Editorial
Volume 3 Issue 4 - December 2018
DOI: 10.19080/JTMP.2018.03.555620
J Tumor Med Prev
Copyright © All rights are reserved by Nahla AM Hamed
Polycythemia Vera: Current Therapeutic
Approaches
Nahla AM Hamed*
Department of Hematology, Faculty of Medicine, Alexandria University, Egypt
Submission: November 05, 2018; Published: December 07, 2018
*Corresponding author: Nahla AM Hamed, Department of Hematology, Faculty of Medicine, Alexandria University, Egypt
Introduction
PV is one of the lower-risk subtypes of MPNs [1] that is
characterized primarily by clonal erythrocytosis [2]. It has
an annual incidence of 0.21-2.27 per 100,000. Median age at
diagnosis is estimated at 71 years. A male preponderance is noted.
JAK2 V617F is found in >95% of PV patients and JAK2 exon 12
mutations in ~4% [3]. JAK2 exon 12 mutations such as insertions
or deletions are relatively specific to JAK2V617F-negative PV
and not found in ET and PMF [4]. JAK2 homozygosity is neither
necessary nor sufficient for a PV phenotype as indicated by
presence of small or undetectable homozygous clones in some PV
patients [5]. Heterozygous mutant erythroblasts of PV patients
have distinct transcriptional profiles that precede acquisition of
homozygosity and reflect differential activation of phosphorylated
STAT1, which modulate erythropoiesis [5]. CALR and MPL have
distribution frequency of 0 and 0% [6]. Other mutations (e.g.,
LNK) have been reported [2]. Co-occurrence of CALR mutation
exon 9 with JAK2V617F, usually occurs in less than 1% of PV [4].
JAK2, MPL, and calreticulin mutations are driver mutations
that activate the JAK2 pathway, but additional recurrent somatic
mutations in several genes (TET2, ASXL1, DNMT3A, CBL, LNK,
IDH1/2, IKF1, EZH2, TP53, and SRSF2), encoding transcriptional
and epigenetic regulators and signaling proteins, modulate
disease progression and can also occur as a primary mutation [3].
Clinical features of PV include mild-to moderate degree
of splenomegaly, mild-to-moderate degree of constitutional
symptoms, including fatigue and pruritus [6] (usually after bathing)
[2], symptoms of hyperviscosity, leukocytosis, thrombocytosis,
microvascular symptoms (e.g., headaches, lightheadedness,
visual disturbances [6] e.g., blurry vision [2], atypical chest pain,
acral paresthesia and erythromelalgia [6] i.e., erythema, warmth,
and pain in distal extremities) [2], and thrombotic and bleeding
complications [6].
The risk of thrombosis exceeds 20% [7]. History of
hypertension predicted arterial thrombosis and advanced age
venous thrombosis [7]. Hepatic and portal vein thrombosis
is a well-recognized phenotypic association with JAK2 V617F
and is often observed in younger women, with either a masked
phenotype, or lower leukocyte counts and lower allelic burdens.
The affected patients present a unique exception with regard to
demographics, clinical phenotype, and allelic burden [8]. Some
patients may develop AvWS, especially in the presence of extreme
thrombocytosis (platelets >1000 x 10
9
/L) and are at risk for
aspirin associated bleeding [7].
JAK2 oncogenes homozygosity may result in distinct
tumorigenic consequences, but direct evidence has been elusive
(5). JAK2V617F homozygosity was associated with reduced
platelet survival that is likely to reflect increased platelet apoptosis
and/or clearance. Moreover, homozygosity results in reduced
platelets numbers, consistent with the lower platelet levels seen
in PV compared with ET [5]. Increased JAK2V617F signaling
may enhance (or reduce) platelet reactivity, a concept that may
be relevant to thrombotic (or hemorrhagic) complications in PV
patients [5].
JAK2 V617F homozygous allele burden has been associated
with older age, male sex, pruritus, and splenomegaly; associations
between homozygous or increasing allelic burdens and thrombosis
J Tumor Med Prev 3(4): JTMP.MS.ID.555620 (2018) 0068
Abstract
The hemoglobin level threshold required to diagnose PV is now established at 16.5 g/dL for men and 16 g/dL for women by the 2016 WHO
classification for MPNs. The clinical course of PV might be interrupted by thrombohemorrhagic complications and disease transformation to MF
or acute myeloid leukemia. JAK2 oncogenes homozygosity may result in distinct tumorigenic consequences. Different therapeutic strategies and
good candidates’ patients for such therapies are illustrated.
Keywords: Polycythemia vera; Thrombohemorrhagic; Tumorigenic; Myeloid leukemia
Abbreviations: PV: Polycythemia Vera; STAT: Signal Transducer and Activator of Transcription; AML: Acute myeloid leukemia; MF: Myelofibrosis;
MPNs: Myeloproliferative neoplasms