Patients with spinal muscular atrophy (SMA) receiving nusinersen appear to improve motor function as well as their nutritional status, according to a recently published study in BMC Neurology.
Nusinersen is an antisense oligonucleotide (ASO), the first of its kind to receive food and drug administration (FDA) approval for the treatment of SMA, the researchers wrote. Although several studies have assessed its efficacy and safety, there needs to be more data regarding its impact on the nutritional status of patients with SMA, they continued.
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Previous studies have pointed out the prevalence of nutritional disorders in patients with SMA, emphasizing the importance of close nutritional monitoring in these patients, the study authors noted.
Therefore, the authors aimed to assess the motor function and nutritional status of pediatric patients with SMA who had received the first 4 doses of nusinersen between 2019 and 2022 through a retrospective study.
The study included 46 patients with confirmed SMA. The majority of patients (n=31) had SMA type; however, SMA types 1 and 3 were also represented in the study, with 8 and 7 patients, respectively. Patients began treatment between 0.7 and 15 years of age. Four patients required ventilator support.
Motor function was evaluated using different standardized tools depending on SMA type. The modified Hammersmith Infant Neurologic Examination-Part 2 and the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders were used in patients with SMA type 1.
The Revised Upper Limb Module and Hammersmith Functional Motor Scales Expanded for patients with SMA type 2. In patients with SMA type 3, RULM, HFMSE, and 6-minute walk were used.
Due to the prevalence of scoliosis among patients with SMA and its impact on body length measuring, the authors used weight/age Z-scores to assess the nutritional status of patients. Results showed statistically significant improvement in motor function in all SMA types.
The authors noted significant nutritional improvement in patients with SMA type 2 after receiving the first two doses. Nonetheless, no statistically significant change occurred in a patient with SMA types 1 and 3.
“Our findings on the outcomes of patients with SMA treated with nusinersen can provide a better understanding of the disease, contribute to improved clinical and nutritional management of patients, and add to the assessment of disease-modifying treatment effects on SMA,” the authors concluded.
Yang H, Tao Q, Li D, et al. Assessment of motor function and nutritional status in children with spinal muscular atrophy treated with nusinersen after loading period in Western China: a retrospective study. BMC Neurol.Published online January 23, 2023. doi:10.1186/s12883-023-03063-3