Rare Blood

Investigators said the benefits appeared relatively early in treatment for many patients and persisted throughout the study period.

A systematic review and meta-analysis found no clear advantage for automated red cell exchange.

Prior to the pandemic, youth with sickle cell disease were more likely to report depressive symptoms, but the opposite was true during the pandemic.

A cure for sickle cell disease could more than double the lifetime incomes of patients, the study found.

A panel of experts suggests adding caplacizumab to the historic treatment of plasma exchange and immunosuppression could lead to significant improvement in outcomes for patients with immune-mediated thrombotic thrombocytopenic purpura (iTTP).

The findings highlight the significant financial burden of sickle cell disease.

While suggesting that the disorder be included in the differential diagnosis of thrombocytopenia following vaccination, the group emphasized that the benefits of global vaccination against the virus outweigh the risk of this rare complication.

Patients with cold agglutinin disease faced a 4.5-times greater risk of death during winter months, new data show.

Among their sample cohort of 49 patients, the researchers found that scores on the Montreal Cognitive Assessment correlated with measures of both literacy and health literacy.

Improved management of sickle cell disease has vastly improved life expectancy and reduced morbidity for affected patients, but the disease itself and its treatments carry fertility risks that many young patients and their caregivers are unaware of.

During her presentation at the Academy of Managed Care Pharmacy Nexus 2021 meeting, Aimee Tharaldson, PhD, highlighted the most anticipated specialty pharmacy drugs awaiting FDA approval, including several treatments for cancer and rare diseases.

After a more than 35-year pursuit, the licensing of a gene therapy product for treating hemophilia may be available within 2 years.