Myeloproliferative neoplasms working group consensus recommendations for diagnosis and management of primary myelofibrosis, polycythemia vera, and essential thrombocythemia

Abstract

According to the 2008 revision of the World Health Organization (WHO) classification of myeloid malignancies, philadelphia chromosome (Ph)-negative myeloproliferative neoplasms (MPNs) include clonal, hematologic disorders such as polycythemia vera, primary myelofibrosis, and essential thrombocythemia.Recent years have witnessed major advances in the understanding of the molecular pathophysiology of these rare subgroups of chronic, myeloproliferative disorders. Identification of somatic mutations in genes associated with pathogenesis and evolution of these myeloproliferative conditions (Janus Kinase 2; myeloproliferative leukemia virus gene; calreticulin) led to substantial changes in the international guidelines for diagnosis and treatment of Ph-negative MPN during the last few years.The MPN-Working Group (MPN-WG), a panel of hematologists with expertise in MPN diagnosis and treatment from various parts of India, examined applicability of this latest clinical and scientific evidence in the context of hematology practice in India.This manuscript summarizes the consensus recommendations formulated by the MPN-WG that can be followed as a guideline for management of patients with Ph-negative MPN in the context of clinical practice in India.

Essential Thrombocythemia in a 15-Year-Old Female: Presentation, Workup, and Treatment Considerations in the Pediatric Population

“…Risk for thrombosis or hemorrhage also occurs at a lower rate in children compared with adults with ET; however, this is still a major complication ( Agarwal et al, 2015 ; Aladily et al, 2017 ; Ianotto et al, 2019 ). Risk for vascular events is increased with the presence of clonal mutations or very high platelet counts of over 1,500,000/L.…”

Are patients with high-risk polycythemia vera receiving cytoreductive medications? A retrospective analysis of real-world data

“…The Philadelphia chromosome-negative myeloproliferative neoplasms (MPNs), including polycythemia vera (PV), essential thrombocythemia, and myelofibrosis, are a group of clonal hematologic malignancies with overlapping pathology and clinical features [ 1 ]. PV is primarily characterized by erythrocytosis and constitutively active mutations in Janus kinase 2 [ 2 ].…”

Transfusion Independency and Histological Remission in a Patient with Advanced Primary Myelofibrosis Receiving Iron-Chelation Therapy with Deferasirox

“…At present, ruxolitinib is the only approved JAK2 inhibitor that has shown to reduce a symptomatic splenomegaly by 35% in 40% of patients, and to improve quality of life due to reduction of constitutional symptoms like weight loss, fever, itching, fatigue and abdominal discomfort [1]. In our patient, ruxolitinib was not considered because of a normal spleen size and lack of constitutional symptoms.…”

“…Anemia and transfusion dependency is a common problem in patients with primary myelofibrosis (PMF) [1,2]. The occurrence of anemia and transfusion dependency at diagnosis of PMF heralds a shortened survival [3].…”

“…However, for patients with myeloproliferative syndromes, especially PMF, no clear benefit of chelation therapy has been shown [12]. Current treatment guidelines in PMF do not recommend ICT in patients with transfusion dependency and iron overload [1], although an increasing number of publications indicate possible positive effects on hematopoiesis and also possible transfusion independency during ICT with deferasirox [2,9,13,14,15]. …”