Clinical outcomes under hydroxyurea treatment in polycythemia vera: a systematic review and meta-analysis

Alberto Ferrari, Alessandra Carobbio, Arianna Masciulli, Arianna Ghirardi, Guido Finazzi, Valerio De Stefano, Alessandro Maria Vannucchi, Tiziano Barbui

Research output: Contribution to journalArticlepeer-review

Abstract

Hydroxyurea is the standard treatment in high-risk patients with polycythemia vera. However, estimates of its effect in terms of clinical outcomes (thrombosis, bleeding, hematologic transformations and mortality) are lacking. We performed a meta-analysis to determine the absolute risk of events in recent cases of patients under hydroxyurea treatment. We searched for relevant articles or abstracts in the following databases: Medline, EMBASE, clinicaltrials.gov, WHO International Clinical Trials Registry, LILACS. Sixteen studies published from 2008 to 2018 reporting number of events using World Health Organization diagnosis for polycythemia vera were selected. Through a random effect logistic model, incidences, study heterogeneity and confounder effects were estimated for each outcome at different follow ups. Overall, 3,236 patients were analyzed. While incidences of thrombosis and acute myeloid leukemia were stable over time, mortality and myelofibrosis varied depending on follow-up duration. Thrombosis rates were 1.9%, 3.6% and 6.8% persons/year at median ages 60, 70 and 80 years, respectively. Higher incidence of arterial events was predicted by previous cardiovascular complication. Leukemic transformation incidence was 0.4% persons/year. Incidence of transformation to myelofibrosis and mortality were significantly dependent on age and follow-up duration. For myelofibrosis, rates were 5.0 at five years and 33.7% at ten years; overall mortality was 12.6% and 56.2% at five and ten years, respectively. In conclusion, we provide reliable risk estimates for the main outcomes in polycythemia vera patients under hydroxyurea treatment. These findings can help design comparative clinical trials with new cytoreductive drugs and prove the feasibility of using critical end points for efficacy, such as major thrombosis.

Original languageEnglish
Pages (from-to)2391-2399
Number of pages9
JournalHaematologica
Volume104
Issue number12
DOIs
Publication statusPublished - Dec 2019

Fingerprint

Dive into the research topics of 'Clinical outcomes under hydroxyurea treatment in polycythemia vera: a systematic review and meta-analysis'. Together they form a unique fingerprint.

Cite this