The primary end point was time to readiness for discharge, defined as the difference between the readiness-for-discharge date and time and the start date and time of the first infusion of study drug.
Priority Review shortens the FDA review time from 10 months to 6 months
Patients presenting at emergency departments were nearly 4 times as likely to be admitted to a hospital as patients presenting at a day hospital.
Young patients with sickle cell disease underwent dosing schedules guided by analysis of pharmacokinetic data using a population pharmacokinetic model.
Voxelotor may be a safe and efficacious treatment for sickle cell disease, especially at a dosage of 1500 mg/d.
Patients with severe sickle cell disease were more than twice as likely to experience recurrent thromboembolism compared with patients with less severe disease.
A multidisciplinary intervention strategy led to a 61% decrease in hospital stay duration for patients with sickle cell disease and vaso-occlusive pain.
Quantitative sensory testing may facilitate objective evaluation of feelings of pain and function in patients with sickle cell disease.
Mometasone usage was found to correlate with decreased levels of macrophage markers but not of asthma-related markers.
Infants with sickle cell disease who started hydroxyurea before 12 months of age had better long-term outcomes.
