Results of a healthcare transition learning collaborative for emerging adults with sickle cell disease: the ST3P-UP study transition quality improvement collaborative.

Background: Individuals with sickle cell disease (SCD) experience poor clinical outcomes while transitioning from paediatric to adult care. Standards for SCD transition are needed. We established a Quality Improvement (QI) Collaborative that aimed to improve the quality of care for all young adults with SCD by establishing a standardised SCD transition process.

Immunization adherence among children with sickle cell disease and sickle cell trait: Results of a population-based study.

Introduction: Despite the importance of timely vaccine completion for protection from infectious disease, there is limited knowledge of the immunization adherence rates of children with sickle cell disease (SCD).

Methods: This is a retrospective cohort study comparing the immunization rates of children with SCD to those with sickle cell trait between 2008 and 2019 in Georgia. Completion rates for each vaccine and the proportion of children with up-to-date status at 24 and 35 months were calculated and compared between the cohorts.

Sickle Cell Trevor Thompson Transition Project (ST3P-UP) protocol for managing care transitions: Methods and rationale.

Background: Emerging adults with sickle cell disease (EASCD) experience significant challenges transitioning from pediatric to adult care. Acute care utilization increases, quality of life (QOL) declines, with an increased risk of mortality. Currently, there are no practice standards to guide emerging adults through the transition process.

Oversight of Right-to-Try and Expanded Access Requests for Off-Trial Access to Investigational Drugs.

For decades, the U.S. Food and Drug Administration (FDA) has provided an "expanded access" pathway that allows patients who meet qualifying conditions to gain access outside a clinical trial to an investigational medical product being tested to see if it is safe and effective for a specific use.