30-year 'kidneyversary' highlights need for protocols for CAKUT patients

March 12 is an important date for Maria Hoffman. It’s the anniversary of the day, 30 years ago, that she received a kidney transplant at the age of 9.

Thirty-year kidneys are unusual, though not unheard of – one woman in France recently celebrated 50 years with a transplanted kidney. But Hoffman’s just might be the longest-lasting kidney that was transplanted into a child because of CAKUT, or congenital anomalies of the kidneys and urinary tract, said her doctor, MUSC Children’s Health’s Shumyle Alam, M.D.

The remarkable milestone is something to celebrate – but it’s also incentive to develop better protocols for the care of CAKUT patients so that more of them can enjoy stable health and longer-lasting kidneys, Alam said.

“The literature would suggest she had a greater than 50% chance of retransplant within 10 years, and here we are 30 years later. That’s extraordinary,” he said.

He advocates for a multidisciplinary team approach to care as well as protocols that follow these patients into adulthood. Right now, patients who age out of the pediatric system find themselves adrift amid a disconnected set of specialists, including urologists, nephrologists, transplant surgeons and even gynecologists, none of whom have extensive experience with CAKUT patients.

Protocols developed for cystic fibrosis and heart patients making the transition from pediatric care to adult doctors have led to healthier patients with longer life spans, and CAKUT care should have the same types of standardized guidelines, Alam said.

Hoffman experienced that disconnect when she aged out of pediatric care.

“The adult world is completely different,” she said. She found that adult doctors typically didn’t order the labs and ultrasounds that pediatric doctors would to monitor her health. That’s why she, and so many of Alam’s other patients, continue to see him into adulthood. Hoffman has followed Alam from Cincinnati Children’s, where she met him during his fellowship, to New York and now to MUSC Children’s Health. The last move was serendipitous for Hoffman, who grew up in Greer, South Carolina, and attended Clemson University. She had been looking for a way to return to her home state.

“When he announced he was coming to South Carolina, it was a no-brainer to start applying for jobs and getting back here,” she said.

Having grown up in the medical world, Hoffman had become interested in quality improvement and earned her Master in Health Administration degree while working at Cincinnati Children’s. When she returned to South Carolina, she found a job as program manager in the Susan Pearlstine Sarcoidosis Center of Excellence at MUSC Health.

Alam said Hoffman’s extraordinary outcome can be traced to the beginnings of her care. Her kidney transplant surgeon, Curtis Sheldon, M.D., was founding director of the Urogenital Center at Cincinnati Children’s. Alam described him as something of a “unicorn,” the rare doctor who was a board certified pediatric surgeon, pediatric urologist and a transplant surgeon, meaning Hoffman had multidisciplinary care from the beginning.

"There’s yet to be a protocolized approach for how we should monitor patients, and that’s what we’re trying to find out. We’re trying to develop a protocol that allows us to best monitor and follow patients with CAKUT to prevent deterioration of kidney function but also to promote a holistic pathway for the best possible outcomes."

Dr. Shumyle Alam

Nonetheless, Hoffman said, when she prepared to transition out of pediatric nephrology care, she was told, “Your kidney probably won’t last another year. You're lucky if you get two more years out of it.”

She attributes her kidney’s longevity since then to Alam and to Lisa Creelman, R.N., who has cared for Hoffman since she was a child and who transferred with Alam to New York and then to MUSC Children's Health.

“Getting to 30 years post-transplant hasn't always been sunshine and rainbows, but you would never know that because Lisa is behind the scenes making the impossible possible,” Hoffman said.

Alam said children need the full range of care management, encompassing areas like nutrition, growth and development and bladder and bowel management. Unfortunately, children’s outcomes are still measured the same as adult outcomes, he said. For example, an adult can have 10% to 20% kidney function and not need dialysis.

“Now imagine an 8-year-old child who’s metabolically active. If they have 10% to 20% kidney function, they're not going to grow,” he said.

Yet because both patients are off dialysis, their outcomes are considered equivalent. Most of the research literature focuses on such immediate outcomes related to the transplanted kidney’s survival and time off of dialysis.

“There are immediate outcomes, and there are outcomes based on measurements that are standardized. For example, with transplant, does the graft have blood flow? Is the patient on dialysis? It’s not: Has the patient grown? Has the patient reached their milestones? Has the patient done well in school? Those types of things aren’t really talked about.”

At MUSC Children’s Health, Alam’s team is working to create a model that can assess each patient’s risk level and from there determine how much and what type of monitoring each patient needs.

“There’s yet to be a protocolized approach for how we should monitor patients, and that’s what we’re trying to find out. We’re trying to develop a protocol that allows us to best monitor and follow patients with CAKUT to prevent deterioration of kidney function but also to promote a holistic pathway for the best possible outcomes and to prepare them for a transition plan to adult care because, again, the biggest problem with transition plans is not knowledge of the disease but best practices for how to manage,” he said.

But Alam’s thoroughness isn’t the only reason that Hoffman and so many other patients have followed him. Hoffman especially appreciates the personal touch that Alam brings into each patient encounter.

“He always said, ‘I’m not here to treat “the patient.” I’m here to treat Maria.’ And I’m not the only patient of his who says that," she said.