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Open‐labelled study to monitor the effect of an amino acid formula on symptom management in children with spinal muscular atrophy type I: The SMAAF pilot study

Bowerman, Melissa

Open‐labelled study to monitor the effect of an amino acid formula on symptom management in children with spinal muscular atrophy type I: The SMAAF pilot study Thumbnail


Authors



Abstract

Background: An increasing number of families of spinal muscular atrophy (SMA) children are incorporating an amino acid-based enteral formula into their child’s feeding regimens. Other components of the amino acidbased formula include added carbohydrates, electrolytes, and probiotics. Due to insufficient evidenced based research regarding the efficacy of the formula clinicians are unable to prescribe or endorse. The aim was to assess the tolerability and safety of an adapted version of the traditional SMA amino acid formula (SMAAF) in children with SMA Type I.

Methods: Children with SMA Type I were recruited if exclusively enterally fed and experiencing at least one gastrointestinal symptom (reflux, vomiting, constipation and/ or diarrhoea). Children were transferred on to an amino acid formula (Neocate® Syneo™- Nutricia) for eight weeks. Primary outcome was feed tolerance, measured weekly by telephone consultation to monitor gastrointestinal tolerance.

Results: Fourteen children were recruited, mean age 4.1 years (1.2SD). The mean resting energy expenditure measured by indirect calorimetry was 51kcal/ kg (7SD). The most common gastrointestinal complaint prior to switching to SMAAF was constipation (n=12). Ten of the 12 children who reported constipation symptoms were prescribed stool softeners/laxatives daily and after 8 weeks on SMAAF, 8 of these 10 (80%) children reduced or discontinue stool softener/ laxatives. Parents reported an improvement with reflux symptoms in 4 out of 5 children whilst on SMAAF.

Conclusion: Our pilot study suggests that children with SMA Type I who are displaying gastrointestinal symptoms, such as constipation and reflux may benefit from an amino acid formula.

Citation

Bowerman, M., O'Connor, G., Edel, L., Raquq, S., Szmurlo, A., Simpson, Z., …Baranello, G. (2023). Open‐labelled study to monitor the effect of an amino acid formula on symptom management in children with spinal muscular atrophy type I: The SMAAF pilot study. Nutrition in Clinical Practice, 38(4), 871-880. https://doi.org/10.1002/ncp.10940

Journal Article Type Article
Acceptance Date Oct 11, 2022
Online Publication Date Dec 12, 2022
Publication Date 2023-08
Publicly Available Date Dec 13, 2023
Journal Nutrition in Clinical Practice
Print ISSN 0884-5336
Publisher SAGE Publications
Volume 38
Issue 4
Pages 871-880
DOI https://doi.org/10.1002/ncp.10940
Keywords Nutrition and Dietetics, Medicine (miscellaneous)
Publisher URL https://aspenjournals.onlinelibrary.wiley.com/doi/10.1002/ncp.10940