Red Blood Cell & Iron Disorders

Red Blood Cell & Iron Disorders

A collection of features and news articles published in ASH Clinical News related to red blood cell and iron disorders.

Givosiran Reduces Porphyria Attacks Among Patients With Acute Intermittent Porphyria

Compared with placebo, treatment with the RNA interference therapy givosiran lowered the rate of porphyria attacks in patients with acute intermittent porphyria, the most...

Higher Hydroxyurea Dose Does NOHARM in Sub-Saharan African Children With Sickle Cell Disease

According to results from the NOHARM (Novel use Of Hydroxyurea in an African Region with Malaria) maximum tolerated dose (MTD) trial, dose-escalated hydroxyurea controlled...
On location

Pegcetacoplan Outperforms Eculizumab for Paroxysmal Nocturnal Hemoglobinuria

Results from the phase III PEGASUS trial suggest that the targeted C3 inhibitor pegcetacoplan improved hemoglobin levels better than eculizumab in patients with paroxysmal...

Global Blood Therapeutics Seeks Approval of Voxelotor for Pediatric Sickle Cell Treatment

Next year, Global Blood Therapeutics Inc. (GBT) will seek expanded approval of the use of its treatment for sickle cell disease (SCD), voxelotor, in...

Luspatercept-aamt Approved for Anemia in Adults With MDS

The FDA has approved luspatercept-aamt for the treatment of anemia that requires 2 or more red blood cell transfusions over 8 weeks in adults...
You Make the Call

You Make the Call: Would rituximab re-treatment help this patient with hemolytic anemia?

This month, Edward Benz Jr., MD, discusses treatment of Coombs-positive hemolytic anemia in a patient who declines to undergo splenectomy.I am caring for a...

Comparing Risks of Hypophosphatemia With Intravenous Formulations in Iron-Deficiency Anemia

For patients with iron-deficiency anemia who are intolerant of or unresponsive to oral iron, intravenous formulations can rapidly correct iron deficiency, but certain formulations...

Luspatercept Reduces Anemia Severity, Improves Rate of Transfusion Independence in MDS

Compared with placebo, treatment with luspatercept improved the 12- to 16-week rate of red blood cell (RBC) transfusion independence in patients with lower-risk myelodysplastic...

Sickle Cell Disease Gene Therapy Receives Orphan Drug Designation

ARU-1801, an investigational gene therapy for the treatment of sickle cell disease (SCD) and beta thalassemia, was granted orphan drug designation by the FDA. ARU-1801...
Sickle cell anemia

ICER Report Finds That Sickle Cell Drugs Are Too Expensive

According to a draft report by the Institute for Clinical and Economic Review (ICER), SCD drugs made by Novartis, Global Blood Therapeutics (GBT), and...
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