Journal article

Proof of concept for the clinical effects of oral rilzabrutinib, the first Bruton tyrosine kinase inhibitor for pemphigus vulgaris: the phase II BELIEVE study

DF Murrell, A Patsatsi, P Stavropoulos, S Baum, T Zeeli, JS Kern, A-V Roussaki-Schulze, R Sinclair, ID Bassukas, D Thomas, A Neale, P Arora, F Caux, VP Werth, SG Gourlay, P Joly

BRITISH JOURNAL OF DERMATOLOGY | WILEY | Published : 2021

Abstract

BACKGROUND: Bruton tyrosine kinase (BTK) inhibition targets B-cell and other non-T-cell immune cells implicated in the pathophysiology of pemphigus, an autoimmune disease driven by anti-desmoglein autoantibodies. Rilzabrutinib is a new reversible, covalent BTK inhibitor demonstrating preclinical efficacy as monotherapy in canine pemphigus foliaceus. OBJECTIVES: To evaluate the efficacy and safety of oral rilzabrutinib in patients with pemphigus vulgaris in a multicentre, proof-of-concept, phase II trial. METHODS: Patients with Pemphigus Disease Area Index severity scores 8-45 received 12 weeks of oral rilzabrutinib 400-600 mg twice daily and 12 weeks of follow-up. Patients initially received..

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