Myeloproliferative Neoplasms

The priority review designation for pacritinib is based off results from 3 trials investigating pacritinib 200 mg twice daily for patients with myelofibrosis and severe thrombocytopenia.

CancerNetwork® spoke with Abdulraheem Yacoub, MD, during the American Association for Cancer Research Annual Meeting 2021 to discuss how predictors of response to therapy may be just as important as the development of new therapies for advancing outcomes in myelofibrosis and myeloproliferative neoplasms.

Adding parsaclisib to the ruxolitinib treatment of patients with myelofibrosis who had a suboptimal response on a standard dose of ruxolitinib alone led to improvements in spleen volume reduction and symptom burden.

The telomerase inhibitor demonstrated improved overall survival spleen response, and symptom response in patients with myelofibrosis.

Study results showed that adding navitoclax to ruxolitinib resulted in a clinically meaningful improvement in spleen volume and total symptom score in patients with myelofibrosis who no longer benefited from prior ruxolitinib therapy.

Treatment with momelotinib improved overall survival and sustained efficacy outcomes in patients with intermediate- or high-risk myelofibrosis.

The FDA approved the first and only erythroid maturation agent for the treatment of anemia in patients with lower-risk myelodysplastic syndromes.

This study highlighted the need for novel therapies in myelofibrosis treatment, given that salvage therapies could potentially improve patient outcomes.

In this second part of our two-part review, we discuss the use of mutation profiling in the diagnosis, prognosis, and treatment of patients with myeloproliferative neoplasms and other myeloid diseases.

A recent phase III study revealed a benefit with pacritinib for patients with myelofibrosis and thrombocytopenia who have experienced treatment failure with previous therapies.

This video highlights a study that establishes mutated CALR as a myeloproliferative neoplasms–specific tumor antigen and provides a rationale for the development of immunotherapies targeting mutated CALR.

This video reviews the phase I portion of a trial testing glasdegib in patients with primary/secondary myelofibrosis previously treated with JAK inhibitors.

This video highlights results of a phase I study of idasanutlin (RG7388) in patients with refractory polycythemia vera and essential thrombocythemia.

Survival of patients with myelofibrosis who undergo splenectomy is adversely affected by older age, the need for transfusion, and leukocyte and circulating blast cell counts, according to a new analysis.

A new study including two separate cohorts found that male gender is a significant risk factor for mortality in patients with essential thrombocythemia. Gender may play a bigger role than thrombosis history in determining risk.

In this review, we demonstrate the evolving landscape of clinical investigation as researchers and clinicians attempt to address the important therapeutic needs of patients with myeloproliferative neoplasms.

Patients with myelofibrosis resistant or intolerant to ruxolitinib may have an alternative treatment option with the JAK2-selective inhibitor fedratinib, according to the results of a phase II study.

The JAK2 and FLT3 inhibitor pacritinib induced significant and sustained spleen volume reduction and symptom reduction in patients with myelofibrosis.

A 60-year-old man with a history of coronary artery disease and JAK2 V617F–positive polycythemia vera presented to our bone marrow transplantation clinic with progressive fatigue, splenomegaly, and cytopenias.

Myeloproliferative neoplasms are a group of clonal myeloid cell–derived disorders characterized by myeloproliferation without dysplasia, bone marrow hypercellularity, and predisposition to thrombosis, hemorrhage, and bone marrow fibrosis.

A new study has confirmed that the symptom burden of patients with polycythemia vera is substantial, and is independent of a patient’s disease features.

A study found that patients with myeloproliferative neoplasms have a higher overall mortality rate due to death from hematologic malignancies and infections.

New data indicates that the order in which people acquired certain somatic mutations influenced several clinical features of myeloproliferative neoplasms.

Ruxolitinib was significantly better than the best available therapy in a phase III, open-label trial of patients with polycythemia vera who were resistant to or intolerant of hydroxyurea.

A rare subtype of disease known as atypical chronic myeloid leukemia (aCML) has been shown to be clinically distinct from a related condition known as unclassifiable myelodysplastic/myeloproliferative neoplasm.

In a late-breaking abstract presentation, Thorsten Klampfl, PhD, of the CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences in Vienna, presented data from a whole exome study of primary myelofibrosis patients, identifying a new specific molecular mutation

Palliation is a laudable concept and an important goal in the therapy of all patients with malignant disease. Unfortunately, in the current day and age, the adjective “palliative” is being used in a derogatory manner that suggests palliation of suffering somehow lessens the importance or impact that such a therapy has upon individuals with the disease.

By inhibiting inflammatory cytokines and controlling the signs and symptoms of myelofibrosis, the patient’s body condition improves as the disease is kept under good control. Ultimately, prolonged exposure of a patient with myelofibrosis to ruxolitinib at a clinically effective dose results in prolongation of the his or her life.

In this article, we provide updated data on ruxolitinib therapy for patients with myelofibrosis and offer expert opinion on the appropriate use of this agent in the community practice.

Is it time to revisit the therapeutic role of interferon alfa2 (IFN-α-2) in early myelofibrosis (MF) since new data show promising results? Silver et al from Weil-Cornell Medical College in New York has recently published the results of a prospective study, which used IFN-α-2 in the treatment of 17 patients with low and intermediate-1 risk categories, and >80% of patients showed either response or had stable disease with 2 patients achieving complete remission (CR), and seven partial remission (PR).