April 18th 2024
A cost-utility analysis conducted from the perspective of the Italian health system found that pegcetacoplan was more effective and less costly than 2 complement 5 (C5) inhibitors for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
Patients With SCD Prefer Digital CBT if It’s Personalizable and Social
May 17th 2023Focus groups show patients who have sickle cell disease (SCD) like the idea of a digital health app for cognitive behavioral therapy (CBT) that allows them to make meaningful connections with people with shared experiences.
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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.
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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.
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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.”
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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.
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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.
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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.
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