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Invertase is an effective and cost-efficient alternative to sacrosidase oral solution for sucrase deficiency: a
Jennifer Morris1, Eve Moreland1, David I F Wands2
1School of Medicine, University of Glasgow, Glasgow, UK.
Insights
Invertase offers an effective and affordable treatment for paediatric sucrase deficiency, showing significant symptom improvement and cost savings compared to sacrosidase. Further research is recommended for optimal strategies.
Area of Science:
- Gastroenterology
- Paediatric Medicine
- Biochemistry
Background:
- Disaccharidase deficiencies, particularly sucrase deficiency, impact paediatric health.
- Sacrosidase oral solution is a current treatment option for sucrase deficiency.
- Understanding the prevalence of these deficiencies in tertiary care is crucial.
Purpose of the Study:
- To evaluate invertase as a cost-effective alternative to sacrosidase for paediatric sucrase deficiency.
- To assess the prevalence of various disaccharidase deficiencies in a tertiary care setting.
Main Methods:
- Retrospective analysis of duodenal biopsy and enzyme assay results from paediatric patients (2011-2019).
- Inclusion criteria: low disaccharidase activity, confirmed biopsy, normal histology.
- Efficacy assessment of invertase based on clinical outcomes and symptom improvement, with cost analysis.
Main Results:
- 38% of patients exhibited low disaccharidase activity; sucrase, maltase, and lactase deficiencies were noted in 19.4%, 25.0%, and 29.9% respectively.
- Invertase treatment in nine sucrase-deficient patients led to symptom resolution or improvement in 89% (diarrhoea) and 88% (overall symptoms).
- Invertase treatment cost was substantially lower (£1972/year) than sacrosidase oral solution (£51,938/year).
Conclusions:
- Invertase presents a viable, cost-efficient alternative to sacrosidase for treating paediatric sucrase deficiency.
- The study underscores the need for prospective research on disaccharidase deficiency prevalence and treatment optimization.
Objectives:
This study aims to evaluate the effectiveness of invertase as an alternative to sacrosidase solution for treating sucrase deficiency in paediatric patients and assess the prevalence of disaccharidase deficiencies in a tertiary care setting.
Design:
A single-centre, retrospective study was conducted at the Royal Hospital for Children, Glasgow. Patients with low disaccharidase enzyme assay results, confirmed by duodenal biopsy and with normal duodenal histology, taken between 01 January 2011 and 31 August 2019, were included. Data collected included patient demographics, symptomatology, laboratory results and outcomes. The efficacy of invertase supplementation was assessed based on global clinician assessment and improvements in diarrhoea. An estimated treatment cost of invertase versus Sucraid (sacrosidase oral solution) is provided.
Results:
Of 268 biopsy results from 245 patients, 38% showed low disaccharidase activity, with combinations of sucrase, maltase and lactase deficiencies observed in 19.4%, 25.0% and 29.9% of cases, respectively. Invertase was administered to nine patients with low sucrase. Following invertase treatment, 4/9 (44%) of patients experienced symptom resolution, 4/9 (44%) had a partial response and 1/9 (11%) had no response. Diarrhoea resolved or significantly improved in 8/9 (89%) of patients. The cost of invertase treatment was significantly lower than sacrosidase oral solution, with an estimated yearly cost of £1972 compared with £51 938 for sacrosidase oral solution.
Conclusions:
Invertase is an effective and cost-efficient alternative to sacrosidase for treating sucrase deficiency in paediatric patients. The study highlights the need for further prospective research to establish the prevalence of disaccharidase deficiencies and to optimise treatment strategies.
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