Iptacopan Meets Primary End Point of APPOINT-PNH Trial in Paroxysmal Nocturnal Hemoglobinuria
May 4th 2023Iptacopan elicited improved hemoglobin levels measuring at least 2 g/dL higher vs baseline, leading to transfusion independence after 24 weeks in approximately 92.2% of patients with complement inhibitor–naïve paroxysmal nocturnal hemoglobinuria.
Read More
Brain Volume May Serve as Biomarker of Brain Injury in Patients With Sickle Cell Disease
April 26th 2023The data highlight that silent cerebral infarcts represent just a portion of brain injury that occurs in patients with sickle cell disease (SCD), and brain volume can serve as another potential biomarker of brain injury in those with the disease.
Read More
Joseph Alvarnas, MD, Discusses New CMS Drug Pricing Models, Impact for Blood Diseases
April 19th 2023CMS in February 2023 announced 3 new models for testing by the Center for Medicare & Medicaid Innovation—all of which “aim to lower the cost of drugs, promote accessibility to life-changing drug therapies, and improve quality of care.”
Read More
TCD Screening and Spending Among Children With Sickle Cell Anemia
A substantial proportion of families of privately insured children with sickle cell anemia pay more than $100 for essential stroke screenings, a high-value service.
Read More
FDA Approves Efanesoctocog Alfa for Hemophilia A
February 24th 2023Efanesoctocog alfa is the first factor VIII therapy to overcome the interaction with endogenous von Willebrand factor, which creates a ceiling of 8 to 19 hours on the half-life of current factor VIII replacement products, which in turn creates the need for more frequent dosing.
Read More
The reality of a sickle cell disease cure brings fear, optimism, and questions to patients’ lives; veterans who are in suicidal crisis can receive free emergency care at any Department of Veterans Affairs or private facility; Pfizer to increase access to progressive treatments through sale of drugs at non-profit prices to poor countries.
Read More
Analysis of Beti-Cel for Severe β-Thalassemia Shows Extensive, Long-term Patient Improvement
December 20th 2022A pair of abstracts presented at this year’s 64th American Society of Hematology Annual Meeting and Exposition bear out the significant transfusion-free rate at 3 years following beti-cell administration and marked improvements in patient-reported outcomes, including the ability to work and be physically active.
Read More
Some Patients With CAD Experience Sustained Remission After Sutimlimab Discontinuation
November 18th 2022A research letter published in Blood Advances details the first clinical evidence of sustained hematologic remission for cold agglutinin disease (CAD) after sutimlimab treatment discontinuation.
Read More
Study Highlights Value of PROs in Children With Sickle Cell Disease During Acute Pain Episodes
November 14th 2022Sufficiently powered studies are necessary to investigate associations between clinical measures and patient-reported outcomes (PROs) in children with sickle cell disease experiencing acute pain episodes, according to a recent study.
Read More
Report Suggests Best Practices for Care of Pediatric Sickle Cell Disease
November 12th 2022A panel of specialists proposed 26 essential elements for comprehensive pediatric sickle cell disease care, laying groundwork for standardized guidelines and the establishment of accredited care centers in the future.
Read More