Changing the face of Midaortic Syndrome treatment to save Sofia

The American Journal of Transplantation recently published a paper, documenting a first-of-its-kind treatment for midaortic syndrome, as performed by Khashayar Vakili, MD, of Boston Children’s Department of Surgery and Heung Bae Kim, MD, director of Boston Children’s Pediatric Transplant Center.

In the following blog Marcus Groff, father of the patient described in the Journal of Transplantation, writes about his daughter’s revolutionary surgery at Boston Children’s.

Sofia

Two years ago my wife and I were anxiously waiting outside the NICU of our local hospital at four in the morning. Inside, our daughter Sofia was being monitored for several medical concerns, including dangerously high blood pressure. The nurses on staff assured us that Sofia was “a fighter” so when we drifted off to sleep later that morning we were exhausted, but joyful. We never saw what was coming, but then, you rarely do.

It would take a few days before the gravity of the situation became too obvious to ignore. While we fretted over our daughter’s high bilirubin and high blood pressure, something ominous was lurking. I couldn’t put my finger on it, but something wasn’t quite right. Still, we took our queues from the medical staff, which seemed confident our Sofia would pull-through. Only later, through tearful goodbyes, would the nurses confide that privately they feared Sofia wouldn’t make it.

We came to Boston Children’s for a second opinion of what was making Sofia so sick—what we got was a name for the phantom malady that was plaguing our daughter: midaortic syndrome (MAS). MAS occurs when the aorta, the main artery that delivers oxygen-rich blood throughout the body, is narrowed or damaged, which can often cause serious spikes in blood pressure by restricting blood flow to the kidneys. (A condition known as renovascular hypertension.)

We finally had a name for Sofia’s illness, but a larger question still loomed: How do you treat it?

Before treatment, Sofia was a failure to thrive baby

As it turns out, even the most accomplished specialists in the world don’t have a ready-made answer to that question. There are few established corrective treatments for MAS—and none that avail themselves to a 5-pound infant. Sofia would require a novel approach; something as unique as she is, something that couldn’t be anticipated. She needed something…fantastic.

Given that they are in the business of evolving medicine, it’s not surprising that the specialists at Boston Children’s wouldn’t shrink from this challenge. But, no one could have expected just how ready they were.  Much like an accomplished group of musicians, formed into an orchestra to perform a masterpiece, Sofia’s treatment became their latest opus.

It began with an angioplasty on Sofia’s Celiac Artery. A tiny balloon was inserted in the artery and inflated, manually expanding the narrowed passage. Several months, multiple angioplasties and ultimately a few stents later and Sofia had grown from a 5-pound infant to a 21-pound toddler who was robust enough to undergo surgery.

But like Sofia herself, the upcoming surgery would be anything but typical.

Sofia saw many doctors so she could grow big and strong enough for surgery to treat her MAS

On Thursday, October 28th, 2010, Sofia received the gift of a new kidney, with several inches of healthy aorta attached—which is what made her procedure so special. Typical kidney transplants consist solely of a donor organ being placed inside the recipient, but Sofia needed a bit more. Lead by Dr. Vakili and Dr. Kim a talented team of doctors performed the first ever organ transplant where a length of aorta was transplanted along with a kidney, simultaneously treating both Sofia’s renal failure and MAS.

13 days later Sofia was home and enthusiastically crawling up and down the stairs of our house. A far cry from the sick, bedridden little girl she had been weeks earlier.

 

With a new a kidney and length of aorta Sofia is feeling much better

It’s been a few years since her treatment, but memories of that time are still very fresh in my mind. For two years we watched as our daughter slowly gained weight, fought off illness and worked to overcome developmental hurdles. As a parent it was very difficult to watch, but the careful supervision and coordination on the part of Boston Children’s Cardiology, Nephrology, Gastroenterology and Nutrition teams saw her through. All the while we marveled at her good cheer and resilience. Before her operation, Sofia was the textbook definition of “failure to thrive”, but in terms of overcoming obstacles, she demonstrated a willfulness and determination that we openly envied.

Sofia carries the hopes and dreams of two families inside her now—and the promise of a better day for other children suffering from MAS. Thanks to Dr. Vakili’s vision and surgical skill she is ready—and finally able—to thrive.

Boston Children’s has a history of designing new and innovative ways to treat children with MAS. Learn more about our MAS program, and the multiple approaches our specialists have for caring for children with midaortic syndrome.

Interested in speaking with a member of Boston Children’s Midaortic Syndrome and Renovascular Hypertension (MAS/RVH) Program? Please click to request an appointment with our MAS specialists.