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.

Safety and efficacy of rilzabrutinib vs placebo in adults with immune thrombocytopenia: the phase 3 LUNA3 study.

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

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

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 

Related studies

https://www.nejm.org/doi/full/10.1056/NEJMcp1810479