Pharmacotherapy of essential thrombocythemia.
BACKGROUND
The natural history of essential thrombocythemia is characterized by an increased incidence of thrombotic and hemorrhagic events and, in the long-term, a tendency for disease transformation to myelofibrosis or acute leukemia. Advanced age and a prior history of thrombosis are the major predictors of thrombotic complications.


OBJECTIVE
The aim of this study was to outline the current evidence and the authors' opinion regarding the clinical management of patients with essential thrombocythemia.


METHODS
The study reviewed the pertinent literature addressing the management options for essential thrombocythemia patients.


CONCLUSIONS
Cytoreductive agents can reduce the rate of thrombotic events, but do not affect the overall survival or rate of disease transformation. Thus, a risk-adapted strategy is recommended for the management of patients with essential thrombocythemia. High-risk patients with essential thrombocythemia should be treated with cytoreductive therapy and low-dose aspirin. Hydroxycarbamide is the agent of choice in most patients: however, interferon-alpha is a reasonable alternative in young patients, pregnancy or those intolerant of hydroxycarbamide. Low-risk patients can be safely observed. The management of intermediate-risk patients needs to be individualized: however, low-dose aspirin can be used after excluding acquired von Willebrand's disease. In the future we await the role of molecularly targeted therapy with JAK2 inhibitors in high-risk essential thrombocythemia patients.
Gangat
Naseema
N
Mayo Clinic, Division of Hematology, 200 First Street SW, Rochester, MN 55905, USA.
Tefferi
Ayalew
A
D000367:Age Factors; D000970:Antineoplastic Agents / Q000627:therapeutic use; D001241:Aspirin / Q000627:therapeutic use; D018450:Disease Progression; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use; D016898:Interferon-alpha / Q000627:therapeutic use; D053614:Janus Kinase 2 / Q000037:antagonists & inhibitors; D010975:Platelet Aggregation Inhibitors / Q000627:therapeutic use*; D012307:Risk Factors; D013920:Thrombocythemia, Essential / Q000097:blood / Q000188:drug therapy*
D000970:Antineoplastic Agents; D016898:Interferon-alpha; D010975:Platelet Aggregation Inhibitors; D053614:Janus Kinase 2; D001241:Aspirin; D006918:Hydroxyurea