Management of symptoms in polycythemia vera and essential thrombocythemia patients

Abstract

The BCR-ABL-negative myeloproliferative neoplasms (MPNs) are clonal stem cell derived malignancies, which include polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF). The MPNs are characterized by dysregulated JAK-STAT signaling pathways. PV and ET are associated with an increased risk of thrombo-hemorrhagic complications, risk of progression to MF and leukemia. Presentation of patients with PV and ET is variable and usually as a result of abnormal full blood count indices (raised hemoglobin and hematocrit, leukocytosis, and thrombocytosis). Presentation with thrombosis or splenomegaly occurs in ϳ30% of patients. Historically thought of as indolent compared with MF, patients with PV and ET have significant disease symptom burden which does not directly correlate to the current clinical prognostic classifications. The mainstay of therapy is reserved for patients with high-risk disease and thus excludes a population of patients with significant symptom related morbidity impacting their quality-of-life and survival. Recent treatment strategies have aimed to incorporate disease burden assessment into the selection of therapeutic interventions such as JAK2 inhibitors and HDAC inhibitors. We will review the advances in the field of MPN symptom assessment and symptom burden experienced by ET and PV patients. We will also discuss the risk-stratified management of ET and PV patients alongside symptom assessment and the impact of potential novel therapies, for patients who fail to respond to conventional treatment. Learning Objectives• To recognize the symptom burden in patients with PV and ET • To review the current management of symptoms in patients with PV and ET • To consider impact of novel treatments on symptom burden Polycythemia vera (PV) and essential thrombocythemia (ET) are 2 of the 3 classical bcr-abl-negative myeloproliferative neoplasms (MPNs) characterized by clonal stem-cell proliferation and a dysregulated JAK-STAT pathway.

Risk of venous thromboembolism in pregnant women with essential thrombocythemia: a systematic review and meta-analysis

“…2,3 Published studies in women with ET have primarily focused on the outcomes of live birth rate and pregnancy complications, 4-8 with little data available on the absolute risk of VTE (deep vein thrombosis [DVT] and pulmonary embolism [PE]) during pregnancy or the 6-week postpartum period.…”

Profile of Polycythemia Vera in Campus Teaching Hospital of Lome - Togo

“…Similar results were found by several authors who concluded that the presence of this mutation may play a role in the development of vascular complications. [22,25,26] These results show that Jak2V617 mutation would affect the severity of signs and occurrence would be a factor of progressive complications.…”

Partial status epilepticus in cerebral venous sinus thrombosis, initial manifestation of polycythemia vera

“…Proposed mechanisms are increased blood viscosity (increased number of clonal cells), easy activation of platelets, erythrocyte, and plate abnormalities that were found to be associated with JAK-2 V617F mutation and endothelium impairment. [4] Accordingly, JAK-2 V617F mutation is confirmed as an independent risk factor for thrombosis. [4] Dentali et al investigated the frequency of PV in a series of 706 patients with cerebral vein thrombosis (CVT) and the frequency of CVT in a cohort of 2,143 MPNs (735 with PV) patients.…”

“…[4] Accordingly, JAK-2 V617F mutation is confirmed as an independent risk factor for thrombosis. [4] Dentali et al investigated the frequency of PV in a series of 706 patients with cerebral vein thrombosis (CVT) and the frequency of CVT in a cohort of 2,143 MPNs (735 with PV) patients. [5] The incidence of MPN in CVT patients was 3.8%; however, among 735 PV patients, only 5 (0.7%) were documented to have CVT.…”

“…[7] Rather than splanchnic venous thrombosis, CVT was the presenting sign of PV in our case. This patient was also a high-risk PV patient (leukocyte count >15 × 10 9 /L, age 57–66, venous thrombosis)[4] demanding careful follow-up.…”