‘Sickle cell needs more funding’: An 18-year-old patient advocate calls on Washington to improve children’s health care

Sydney McLeod left her college dorm in New York to rush to the emergency room, hoping to get treatment for the blinding pain she felt in her legs and chest and the swelling in her ankles.

But this wasn’t the hospital back home, where she’s been receiving treatment for sickle cell disease for over a decade. These weren’t the doctors who knew her case. So 18-year-old McLeod found herself taking charge — detailing to the doctors and nurses what kind of medication she needed, the dosage, even checking to make sure the staff was keeping her hydrated.

“The doctor didn’t really know what he was doing,” McLeod said. “He was clueless as to how to help me so I was just telling him everything.”

One nurse who didn’t understand pain triggers for sickle cell patients even suggested McLeod was lying about her pain.

“The nurse was like ‘I don’t really think you need it because you don’t look like you’re in pain,’” McLeod said. “And I was just sitting there like, ‘I don’t know how else you want me to look?’”

It’s not the kind of ER visit most 18-year-olds experience — but it’s not that rare for patients with sickle cell, an inherited blood disorder that affects some 100,000 people in the United States, many of them African American. Treatment options are limited and care for patients is notoriously problematic.

Now, McLeod is joining lobbyists and other patients to fight to make sure other kids don’t have to go through what she’s gone through. She and her family visited several Capitol Hill offices this week as part of an advocacy day organized by the Children’s Hospital Association, an annual event created to give patients and their families a platform to raise awareness about children’s health care.

“Sickle cell needs more funding, and more people need to be aware of what the disease actually is, and how it affects many people across the country,” she said.

Sickle cell disease is excruciatingly painful  — it can cause chronic pain, multi-organ failure and stroke. Many patients don’t live past their early 40’s.

Without many directed treatment options, many patients rely on opioids to manage episodes of pain known as pain crises, like the one McLeod experienced near her college. Like her, many patients experience skeptical hospital staff. In fact, at one hospital researchers found that sickle cell patients waited 60 percent longer to get pain medication than other patients who reported less severe pain.

This is exacerbated by a misunderstanding of the disease, a lack of a national data registry to track the impact of the disease, and a lack of qualified doctors that understand it. A 2015 survey of more than 3000 family physicians showed that only 20 percent felt comfortable treating patients with sickle cell disease.

Currently, progress related to treatments for sickle cell disease have been stagnant — there have been just two treatments in almost the last three decades.

Advocates for more research funding often highlight the difference between the disease and cystic fibrosis, another autosomal recessive disorder for which drug makers have developed numerous new treatments since 2010. In the same span, just one has been developed for sickle cell. Cystic fibrosis has also seen more funding from the NIH.

“A lot of this funding tends to be disproportionately directed towards diseases where advocacy is done well,” McLeod’s hematologist, Dr. Deepa Manwani, said. “There are many reasons why the community that’s affected by sickle cell disease, which predominantly affects African Americans and Hispanics, has not been able to really have that voice.”

“The bottom line is, it is an issue of disparities. And this disparity has to be made absolutely clear to our politicians,” Manwani continued.

McLeod wasn’t advocating for a specific bill on Wednesday — instead, she was on Capitol Hill to tell her story to lawmakers including the offices of Sen. Chuck Schumer (D- N.Y.) and Sen. Kristen Gillibrand (D-N.Y.). She spoke most frequently about the importance of funding sickle cell research, but also emphasized the importance of funding physicians who specialize in pediatric conditions like hers.

“Having a hospital that has the skill set, the staff that understands those pediatric conditions, is actually absolutely critical,” said Jim Kaufman, vice president of public policy at the Children’s Hospital Association. “We’re always reminding policymakers, there’s a uniqueness to children’s healthcare.”

She encouraged lawmakers and staffers, too, to protect Medicaid, the hybrid state-federal health insurance program that covers some 72 million low-income Americans.

Hospitals might be less willing to take on patients like McLeod if they are covered by Medicaid, because reimbursement rates in that program are lower than those for privately insured patients, said Manwani, the director of hematology in the department of pediatrics at Montefiore.

McLeod’s mother, Trisha Gibbs, echoed the importance of Medicaid. Without it, she said, McLeod wouldn’t be able to receive the quality of care that she does.

For now, McLeod has decided to make the two-hour drive from her college campus back to her home in the Bronx for her medical care. She needs a check-up every three weeks, though she said she goes back more often than that because of unexpected bouts of pain.

“I have a lot of sleepless nights when she’s on campus … especially during the winter time,” McLeod’s mother said. “It’s hard for me to sleep comfortably at home when I know she’s two hours away and in pain.”

Correction: an earlier version of this story misstated the number of treatments the FDA has approved for sickle cell disease since 2010 and misidentified the state in which McLeod attends college. It also incorrectly stated that Montefiore’s adult division for sickle cell care had closed.