N16 — April 2013 — Sisters Bone Marrow Transplant
Intro: Imagine what it would be like to give birth to healthy twin baby girls, only to find out that both of them were born with a potentially fatal disease. That happened to the family you're about to meet. Both girls needed a bone marrow transplant to hopefully cure a serious blood disorder. Vivien Williams has their story of survival.
"When they were born we thought there was nothing wrong with them."
And when you look at 5-year-old twins Elizabeth and Kathryn Girtler play with their big brother LeeRoy, you'd never suspect they were born with a deadly disease.
"I was still little."
Hours after birth, doctors told parents Michele and Brett that standard newborn screening blood tests revealed their precious baby twins had something wrong with their blood. A condition that could develop into leukemia if not treated. Amegakaryocytic thrombocytopenia [Ameg'a-care-it-oh-sit'-ick throm'-bow-site-oh-pee'-nee-ah].
"It was devastating actually."
Mayo Clinic pediatric hematologist Dr. Shakila Khan [Shah-Kee'-lah Kahn] says it's an inherited disease that causes your bone marrow to malfunction and not make enough platelets, which are the components of blood responsible for clotting.
"The curative treatment is bone marrow transplant for these patients."
For their first four years the girls stayed relatively healthy with transfusions of platelets. But as the disease progressed, the Girtlers decided it was time for a transplant. First it was Elizabeth's turn. Then Kathryn's. Dr. Khan and a medical team that included Dr. Vilmarie Rodriguez and nurse practitioner Julia Gourde prepared the girls for the procedure.
It involves wiping out the bone marrow and immune system with chemotherapy and sometimes radiation. Then healthy donor stem cells are infused into the blood stream via an IV. As if equipped with a homing device, the new cells travel to the bone marrow where new healthy cells grow.
It wasn't an easy journey; the girls spent lots of time in the hospital.
"It was very hard."
But the bone marrow transplants were successful.
"They can now just be normal little kids again. That's what I want for them."
Healthy and strong and able to keep up with their big brother LeeRoy. For Mayo Clinic News Network, I'm Vivien Williams.
Dr. Khan says if all continues to go well, Elizabeth and Kathryn will be cured with no need for medication in the future. She also says bone marrow transplant has come a long way in recent years. Better medicines and better ways to treat complications mean more kids are surviving and living.
For more information, visit our website at…
STATIONS: Per the licensing agreement, please provide a link from your station's website to http://www.MayoClinic.org or voice tag "MayoClinic.org" for more information.