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

BACKGROUND 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. OBJECTIVE To determine efficacy and safety of a new uncooked starch (WMHMS) compared to UCCS over 16 weeks treatment with each. METHOD A 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. RESULTS 4 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. CONCLUSION WMHMS leads to significant reduction in insulin release and reduced starch use in some GSD patients.