“We hypothesized that rilzabrutinib would be effective in both reducing platelet-attacking antibodies as well as minimizing the rate of platelet destruction by macrophages.Lead author David J. Kuter, MD, DPhil, director of the Hematology program at MGH and professor of Medicine at Harvard Medical School.
In a 60-patient trial involving a series of doses of rilzabrutinib, all treatment-related adverse events were mild and transient. At a mean follow-up of about 5.5 months of treatment, 24 of 60 patients (40%) overall and 18 of 45 patients (40%) who had started rilzabrutinib at the highest dose had a response. significant platelet response.
The average time to develop a healthy platelet count is 10.5 days. Among patients with a platelet response, the median percentage of weeks in which they had a healthy platelet count was 65%. A dose of 400 mg twice daily was determined to be the dose for further testing.
The patients in this study tried many therapies and their disease was considered very difficult to treat.
A large phase 3 clinical trial is currently underway in multiple settings, including MGH, to test the effectiveness of rilzabrutinib in patients with less resistant ITP.
“If the findings of our study were to be replicated in other studies, rilzabrutinib may cause a rapid and sustained increase in platelet counts to a safe platelet count, thereby possibly minimizing bleeding and The drug can produce antibodies that cause disease. disappear“Kuter said.
“What is impressive is that this drug does not have the major negative effects that have been historically associated with this class of drugs. We did not find patients at increased risk of bleeding, infection, liver dysfunction or intolerance. “