In October 2017, two physicians from Lucile Packard Children’s Hospital Stanford traveled to another California hospital to evaluate a little boy with a very rare genetic disease. Ronnie Dogan had an autoimmune condition called IPEX syndrome. From his second week of life, his immune system waged severe attacks on his digestive tract. He was very ill.
When doctors Alice Bertaina, MD, PhD, and Rosa Bacchetta, MD, met him, he’d just turned 1 and had spent nearly all his life in hospitals.
But Bertaina and Bacchetta determined that Ronnie was a good candidate for a stem cell transplant, which had the potential to cure him. A transplant would replace some of the abnormal immune stem cells in his bone marrow with those from a healthy person, enabling his immune system to function normally. The risks of a standard stem cell transplant had deterred his other doctors from pursuing this option, but the Stanford team could offer an improved transplant technique to treat his IPEX syndrome that was not available anywhere else in the United States.
As a new story in Stanford Medicine magazine explains, considering this option required courage from Ronnie’s family. When they first heard about the possibility of a transplant:
Ronnie’s family was skeptical. They’d recently been told there was no hope.
The physicians weren’t surprised by this reaction. “Before we met them, they went through so much, so many different therapies, and nothing was enough to cure the disease or to really overcome the clinical manifestations,” Bacchetta said. “Usually it’s very, very traumatic for a family to see their child so sick, to have a disease which is basically incurable or curable only with a very steep pathway, an invasive treatment.”
Once the Stanford physicians provided a detailed explanation of the proposed treatment, Ronnie’s grandmother and legal guardian, Rosemary Anama, was thrilled that he could be helped. “I don’t even have the words for it — I was past happy and excited,” she said.
There were still challenges ahead. In late 2017, Ronnie’s condition worsened, and it took months to stabilize him enough to bring him to Packard Children’s. Meanwhile, in early 2018, Ronnie’s little brother, Levi, was born. He, too, was soon diagnosed with IPEX syndrome and transferred to Packard Children’s.
Ronnie received his stem cell transplant in April 2018, and Levi’s was performed in August. The procedure required several more months in the hospital while the medical team waited to see if the donor stem cells would become established in the boys’ bone marrow.
As the months passed and Ronnie and Levi’s health improved, Anama took great joy in watching her grandsons grow. Again, from the story:
She loves seeing the boys show who they are, such as when they’re dancing to the “Baby Shark” song that exploded on social media over the last year. “Ronnie sways side to side and Levi goes back and forth,” she said with a chuckle.
Best of all, both brothers are now doing well and are expected to be out of the hospital soon.
