Life-changing Sickle Cell Disease Treatment Gives Toddler Fresh Start

By Heather Duge

Four-year-old Winnie Babalola smiles all the time and talks nonstop. But it wasn’t always that way.

Diagnosed with Sickle Cell Disease at two weeks old, Winnie faced a condition that can cause damage to organs and intense pain episodes. She began treatment at the Cook Children’s Sickle Cell Center where the hematology team followed her. “Every time Winnie had a fever or got sick, she needed to be in the hospital for several days of monitoring,” said Ademilola Awoyemi, Winnie’s mom.

Bone Marrow Transplant Brings Hope

In 2019, Winnie landed in the hospital where doctors discovered her spleen was markedly enlarged, requiring a blood transfusion. Soon after, the family met with Richard Howrey, M.D., associate medical director of the Stem Cell Transplant Program, to discuss curative therapy for 10-month-old Winnie. The family decided Winnie would undergo a bone marrow transplant after discovering Winnie’s brother, Wisdom, was a perfect match.

“The goal of a transplant is to wipe out her diseased bone marrow that only makes sickle cells and replace it with healthy bone marrow essentially curing her of Sickle Cell Disease,” Dr. Howrey said. 

Transplanting patients early in life helps to prevent long-term complications, according to Gretchen Eames, M.D., M.P.H., medical director of the Stem Cell Transplant Program. Those who do not undergo transplants have a much harder time in adulthood with access to healthcare, insurance issues and difficulty navigating a chronic disease. They also have a shorter life expectancy due to progressive complications of Sickle Cell Disease

Journey to Healing

Because of the pandemic, Winnie’s transplant was delayed until June 2021 when the process began with surgery to place a central line. She then received chemotherapy over the span of a week before the transplant. During the first phase of the transplant, Winnie experienced the typically expected side effects including severe mouth sores which required IV narcotics for pain and a feeding tube to maintain nutrition, along with multiple transfusions for low blood counts.

“Transplant comes with risks, and Winnie experienced a few complications including thrombotic microangiopathic anemia (TMA) which caused high blood pressure and a breakdown of the red blood cells,” Dr. Howrey said. “We adjusted her medications and gave her a specific medication which targets the pathway that causes TMA and thankfully, she had a great response.”

Throughout all of this, Ademilola said everyone at Cook Children’s helped her to focus on taking it one day at a time.

“From day one, the doctors and nurses were tremendous and gave Winnie the best treatment,” Ademilola said. “It was hard, but they really brightened our days.” 

Winnie was discharged two months after her transplant, has continued to do well, and is now fully recovered.

Brighter Days Ahead

The prognosis of patients with Sickle Cell Disease who undergo matched sibling bone marrow transplants is very good, with a survival rate of about 95%. Having a matched sibling was a great advantage for Winnie. Compared with other types of bone marrow transplants, matched sibling transplants have a lower risk of graft versus host disease, which is when the donated bone marrow attacks the body.

Winnie’s young age and lack of other complications such as stroke or end organ damage prior to transplant was another factor in her favor.

“Now Winnie is off all medications and doing great,” Dr. Howrey said. “Her immune system has fully recovered. I fully anticipate Winnie will be cured from Sickle Cell Disease.”

Ademilola said the bone marrow transplant changed their lives. Last month, Winnie walked into school for the first time, and the preschooler couldn’t be happier.

“Winnie is a fighter,” Dr. Eames said. “Providing life-saving therapy and carrying patients like Winnie through this journey to a successful outcome is very rewarding.”