SMA Disease Management Guidelines Released

A new “consensus statement” on clinical care in spinal muscular atrophy (SMA), published in the August issue of the Journal of Child Neurology, aims to improve and standardize the management of this disease with respect to diagnosis, pulmonary care, gastrointestinal and nutritional issues, orthopedics and rehabilitation, and end-of-life decisions. (For the entire document, see “Consensus Statement”.)

Establishing standards of SMA care was identified as a priority by a patient advocacy group of the International Coordinating Committee (ICC) for Spinal Muscular Atrophy Clinical Trials. The ICC was formed in January 2005 under the auspices of the National Institutes of Health (NIH).

After discussions at an NIH-sponsored conference in 2004 revealed wide variations in medical practices in this disease, MDA supported the formation of an SMA Standard of Care Committee through the ICC’s patient advocacy group.

The committee divided SMA patients into “nonsitters,” “sitters” and “walkers” and addressed the care needs of the three groups separately and in detail.

Here are some of the group’s general conclusions.

• Even young children with SMA should be offered independent mobility and activities of daily living, including play.
• Walking should be encouraged, with the use of appropriate assistive devices and orthotics.
• Spinal orthoses (back braces) may provide support but don’t prevent progression of spinal curvatures and may impair breathing.
• Surgery for scoliosis (spinal curvature) appears to benefit patients who survive beyond age 2 when curves are severe and progressive.
• Scoliosis surgery should be performed while pulmonary function is adequate.
• Special preoperative respiratory care and neurological monitoring during surgery are desirable.