Spinal muscular atrophy (SMA) affects approximately one out of as many as 10,000 children. The rarity of this condition can pose challenges for families searching for appropriate specialty care or opportunities for innovative treatment.
At Arkansas Children's Hospital (ACH), a specialty clinic provides both individualized services and access to clinical trials. Using a multidisciplinary approach, the SMA clinic cares for approximately 30 patients who are seen between two and four times per year.
“If we don’t treat these children, we would expect them to experience a rapid decline,” said Kapil Arya, M.D., a pediatric neurologist and medical director of physical medicine and rehabilitation at AC and associate professor of pediatrics and child neurology residency program director at the University of Arkansas for Medical Sciences. “What we see instead is improvement.”
During each visit to the SMA clinic, families meet with a team that includes a neurologist, pulmonologist, rehabilitation specialist, physical therapist, nutritionist, social worker and orthopedic specialist. Patients undergo motor function and pulmonary testing, followed by an evaluation of their rehabilitative needs. When appropriate, laboratory tests monitor medication response to quickly identify any potential side effects. Bone densitometry detects bone degradation, and swallow studies and sleep assessments diagnose issues that reduce quality of life.
This single three-to-four-hour visit allows families to access all the specialists they need to see in one location. Between visits, patients and families are urged to contact the clinic with any questions they may have.
A neurology nurse coordinates the SMA clinic. One of their responsibilities is phone triage. When a patient experiences worrisome symptoms, the parent contacts the clinic. Experts communicate the change of status to providers and return to the parents with provider recommendations. This service enables families to address medical concerns without needing to drive long distances for evaluation.
ACH houses the state's only dedicated clinic for pediatric SMA patients. SMA clinic patients have access to novel therapies currently undergoing clinical research. In the past, ACH participated in studies that have become accepted treatments. Today, pediatric neurologists at ACH continue to push the field. Current studies seek to determine whether combination therapy or increased medication dosage could offer enhanced benefit for SMA patients.
Always hopeful that clinical trials will advance care and improve patient quality of life, Arkansas Children’s Hospital clinicians rely on current treatment protocols that provide the best outcomes.
"We have been extremely pleased with how our patients are doing," Dr. Arya said. "They are happy. They maintain motor function, experience more stamina and are better able to play with friends and participate in family activities."
To complement the SMA clinic, ACH also houses a spasticity clinic. Developed in close cooperation with the hospital's rehabilitation services, the spasticity clinic is the state's only such clinic providing comprehensive care for children with spasticity. Common treatments include botulinum toxin injections, oral medications and orthopedic procedures.
When these are insufficient, neurologists may recommend intrathecal baclofen. With this approach, patients with spasticity are initially given a specific baclofen dose during a trial period, during which ACH neurologists and the spasticity clinic team monitor patients' progress and review responses at predetermined time increments.
Assuming a positive outcome, clinic neurologists recommend implantation of a baclofen pump. Once this has been placed, they continue to assess the effects and refill as required to provide ongoing spasticity management.
