Use of waxy maize heat modified starch in the treatment of children between 2 and 5 years with glycogen storage disease type I: A retrospective study

Ghada Hijazi; Nisha Pai; Laura L Nagy; Sarah Herd; Jolynn Dickson; Maya Ram; Michal Inbar-Feigenberg

doi:10.1016/j.ymgmr.2019.100536

Use of waxy maize heat modified starch in the treatment of children between 2 and 5 years with glycogen storage disease type I: A retrospective study

Mol Genet Metab Rep. 2019 Nov 6:21:100536. doi: 10.1016/j.ymgmr.2019.100536. eCollection 2019 Dec.

Authors

Ghada Hijazi¹, Nisha Pai², Laura L Nagy², Sarah Herd², Jolynn Dickson², Maya Ram², Michal Inbar-Feigenberg¹

Affiliations

¹ Division of Clinical and Metabolic Genetics, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
² Division of Clinical and Metabolic Genetics, the Department of Clinical Dietetics, The Hospital for Sick Children, Toronto, ON, Canada.

Abstract

Background: Glycogen storage disease type I (GSDI) is caused by deficiency of the enzyme glucose-6-phosphatase or glucose-6-phosphate transporter. Mainstay of treatment is provision of uncooked cornstarch (and/or continuous nocturnal pump feed (CNPF) to maintain normoglycemia). Waxy maize heat modified starch (WMHMS) is another treatment option to maintain normoglycemia overnight. Our objective was to describe our experience treating children 2-5 years of age with GSDI using WMHMS overnight.

Method: This is a retrospective case series review (n = 5) comparing the overnight feeding regimen and biochemical control one year before and after nocturnal WMHMS therapy. The WMHMS trial, in which blood glucose and lactate levels were monitored hourly, is reported in detail.

Results: Most patients successfully transitioned to nocturnal WMHMS feeds. These patients had stable glucose and lactate throughout the overnight period, permitting a fasting period of 6.5-8 h overnight. Within the time period studied, WMHMS appeared to have improved overnight control of blood glucose levels with fewer reported episodes of hypoglycemia compared to CNPF.

Conclusion: WMHMS can be an effective substitute treatment to achieve stable nocturnal glucose control in children younger than five years of age. A larger multicenter prospective study is recommended to establish stronger evidence of the efficacy and safety of using WMHMS in treatment of young children with GSDI.

Keywords: ALT, alanine aminotransferase; AST, aspartate aminotransferase; BMI, body mass index; CNPF, continuous nocturnal pump feed; Continuous nocturnal pump feed (CNPF).; GI, gastrointestinal; GSD, Glycogen storage disease; Glycogen storage disease (GSD); TGs, triglycerides; UCCS, uncooked cornstarch; Uncooked cornstarch (UCCS); WMHMS, waxy maize heat modified starch; Waxy maize heat modified starch (WMHMS); abdominal US, abdominal ultrasound; cm, centimeter; hrs, hours; kg, kilogram.