Cohen Children’s, CHOP share $8.5M sickle cell study

Transitional tools: With more and more pediatric sickle cell disease patients surviving into adulthood, a new $8.5 million study will explore interventions and other tools for easing their shift into adult healthcare.

The Cohen Children’s Medical Center will share $8.5 million in funding for a study targeting children with sickle cell disease.

The New Hyde Park-based Steven and Alexandra Cohen Children’s Medical Center, a 202-bed hospital managed by the Northwell Health system, will share the Patient-Centered Outcomes Research Institute award with the Children’s Hospital of Philadelphia.

The institutions will focus on improving the quality of life for sickle cell youth as they transition to adults. Specifically, the study will assess the effectiveness of two “support interventions” designed to help sickle cell patients ages 17 and older adjust from pediatric to adult healthcare.

Each intervention includes “self-management tools” for the transitioning patients, according to Northwell Health. One involves a six-month community health worker program while the other utilizes a mobile health app.

Both will be compared to “usual care” to see if either intervention “improves the patients’ ability to care for themselves, stay connected with their doctors, avoid visits to the emergency room and, ultimately, experience better quality of life,” the health system said.

“Sickle cell disease” is an umbrella term for multiple red blood cell disorders that cause severe pain and anemia, increase risk of infections and damage organs throughout the body. Sickle cell, which usually presents in childhood, affects roughly 100,000 Americans – and young adults with the disease experience “a seven-fold increase in mortality rates during the years that they transition to adult care,” according to Northwell Health.

While the health of those patients is a primary concern, the study will also consider a second challenge facing sickle cell patients and their families during the patients’ transition out of pediatric care: the all-too-common loss of health insurance.

“We’ve heard from youth with sickle cell disease and their families that the transition of their care is their greatest concern, beyond their health,” noted David Rubin, CHOP’s medical director of population health. “With this study, we’re seeking to understand what tools we can provide outside of the doctor’s office so these patients feel actively involved in the transfer of their healthcare.”

“Although young people with sickle cell disease are more likely than ever to live into adulthood, we know that their transition to adult healthcare is often accompanied by loss of insurance coverage,” added Kim Smith-Whitley, director of CHOP’s Comprehensive Sickle Cell Center, and that drop in coverage creates “a decrease in the quality of life and other challenges that endanger their health.”

“As a pediatrician who specializes in sickle cell disease, I want to know what are the best tools that I can provide my patients so that when they progress out of the children’s hospital, they are supported and empowered to manage their care,” Smith-Whitley said.

Such concerns make those potential interventions, and the sickle cell adult transition as a whole, prime meat for the PCORI, a Washington-based, independent nonprofit organization authorized by Congress in 2010 to investigate and quantify the relative effectiveness of various medical treatments.

Sophia Jan: Sickle cell is just the start.

Although it was created by modifications to the Social Security Act by clauses in the Affordable Care Act – and while it may have Medicare’s ear, insofar as therapies the federal insurance program will and won’t cover – the patient-centered research institute is classified is a nongovernmental organization.

“This project was selected for PCORI funding for its potential to fill an important gap in our understanding of how to improve care for older adolescents and young adults with sickle cell disease,” Joe Selby, PCORI’s executive director, said in a statement. “It will provide useful information to help patients weigh the effectiveness of their care options.”

And Cohen Children’s Medical Center looks forward to helping provide that information, according to Sophia Jan, Cohen Children’s chief of general pediatrics, who noted the scope of the joint study is right in the medical center’s wheelhouse.

“We’ve done a lot of research on the types of interventions – namely around youth community health workers and mobile health interventions – that could assist young adults with sickle cell disease in their transition to adult care,” said Jan, who also directs Northwell Health’s Health Home Serving Children program. “We’re excited to continue partnering with these young adults to learn which of these tools work best for them.”

The Cohen Children’s-CHOP research could ultimately prove beneficial to patients and caregivers beyond the sickle cell realm, Jan added.

“What we learn will not only help patients make informed decisions and feel more prepared for the transition from pediatric to adult care,” she said. “[It] will provide everyone involved in patients’ wellbeing – their families, pediatric and adult healthcare providers, health systems and policymakers – with better tools and more knowledge to build a successful model for transition of care for all youth with complex medical needs.”