TY - JOUR
T1 - Disease characteristics and clinical outcome in young adults with essential thrombocythemia versus early/prefibrotic primary myelofibrosis
AU - Barbui, Tiziano
AU - Thiele, Jürgen
AU - Carobbio, Alessandra
AU - Passamonti, Francesco
AU - Rumi, Elisa
AU - Randi, Maria Luigia
AU - Bertozzi, Irene
AU - Vannucchi, Alessandro M.
AU - Gisslinger, Heinz
AU - Gisslinger, Bettina
AU - Finazzi, Guido
AU - Ruggeri, Marco
AU - Rodeghiero, Francesco
AU - Rambaldi, Alessandro
AU - Gangat, Naseema
AU - Tefferi, Ayalew
PY - 2012/7/19
Y1 - 2012/7/19
N2 - In the present study, we investigated disease characteristics and clinical outcome in young patients (< 40 years) with World Health Organization (WHO)-defined essential thrombocythemia (ET) compared with early/prefibrotic primary myelofibrosis (PMF) with presenting thrombocythemia. We recruited 213 young patients (median age, 33.6 years), ncluding 178 patients (84%) with WHO-defined ET and 35 patients (16%) showing early PMF. Median follow-up time was 7.5 years. A trend for more overall thrombotic complications, particularly arterial, was seen in early PMF compared with ET. Progression to overt myelofibrosis was 3% in ET and 9% in early PMF, but no transformation into acute leukemia was observed. Combining all adverse events (thrombosis, bleeding, and myelofibrosis), the rate was significantly different (1.29% vs 3.43% of patients/year, P = .01) in WHO-ET and early PMF, respectively. In multivariate analysis, early PMF and the JAK2V617F mutation emerged as independent factors predicting cumulative adverse events.
AB - In the present study, we investigated disease characteristics and clinical outcome in young patients (< 40 years) with World Health Organization (WHO)-defined essential thrombocythemia (ET) compared with early/prefibrotic primary myelofibrosis (PMF) with presenting thrombocythemia. We recruited 213 young patients (median age, 33.6 years), ncluding 178 patients (84%) with WHO-defined ET and 35 patients (16%) showing early PMF. Median follow-up time was 7.5 years. A trend for more overall thrombotic complications, particularly arterial, was seen in early PMF compared with ET. Progression to overt myelofibrosis was 3% in ET and 9% in early PMF, but no transformation into acute leukemia was observed. Combining all adverse events (thrombosis, bleeding, and myelofibrosis), the rate was significantly different (1.29% vs 3.43% of patients/year, P = .01) in WHO-ET and early PMF, respectively. In multivariate analysis, early PMF and the JAK2V617F mutation emerged as independent factors predicting cumulative adverse events.
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U2 - 10.1182/blood-2012-01-407981
DO - 10.1182/blood-2012-01-407981
M3 - Article
C2 - 22700720
AN - SCOPUS:84864136100
SN - 0006-4971
VL - 120
SP - 569
EP - 571
JO - Blood
JF - Blood
IS - 3
ER -