CPX-351 (Vyxeos™) treatment in blast-phase myeloproliferative neoplasm (MPN-BP): real-world experience in 12 consecutive cases
Rimal Ilyas, Kristen McCullough, Talha Badar, Mrinal M. Patnaik, Hassan B. Alkhateeb, Abhishek A. Mangaonkar, Animesh Pardanani, Ayalew Tefferi, Naseema Gangat
Abstract
Blast-phase transformation in myeloproliferative neoplasms (MPN-BP) occurs at rates of 3.9, 2.6, and 9.3%, after median follow-up periods of 8.2, 9.9, and 3.2 years, in polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF), respectively [ 1 , 2 , 3 ]. A Mayo Clinic and University of Florence, Italy collaborative study of 410 patients with MPN-BP included 248 Mayo Clinic cases in whom treatment details were available [ 4 ]; 96% of the patients were dead after a median follow-up of 3.6 months with 1-, 3- and 5-year survival rates of 17, 6, and 4%, respectively; treatment included supportive care ( n = 121; 49%), chemotherapy ( n = 103; 42%) with ( n = 24) or without ( n = 79) achieving complete remission (CR) or CR with incomplete count recovery (CRi), and allogeneic stem cell transplant (ASCT; n = 24;10%); 1- and 3-year survival rates were 66% and 32% for ASCT, 37% and 19% for chemotherapy-treated patients achieving CR/CRi but were not transplanted, and 8% and 1% in the absence of both ASCT and CR/CRi, respectively [ 4 ].