Ruxolitinib versus Standard Therapy for the Treatment of Polycythemia Vera

• Published in: The New England journal of medicine Vol. 372; no. 5; pp. 426 - 435
• Main Authors: Vannucchi, Alessandro M, Kiladjian, Jean Jacques, Griesshammer, Martin, Masszi, Tamas, Durrant, Simon, Passamonti, Francesco, Harrison, Claire N, Pane, Fabrizio, Zachee, Pierre, Mesa, Ruben, He, Shui, Jones, Mark M, Garrett, William, Li, Jingjin, Pirron, Ulrich, Habr, Dany, Verstovsek, Srdan
• Format: Journal Article
• Language: English
• Published: United States Massachusetts Medical Society 29-01-2015
• Subjects: Adult; Aged; Aged, 80 and over; Antineoplastic Agents - adverse effects; Antineoplastic Agents - therapeutic use; Blood cancer; Blood Cell Count; Blood diseases; Cancer; Drug dosages; Drug therapy; Female; Hematocrit; Herpes zoster; Humans; Hydroxyurea; Hydroxyurea - adverse effects; Hydroxyurea - therapeutic use; Inhibitor drugs; Janus kinase; Janus Kinases - antagonists & inhibitors; Kaplan-Meier Estimate; Male; Middle Aged; NMR; Nuclear magnetic resonance; Organ Size - drug effects; Patients; Phlebotomy; Polycythemia; Polycythemia vera; Polycythemia Vera - drug therapy; Pyrazoles - adverse effects; Pyrazoles - pharmacology; Pyrazoles - therapeutic use; Remission; Remission Induction; Restless legs syndrome; Side effects; Spleen; Spleen - drug effects; Spleen - pathology; Splenomegaly; Thrombocytopenia; Thrombocytopenia - chemically induced; Thromboembolism; Thrombosis; Tumors
• ISSN: 0028-4793; 1533-4406
• DOI: 10.1056/NEJMoa1409002

Ruxolitinib, an oral inhibitor of Janus kinase (JAK) 1 and 2, was associated with hematocrit control and spleen size reduction in 21% of patients with polycythemia vera who had an inadequate response to or unacceptable side effects from hydroxyurea.
Polycythemia vera is a chronic clonal myeloproliferative neoplasm characterized by increased red-cell mass; elevated white-cell and platelet counts are also common.
1
Patients have an increased risk of thrombotic and cardiovascular events
2
and a substantial symptom burden that includes pruritus, fatigue, and night sweats.
3
Splenomegaly often develops as the disease progresses.
4
The main goal of therapy is to prevent thrombotic events while avoiding iatrogenic harm and minimizing the risk of transformation to post–polycythemia vera myelofibrosis or acute myeloid leukemia (AML).
5
,
6
Most patients receive low-dose aspirin and undergo phlebotomy,
7
with a goal of maintaining hematocrit values of less than 45%. Aggressive . . .