A pilot longitudinal study of the use of waxy maize heat modified starch in the treatment of adults with glycogen storage disease type I: a randomized double-blind cross-over study.

Kaustuv Bhattacharya,Maggie F Lilburn,François Maillot,Helen Mundy,Michael P Champion,David W Morley

doi:10.1186/s13023-015-0229-6

Abstract

BackgroundUncooked corn-starch (UCCS) has been the mainstay of therapy for the hepatic glycogen storage diseases (GSD) but is not always effective. A new starch (WMHMS) has demonstrated a more favourable short-term metabolic profile.ObjectiveTo determine efficacy and safety of a new uncooked starch (WMHMS) compared to UCCS over 16 weeks treatment with each.MethodA double-blind cross-over study of 10 adults (aged 16 – 38 years, six male) with GSD Ia and Ib. After an individualised fast, subjects were randomised to take a 50 g starch-load of either WMHMS or UCCS. Starch-loads terminated when blood glucose was < 3.0 mmol/L or the subject felt subjectively hypoglycaemic. Anonymous biochemical profiles were assessed by 2 investigators and a starch administration schedule recommended. Each starch was delivered in coded sachets and intake was monitored for the following 16 weeks. After a washout period, the protocol was repeated with the alternative product.Results4 subjects failed to establish therapy on the cross-over limb. Data from 7 paired starch load showed: longer median fasting duration with WMHMS (7.5 versus 5 hours; p = 0.023), slower decrease in the glucose curve (0.357 versus 0.632 mmol/hr p = 0.028) and less area under insulin curves for the first 4 hours (p = 0.03). Two of six subjects took 50% or less WMHMS compared to UCCS and one took more. Plasma triglycerides, cholesterol and uric acid were unchanged after each study phase.ConclusionWMHMS leads to significant reduction in insulin release and reduced starch use in some GSD patients.

Highlights

Uncooked corn-starch (UCCS) has been the mainstay of therapy for the hepatic glycogen storage diseases (GSD) but is not always effective
WMHMS leads to significant reduction in insulin release and reduced starch use in some GSD patients
This study with a small number of patients with GSD Ia and Ib, demonstrated that WMHMS can be safely incorporated into the dietary regimen of these patients

Summary

Introduction

Uncooked corn-starch (UCCS) has been the mainstay of therapy for the hepatic glycogen storage diseases (GSD) but is not always effective. A new starch (WMHMS) has demonstrated a more favourable short-term metabolic profile. The glycogen storage diseases (GSDs) comprise a group of rare inherited disorders of glycogen metabolism. The subsequent introduction of uncooked corn-starch (UCCS) into the daily dietary treatment at least. Bhattacharya et al Orphanet Journal of Rare Diseases (2015) 10:18 matched this improvement and improved the quality of life of many patients [4,5]. Whilst the introduction of UCCS has benefited many, its use does have problems. UCCS is only partially utilized and can be associated with malabsorption in GSD I. Diarrhoea may be a feature of GSD I itself or its treatment with corn-starch, and inflammatory bowel disease is a feature of GSD Ib [8]

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