When 15-year-old Grayson paddles a canoe, plays on the beach or competes on his school trap-shooting team, there is little evidence of the challenges he has overcome. His adrenal glands do not make the hormones essential to help him grow, endure rigorous exercise and cope with stress.

Thanks to ongoing care he found at Children’s Hospital of Philadelphia for Addison’s disease, an extremely rare condition that affects an estimated one to four children out of a million, Grayson is leading a normal life.

“At the end of the day, this is something that people can manage,” says Maria G. Vogiatzi, MD, Medical Director at CHOP’s Adrenal and Puberty Center. 

Every six months, Grayson and his mom journey from Fayetteville, Ark., to Philadelphia. At each visit, CHOP specialists monitor his progress and adjust the suite of medications that spur bone growth and replace necessary hormones.  

Long trip worth the expertise at CHOP

The long trip is worth it, Grayson’s mother Lindsey says, for the expertise and reassurance provided by CHOP specialists for his extremely rare disorder.

“There is definitely a way to manage it and live a completely normal life with some modifications. We are very thankful and blessed that his disease process is so stable and well controlled with some minor changes. We could not be happier,” Lindsey says. “With Dr. Vogiatzi, there is a real sense of peace and safety and reassurance that we are doing everything right.”

Although Addison’s disease is rare, adrenal insufficiency also accompanies other conditions treated by CHOP specialists. The approach, Dr. Vogiatzi says, is personalized to each patient. For example, when children with adrenal insufficiency like Grayson arrive at CHOP, Dr. Vogiatzi says, “there is an initial phase of learning for everyone.”

CHOP specialists come to understand, she says, “how the patient responds to treatment. The family learns how to manage it. And then we decide how often we need to see them.”

Having tried other doctors before finding CHOP, Grayson says, “we had not such a great experience a few times. Now I am comfortable and my whole family can be comfortable. I want kids like me to know about ‘Dr. V,’ so they know they are not alone with this disease.”

Coming from Arkansas to see Dr Vogiatzi, Grayson adds, is worth it. “I would follow her if she was farther away. From the very first moment we came here, we could not have had a better experience.”

Grayson’s journey to CHOP

Grayson was 11 years old when his first symptoms appeared. He became nauseous after eating a breakfast granola bar. Over the next few months, unexplained bouts of nausea and vomiting continued, accompanied by increasing muscle weakness. Grayson struggled to climb stairs. He lost 20 pounds, despite a voracious appetite for salt. He would eat three jars of pickles and drink the pickle juice, his mother remembers. One night in 2019, the fourth grader was found unconscious on the couch, prompting his first hospitalization. 

Bloodwork at a children’s hospital in Arkansas showed adrenal deficiency. The family found an endocrinologist in Tulsa who explained the extreme rarity of the condition in children. The family sought care from a pediatric specialist in Kansas City. Grayson was soon taking hormone medication, morning, afternoon and night, a regimen to continue for life. Two years following diagnosis, his mother became worried about Grayson’s overall health. He was not growing as expected even though his adrenal insufficiency was under control. It became clear, Lindsey says, that Grayson needed specialists who could provide a more comprehensive approach to meet all his needs.

Grayson’s local pediatrician recommended the family get evaluated at CHOP. In 2021, Grayson traveled to CHOP for an evaluation that resulted in the successful treatment plan he still follows to help his body grow and thrive.

Before Grayson found comprehensive care in Philadelphia, “I felt like he was falling through the cracks,” Lindsey says. “As a child with such a rare condition, Grayson is a unicorn. He needed a unicorn doctor and that is what we found at CHOP.”

From fear and confusion to normalcy

As he prepares to finish ninth grade, Grayson says he wants children like him to know the disease can be treated and that it is possible to live a fairly normal life.

After each visit, CHOP specialists relay a report back home to Grayson’s pediatrician in Fayetteville. They remain in contact to coordinate care as he grows to adulthood.

“Dr. V gave us a sense of peace, a sense of hope,” Grayson says. “Having a disease like this is frightening. It can leave you confused and scared. I want people to know they don’t have to feel afraid. They are not alone.”