Disease characteristics and clinical outcome in young adults with essential thrombocythemia versus early/prefibrotic primary myelofibrosis.
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), including 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.
Ospedali Riuniti di Bergamo, Largo Barozzi 1, Bergamo, Italy. tbarbui@ospedaliriuniti.bergamo.it
Tiziano
T
Barbui
Jürgen
J
Thiele
Alessandra
A
Carobbio
Francesco
F
Passamonti
Elisa
E
Rumi
Maria Luigia
ML
Randi
Irene
I
Bertozzi
Alessandro M
AM
Vannucchi
Heinz
H
Gisslinger
Bettina
B
Gisslinger
Guido
G
Finazzi
Marco
M
Ruggeri
Francesco
F
Rodeghiero
Alessandro
A
Rambaldi
Naseema
N
Gangat
Ayalew
A
Tefferi
D000293:Adolescent; D000328:Adult; D017668:Age of Onset; D016208:Databases, Factual / Q000706:statistics & numerical data*; D018450:Disease Progression; D018572:Disease-Free Survival; D005500:Follow-Up Studies; D006801:Humans; D053614:Janus Kinase 2 / Q000235:genetics; D053208:Kaplan-Meier Estimate; D055728:Primary Myelofibrosis / Q000401:mortality* / Q000628:therapy; D016016:Proportional Hazards Models; D012307:Risk Factors; D013920:Thrombocythemia, Essential / Q000401:mortality* / Q000628:therapy; D055815:Young Adult
C507924:JAK2 protein, human; D053614:Janus Kinase 2