For 15 years, the Comprehensive Neuromuscular Center at Cincinnati Children’s has been a leader in providing multidisciplinary care for children with Duchenne muscular dystrophy, spinal muscular atrophy and other neuromuscular disorders. In 2016, the center was recognized by the National Committee for Quality Assurance (NCQA) as a Level 3 Patient-Centered Specialty Practice, the highest achievable status. Here, the center’s director, Brenda Wong, MD, MBBS, talks about how her team is advancing care and providing hope to patients with neuromuscular disease.

What does the NCQA designation signify? 

When patients are dealing with a progressive, multisystem disease, they need to have a specialty medical home where all of their care needs can be met. That is what our center is – we coordinate and integrate care across all elements of the complex healthcare system. So when Duchenne patients come to us, for instance, they are going to see the multiple pediatric specialists needed for their multisystem health problems at one appointment. This can include seeing a pediatric neurologist, endocrinologist, cardiologist, pulmonologist, orthopedic surgeon, physiatrist, genetic counselor, nutritionist and physical therapist, depending on their needs. A coordinated, collaborative, patient-centered specialty care program like ours improves outcomes and the patient experience.

How does this type of care improve outcomes?

Here is one recent example. Last year, we published a retrospective study in The Journal of Pediatrics that demonstrated the value of a coordinated, interdisciplinary care approach for Duchenne patients on long-term glucocorticoid therapy. Our care approach strives to maximize the benefits of long-term glucocorticoid therapy while minimizing glucocorticoid side effects. In our study, 97 patients ages 10 to <16 years were treated with daily glucocorticoid therapy for a mean of 8.5 years. Outcomes measures were motor, pulmonary and cardiac function, and scoliosis, and we found strong evidence that the glucocorticoid therapy provided meaningful benefit.

For instance, patients who were 13 to 16 years old experienced significant improvement in motor function, with 40 percent being able to rise from the floor and 50 percent performing a 30-foot run test. This is particularly important because Duchenne patients not treated with glucocorticoids generally lose the ability to walk from place to place by age 13. Side effects were evident but manageable, which we attribute to the well-coordinated, multidisciplinary care these patients received at our center.

Have other programs expressed interest in learning about the center’s model of care?

We have hosted clinical teams from around the country who were interested in spending a few days with us to observe our center in action. All of us have a desire to provide the best possible care for children and young adults with Duchenne and other neuromuscular diseases, and sharing expertise is one way to do that.

Who are your patients?

We have about 1,200 patients from all over the world, including Europe, South America, Canada, Asia, Africa, Russia and even Australia. About 500 have Duchenne, and the others have spinal muscular atrophy, other types of muscular dystrophy or another congenital myopathy. More than two-thirds of our patients come to the center from outside our immediate four-state region at least once a year, seeking the most up-to-date treatment and support. Many are eager to participate in our clinical trials. For patients with Duchenne and other debilitating neuromuscular diseases, providing access to experimental therapies and medications allows us to give families hope.

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