We evaluated the effects of current therapy on the 24-h biochemical profile, the occurrence of significant hypoglycemia, and the long-term effects of CGT on physical growth and development, and the complications of GSD-1 in 17 patients (11 male), median age 14.2 (5.3 - 21.4) y.
CGT was started at a median age of 0.9 (0.1 - 1.6) y. At their most recent evaluation, subjects had received CGT for a median duration of 13.9 y (4.6 - 20.7 y) and uncooked cornstarch (UCS) for a median of 11 (3.5 - 12.9) y. Subjects followed their usual home dietary regimens with UCS supplements at 2- 4 h intervals during the day and at 4 - 8 h intervals during the night. Blood samples were obtained at hourly intervals for 24-h during which mean plasma glucose (PG) and blood lactate concentrations were >70 mg/dl and 2 - 4 mmol/L, respectively. From 24:00 - 08:00 h, 7 (41%), 10 (59%), and 13 (76%) subjects had PG ≥70, ≥60, and ≥50 mg/dl, respectively; 4 subjects had transient PG levels ≤50 mg/dl. The highest mean blood lactate levels (≥3 mmol/L) were between 02:00 - 09:00 h. Median height SDSCA was -0.8(-3.3 - 1.2), significantly (P = 0.002) less than median mid-parental height SDS of -0.1 (-2.1 - 1.4); median weight SDS was 0.1 (-1.3 - 3.3).
Six (35%) subjects (12.2 - 21.4 y) had anemia of chronic disease with hemoglobin concentrations of 9.8 - 11.6 g/dl. Ultrasound examination showed≥1 focal hepatic lesion consistent with an adenoma in 5 (29%) subjects(10.4 - 21.4 y); serum alpha-fetoprotein and carcinoembryonic antigen levels were normal in all subjects. Glomerular hyperfiltration was present in 16/17 subjects and urinary albumin excretion was increased (50 and 32 mg/24-h) in 2 subjects, 15.9 and 21.1 y, respectively.
Conclusion: near normal 24-h plasma glucose and blood lactate concentrations are attainable with UCS. Transient nocturnal hypoglycemia, however, was not completely prevented. No subject treated with UCS had a hypoglycemic seizure or loss of consciousness. Long-term CGT with UCS, begun in infancy, resulted in median height 0.7 SDS below target height. Renal glomerular dysfunction and formation of hepatic adenomata remain serious long-term complications.
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
Wolfsdorf, J., Crigler, J. Effect of Continuous Glucose Therapy (CGT) Begun in Infancy on the Long-Term Clinical Course of Patients with Type I Glycogen Storage Disease (GSD-I)† 502. Pediatr Res 43 (Suppl 4), 88 (1998). https://doi.org/10.1203/00006450-199804001-00523
Issue Date:
DOI: https://doi.org/10.1203/00006450-199804001-00523