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
Study Examines Relapse Risk in GVHD After Allo-HCT Using 2 Therapy Options
December 9th 2021Despite the increase in relapse risk, 2-year survival for patients with graft-versus-host disease (GVHD) who received matched sibling donor (MSD) allogeneic hematopoietic cell transplant (allo-HCT) administered post-transplant cyclophosphamide remained similar compared with patients receiving cyclosporine A with methotrexate prophylactically.
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Researchers Identify Novel Biomarker, Therapeutic Target for Polycythemia Vera
November 24th 2021The data set included 177 samples from polycythemia vera, essential thrombocytopenia, primary myelofibrosis, and healthy donors, from which the researchers found that MAPK14 was overexpressed in PV samples and was associated with more symptoms and worse outcomes.
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Significant Amount of Patients Remain on Sick Leave 1 Year Following Allo-HSCT, Study Says
November 17th 2021The findings showed that between 2009 to 2016, 76% of the 122 patients included in the study were on sick leave a year following their allogeneic hematopoietic stem cell transplantation (allo-HSCT).
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GVHD Insights Point to Importance of Immunometabolism to Control Disease
November 13th 2021According to the study authors, a growing number of findings from preclinical studies have suggested a focus on immunometabolism to modulate alloreactive donor T cell responses to control graft-versus-host disease (GVHD) and promote graft-versus-tumor effect.
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Patients With MPNs Face Higher Cardiovascular Risks, in Addition to AML Risk
October 30th 2021A new analysis suggests physicians should be on the lookout for cardiovascular disease and other types of cancer, in addition to acute myeloid lymphom, when caring for patients with certain myeloproliferative neoplasms (MPNs).
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Ruxolitinib Has Cost-effectiveness, Survival Benefits for Myelofibrosis vs Competitors
October 6th 2021Research presented at EHA2021 shows that the JAK 1/2 inhibitor ruxolitinib produced cost-effective benefits per quality-adjusted life-year and increased the overall survival rate for patients with myelofibrosis.
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Management of Low-risk ET and PV Has Room for Improvement
October 3rd 2021Despite excellent prognoses in low-risk patients with essential thrombocytopenia (ET) and polycythemia vera (PV), knowledge gaps remain and novel, more tolerable therapies require reevaluation of treatment algorithms.
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Pediatric AADC Deficiency Improves With New Gene Therapy
October 1st 2021This new analysis of 5-year data on a novel gene therapy from PTC Therapeutics shows both cognitive and motor function improvement in children with aromatic L-amino acid decarboxylase (AADC) deficiency, a rare central nervous system disorder.
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Researchers Detail Case of Statin-Induced Necrotizing Myositis
September 26th 2021Immune-mediated necrotizing myopathy typically presents with progressive symmetrical proximal muscle weakness and myalgias, although some cases can present with respiratory and esophageal muscle weakness, as seen in this reported case study.
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Managing Cerebrovascular Complications in Patients With MPN
September 18th 2021Complications of note in patients with myeloproliferative neoplasm (MPNs) include ischemic stroke, intracerebral and subarachnoid hemorrhage, microbleeds, posterior reversible encephalopathy syndrome, and dural sinus and cerebral vein thrombosis.
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Multistakeholder Panel Weighs In on Which Factors to Consider for Reimbursement of Orphan Drugs
September 11th 2021From the decision analysis came an agreement that reimbursement for an orphan drug should be based primarily on its effect on health-related quality of life, its effectiveness, and the availability of other treatment options.
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Family Caregivers of Patients With Gaucher Disease Report Lower Quality-of-Life Scores
September 4th 2021Gaucher disease is a rare metabolic condition that causes a fatty substance to build up in the organs and bones. Patients can suffer liver enlargement, anemia, and reduced platelets; they experience fatigue, bone infarctions, and permanent disability or death.
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Patients, Caregivers Report Lack of Supportive Services for Inherited Metabolic Diseases
August 25th 2021The European-based survey collected information from over 900 patients and caregivers from 25 countries; the responses show a need for both local and national governments to scale up their services for patients with rare metabolic diseases, which also have a positive spillover effects for caregivers.
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Using Ruxolitinib to Target JAK1/2 in T-LBL May Prevent Treatment Resistance
August 21st 2021For pediatric patients with T-cell origin lymphoblastic leukemia (T-LBL) with disease progression following second-line treatment, investigators found that the JAK1/2 pathway may be a new actionable target through treatment with ruxolitinib.
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