The European Medicines Agency (EMA) has confirmed the continuing suspension of marketing authorization for Pfizer’s sickle cell disease (SCD) treatment Oxbryta (voxelotor) — withdrawn from the market last year due to safety concerns — following a committee’s recommendation that “concluded that the benefit-risk balance … is no longer favourable.” The EMA’s…
News
Using tocilizumab off-label in four young adults with sickle cell disease (SCD) who developed hyperhemolysis syndrome as a complication of a blood transfusion stopped the premature destruction of red blood cells, suggesting it may be safe and effective when other options fail. While tocilizumab is not approved for…
New research shows that Black men with sickle cell disease (SCD) become biological parents at a lower rate than similarly aged Black men in the U.S. general population. The analysis found about 25% fewer Black men enrolled in the Sickle Cell Disease Implementation Consortium (SCDIC) registry were parents compared…
People with sickle cell disease (SCD) seeking emergency care for vaso-occlusive crises (VOCs) are often not triaged appropriately, leading to unnecessarily long waits for pain relief. A recent study finds that assigning the correct Emergency Severity Index score, a tool used in emergency departments to prioritize care based…
Most Gambian children with sickle cell anemia — the most common and severe form of sickle cell disease (SCD) — have mild to moderate disease, but early diagnosis and wider access to hydroxyurea may be key to reducing complications, a study found. “The findings underscore the need for…
Mortality rates related to sickle cell disease (SCD) have increased in the U.S. in recent decades, while mortality for people with cystic fibrosis (CF) has declined over the same time period, a study shows. “Several factors contribute to this stagnation, including under-resourced care systems, limited access to first-line…
Blood levels of heme oxygenase-1, or HO-1, an enzyme that helps protect cells from certain types of damage, are elevated in people with sickle cell disease (SCD), especially children, a new study showed. HO-1 serves as a safeguard against the toxic effects of free heme, a molecule present in…
Changes in the gut microbiome, the group of bacteria and other microbes living in the digestive tract, may drive chronic pain in people with sickle cell disease (SCD), a new study reveals. In a study using mice and human tissues, a team of U.S. researchers investigated how such changes,…
Scientists at New York University (NYU) are teaming up with Nigerian-born music artist Adekunle Gold on a project to improve the lives of people affected by sickle cell disease (SCD). The collaboration was announced at an event held earlier this month as part of National Sickle Cell Awareness…
Young people with sickle cell disease (SCD) who live in areas with more air pollution are more likely to be hospitalized due to acute chest syndrome (ACS), an SCD complication marked by symptoms that can include chest pain and difficulty breathing, a study found. “As ACS is a…
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