https://doi.org/10.55788/2d100bb8
Part B of the adaptive 2-part, open-label, phase 1/2 LUNA 2 trial (NCT03395210) tested whether the oral BTK inhibitor rilzabrutinib could induce a sustained increase in platelet counts in 26 adult participants with R/R ITP. Prof. Michael Tarantino (University of Illinois, IL, USA) shared data on the effects of rilzabrutinib on bleeding outcomes and health-related QoL (HRQoL) [1].
After 24 weeks of therapy, 9 participants (35%) had achieved the primary endpoint, which was a durable platelet response, defined as having at least 8 platelet counts ≥50 x 109/L in the last 12 weeks without using rescue medication. “Fortunately, we did not see treatment-related bleeding events,” expressed Prof. Tarantino. Grade ≥2 bleeding events due to any cause were seen in 4 participants. “All these events were resolved,” added Prof. Tarantino. Yet, 1 of these 4 participants presented with a grade 3 post-procedural haemorrhage, leading to a dose interruption.
ITP Bleeding Score (IBLS) was reduced with a mean of -0.07, dropping from 0.27 at baseline to 0.20 at week 25. Especially the number of skin and oral bleedings was reduced. Finally, a median +7 increase in self-rated health (EQ-VAS) was reported from baseline (median 78) to week 25 (median 85) and 4 domains of the ITP-Patient Assessment Questionnaire displayed clinically meaningful changes from baseline to week 25, namely women’s reproductive health, overall HRQoL, fatigue/sleep, and activity.
Thus, quick and durable platelet responses were seen with rilzabrutinib treatment in participants with primary ITP, without worrisome safety issues. “Bleeding and QoL will be further analysed in the LUNA 3 trial (NCT04562766), which tests rilzabrutinib in participants with persistent or chronic ITP,” informed Prof. Tarantino.
- Tarantino MD, et al. The effects of rilzabrutinib, an oral Bruton tyrosine kinase inhibitor, on bleeding symptoms and health-related quality of life in patients with immune thrombocytopenia. Abstract 690, 65th ASH Annual Meeting, 9–12 December 2023, San Diego, CA, USA.
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Table of Contents: ASH 2023
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