-escalating study enrolled 11 adult SLE patients with a non-organ threatening disease, clinically active despite conventional therapy. Patients received 10 IV infusions of ATO within 24 days. The first group received 0.10 mg/kg per injection, with dose-escalating to 0.15 mg/kg in a second group, and to 0.20 mg/kg in a third group. The primary endpoint was the occurrence of adverse events (AEs) and secondary endpoints were the number of SLE Responder Index 4 (SRI-4) responders at week 24 and reduction of corticosteroid dosage. In an exploratory analysis, we collected long-term data for safety and attainment of lupus low disease activity state (LLDAS).ResultsFour serious AEs occurred (grade 3 neutropenia, osteitis, neuropathy), 2 of which were attributable to ATO (neutropenia in the 2 patients treated with mycophenolate). Two patients suffered a severe flare during the last 4 weeks of the trial. At W24, five patients among 10 were SRI-4 responders. Overall, mean corticosteroid dosage decreased from 11.25 mg/day at baseline to 6 mg/day at W24 (P < 0.01). In the long term, 6 patients attained LLDAS at W52, which continued at last follow-up (median LLDAS duration 3 years, range 2–4).ConclusionsA short course of ATO has an acceptable safety profile in SLE patients and encouraging efficacy.Trial, NCT01738360 registered 30 November 2012

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