The observational, noninterventional study was part of an emicizumab clinical development program in which a daily bleed and medication questionnaire developed by the sponsor on a handheld device was used to prospectively collect data on treatment with factor VIII or bypassing agents in adult and adolescent people with hemophilia A, with and without inhibitors to FVIII.
Study Confirms Safe Use of Opioids for Pain Control in Sickle Cell Disease
Continuous Infusion of Recombinant Activated Factor VII Safe, Effective in Management of Congenital Hemophilia
Pulmonary hypertension (PH) has been reported to be associated with myeloproliferative neoplasms (MPN) in 5% to 48% of MPN patients. Now, authors of the largest PH study in patients with Philadelphia chromosome-negative MPN have concluded that the prevalence of PH is lower than has been previously reported.
The British Society for Haematology has released a new updated guideline that offers practical guidance for the management of specific situations and complications of polycythemia vera and outlines management of the diverse types of secondary erythrocytosis.
There have been few therapeutic options for treating thrombocytopenia in MDS patients. Now, early phase data suggest that CC-486 (oral azacytidine, an investigational drug sponsored by Celgene) is a relatively safe and effective treatment for thrombocytopenic patients with MDS.
Treatment of acute myeloid leukemia (AML) has remained a challenge, partly because of an insufficient understanding of the molecular mechanisms that promote and maintain the leukemic state of AML cells.
Treatment with the investigational drug crizanlizumab (SEG101) reduced pain in patients with sickle cell disease (SCD) who were experiencing vaso-occlusive crisis according to posthoc results of the phase II SUSTAIN study published online in The American Journal of Hematology. The study found that more than twice as many patients with SCD treated with crizanlizumab did not experience a VOC compared with those treated with placebo.
If the trial is successful, venglustat could be the first treatment to target the mechanism of action in autosomal dominant polycystic kidney disease (PKD), which affects 120,000 people in the United States.
Treatments 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.
Researchers at Cornell University have developed a new tool to study genetic switches active in human glioblastoma tumors that drive the growth of the cancer.
PZ-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.
Guidelines 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.
In patients with progressive multifocal leukoencephalopathy (PML), BK virus-specific T-cell infusions have been shown to clear JC virus in the cerebrospinal fluid (CSF) and relieve clinical symptoms, according to results of a study by Muftuoglu et al.
A 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.
With 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.
Myeloproliferative neoplasms have varied progression rates, and a complex genetic landscape may contribute to heterogeneity in the outcomes of patients with these diseases.
Abnormal 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.
A 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.
The 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.
A 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.
The new initiative will not focus on donor-related bone marrow transplants in sickle cell disease, but instead will home in on genetic modifications of a patient’s own stem cells.
The 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.
In 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.
