Medical world watching as Kansas baby battles disease / LJWorld.com

Archive for Monday, February 4, 2008

Associated Press

February 4, 2008

Kansas City, Mo. — Medical professionals have been watching closely how an 11-month-old Kansas boy responds to treatment for a life-threatening genetic syndrome.

"There's a lot of curiosity about what happens to him," said Dr. Laurie Smith, a genetics expert at Children's Mercy Hospital. "I would say all the metabolic doctors in the world are interested in what will happen."

Jarin Burkett of Fort Riley, Kan., has Hunter syndrome, which prevents his body from producing enzymes needed to break down certain waste products in cells, according to The Kansas City Star. The syndrome can lead to coarsened facial features, stiffened joints, enlarged internal organs, deafness and eye problems. In severe cases, children develop mental retardation, aggressive behavior and hyperactivity.

How Jarin responds to the weekly intravenous infusion of a recently approved drug called Elaprase could mean hope for other infants with Hunter syndrome, which affects about one in 100,000 births.

Elaprase supplies the missing enzyme and is the only treatment available for Hunter syndrome. Children's Mercy has become one of the leading hospitals in the nation providing the therapy.

Elaprase has been tested only on patients 5 years and older. It has improved their conditions, but by that age children often have already suffered the disease's damage.

Jarin's role is important because he started taking Elaprase when he was 3 months old.

He and his mother, Krista Burkett, 28, drive from their home at Fort Riley, Kan., to Children's Mercy for the treatment. Jarin's father, Army Sgt. Jeff Burkett, 31, has been stationed at a military base north of Baghdad since September.

If Jarin shows that early treatment works, that may be a justification for newborn screening for Hunter syndrome, Smith said.

So far, Jarin is doing remarkably well. He's meeting his developmental milestones and his Hunter syndrome symptoms have been mild.