Journal Review: Nutrition outcomes of disease modifying therapies in spinal muscular atrophy: A systematic review
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Abstract
The nutritional implications of spinal muscular atrophy (SMA) are profound. Disease modifying therapies (DMT) have improved clinical outcomes. This review describes the impact of DMT on nutrition outcomes. A systematic search strategy was applied across seven databases until May 2023. Eligible studies measured nutrition outcomes in individuals with SMA on DMT (nusinersen, risdiplam or onasemnogene abeparvovec [OA]) compared to untreated comparators. Nutrition outcomes included anthropometry, feeding route, swallowing dysfunction, dietary intake, dietetic intervention, nutritional biochemistry, metabolism, gastrointestinal issues and energy expenditure. Articles retrieved were screened in duplicate, data were extracted and appraised systematically. Sixty three articles from 54 studies were included; 41% (n = 22) investigated nusinersen in pediatric participants with SMA type 1. Anthropometry (n = 18), feeding route (n = 39), and swallowing dysfunction (n = 18) were the most commonly reported outcomes. In combined pediatric and adult cohorts, BMI z-score remained stable post nusinersen therapy. The proportion of children with SMA requiring enteral nutrition was stable post nusinersen therapy. Ability to thrive at age 1.5 years was higher in children treated in early infancy with OA compared to historical controls. Significant heterogeneity existed across study population characteristics and outcome measures. Nusinersen may prevent deterioration in some nutrition outcomes; and OA in early infancy may be associated with improved nutrition outcomes. Timing of DMT initiation is an important consideration for future nutrition research. Studies investigating nutrition as a primary outcome of DMT, using consistent outcome measures are required for nutritional management strategies for this cohort to be appropriately tailored.
Objectives:1) Incorporate knowledge into clinical practice regarding the complex interaction between disease-modifying therapies for spinal muscular atrophy and potential changes in body composition, feeding route, and swallowing dysfunction; 2) Understand the impact of the disease-modifying therapy nusinersen on nutritional outcomes in patients with spinal muscular atrophy and consider this information when developing management plans; 3) Understand the impact of the disease-modifying therapy onasemnogene abeparvovec on nutritional outcomes in patients with spinal muscular atrophy and consider this information when developing management plans.
ACCREDITATION STATEMENT The AANEM is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
CREDIT DESIGNATION The AANEM designates this enduring material for a maximum of 1
AMA PRA Category 1 Credits TM. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
DISCLOSURE INFORMATIONDr. Zach Simmons (editor) has no conflicts of interest. Relevant financial relationships of the article’s authors have been disclosed and managed through the journal’s editorial review process.
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Katie O'Brien MSc; Kay Nguo PhD; Eppie M. Yiu PhD; Ian R. Woodcock MSc; Natassja Billich PhD; Zoe E. Davidson PhD