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."
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