Evaluation of liquid yeast-derived sucrase enzyme replacement in patients with sucrase-isomaltase deficiency.
BACKGROUND
No enzyme replacement therapy exists for patients with congenital sucrase-isomaltase deficiency (CSID). A by-product of the manufacture of baker's yeast is a liquid preparation containing high sucrase activity. The aim of the present study was to investigate the activity and stability of this preparation and its effect on breath hydrogen excretion and gastrointestinal symptoms after sucrose ingestion in 14 patients with CSID.


METHODS
The homogeneity of yeast sucrase was studied by sodium dodecyl sulfate-polyacrylamide gel electrophoresis, and its activity was measured. Stability at various temperatures and pH ranges and in the presence of gastric aspirate, pepsin, and bovine serum albumin was assessed. Fourteen patients with CSID underwent double-blind placebo-controlled breath tests with yeast sucrase. They then completed an 8-week dose response study that used different enzyme concentrations while consuming a sucrose-containing diet.


RESULTS
Liquid yeast sucrase is highly glycosylated, contains no lactase activity, and is stable at 4 degrees C and over a wide range of pH. Pepsin digestion of the enzyme in vitro can be blunted by bovine serum albumin and by increasing the pH. Yeast sucrase reduces breath hydrogen excretion in patients with CSID who are given a sucrose load (P < 0.001) and allows most patients to consume a sucrose-containing diet.


CONCLUSIONS
Liquid yeast sucrase offers effective enzyme replacement therapy for patients with CSID.
Treem
W R
WR
Division of Pediatric Gastroenterology and Nutrition, Hartford Hospital, Connecticut.
Ahsan
N
N
Sullivan
B
B
Rossi
T
T
Holmes
R
R
Fitzgerald
J
J
Proujansky
R
R
Hyams
J
J
D000284:Administration, Oral; D000293:Adolescent; D000328:Adult; D000704:Analysis of Variance; D002239:Carbohydrate Metabolism, Inborn Errors / Q000188:drug therapy* / Q000378:metabolism; D002648:Child; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D004591:Electrophoresis, Polyacrylamide Gel; D005260:Female; D006801:Humans; D006859:Hydrogen / Q000032:analysis / Q000378:metabolism; D006863:Hydrogen-Ion Concentration; D007223:Infant; D008297:Male; D010434:Pepsin A / Q000494:pharmacology; D012441:Saccharomyces cerevisiae / Q000201:enzymology*; D012709:Serum Albumin / Q000494:pharmacology; D013393:Sucrase / Q000008:administration & dosage / Q000378:metabolism / Q000627:therapeutic use*; D013394:Sucrase-Isomaltase Complex / Q000172:deficiency*; D013395:Sucrose / Q000008:administration & dosage / Q000494:pharmacology; D013696:Temperature
D012709:Serum Albumin; D013395:Sucrose; D006859:Hydrogen; D013394:Sucrase-Isomaltase Complex; D013393:Sucrase; D010434:Pepsin A