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United States Department of Agriculture

Agricultural Research Service

Research Project: CLINICAL NUTRITION IN CHILDREN Title: Congenital sucrase-isomaltase deficiency (CSID) in the era of sucraid

Authors
item Treem, W -
item Douglas, M -
item Duong, S -
item Ford, R -
item Hochman, J -
item Nichols, B -
item Teitelbaum, J -
item Nguyen, K -
item Xu, J -
item Gill, R -
item Schwarz, S -

Submitted to: Journal of Pediatric Gastroenterology and Nutrition
Publication Type: Abstract Only
Publication Acceptance Date: November 1, 2009
Publication Date: November 1, 2009
Citation: Treem, W.R., Douglas, M., Duong, S., Ford, R., Hochman, J., Nichols, B., Teitelbaum, J., Nguyen, K., Xu, J., Gill, R., Schwarz, S. 2009. Congenital sucrase-isomaltase deficiency (CSID) in the era of sucraid [abstract]. Journal of Pediatric Gastroenterology and Nutrition. 49(Suppl.1):E25.

Technical Abstract: Yeast-derived Sucraid was FDA-approved for CSID treatment in 2001. The aim of our study was to collect epidemiologic, natural history, and quality of life data in CSID patients on Sucraid. An anonymous questionnaire was mailed to 226 patients who took Sucraid within the last 5 years. Patients supplied data in collaboration with their treating physicians. Questionnaires from 57 patients (32 M) were returned. Symptoms began at < 1 yr of age in 79%, but only 28% were diagnosed by 1 yr. Median delay in diagnosis was 15 mo and 39% experienced > 2 yrs delay. The median age of starting Sucraid was 2.5 yr but 36% were >5 yr of age. Median duration of therapy was 3 yr (range 1 mo–17 yr) with 33% treated for > 5 yr. 55/57 patients continued Sucraid, with 1 discontinuing due to cost, and 1 because of poor response. 9/55 patients (16%) exceeded the maximum recommended dose in order to achieve efficacy. Two thirds taking Sucraid consumed either an unrestricted or a mild starch- and sucrose-restricted diet. However, 25% continued to require strict sucrose restriction even with Sucraid therapy. Only 4/55 patients experienced >3 bowel movements/d; 27% had diarrhea >1/wk and 38% had gas/bloating >1/wk. Associated symptoms while on Sucraid included 7/55 with sleep disturbances, 4/55 with constipation, and 3/55 with headaches. Parents rated the effect of CSID on their child’s overall health while on Sucraid as no effect in 45%; mild in 38%; moderate in 7%; and severe in none. In 8/57 families, other relatives were diagnosed after the index case including 5 siblings, 3 mothers, 3 grandparents, and 1 cousin. The diagnosis of CSID is delayed in a large number of patients even in families with a known index case. Once treated, the majority of patients consume a near-normal diet with minimal symptoms that do not significantly reduce quality of life, although 1/6 requires super therapeutic doses. One quarter still, require strict sucrose restriction to achieve symptomatic control even while treated with Sucraid.

Last Modified: 12/22/2014
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