Presentation Details
| Long-Term Efficacy and Safety With Oral Bruton Tyrosine Kinase Inhibitor Rilzabrutinib in Patients With Immune Thrombocytopenia David J Kuter1, Terry Gernsheimer2, Waleed Ghanima3, Umer Khan4, Brad Ward5, Ahmed Daak5, Nichola Cooper6. 1Hematology Division, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.2University of Washington Medical Center, Seattle, WA, USA.3Østfold Hospital Foundation, Gralum, Norway.4Biostatistics, Sanofi, Bridgewater, NJ, USA.5Sanofi, Cambridge, MA, USA.6Department of Medicine, Hammersmith Hospital, London, United Kingdom |
Abstract
Background: Adult patients with immune thrombocytopenia (ITP) treated with rilzabrutinib showed rapid and durable platelet responses and acceptable safety profiles in a global phase I/II trial (NCT03395210). Objective: To report updated efficacy and safety of rilzabrutinib in patients receiving 400 mg BID for 2 years in a long-term extension (LTE) study. Methods: ITP patients with 2 baseline platelet counts <30×109/L who responded to ≥1 prior ITP therapy but were unable to maintain adequate response to prior/concomitant therapies enrolled in the main study. Primary endpoints were safety and platelet responses (≥2 consecutive platelet counts ≥50×109/L and increased ≥20×109/L from baseline without rescue medication). Patients in the main 24-week study were eligible for LTE if platelet count was ≥50×109/L for ≥50% of last 8 weeks of treatment. Results: As of 21 December 2022, 16 of 60 patients from the main study enrolled in LTE on rilzabrutinib 400 mg BID (n=11 with concomitant CS and/or TPO-RA). At enrollment, patients had median duration of ITP for 4.3 years, received a median of 3 (range, 1-9) unique prior therapies; 3 were splenectomized. The median treatment duration for main+LTE periods was 1032 days (range, 318-1506). Figure 1 shows median platelet counts over time. All LTE patients met the primary endpoint with median platelet count at LTE entry of 87×109/L. After LTE entry, median platelet counts for LTE patients were 92×109/L, 71×109/L, 61×109/L, and 64×109/L at 3, 6, 12, and 24 months, respectively. Table 1 shows platelet counts above various LTE thresholds; 14 (88%) patients achieved platelet counts ≥100×109/L during the LTE. Two patients received rescue medication during the LTE. All treatment-related adverse events were transient, grade 1/2 events with no related bleeding or thrombotic events, serious adverse events, or deaths. Conclusions: With continued treatment through the LTE, rilzabrutinib 400 mg BID demonstrated durable clinical activity and remains well tolerated in patients with ITP.
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