The clinical team from the Roswell Park Oishei Children’s Cancer and Blood Disorders Program has successfully performed the region’s first pediatric bone marrow transplant for 19-year-old sickle cell patient Kayla Rushing, effectively curing her of the inherited blood disease.
The successful transplant, made possible through a joint program with Oishei Children’s Hospital and Roswell Park Comprehensive Cancer Center, offers hope and more accessible treatment options for those suffering from the disease in Western New York and the surrounding regions.
Steven Ambrusko, MD, MS, director of sickle cell and hemoglobinopathy services within the joint program, said he is pleased with the outcome of the transplant and what it means for patients suffering from sickle cell disease.
“We’ve created a successful and collaborative joint program to ensure that children and their families suffering from this disease don’t have to look elsewhere for treatment — it’s available to them right here in Western New York on the Buffalo Niagara Medical Campus,” Ambrusko said.
The procedure, the program’s first pediatric bone marrow transplant for sickle cell disease, was conducted as part of a clinical trial using a haploidentical donor, meaning the stem cells donated — by the patient’s younger sister, Amaya Rushing — were only half a genetic match to the patient’s own stem cells. The transplant team, led by Barbara Bambach, MD, and Meghan Higman, MD, infused Amaya’s cells directly into the patient after conditioning chemotherapy to replace the unhealthy blood-forming stem cells.
Sickle cell disease causes the body’s red blood cells to change shape, making them break apart rapidly and block blood vessels, resulting in poor oxygen supply to muscles, bones, and other organs. Without oxygen, these organs become damaged, resulting in excruciating pain episodes and other serious complications, such as life-threatening infection, lung crises, and stroke.
Kayla had lived with sickle cell disease since birth. She and her family decided on the bone marrow transplant treatment option last year after she became unable to continue daily activities such as attending school, getting in a car, and even walking at times. Doctors said she had debilitating chronic pain, occurrences of sickle cell crisis in her lungs, and a need for periodic blood transfusions.
“She is now disease-free and will soon be able to go back out into the world to do what a 19-year-old should be doing,” Ambrusko said.
“Amaya is the real star of the show,” Rushing says of her 14-year-old sister, whose donation of stem cells made this treatment possible.
According to Ambrusko, the clinical team treats approximately 160 children and adolescents with sickle cell disease as part of the joint Roswell Park Oishei Children’s Cancer and Blood Disorders Program. “Only 11% of sickle cell disease patients have a fully-matched potential donor, so if haploidentical bone marrow transplant is proven safe and effective, it may open the door to cure for many of our patients in need.”
“Sickle cell disease can be such a devastating chronic illness for children and their families to have to endure,” notes Dr. Bambach, clinical director of pediatric bone marrow transplant for the joint program. “The tremendous collaboration between Roswell Park and Oishei Children’s Hospital has allowed for us to be able to offer leading edge therapies such as hematopoietic stem cell transplantation for sickle cell disease to these patients, and offer them a chance for a better life and cure.”
Blood and bone marrow transplant is currently the only cure for sickle cell disease, a group of inherited red blood cell disorders typically diagnosed at birth, and most common in people of African or Hispanic heritage.