Abstract
Background: Glycogen storage disease type I (GSD I) causes severe hypoglycemia during periods of fasting since both glycogenolysis and gluconeogenesis are impaired. Primary treatment in North America consists of cornstarch therapy every 3–4 h. Waxy maize extended release cornstarch was introduced for maintaining overnight glucose concentrations, but no studies have assessed long-term safety and efficacy of the product.
Objective: To demonstrate the safety and efficacy of modified cornstarch in GSD I.
Design: An open-label overnight trial of extended release cornstarch was performed. Subjects with a successful trial (optimal metabolic control 2 or more hours longer than with traditional cornstarch) were given the option of continuing into the chronic observational phase. Subjects were assessed biochemically at baseline and after 12 months.
Results: Of the 106 subjects (93 GSD Ia/13 GSD Ib), efficacy was demonstrated in 82 patients (88%) with GSD Ia and 10 patients (77%) with GSD Ib. The success rate for extending fasting was 95% for females and 78% for males. Of the patients who entered the longitudinal phase, long-term data are available for 44 subjects. Mean duration of fasting on traditional cornstarch prior to study for the cohort was 4.1 and 7.8 h 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 appears to improve the quality of life of patients with GSD I without sacrificing metabolic control. Avoiding the overnight dose of cornstarch should enhance safety in this population.
Competing interests: None declared
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Abbreviations
- ALT:
-
Alanine aminotransferase
- AST:
-
Aspartate aminotransferase
- GSD:
-
Glycogen storage disease
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Acknowledgments
The authors would like to thank Connie L. Cauthen for her help organizing in-patient visits for the participants of the study and University of Florida Health Clinical Research Center in-patient nurses (Charles J. Church, Emma B. Labrador, Dorothy G. Nichols, and Elizabeth A. Potocik) for the great care that they provide. The authors also thank Dr. Monika Dambska for assisting with the editing of the revision.
This research was supported by philanthropic support provided by the Furtherance Fund and the following funds managed through the University of Florida Office of Development: Scott Miller GSD Program Fund, GSD Dream Fund, and Green Family Fund for GSD Research. This work was also supported in part by the NIH/NCATS Clinical and Translational Science Award UL1 TR000064 granted to the University of Florida.
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Katalin M. Ross, Laurie M. Brown, Michelle M. Corrado, Tayoot Chengsupanimit, Latravia M. Curry, Iris A. Ferrecchia, Laura Y. Porras, Justin T. Mathew, and David A. Weinstein declare that they have no conflict of interest.
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All procedures followed were in accordance with the ethical standards of the responsible committee on human experimentation (institutional and national) and with the Helsinki Declaration of 1975, as revised in 2000 (5). Informed consent was obtained from all patients for being included in the study.
Details of the Contributions of Individual Authors
Katalin Ross designed the research study, conducted the research, and contributed to the data collection, data analysis, writing of the manuscript, and approval of the final content of manuscript.
Laurie Brown conducted the research, data analysis, data collection, writing of the manuscript, and approval of the final content of manuscript.
Michele Corrado designed the research study and data analysis and contributed to the data collection, writing of the manuscript, and approval of the final content of manuscript.
Tayoot Chengsupanimit contributed with the data analysis, writing of the manuscript, and approval of the final content of manuscript.
Latravia Curry contributed to the data collection, data analysis, writing of the manuscript, and approval of the final content of manuscript.
Iris Ferrecchia contributed to the data collection, data analysis, writing of the manuscript, and approval of the final content of manuscript.
Laura Porras contributed to the data collection, data analysis, writing of the manuscript, and approval of the final content of manuscript.
Justin Mathew contributed with the statistical analysis, writing of the manuscript, and approval of the final content of manuscript.
David Weinstein designed the research study, conducted the research, and contributed to the data collection, data analysis, writing of the manuscript, and approval of the final content of manuscript.
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Ross, K.M. et al. (2015). Safety and Efficacy of Chronic Extended Release Cornstarch Therapy for Glycogen Storage Disease Type I. In: Morava, E., Baumgartner, M., Patterson, M., Rahman, S., Zschocke, J., Peters, V. (eds) JIMD Reports, Volume 26. JIMD Reports, vol 26. Springer, Berlin, Heidelberg. https://doi.org/10.1007/8904_2015_488
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DOI: https://doi.org/10.1007/8904_2015_488
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