Long‐term cornstarch therapy in glycogen storage disease types I, Ib and III

The purpose of treatment of hepatic glycogen storage disease (GSD) is to prevent fasting hypoglycaemia and its clinical and metabolic consequences: growth retardation, hepatomegaly, metabolic acidosis, hyperuricaemia and hyperlipidaemia. This problem is particularly severe for patients with GSD type I (McKusick 23220) and for young patients with GSD type III (McKusick 23240). In recent years frequent day feedings associated with continuous intragastric infusion have been proposed as the treatment of choice (Burr et al., 1979; Fernandes et al., 1984b; Greene et al., 1980). Gastric drip night feeding is effective, but serious complications such as symptomatic hypoglycaemia and acidosis resulting from acute gastroenteritis, vomiting and delays in feeding are reported (Greene et al., 1980). The regimen requires monitoring by infusion pump and specific training for parents; furthermore, some patients have psychological problems in accepting it. Recently the use of uncooked cornstarch has been proposed as an alternative therapy for patients with GSD I when continuous intragastric feeding is impracticable (Chen et al., 1984; Sloan and Zipt, 1983; Smit et al., 1984), but long-term experiences are still not available. In this paper we report the results in 10 children with GSD I, Ib and III after one year of cornstarch therapy.