Effect of an oral Shiga toxin-binding agent on diarrhea-associated hemolytic uremic syndrome in children: a randomized controlled trial.
BACKGROUND
Diarrhea-associated hemolytic uremic syndrome (HUS) is the most common cause of acute renal failure in children. Most cases are caused by an intestinal infection with Shiga toxin-producing strains of Escherichia coli.


OBJECTIVE
To determine if administration of an oral agent that binds Shiga toxin could diminish the severity of diarrhea-associated HUS in pediatric patients.


METHODS
Multicenter, randomized, double-blind, placebo-controlled clinical trial of 145 children (96 experimental and 49 placebo) aged 6 months to 18 years with diarrhea-associated HUS conducted between July 27, 1997, and April 14, 2001, at 26 tertiary care pediatric nephrology centers in the United States and Canada. Trial included 2 phases, the hospital course for treatment of the acute illness and a 60-day outpatient follow-up period after discharge from the hospital.


METHODS
Patients were assigned to receive the binding agent, 500 mg/kg daily, or cornmeal placebo orally for 7 days in a 2:1 randomization scheme.


METHODS
Combined frequency of death or serious extrarenal events and need for dialysis in the experimental vs placebo group.


RESULTS
A total of 62 patients (43%) were male and 123 (85%) were white. The median age of the patients was 4.2 years. Most patients (59%) were transferred from other hospitals to participating sites. The severity of disease at the time of randomization was comparable in the 2 groups. The prevalence of death or serious extrarenal events was 18% and 20% in the experimental and placebo groups, respectively (P =.82). Dialysis was required in 42% of experimental and 39% of placebo groups (P =.86).


CONCLUSIONS
Oral therapy with a Shiga toxin-binding agent failed to diminish the severity of disease in pediatric patients with diarrhea-associated HUS.
Trachtman
Howard
H
Department of Pediatrics, Schneider Children's Hospital of the North Shore-Long Island Jewish Medical Center, New Hyde Park, NY 11040, USA. trachtma@lij.edu
Cnaan
Avital
A
Christen
Erica
E
Gibbs
Kathleen
K
Zhao
Sanyi
S
Acheson
David W K
DW
Weiss
Robert
R
Kaskel
Frederick J
FJ
Spitzer
Adrian
A
Hirschman
Gladys H
GH
D000284:Administration, Oral; D000293:Adolescent; D000553:Ambulatory Care; D002648:Child; D002675:Child, Preschool; D003967:Diarrhea / Q000188:drug therapy* / Q000209:etiology* / Q000382:microbiology; D004311:Double-Blind Method; D005243:Feces / Q000737:chemistry / Q000382:microbiology; D005260:Female; D006463:Hemolytic-Uremic Syndrome / Q000150:complications* / Q000188:drug therapy* / Q000382:microbiology; D006760:Hospitalization; D006801:Humans; D007223:Infant; D008297:Male; D017646:Organosilicon Compounds / Q000008:administration & dosage / Q000627:therapeutic use*; D022603:Shiga Toxins / Q000235:genetics / Q000378:metabolism; D014312:Trisaccharides / Q000008:administration & dosage / Q000627:therapeutic use*
D017646:Organosilicon Compounds; C119238:SYNSORB Pk; D022603:Shiga Toxins; D014312:Trisaccharides