The PCORI Board Approves $18 Million For Sickle Cell Research


The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors approved roughly $18 million to fund two studies comparing different ways to manage the transition of people with sickle cell disease from childhood to adult care.

Though children with sickle cell disease broadly receive the care they need to manage their condition, the transition to adult care can be dangerous. A number of primary care clinicians who treat adults are unfamiliar with caring for this rare condition, and patients can find it difficult to access care across specialists. Losing a source of care, such as pediatric specialty care, drops the chances that young adults with sickle cell disease will receive preventive care and screening visits, and it increases the probability they will look for emergency department care when painful symptoms ignite.

“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,” stated PCORI Executive Director Joe Selby, MD, MPH. “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.”

The two awards the Board approved are:

A $9.8 million study based at Carolinas Medical Center comparing two interventions that have proven effective in simplifying care transitions for people with other conditions. One intervention entails peer support in addition to an education program created to help young people learn to manage their condition. The other includes the education program solely. The study will determine how effective each approach is in cutting emergency department visits and hospital admissions and in improving quality of life amid young adults with sickle cell disease.

An $8.5 million project spearheaded by the Children’s Hospital of Philadelphia that will compare three methods: community health workers engaging with patients, use of mobile apps to help individuals manage their condition, and enhanced usual care. The study will test which approach is most effective at refining patients’ quality of life during their transition to adulthood and trimming their dependence on the emergency department.

These two projects join forces with five others previously awarded that target sickle cell disease. Collected they are a part of PCORI’s $80 million investment dedicated to research of rare diseases.

In addition to these awards, the Board approved more than $80 million to endorse nine studies on other conditions and problems that inflict high burdens on patients, caregivers and the healthcare system. These other studies include five testing strategies to treat multiple sclerosis.


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