Inhibition of FcRn with rozanolixizumab in adults with immune thrombocytopenia: Two randomised, double-blind, placebo-controlled phase 3 studies and their open-label extension.

Autor: Cooper N; Imperial College London, London, UK., Bussel JB; Weill Cornell Medicine - New York Presbyterian Hospital, New York, New York, USA., Kaźmierczak M; Poznań University of Medical Sciences, Poznań, Poland., Miyakawa Y; Saitama Medical University, Saitama, Japan., Cluck S; UCB, Slough, UK., Lledó García R; UCB, Slough, UK., Haier B; UCB, Monheim, Germany., Lavrov A; UCB, Monheim, Germany., Singh P; UCB, Slough, UK., Snipes R; UCB, Morrisville, North Carolina, USA., Kuter DJ; Hematology Division, Massachusetts General Hospital, Boston, Massachusetts, USA.
Jazyk: angličtina
Zdroj: British journal of haematology [Br J Haematol] 2024 Nov 18. Date of Electronic Publication: 2024 Nov 18.
DOI: 10.1111/bjh.19858
Abstrakt: Primary immune thrombocytopenia (ITP) is an antiplatelet-antibody-mediated disorder with accelerated platelet clearance and decreased platelet production. Rozanolixizumab, a monoclonal IgG4 anti-FcRn antibody, blocks IgG recycling and decreases IgG levels. We report efficacy and safety of rozanolixizumab in adults with persistent/chronic ITP in 24-week phase 3 studies (TP0003; TP0006), and their 52-week open-label extension (OLE). Primary end-point was durable clinically meaningful platelet response (DCMPR) of ≥50 × 10 9 /L for 8/12 weeks during Weeks 13-25 in the double-blind studies. Operational delays and evolving ITP treatment landscape led the sponsor to terminate these studies early; thus, only 21 and 12 (TP0003) and 20 and 10 (TP0006) patients were randomised to rozanolixizumab or placebo. Forty-three patients enrolled in the OLE: 42 started on every 2-week dosing; 21 later switched to weekly dosing. More rozanolixizumab-treated than placebo-treated patients achieved DCMPR: 4/21 versus 0 (TP0003) and 1/20 versus 0 (TP0006). Platelet increases to ≥50 × 10 9 /L were observed on Day 8 in 52.4% (TP0003; 2/12 placebo) and 45.0% (TP0006; 1/10 placebo) of rozanolixizumab-treated patients. OLE platelet increases were maintained while on weekly dosing. The most frequent treatment-emergent adverse events overall were headache, pyrexia and nausea, as seen previously. Weekly dosing appears more efficacious than every 2-week dosing.
(© 2024 The Author(s). British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.)
Databáze: MEDLINE
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