Myeloproliferative neoplasms (MPNs) are rare, clonal blood disorders which originate from mutated hematopoietic stem cells.1,2 JAK2-activating mutations drive most MPNs, including the most common MPN, polycythemia vera (PV).2,3 PV is a progressive, chronic, and acquired blood cancer characterized by an overproduction of red blood cells, and is often associated with excess production of white blood cells and platelets.3 Patients with PV have an increased risk of thrombosis, and myelofibrotic and/or leukemic progression. Symptoms may interfere with daily activities, family/social life, and work productivity.4-7
For all patients with PV, the goal of treatment is hematocrit (Hct) control of less than 45%, which lowers the risk of thrombosis and the severity of symptoms.8 Most patients with low-risk PV are phlebotomy dependent, but the effect is transient and repeated phlebotomy may exacerbate PV-related symptoms.9,10
The INTREPID study (NCT07429266) is a Phase 3, randomized, double-blind, placebo-controlled, global study to evaluate the efficacy and safety of sapablursen when added to current PV therapy in patients with phlebotomy-dependent PV.
For a full list of study locations, please visit clinicaltrials.gov.
References
1) Khoury JD, et al. Leukemia. 2022;36(7):1703-19.
2) Gerds AT, et al. JNCCN. 2022;20(9):1033-62.
3) Tefferi A, et al. Am J Hematol. 2023;98(9):1465-87.
4) Hultcrantz M, et al. Ann Intern Med. 2018;168(5):371-25
5) Szuber N, et al. Am J Hematol. 2018;93(12):1474-84.
6) Cerquozzi S, et al. Blood Cancer J. 2014;5(11):e366.
7) Grunwald MR, et al. Clin Lyomphoma Myeloma Leuk. 2019;19(9):579-84.
8) Marchioli R, et al. N Engl J Med. 2013;368(1):22-23.
9) Vannucchi AM, et al. N Engl J Med. 2015;372(5):426-35.
10) Casu C, et al. Blood. 2018;131(16):1790-4.