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A serendipitous save that changed treatment of the most common tumor of infancy

IsabellaManley1stgrade-cropAt research institutions like Stanford, we often talk about the value of evidence-based medical care, the kind based on careful scientific comparisons of which treatments work best.

But sometimes, even the best-studied treatments fail. That's what happened in 2008, when a baby named Isabella Manley was brought by her parents from their Sacramento, Calif., home to Lucile Packard Children's Hospital Stanford because of a tumor in her trachea that threatened to block her breathing. Her case illustrates that serendipity sometimes plays a key role in medical success.

Isabella had a hemangioma, the most common tumor of infancy. Most hemangiomas, which consist of extra blood vessels, create harmless red marks on a baby's skin that fade with time. Isabella's was much more serious. Although her doctors tried all the hemangioma treatments then reported in the medical literature, including high-powered steroid drugs and two types of surgery, her breathing problems persisted. Pediatric otolaryngologist Kay Chang, MD, who oversaw her care at Stanford, ordered an MRI to find out why. A story I wrote about her case explains what happened next:

“We found, to our horror, that this hemangioma was massive, surrounding her entire windpipe and also her heart,” Chang said.

“It was becoming tangled into every structure in her neck and crawling down into her chest,” said Mai Thy Truong, MD, a pediatric otolaryngologist now with the hospital’s vascular malformations clinic.

... The tumor was too extensive for surgery and was still growing. Truong and Chang feared that it would soon block Isabella’s airway. They were not sure they could save her.

Fortunately for Isabella, Chang recalled hearing a conversation at a recent medical conference about an accidental discovery by a group of French physicians: They saw that a blood pressure medication, propranolol, shrank skin hemangiomas in a few of their patients. Chang wondered if the drug would have the same effect on a hemangioma in the airway. Isabella's parents, Todd and Julie Anna, told me what happened when their baby daughter started receiving propranolol:

To everyone’s surprise, Isabella’s breathing improved in the first 24 hours on the medication. A week later, she received another MRI.

“Dr. Chang came and found us and said, ‘You’ve got to see this; the tumor shrunk in half,’” Julie Anna recalled. “We were just beside ourselves.”

After enduring such a long struggle to help Isabella breathe, the Manleys cried happy tears. “It was one of the best days of our lives,” Todd said.

Chang, meanwhile, couldn’t stop thinking about what the success meant for other children. “I was totally excited,” he said. “It’s rare in medicine that you see something have such a dramatic effect so fast.”

And that's my favorite part of this story: Not only is Isabella now a healthy first-grader, but her case helped alert doctors to an entirely new line of scientific investigation. Today, a search of the medical literature reveals hundreds of studies documenting the value of propranolol for treating severe hemangiomas.

The consensus of this research is clear. The drug works better and has far fewer side effects than the previous generation of therapies. A chance discovery, and its serendipitous use, seeded a revolution in this corner of evidence-based medicine.

Previously: Why researchers need to communicate the story behind the science
Photo of Isabella Manley courtesy of Todd and Julie Anna Manley

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