Over $18 million has been given to support research into how best to assist young people with sickle cell disease (SCD) moving from pediatric to adult care. The award comes from the board of governors of the Patient-Centered Outcomes Research Institute (PCORI).
Transiting to adult care can be difficult, as children usually are guided in receiving the care they need and are given necessary care. But many primary care physicians working with adults, who serve as patients’ first and continuing contact for treatment, are not familiar with managing this rare condition.
As a result, patients may have difficulty accessing needed specialists, PCORI notes in its press release on the award. Young adults with SCD are more likely to use emergency services for flares of painful symptoms when preventive care and screening visits are lacking.
The funding from PCORI, an independent non-profit supporting research into patient care and education, was awarded to two projects.
The first is a $9.8 million study based at Carolinas Medical Center that will compare the effectiveness of two approaches in managing care transitions for SCD patients. Peer support — a kind of mentoring program that pairs new patients with those who have already made the change — plus a specially designed education program will be compared to the education program alone. Researchers will evaluate the two by measure that include a reduction in emergency hospital visits and admissions, and reported improvement in the quality of life among these SCD patients.
This study, which begins this month, will run through July 2022.
The second awards $8.5 million to a project led by researchers at Children’s Hospital of Philadelphia. It will compare three approaches to care: community health workers who engage with patients, mobile apps that help patients manage their condition, and increased but customary care. Outcomes will be evaluated according to how well each approach is seen to improve quality of life during a patient’s transition to adult care, and the extent to which an adult is less dependent on emergency care. This study will also run through July 2022.
Both projects are among seven PCORI awards for SCD, part of a total $80 million investment in research into the treatment of people with rare diseases.
“These newly funded studies will address important gaps in our understanding of how to improve care for older adolescents and young adults with sickle cell disease,” said Joe Selby, PCORI executive director. “The good news is that young people with this serious disease are more likely than ever to live well into adulthood.
“The comparative clinical effectiveness research that PCORI funds can give these patients, their families and clinicians the information they need to make better-informed decisions about their care options,” he said.
PCORI defines its mission as supporting research that provides patients, their caregivers, and clinicians with the information they need to make better-informed healthcare decisions.