Safety and Efficacy of Long-Term Use of Extended Release Cornstarch Therapy for Glycogen Storage Disease Types 0, III, VI, and IX

Authors

  • Katalin M. Ross Glycogen Storage Disease Program, Division of Pediatric Endocrinology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA
  • Laurie M. Brown Glycogen Storage Disease Program, Division of Pediatric Endocrinology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA
  • Michelle M. Corrado Glycogen Storage Disease Program, Division of Pediatric Endocrinology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA
  • Tayoot Chengsupanimit Glycogen Storage Disease Program, Division of Pediatric Endocrinology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA
  • Latravia M. Curry Glycogen Storage Disease Program, Division of Pediatric Endocrinology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA
  • Iris A. Ferrecchia Glycogen Storage Disease Program, Division of Pediatric Endocrinology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA
  • Laura Y. Porras Glycogen Storage Disease Program, Division of Pediatric Endocrinology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA
  • Justin T. Mathew Glycogen Storage Disease Program, Division of Pediatric Endocrinology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA
  • Monika Dambska Glycogen Storage Disease Program, Division of Pediatric Endocrinology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA
  • David A. Weinstein Glycogen Storage Disease Program, Division of Pediatric Endocrinology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA

DOI:

https://doi.org/10.6000/1929-5634.2015.04.04.5

Keywords:

, Glycogen storage disease, uncooked cornstarch, extended release cornstarch, ketotic hypoglycemia

Abstract

Background: Impaired glycogen release with fasting results in hypoglycemia in the glycogen storage diseases. A waxy-maize extended release cornstarch was introduced in the United States in 2012 to maintain glucose concentrations during the overnight period, but no studies have assessed the long-term safety and efficacy of this product in the ketotic forms of GSD.

Objective: To assess long-term safety and efficacy of modified cornstarch in patients with ketotic forms of GSD.

Design: An open label overnight trial of extended release cornstarch was performed. Subjects who had a successful trial (defined as optimal metabolic control lasting 2 or more hours more than with traditional cornstarch) were given the option of continuing into the long-term observational phase. Participants were assessed biochemically at baseline and after 12 months.

Results: A total of 16 subjects participated in the open label trial. Efficacy was demonstrated in 100% of the subjects with GSD 0, III, VI, and IX. Of the patients who entered the longitudinal phase, long-term data are available for all subjects. The mean duration of overnight fasting on traditional cornstarch prior to the study for the cohort was 4.9 hours and 9.6 hours on the extended release cornstarch (P < 0.001). All laboratory markers of metabolic control have remained stable in the chronically treated patients.

Conclusion: Extended release cornstarch dramatically prolongs the overnight fast duration, maximizes safety from hypoglycemic events, reduces the possibility of sleep deprivation, and improves the quality of life of patients by eliminating the need to awaken without fail for middle of the night therapy without sacrificing metabolic control.

References

Wolfsdorf JI, Weinstein DA. Glycogen storage diseases. Rev Endocr Metab Disord 2003; 4: 95-102. http://dx.doi.org/10.1023/A:1021831621210 DOI: https://doi.org/10.1023/A:1021831621210

Kishnani PS, Austin SL, Arn P, Bali DS, Boney A, Case LE, Chung WK, Desai DM, El-Gharbawy A, Haller R, et al. Glycogen storage disease type III diagnosis and management guidelines. Genet Med 2010; 12: 446-63. http://dx.doi.org/10.1097/GIM.0b013e3181e655b6 DOI: https://doi.org/10.1097/GIM.0b013e3181e655b6

Brown LM, Corrado MM, van der Ende RM, Derks TG, Chen MA, Siegel S, Hoyt K, Correia CE, Lumpkin C, Flanagan TB, et al. Evaluation of glycogen storage disease as a cause of ketotic hypoglycemia in children. J Inherit Metab Dis 2015; 38(3): 489-93. http://dx.doi.org/10.1007/s10545-014-9744-1 DOI: https://doi.org/10.1007/s10545-014-9744-1

Chen YT, Cornblath M, Sidbury JB, Cornstarch therapy in type I glycogen-storage disease. N Engl J Med 1984; 310: 171-5. http://dx.doi.org/10.1056/NEJM198401193100306 DOI: https://doi.org/10.1056/NEJM198401193100306

Correia CE, Bhattacharya K, Lee PJ, Shuster JJ, Theriaque DW, Shankar MN, Smit GP, Weinstein DA. Use of modified cornstarch therapy to extend fasting in glycogen storage disease types Ia and Ib. Am J Clin Nutr 2008; 88: 1272-6.

Ross KM, Brown LM, Corrado MM, Chengsupanimit T, Curry LM, Ferrecchia IA, Porras LY, Mathew JT, Weinstein DA. Safety and efficacy of chronic extended release cornstarch therapy for glycogen storage disease type I. J Inherit Metab Dis [Epub ahead of print].

Crigler JF Jr, Folkman J. Glycogen storage disease: new approaches to therapy. Ciba Found Symp 1977; 55: 331-51. DOI: https://doi.org/10.1002/9780470720363.ch17

Bhattacharya K, Orton RC, Qi X, Mundy H, Morley DW, Champion MP, Eaton S, Tester RF, Lee PJ. A novel starch for the treatment of glycogen storage disease. J Inherit Metab Dis 2007; 30: 350-7. http://dx.doi.org/10.1007/s10545-007-0479-0 DOI: https://doi.org/10.1007/s10545-007-0479-0

Tsilianidis LA, Fiske LM, Siegel S, Lumpkin C, Hoyt K, Wasserstein M, Weinstein DA. Aggressive therapy improves cirrhosis in glycogen storage disease type IX. Mol Genet Metab 2013; 109: 179-82. http://dx.doi.org/10.1016/j.ymgme.2013.03.009 DOI: https://doi.org/10.1016/j.ymgme.2013.03.009

Derks TG, Smit GPA. Dietary management in glycogen storage disease type III: what is the evidence? J Inherit Metab Dis 2015; 38(3): 545-50. http://dx.doi.org/10.1007/s10545-014-9756-x DOI: https://doi.org/10.1007/s10545-014-9756-x

Downloads

Published

2016-02-02

How to Cite

Ross, K. M., Brown, L. M., Corrado, M. M., Chengsupanimit, T., Curry, L. M., Ferrecchia, I. A., Porras, L. Y., Mathew, J. T., Dambska, M., & Weinstein, D. A. (2016). Safety and Efficacy of Long-Term Use of Extended Release Cornstarch Therapy for Glycogen Storage Disease Types 0, III, VI, and IX. Journal of Nutritional Therapeutics, 4(4), 137–142. https://doi.org/10.6000/1929-5634.2015.04.04.5

Issue

Section

General Articles