Better Preventive Stroke Management Needed for SCD
A new study using real-world data of patients with sickle cell disease (SCD) indicates that rates of ischemic stroke are still high in both children and adults, underscoring the need for better screening and ...
SCD Gene Therapies Present Major Challenges to State Medicaid Budgets
An updated financial analysis on gene therapy treatment for sickle cell disease (SCD), published in Blood Advances, reveals an even greater potential impact on state Medicaid budgets ...
Iron Deficiency Remains Unresolved Years After Diagnosis
The majority of patients with iron deficiency did not have resolution of their condition within three years, according to a statewide retrospective study of more than 13,000 Minnesota ...
More Study Needed to Increase Usage of Hydroxyurea in Infants with SCD
Recent data from the first randomized trial on hydroxyurea treatment for preventing silent cerebral infarctions (SCIs) in infants with sickle cell disease (SCD) add support to early initiation of ...
HCT Shows Better Outcomes in Young Pediatric Patients With Fanconi Anemia
Data from the largest series of pediatric patients with Fanconi anemia (FA) to date suggest that hematopoietic cell transplantation (HCT) should be offered at a young age in patients with aplastic anemia (AA) or ...
Bivalent Lentiviral-Based Gene Therapy Might Decrease Costs in Sickle Cell Disease
The results of a recently published study in Molecular Therapy: Methods & Clinical Development describe a new bivalent lentiviral-based gene therapy approach for sickle cell disease ...
Full Results of Exa-Cel Study Show Continued Safety, Efficacy in Sickle Cell Disease
In December 2023, the U.S. Food and Drug Administration (FDA) approved exagamglogene autotemcel (exa-cel) for the treatment of severe sickle cell disease (SCD) in people 12 years or older who ...
Addition of Avatrombopag to Cyclosporine Can Improve Response Rate in Elderly Patients with Transfusion-Dependent, Non-Severe Aplastic Anemia
Combining avatrombopag with cyclosporine improved platelet levels and improved overall response rates (ORR) and complete response (CR) rates among elderly patients with transfusion-dependent, non-severe ...
CDC Guideline Results in Drop in Opioid Prescriptions for Patients with SCD
In 2016, the U.S. Centers for Disease Control and Prevention (CDC) released the Guideline for Prescribing Opioids for Chronic Pain to provide recommendations to primary care clinicians who prescribe ...
Hepcidin Mimetic Rusfertide Nearly Eliminates Need for Phlebotomies in PV
Treatment with rusfertide, an injectable peptide mimetic of the master iron regulatory hormone hepcidin, essentially eliminated the need for therapeutic phlebotomies in patients with polycythemia vera (PV) who were heavily ...
