31 May Pacritinib Improved Disease Control In Myelofibrosis and Reduced Need For Blood Transfusions
MedicalResearch.com Interview with:
Ruben A. Mesa, MD, FACP
Chair, Division of Hematology & Medical Oncology
Deputy Director, Mayo Clinic Cancer Center
Professor of Medicine Mayo Clinic Cancer Center
NCI Designated Comprehensive Cancer Center
Medical Research: What is the background for this study? What are the main findings?
Dr. Mesa: Myelofibrosis is a rare and chronic blood cancer associated with significantly reduced quality of life and shortened survival. In patients with this disease, spleen enlargement (splenomegaly) is a very common and debilitating symptom – and as the disease progresses, the body slows production of important blood cells.
The results presented at ASCO were from the PERSIST-1 study, which is a Phase 3 registration-directed trial designed to compare pacritinib — an investigational oral multikinase inhibitor with specificity for JAK2 and FLT3 – to best available therapy (exclusive of a JAK inhibitor) in patients with myelofibrosis — regardless of their platelet counts. Data from this study showed that compared to best available therapy, pacritinib resulted in a significantly higher proportion of patients with spleen volume reduction and control of disease-related symptoms, regardless of platelet levels at the time of enrollment.
Medical Research: What should clinicians and patients take away from your report?
Dr. Mesa: There remains a high need for effective therapies that treat myelofibrosis while also providing significant improvements in disease-related symptoms without leading to additional treatment-related myelosuppression. These data not only showed improved spleen volume and control of disease-related symptoms, but also improvements in severe thrombocytopenia and severe anemia — eliminating the need for blood transfusions in a quarter of patients who were transfusion dependent at the time of enrollment; this may indicate an improvement in bone marrow function.
Medical Research: What recommendations do you have for future research as a result of this study?
Dr. Mesa: These results warrant further evaluation in patients with myelofibrosis – and across a broad range of hematologic malignancies. In addition, based on data showing improvement in bone marrow function, pacritinib have the potential to modify disease in the sickest patients as monotherapy and warrants further evaluation in combination with other potential disease-modifying agents.
Abstract presented at the 2015 ASCO meeting May 2015
Results of the PERSIST-1 phase III study of pacritinib (PAC) versus best available therapy (BAT) in primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia-myelofibrosis (PET-MF).
MedicalResearch.com Interview with: Ruben A. Mesa, MD, FACP (2015). Pacritinib Improved Disease Control In Myelofibrosis and Reduced Need For Blood Transfusions