Johns Hopkins Gazette: March 4, 1996

Researchers Improve Treatment Of Young Marfan Patients

Michele Fizzano McFarland
JHMI Office of Public Affairs

     Scoliosis, or curvature of the spine, is one of the most
debilitating side effects of Marfan syndrome. For the first time
researchers have a clearer understanding of how best to treat it
in young Marfan syndrome patients. Contrary to standard practice,
delayed treatment of scoliosis may yield better results than
early, aggressive therapy, they said.

     Investigators from the Johns Hopkins Children's Center
studied the long-term  effects of three different approaches to 
correction of the spine in severe cases of Marfan syndrome. Their
findings, presented last week at the American Academy of
Orthopedics Annual Meeting in Atlanta, showed that early
intervention produces only marginal improvement.

     Paul Sponseller, director of pediatric orthopedics at
Hopkins, said, "Generally, other medical conditions, commonly
heart valve repairs, need to be identified and addressed before
spinal correction is helpful in this special group of patients."

     Marfan syndrome, an inherited, life-threatening, connective
tissue disorder, affects one in every 10,000 children with an
array of symptoms from ocular lens dislocation to heart trouble.
Severe scoliosis, present in 5 to 10 percent of Marfan cases, can
compromise height as a child grows, impede mobility, deform front
and back ribcages and in some cases, interfere with breathing.

     Curve progression tends to be faster and worse in patients
with Marfan syndrome than among those whose scoliosis is of an
unknown origin. Surgeons often attempt to stabilize the spine in
kids as young as 3 years old to prevent further collapse, correct
shortening and improve lung function.

     The three most common treatments include a brace worn
outside the body, sometimes 12 hours a day for six months; a rod
surgically implanted onto the spine; and a rod combined with
surgical fusion of vertebrae to limit spinal bend and curve.

     In the study, bracing had only a limited role in patients
with Marfan syndrome. Although proven effective in children
without the disorder, 11 patients reported no improvement.
"Bracing appears to be effective only when curvature is less than
40 degrees," Sponseller said.

     The researchers also found that children with curvature high
in the spinal column, called kyphosis (or hunchback), are not
helped by a rod alone. Although bracing is one of the most used
current treatments, poor bone density and severe spinal curves
often cause implanted rods to slip, Sponseller said. When
attempted in three patients, the bracing rod dislodged in two
children with kyphosis.

     "In general, children do better if spinal fusion is
performed after age 5, Sponseller said. In 10 patients with an
average age of 6 who underwent surgical rod placement and spinal
fusion, correction diminished 31 percent over time. In addition,
complications occurred in more than one-third of cases.

     "This information should encourage us to wait until a child
with Marfan syndrome is older and more medically stable before
performing corrective surgery," Sponseller said. "Surgeries can
help, but they have to be done judiciously."

Go back to Previous Page

Go to Gazette Homepage