January 23rd 2024
Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
December 26th 2023
Therapeutic Phlebotomy, Hydroxyurea Improve Overall Survival in Geriatric Polycythemia Vera
November 9th 2018Treatments with therapeutic phlebotomy and hydroxyurea are associated with improved overall survival and decreased risk of thrombosis in older patients with polycythemia vera, according to a recent retrospective cohort study published in Blood Advances.
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PZ-2891 Offers New Therapeutic Option for Pantothenate Kinase-Associated Neurodegeneration
November 2nd 2018PZ-2891 is a new and unique therapeutic agent that has shown to be able to penetrate the blood-brain barrier and increase coenzyme A levels in mice, leading to increased weight, longer life span, as well as improving locomotor activity, based on results from a study by Sharma et al.
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Phlebotomy, Cytoreductive Treatments Are Effective Yet Underused in Older Patients With PV
October 29th 2018Guidelines indicate that high-risk patients with polycythemia vera (PV) should be treated with phlebotomy and cytoreductive therapy—such as hydroxyurea or interferon-alfa—to reduce the risk of thrombosis, the major cause of death among patients with PV. To assess the effectiveness of these treatment modalities among older US adults with PV, the authors of a newly published paper evaluated a large cohort of patients with PV in the real-world setting.
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Smoking Is Linked With Increased Risk of Myeloproliferative Neoplasms
October 23rd 2018A recently published cohort study with a register-based follow-up of some individuals from the Danish general population sought to investigate whether smokers are at an increased risk for developing MPNs versus those who have never smoked.
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FDA Approves Hemlibra for Hemophilia A With or Without Factor VIII Inhibitors
October 19th 2018With its new FDA approval, Hemlibra (emicizumab-kxwh), a bispecific factor IXa- and factor X-directed antibody, has become the first prophylactic treatment for patients with hemophilia A with or without factor VIII inhibitors.
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Children With B-Cell Precursor ALL Have Dysregulated Immune Function
October 11th 2018Abnormal concentrations of inflammatory markers detected in children with B-cell precursor acute lymphoblastic leukemia (ALL) support the theory that children with ALL are born with dysregulated immune function.
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Thromboembolic Events Are Associated With Higher Costs in Patients With Polycythemia Vera
October 9th 2018A recent paper sought to address the knowledge gap in the literature with respect to the effects of cardiovascular events and thromboembolic events on healthcare cost associated with patients with polycythemia vera receiving hydroxyurea.
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ODC1 Gene Mutation Leads to Alopecia and Developmental Delays
October 5th 2018The ornithine decarboxylase 1 (ODC1) gene, for the first time in humans, has been linked to a new pediatric disorder leading to macrosomia, macrocephaly, developmental delays, alopecia, hypotonia, and hearing loss, according to a new report.
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Alternative Ruxolitinib Dosing Regimen May Mitigate Anemia in Patients With Myelofibrosis
October 2nd 2018A recent open-label, single-arm, phase 2 study sought to assess evaluate an alternative dosing regimen for adults myelofibrosis (MF) to assess whether such a regimen could mitigate anemia.
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PROs From the REVEAL Study Show Impact of PV on Patients' Lives
September 24th 2018The ongoing REVEAL study, a prospective observational study of US patients with the myeloproliferative neoplasm polycythemia vera (PV), is designed to gather data on disease burden, clinical management, patient-reported outcomes (PROs), and healthcare resource utilization of this patient population.
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Epigenomic Analysis Uncovers Link Between Rheumatoid Arthritis and Huntington Disease
September 19th 2018In their study published in Nature Communications, the authors mapped out the epigenomic profiles of rheumatoid arthritis (RA) and osteoarthritis for the first time to provide insight on RA-specific pathways and transcription factor motifs. A surprising discovery was that the Huntingtin-interacting protein-1 may play a role in the pathogenesis of RA.
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Prevalence of Autoimmune Myelofibrosis in Patients With Lupus May Be Underestimated
September 18th 2018While the pathogenesis of autoimmune myelofibrosis is not completely understood, in patients with systemic lupus erythematosus, it may be triggered by circulating immune complexes that induce megakaryocytes to release platelet-derived growth factor.
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Study in Blood Identifies Novel Mutations in Triple-Negative Myeloproliferative Neoplasms
September 12th 2018Using whole-exome sequencing and evaluating germline mutations, researchers have discovered novel mutations in several genes among patients with essential thrombocythemia and primary myelofibrosis, which could influence the treatment they receive.
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A Minority of Patients With High-Risk Polycythemia Vera Receive Cytoreductive Medication
September 9th 2018According to treatment recommendations from the European LeukemiaNet and the National Comprehensive Cancer Network, patients with high-risk polycythemia vera (PV) should receive cytoreductive medication together with standard treatments for low-risk PV: phlebotomy and low-dose aspirin.
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Cerebello-spinal tDCS Simulation Helps Reduce Neurodegenerative Ataxia Symptoms
September 6th 2018Patients with neurodegenerative ataxia that are treated with cerebello-spinal transcranial direct current simulation (tDCS) have significant improvements in gait, upper limb coordination, quality of life, and other neurological evaluations of cerebellar deficits, according to results from the CStDCSAtaxia trial.
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The COMBI study, a prospective, open‐label, single‐arm phase 2 study ongoing in Denmark, is investigating the feasibility of treating patients with low- to intermediate-risk myelofibrosis or polycythemia vera with low-dose pegylated interferon alfa-2 in combination with ruxolitinib.
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Phase 2 STARS Trial Results Demonstrate Efficacy of OV101 for Treating Angelman Syndrome
September 2nd 2018Ovid Therapeutics Inc., a biopharmaceutical company, recently announced that the phase 2 STARS trial, which tested the efficacy of the investigational drug OV101, achieved its primary endpoint of safety and tolerability for treating Angelman syndrome.
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Lymphadenopathy May Be an Early Manifestation of Primary Myelofibrosis
August 29th 2018Extramedullary hematopoiesis—the presence of hematopoietic activity in sites outside of the bone marrow—can occur in malignant and non-malignant hematologic diseases, and they may be a complication of primary myelofibrosis.
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