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THE CONSULT: Rilzabrutinib, Rozanolixizumab, and Beyond: ITP Therapeutics Explored. Professor Paul Klenerman talks to Professor Nichola Cooper about one of her recent papers.
AoPcasts: "The Consult" Bringing you the latest in medical research
Manage episode 521613185 series 2894201
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Immune thrombocytopenia (ITP) is a rare autoimmune disease, but also one where a great deal of progress has been made in development of new therapies. Prof Cooper performs clinical trials to understand the impact of new therapies, based upon and also gaining insights into the underlying pathogenesis. The clear readout of platelet count allows for a trial design which can funnel diverse new types of therapy into the translational pathway, and this sort of study has implications more broadly for how we address auto-immunity more generally. This work is a great example of how fundamental understanding of pathology can be turned into new therapies for patients by clinician scientists working at the interface.
The papers discussed look at the role of immune modulation in auto-immune thrombocytopenia, and trial approaches using inhibition of neonatal Fc receptors, as well as inhibition of the BTK molecule. More broadly we discuss the pathogenesis of this disease, how trials are performed and the landscape for future studies.
Kuter DJ, Ghanima W, Cooper N, Liebman HA, Zhang L, Hu Y, Miyakawa Y, Homenda W, Galindo LEM, Basquiera AL, Tan CW, Saydam G, Hütter-Krönke ML, Chai-Adisaksopha C, Gómez-Almaguer D, Tran H, Shin HJ, Dantas da Cunha Junior A, Lazar Z, Izquierdo CP, Kirgner I, Lucchini E, Kuzmina G, Fillitz M, Audia S, Taparia M, Cordoba M, Diab R, Yao M, Gouia I, Lee M, Daak A.
Blood. 2025 Jun 12;145(24):2914-2926. doi: 10.1182/blood.2024027336.
PMID: 40090011
Cooper N, Bussel JB, Kaźmierczak M, Miyakawa Y, Cluck S, Lledó García R, Haier B, Lavrov A, Singh P, Snipes R, Kuter DJ.
Br J Haematol. 2025 Feb;206(2):675-688. doi: 10.1111/bjh.19858. Epub 2024 Nov 18.
PMID: 39552477
The role of genetic sequencing in the diagnostic workup for chronic immune thrombocytopenia.
Joshi N, Lango-Allen H, Downes K, Simeoni I, Vladescu C, Paul D, Hart A, Ademokun C, Cooper N.
Blood Adv. 2025 Apr 8;9(7):1497-1507. doi: 10.1182/bloodadvances.2024014639.
PMID: 39808791
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