Survival expectation after thrombosis and overt-myelofibrosis in essential thrombocythemia and prefibrotic myelofibrosis: a multistate model approach
Alessandra Carobbio, Alessandro M. Vannucchi, Elisa Rumi, Valerio De Stefano, Alessandro Rambaldi, Giuseppe Carli, Maria Luigia Randi, H Gisslinger, Francesco Passamonti, Jüergen Thiele, Naseema Gangat, Ayalew Tefferi, Tiziano Barbui
Abstract
Ample evidence has been provided that accurate discrimination between essential thrombocythemia (ET) and early prefibrotic primary myelofibrosis (pre-PMF) has an impact not only on presenting laboratory data but also on complications, like thrombosis, progression to overt myelofibrosis (MF), transformation to blast phase (BP), and overall survival [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 ]. However, studies estimating the epidemiology of these critical events in the two entities have mainly focused on one isolated outcome at a time, without considering the entire spectrum of multiple intermediate disease states, possibly affecting probabilities and risk factors of the outcome of interest. This situation calls for a multistate model approach, a technique that allows a more in-depth insight into intermediate factors likely influencing the progressive transitioning from one status to another.