Children’s Wisconsin is changing the way sickle cell disease is treated

Sadly, 9-year-old Robbi Smith is no stranger to pain and the Children's Wisconsin Emergency Department. 

"If we weren't in there every month, it was at least every season," her mother, Cierra Woods, said. 

Cierra and Robbi have been in and out of the Children's Wisconsin Emergency Department (ED) countless times since Robbi was 4 years old. The reason for these visits was pain crises related to Hemoglobin SC disease, a type of sickle cell disease Robbi was born with. 

People who are diagnosed with sickle cell disease — like Robbi — have a protein in their red blood cells that transforms these cells from their naturally smooth, disk-like shape into a bent, crescent shape. Because of their unique shape, these cells often get stuck in their blood vessels, blocking flow and preventing oxygen from traveling through the body. These blockages — known as sickle cell vaso-occlusive crises — can cause intense, recurring pain throughout an individual's life. 

"Since it is a genetic disorder, sickle cell disease doesn't go away," said Amanda Brandow, DO, MS, a pediatric hematologist-oncologist with the MACC Fund Center for Cancer and Blood Disorders and director of the Sickle Cell Disease Program and Research Program at Children’s Wisconsin and the Medical College of Wisconsin. "It actually gets worse with age." The cumulative effects on the organs can lead to kidney failure, heart problems, lung problems, stroke, chronic pain, and premature death. 

To help improve sickle cell care on a national scale, the MACC Fund Center — which includes Dr. Brandow and a multidisciplinary team of physicians, nurse practitioners, nurses, psychologist, social worker, genetic counselor, neuropsychologist and research coordinators — is involved in numerous research efforts to better understand and treat the disease. 

Setting national standards

Often in an emergency room setting, it can take hours for a patient to receive pain medication, which can be excruciating for people with sickle cell disease experiencing a pain crisis. Dr. Brandow said delays in receiving medication are often due to the lack of existing protocols, but can also be due to stigmas associated with the disease. 

On the outside, people with sickle cell disease look and appear healthy. However, their pain can be extreme and with no outward visible sign. As a result, patients are often not taken seriously or treated with urgency. 

Dr. Brandow and former colleague David Brousseau, MD, MS, who served as the chief of Emergency Medicine at Children’s Wisconsin prior to relocating, worked to fix that gap in care. In 2016, they began putting protocols in place to ensure children with sickle cell disease receive their first dose of pain medication within 60 minutes of arriving at Children’s Wisconsin. 

Using intranasal fentanyl, an opioid nasal spray, physicians can provide much faster pain relief to patients. So, while a patient waits for an IV to be placed or other tests, they can begin to feel some relief. 

Through implementation science and a study funded by the National Institute of Health – National Heart, Lung, and Blood Institute the SCIENCE study (Sickle Cell Improvement: Enhancing Care in the Emergency Department), Dr. Brandow and the ED team at Children's Wisconsin are doing work to share these and other protocols with institutions throughout the country. The goal of this work, Dr. Brandow said, is to help other institutions improve upon their ability to quickly deliver guideline-based treatment in the ED to children impacted by sickle cell disease pain.

Children’s Wisconsin is an academic medical center. What that means is, through its affiliation with the Medical College of Wisconsin, it’s an institution where patient care, medical research and education all take place together. Academic medical centers help set quality standards for patient care nationwide, train the health care workforce, evaluate promising treatments through clinical trials, further scientific concepts through research and provide the majority of complex care. For families, what that all means is simple — they receive the absolute best care possible. 

Exploring new treatments

Traditionally, when a child with sickle cell disease is seen for pain in an emergency department, Dr. Brandow said they will experience a relatively routine evaluation that involves a blood panel, an IV placement and a prescribed dosing schedule of opioids to manage pain. But even with medication, a child can still continue to experience high levels of pain. Once a child reaches a certain number of pain medication doses, they're admitted to the hospital.

That’s exactly what happened with Robbi. It was at this stage in her journey that Robbi and her mother were introduced to the Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial, a sickle cell clinical trial that is examining the effect of intravenous arginine — a treatment that has the potential to make a sickle cell pain shorter or less severe. Robbi is one of 12 children who have joined the trial since Children's Wisconsin became one of 10 active study sites in September of 2021.

When asked if she'd like Robbi to participate in the trial, Cierra said she didn't hesitate to say yes. "It seemed like it was very interesting and something new." Cierra added that she was happy to be a part of Children's Wisconsin's efforts to help improve sickle cell care and raise awareness about the disease.

"I don't see a lot of it being explained or talked about," she said. "You hear about other diseases, but you never hear about someone sharing the facts about Black and brown children having sickle cell disease."

Increasing awareness in the community

Of the 100,000 people in the United States living with sickle cell disease, the vast majority are Black — and 99 percent of the 425 patients Children's Wisconsin sees in its Sickle Cell Disease Program are Black. Conscious and unconscious racial biases can sometimes influence the care patients receive or their access to resources.

Children's Wisconsin is in an optimal position to tackle these challenges and make an impact on treatment options for sickle cell disease. Approximately 85 percent of the 1,200 people in Wisconsin diagnosed with sickle cell disease live in the southeast region of the state, making Milwaukee a significant site for driving improvements for treating the disease.

Ideally, Dr. Brandow said she hopes research efforts like SCIENCE and the STArT trial will help reduce pain and lead to a better quality of life for people living with the disease, as well as improve community awareness of the condition.

"There's a lot of pain and suffering that our families and children have," Dr. Brandow said. "I think any awareness we can bring to the disease is part of our job as providers. The more people know about it, the better."  

Laura Bengs Writer Children's Wisconsin

From mending growing bones to helping kids through everyday anxieties, innovation means more here. At Children’s Wisconsin, we’re 100 percent focused on kids and every aspect of their health — physical, social and mental. So whether it’s expert care for growing bones or groundbreaking behavioral therapy, caring for the whole child drives us to go beyond, unlocking innovation in more ways than you might imagine.