Disease characteristics and clinical outcome in young adults with essential thrombocythemia versus early /prefibrotic primary myelofibrosis.

Barbui T
Thiele J
Carobbio A
Rumi E
Randi ML
Bertozzi I
Vannucchi AM
Gisslinger H
Gisslinger B
Finazzi G
Ruggeri M
Rodeghiero F
Rambaldi A
Gangat N
Tefferi A.
PASSAMONTI, FRANCESCO

Publication date

January 2012

DOI

10.1182/blood-2012-01-407981

Abstract

This study investigates disease characteristics and clinical outcome in young patients (<40 years) with WHO-defined essential thrombocythemia (ET) in comparison to early/prefibrotic primary myelofibrosis (PMF) with presenting thrombocythemia. We recruited 213 young patients (median age 33.6 years) including 178 patients (84%) consistent with WHO-defined ET and 35 patients (16%) showing early PMF. Median follow-up was 7.5 years. A trend for more overall thrombotic, particularly arterial, complications was seen in early PMF compared to ET. Progression to overt myelofibrosis was 3% in ET and 9% in early PMF, but no transformation into acute leukemia was observed. By combining all adverse events (thrombosis, bleeding, myelofibrosis) the rate wa...