Holden Guilfoyle, 3, Fort Scott, is unique in the state. He has the only case in Kansas of type-six mucopolysaccharidosis (MPS), also known as Maroteaux-Lamy Syndrome. MPS type-six affects 50 people in the United States and 1,100 worldwide.
The treatment is not easy, either. Holden must get a weekly four-hour enzyme infusion of 20 milliliters of naglazyme. Each milliliter costs $369. Over the course of a month, just the infusion costs would be worth more than $30,000.
And that does not count the supplies, IV, nurse, heparin shots, and other medical costs required to keep Holden in working order. Luckily, the Guilfoyle’s insurance has been helpful, despite the high costs.
“I’m sure our son is one of the top topics of discussion in their board meetings every year,” said Jenny Guilfoyle, Holden’s mother. Holden has an older sister, Addisyn, 6, and a younger brother, Gabriel, 2.
Holden’s Hope
Holden’s parents are hosting the second annual Holden’s Hope Day from 11 a.m. to 4 p.m. Saturday, June 28 at Lincoln Park in Pittsburg. The event is meant to raise awareness for both mucopolysaccharidosis and for Holden.
Holden’s Hope Day will host multiple games, activities, food and contests for attendees. The event will include a petting zoo, two bounce houses, an inflatable slide, an inflatable obstacle course, a speed pitch game, a golf challenge, face paint, hair paint, Spin Art, tattoos, a trackless train, food, music, a raffle, giveaways and prizes.
Wristbands providing access to the event will be $4.
Last year, the inaugural event had to be moved because of the late June flooding. Even so, the event drew 150-200 people and raised about $2,700.
“Because of the flood, we didn’t get the turnout we were expecting,” Guilfoyle said. “We actually had a company from Baxter Springs up here for a company picnic that saw us and donated to the event for their entire group’s kids to come over.”
Behind the disease
In some ways, the Guilfoyles are lucky. There are seven types of MPS, and type-six, which Holden has, is the only one that does not affect the child’s intellect or lead to death. Only three of the seven types are treatable by enzyme infusions.
MPS, and more specifically Holden’s type of the disease, is characterized by several common symptoms. Symptoms include chronic upper respiratory illnesses, recurring ear infections, a large head, “coarse facial features,” a kyphosis (rounded bump) on the spine, short stature, hearing and vision problems, a big or thick tongue, joint problems and thick skin. Simply put, Holden’s body does not properly break down cells, which causes problems throughout the body. There is a potential Holden could stop growing at some point in the future, but doctors said he could be treated with growth hormone if that were the case.
Guilfoyle said many other parents of MPS type six children have noticed larger than normal birth size for their children. Holden was born two weeks early, but he still weighed 10 pounds., 5 oz. and was 22 1/2 inches long.
Guilfoyle said the discovery of Holden’s disease was not a complete surprise.
“I was alone when we found out. My husband was working in Texas at the time,” Guilfoyle said. “The doctor does not offer much when there is a diagnosis like this. They just wait until the results are in before they tell you.
“Holden took a urine test on a Tuesday, and by Friday we had it confirmed he had MPS, we just didn’t know what form. When I came home and looked it up, he had every symptom. So we were prepared, we were more focused on which type he had.”
Treating MPS
For the first nine months of Holden’s enzyme infusions, the Guilfoyles had to drive to Kansas City every week to get the treatment until he was cleared for home treatments. When taking the treatment, Holden cannot run a fever more than 100 degrees. Guilfoyle said Holden often starts to run a slight fever during the first hour of treatment, but it never gets to or above 100 degrees. Holden has not missed a treatment to date.
“Every week, there is the potential for Holden to get hives or have a deadly reaction to the enzymes,” Guilfoyle said. “He has labs every 12 weeks to look out for him. We try not to take it for granted that he’ll be fine every transfusion.”
Holden saw several doctors, including an allergy specialist, an ear, nose and throat doctor, his primary care physician and a few trips to the emergency room, before he was diagnosed. It took a Kansas City orthopedist to recognize the rare disease. That doctor then called an area geneticist, who was on vacation at the time, and told the geneticist, “We’ve got a Maroteaux-Lamy.”
The orthopedist asked the geneticist to pencil the Guilfoyles in for an appointment as soon as possible, despite a four-month wait on the geneticist’s books. The Guilfoyles got in the following Tuesday.
Finding the funds
Guilfoyle said she has had great support from both her insurance company and the company that makes Holden’s enzyme treatments.
Her insurance has not placed a cap on the amount of money spent on Holden’s care because the disease is so rare. The Guilfoyles pay only $28 a month for Holden’s enzyme infusions. The drug company behind the enzyme treatments have taken a direct hand in Holden’s care, too.
“The company that makes his enzyme treatments called three days after we got the diagnosis to let us know that ‘we’re here and we’re going to help you.’ Holden’s drug representative is amazing,” Guilfoyle said. “He was there the first time we had the infusion in Kansas City and was there for the first home infusion. You could not ask for a better company. We never had to call our own insurance, they just took care of us.”
Guilfoyle said the oldest person she knew with MPS recently died at the age of 34. There is one person diagnosed in Missouri and three in Texas, but no currently diagnosed cases in Oklahoma.
Planning for the future
Holden’s Hope Day raises funds that go both toward future events and to raise money for MPS research through the MPS Society. The Guilfoyles are seeking to create a charity in Holden’s name.
“Hopefully, we can become a charity and get eligible for grants,” Guilfoyle said. “Right now, we’re just a family of five trying to deal with a disease that kills most kids before their parents even know about it.”
Guilfoyle said she’s learned lessons from last year’s event and hopes the turnout is even higher, between 200 and 300 people.
“Being the second year of the event, we’ve learned the ins and outs of how to put this on,” Guilfoyle said. “There are more things available to us. We had to bring back the petting zoo, which was huge last year.”
There will be plenty of giveaways and prizes at Holden’s Hope Day, including several personalized items. Guilfoyle said the day is for kids to “run around and do what they want.”
“I hope there is an excitement in people that they want to have a fun day with their kids,” Guilfoyle said. “They can come out and meet a special little guy that is braver than anyone I know.”
“He’s started asking every day about who’s coming to see him today,” Guilfoyle added. “Most kids don’t even have to ask that.”
For more information about MPS and Holden’s Hope Day, visit www.holdenshope.com.
Andrew Nash can be reached at andrew.nash@morningsun.net or (620) 231-2600 ext. 132.
FORT SCOTT —
