In the fall of 2022, 2-year-old Emilia started complaining of arm pain and trouble walking.
On October 7, Emilia’s worried parents, Lillybeth and Steve, took her to UPMC Hamot near their home in Erie for bloodwork. After a long day at the hospital, the family was on their way back home when a hospital physician called. Emilia’s bloodwork indicated acute lymphoblastic leukemia (ALL). She needed to be seen at UPMC Children’s Hospital of Pittsburgh right away.
Sickened and distraught by the unexpected news, Lillybeth and Steve quickly threw a few things into a bag, packed up Emilia, and made the two-hour drive to Pittsburgh.
A Cutting-edge Clinical Trial
At UPMC Children’s, Emilia was immediately started on treatment. She would need intensive outpatient chemotherapy, careful monitoring, and ongoing testing, including monthly spinal taps.
The family was also presented with the opportunity to enroll Emilia in a clinical trial investigating the use of an immunotherapy drug called blinatumomab. Targeting a specific protein in leukemia, the drug essentially helps the body’s immune system identify and kill the leukemia cells.
UPMC Children’s maintains a strong clinical cancer research program. Thanks in part to the community’s support of the Mario Lemieux Institute for Pediatric Cancer Research at UPMC Children’s, an innovative center dedicated to advancing lifesaving childhood cancer research, the hospital can offer more experimental trials like the blinatumomab study to children like Emilia.
Emilia’s oncology team explained that the multi-site blinatumomab clinical trial offered through the Children’s Oncology Group (COG) was designed to investigate the efficacy of using blinatumomab in combination with traditional chemotherapy as a frontline treatment for patients with ALL. The drug had already shown promise in treating ALL recurrence with fewer side effects.
“At first, we weren’t sure about the study,” Lillybeth explains. “After discussing it further with Emilia’s physicians, we decided to enroll her. We hoped to prevent the cancer from ever coming back.”
Managing Treatment
In January 2023, Emilia was started on the blinatumomab. Administered as a continuous infusion via a port in Emilia’s chest, the immunotherapy was contained in a backpack Emilia wore continuously for 28 days straight.
The treatment meant Lillybeth had to drive Emilia to Pittsburgh twice a week for four weeks for bag changes, and then repeat the entire process for another four weeks.
With Lillybeth and Steve both working full-time, Emilia’s big brothers, Nathaniel and Daniel, in school, and the Erie winter weather with which to contend, this period proved to be extremely challenging for the family.
A Turn for the Worse
Things took a turn for the worse in the summer of 2023. As a result of the intensification phase of Emilia’s chemotherapy treatment, her blood cell counts dropped dramatically. And then her hair fell out.
“That was…” Lillybeth pauses. “It was an adjustment,” she says after a moment, her voice shaking. “The hair didn’t faze Emilia or even her brothers, but it was really hard for me. It’s something I had to work through.”
In June, Emilia developed a high fever and had to be rushed to UPMC Hamot. She was transferred by ambulance from there to UPMC Children’s. “Emilia was in septic shock,” Lillybeth recalls. “We were so scared. It was really, really bad.”
Thankfully, physicians were able to stabilize little Emilia. After several days in the hospital, she was once again discharged home.
Brighter Days
Emilia entered the maintenance phase of treatment at the end of 2023. In the time since, she’s done exceptionally well and has experienced few treatment side effects.
Now 4 years old, Emilia loves playing outside, learning about animals and sea creatures, and anything related to princesses and unicorns. She’s also very attached to her big brothers. “Emilia has been smiling throughout all of this,” Lillybeth shares. “She never complains. She’s a little kid, but she’s my rock.”
Lillybeth and Steve remain grateful for the Division of Pediatric Hematology/Oncology at UPMC Children’s, and appreciative of the opportunity to enroll Emilia in a clinical trial.
“The entire oncology floor is awesome — the nurses, physicians, Mr. Mike [Shulock] from Child Life,” Lillybeth says. “Emilia’s physicians, Dr. Randy Windreich and Hematology/Oncology Fellow Dr. Julia Leix, have been super great. Dr. Randy is always poised. He gives us hope and makes us feel like everything is going to be okay.”
Making History
In August 2024, Dr. Windreich contacted the family with good news. Thanks to the chemotherapy/blinatumomab combination treatment, Emilia’s bloodwork looked excellent. She would no longer require additional spinal taps.
Dr. Windreich and his team further shared that the blinatumomab clinical trial Emilia participated in helped to prove the efficacy of the immunotherapy medicine as a frontline treatment for patients with ALL! The trial was a resounding success.
Emilia still has a way to go before she’s done with treatment, but she gets a little closer every day. With her participation in a cancer clinical trial, she’s further helping to inform better treatment options for other children with cancer.
Steve and Lillybeth could not be prouder of their little girl. “Emilia is a superhero,” they share. “She’s making history!”
At UPMC Children’s, Emilia’s battle against leukemia is not only bringing her closer to recovery but also advancing care for children around the world. By participating in a groundbreaking clinical trial, she’s showing how one little girl can make a big difference. Be a part of her incredible story! Contact Alana S. Kulesa, Senior Director of Corporate and Foundation Relations at UPMC Children’s Hospital Foundation to learn more about how you can invest in a child’s future with a gift to research, or donate now.