November 15th 2024
Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve function after 52 weeks.
Early Initiation of Ruxolitinib Reduces Risk, Severity of aGVHD
June 3rd 2021The study of 57 patients with acute graft-versus-host disease (aGVHD) also showed that patients tolerated the treatment, offering promise for preventing and managing the complication of transplantation, which affects up to half of patients.
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Real-world Evidence Supports Conditional Use of IV Formula in Hospitalized Infants With MSUD
May 16th 2021Hospitalized infants with the rare metabolic disorder maple syrup urine disease (MSUD), who are intolerant to oral or enteral administration of branched-chain amino acid-free formula, may benefit from an intravenous formulation.
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Complications of Steroid Use in GVHD Associated With Higher Health Care Usage, Costs
May 7th 2021For patients with acute and chronic graft-vs-host disease (GVHD), systemic treatment with steroids is the standard of care, and although steroid-related complications are common, the associated health care resource utilization and costs are not well documented.
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Novel Approaches to Treating Polycythemia Vera Needed, Say Researchers
April 29th 2021The current treatment landscape of polycycthemia vera (PV) hinges on treatments like hydroxyurea and ruxolitinib, the latter having emerged as an effective second-line therapy in patients with severe pruritis, symptomatic splenomegaly, or post-PV myelofibrosis symptoms.
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Assessing Best Practices in Managing Pregnancy, Myeloproliferative Neoplasms
April 24th 2021Researchers discuss the unique fetal and maternal challenges for pregnant women with myeloproliferative neoplasms, with insight and recommendations provided on the potential benefit of aspirin therapy, cytoreductive therapy, and systemic anticoagulation.
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Assessing Novel Treatments to Reduce Thrombosis in PV, ET Requires New Approaches
March 20th 2021According to the researchers, few treatments have been able to reduce the thrombotic burden in patients with polycythemia vera (PV) and essential thrombocytopenia (ET), and progress on this front has been hindered by a lack of studies designed to assess a treatment’s impact on thrombotic events.
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