Ruben Mesa, MD, spoke about which patients stand to benefit the most from the approval of pacritinib in myelofibrosis with severe thrombocytopenia.
Ruben Mesa, MD, executive director of Mays Cancer Center, home of the UT Health San Antonio MD Anderson Cancer Center, spoke with CancerNetwork® about which population of patients with myelofibrosis would benefit most from the accelerated approval of pacritinib (Vonjo).1 Mesa also spoke about the PERSIST-1 trial (NCT01773187), for which he is lead investigator.2 This trial aimed to determine if pacritinib vs best available therapy would improve outcomes in patients with myelofibrosis, regardless of baseline cytopenia.
The group of individuals with cytopenic myelofibrosis [will benefit the most from pacritinib] and that’ll be a natural part of the discussion, even patients who are JAK inhibitor naïve who have marked thrombocytopenia. There will be important use as second- or third-line therapy for individuals with myelofibrosis, particularly if they have cytopenic myelofibrosis.
If they have a platelet count under 50,000/μl, it will [offer the most unique option]. Any amount of thrombocytopenia under 100,000/μl, it will be a strong consideration both in the frontline and the second-line setting. There clearly is activity in the PERSIST-1 study [NCT01773187], where it was not limited only to patients with thrombocytopenia. It is active for individuals that have a normal platelet count. In those settings, it will be more in the second line alone or potentially in combinations.
1. CTI BioPharma announces FDA accelerated approval of VONJO™ (pacritinib) for the treatment of adult patients with myelofibrosis and thrombocytopenia. News release. CTI BioPharma Corp. February 28, 2022. Accessed February 28, 2022. https://prn.to/3ppk5Cm
2. Mesa RA, Vannucchi AM, Mead A, et al. Pacritinib versus best available therapy for the treatment of myelofibrosis irrespective of baseline cytopenias (PERSIST-1): an international, randomised, phase 3 trial. Lancet Haematol. 2017;4(5):e225-e236. doi:10.1016/S2352-3026(17)30027-3