Examining Real-World Outcomes Among Patients With Myelofibrosis Treated With Pacritinib

John Mascarenhas, MD, Icahn School of Medicine at Mount Sinai, New York, New York, discusses results of a retrospective study examining the real-world treatment patterns among patients with myelofibrosis (MF) who were treated with pacritinib.

These results “[are] one of the largest real-world data sets of pacritinib in the US after its approval in 2022, demonstrating the ability to deliver this drug in MF patients across multiple lines of therapy,” Dr Mascarenhas stated.

These results were presented at the 2024 European Hematology Association (EHA) Meeting in Madrid, Spain.

Transcript:

Hi, I'm John Mascarenhas, professor of medicine at the Icahn School of Medicine at Mount Sinai.

I'm happy to be presenting, on behalf of my colleagues, our [European Hematology Association] EHA 2024 abstract entitled, “Real World Treatment Patterns and Outcomes in Patients with Myelofibrosis, Treated With Pacritinib in the United States.” This was a retrospective study looking at a total of 142 myelofibrosis (MF) patients that were identified in a deidentified electronic medical record and practice management data set with a diagnosis of myelofibrosis [International Classification of Diseases] ICD-coded and treated with pacritinib between June of 2022 and August of 2023.

We identified 142 patients in this database. They had a median follow-up of approximately 6 months, [and] 28% of patients had a baseline platelet count of less than 50,000 and 30% had a hemoglobin less than 8g/dL. The median time from myelofibrosis diagnosis to pacritinib treatment was about 13 months and the use of pacritinib in the first line was more common in those patients with platelet counts less than 50,000, which is the label by the FDA here in the United States, but also used in patients with higher platelet counts, particularly in latter lines of therapy.

We did see stability and even [upward] trends in the platelet count, particularly for those patients starting with platelet counts less than 100,000, so demonstrating the absence of myelosuppression and worsening thrombocytopenia, as well as hemoglobin levels that trended upwards over time with pacritinib, again, even in those patients with prior ruxolitinib exposure.

The 12-month overall survival probability was approximately 69%, which is quite favorable in this advanced and pretreated patient population, when you look at multiple studies that have been conducted and reported previously, and keeping in mind the majority of these patients were patients with thrombocytopenia. The survival benefit that appeared to be there was maintained even in those with multiple lines of therapy, platelet counts less than 50,000, and prior ruxolitinib treatment.

In totality, this is one of the largest real-world datasets of pacritinib in the US after its approval in 2022, demonstrating the ability to deliver this drug in MF patients across multiple lines of therapy and low platelets and prior ruxolitinib therapy while maintaining blood counts, [ ] hemoglobin and avoiding significant myelosuppression—and also hinting towards improvement in survival in this patient population that formally had an unmet need, that was then met with pacritinib.

Source:

Mascarenhas J. Real world treatment patterns and outcomes in patients with myelofibrosis, treated with pacritinib in the United States. Presented at the 2024 European Hematology Association (EHA) Meeting. July 13–16, 2024; Madrid, Spain.