As sickle cell patients live longer, providers and politicians call for more care

ST. PETERSBURG — When patients in the sickle cell program at Johns Hopkins All Children’s Hospital turn 13, their doctors and nurses begin a process that, decades ago, wouldn’t have often been considered: preparing patients for their adult lives.

Once deemed a pediatric disease because of high youth mortality rates, sickle cell disease now affects more adults than children because of better treatments prolonging life expectancy and improving quality of life. So it’s necessary for teenagers with the condition to learn how to ask doctors questions, refill medications and advocate for themselves.

As they approach their 21st birthdays, when they age out of pediatric care, the program helps with a key challenge of living with the disease into adulthood: finding a new doctor.

“The adult providers are sparse,” said Carrie Gann, a nurse practitioner who developed the All Children’s sickle cell transition program. “They’re not convenient for our patients. I feel like access to care is limited.”

A bill filed last month by U.S. Rep. and Florida gubernatorial candidate Charlie Crist aims to expand access to care for adults with sickle cell disease. The Sickle Cell Care Expansion Act would put $200 million a year from 2023 through 2028 toward the effort, with most of the money going toward scholarships and loan forgiveness for hematologists and those studying to become hematologists; it would prioritize recipients from disadvantaged backgrounds. About a quarter of the money would go, as grants, toward sickle cell education and advocacy.

Most sickle cell patients are Black, and Crist said the “significant African American population” in his district made it important for him to push for the funding.

“We have made many advancements in science recently,” he said. “We need to share that kind of knowledge with members of all races.”

Researchers estimate that some 60 percent of sickle cell patients in America are now 18 or older. In Florida, 82 percent are Black, according to a 2014 study, which then placed the number of Black sickle cell patients in the state at between 8,000 and 14,000. About 100,000 people nationwide live with the inherited disease, in which misshapen red blood cells can cause episodes of severe pain, pneumonia-like symptoms and stroke, among numerous other complications.

Despite those high numbers, there’s a shortage of knowledgeable doctors for adults with the disease. Researchers in a 2020 study noted that hemophilia and cystic fibrosis have national registries and more treatment centers than sickle cell despite affecting half as many people. Only one-fifth of family physicians have said in surveys they’re comfortable treating sickle cell patients.

Gann said the Tampa Bay region has so few options that she has to refer patients from Polk County to a hematologist in Tampa and hope that they can regularly make the trip.

“I don’t know an adult hematologist in Lakeland,” she said. “I wouldn’t even know where to start.”

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Even with so little adult care, recent years have been fruitful for sickle cell treatment. Hydroxyurea, the primary drug used to treat the disease, was federally approved for adult use in 1998 and pediatric use in 2017. Since then, a handful of other drugs have emerged that have broadened patients’ options, said Juan Felipe Rico, the medical director of pediatric hematology-oncology at Tampa General Hospital and the division chief in the same field at the University of South Florida.

Though a successful bone marrow transplant remains the only cure for sickle cell — one that’s available to a minority of patients — promising strides have been made in gene therapy, and some experts believe a cure is on the horizon. Rico said the field still needs widespread funding, for developing a cure to improve treatment for patients in the meantime and, as the Crist bill would do, for encouraging more doctors to specialize in the disease.

“I can look back at my fellowship, and when I looked at opportunities in cancer versus benign hematology, there were many more opportunities to study cancer,” Rico said. “Studying cancer is better funded.” More money for sickle cell education could even the balance.

Crist’s legislation, which has the backing of the Black Women’s Health Imperative and the American Society of Hematology, was referred last month to the House Committee on Energy and Commerce. It hasn’t moved since, but he said he has a hard time imagining it will face opposition, and he hopes for it to pass before his term ends later this year.

“For the sake of humanity,” he said, “this is something that needs to be done.”