Related Experiment Video

Updated: Dec 28, 2025

Application of Laparoscopic Ultrasonography in Primary Choledochal Suture during Combined Two-lens Surgery
04:03

Application of Laparoscopic Ultrasonography in Primary Choledochal Suture during Combined Two-lens Surgery

Published on: March 28, 2025

405

Ursodeoxycholic acid use is associated with significant risk of morbidity and mortality in infants with cholestasis:

Magd Ahmed Kotb1, Dalia Mosallam1, Christine William Shaker Basanti1

  • 1Pediatrics Department, Faculty of Medicine, Kasr Alainy, Cairo University.

Medicine
|February 13, 2020
PubMed

Insights

Off-label ursodeoxycholic acid (UDCA) use in infants with cholestasis was ineffective and unsafe. UDCA treatment worsened outcomes, increased complications, and reduced cure rates, suggesting prohibition of its off-label use in this population.

Area of Science:

  • Pediatric Hepatology
  • Neonatal and Infant Health
  • Pharmacology and Therapeutics

Background:

  • Pediatric drug trials are limited, leading to frequent off-label medication use by clinicians.
  • Off-label prescribing is common in pediatrics due to insufficient safety and efficacy data for children.
  • Physicians often rely on clinical judgment for pediatric medication use.

Purpose of the Study:

  • To evaluate the safety and effectiveness of off-label ursodeoxycholic acid (UDCA) in neonates and infants with cholestasis.
  • To determine the impact of UDCA on treatment outcomes and complications in pediatric cholestasis.
  • To provide evidence regarding the prohibition of off-label UDCA use in young children.

Main Methods:

  • Retrospective study of 779 neonates and infants with cholestasis treated between 2005-2015.
  • Analysis of data from patients attending the Pediatric Hepatology Unit, Cairo University.
  • Comparison of outcomes between a group receiving off-label UDCA (15-30 mg/kg/d) and a control group.

Main Results:

  • Patients receiving UDCA experienced worse outcomes and significantly more complications (P < .001).
  • Cure rates were lower in the UDCA group (45.8%) compared to the no-UDCA group (73.1%) (P < .001).
  • Off-label UDCA use was associated with increased morbidity, disease progression, and mortality.

Conclusions:

  • Off-label ursodeoxycholic acid (UDCA) is ineffective and unsafe for treating cholestasis in Egyptian neonates and infants.
  • UDCA treatment compromises cure rates and is linked to severe adverse events, including death.
  • The off-label use of UDCA in this pediatric population should be prohibited, with data collection in registries.

More Related Videos

Laparoscopic Choledochal Cyst Excision and Roux-en-Y Choledochojejunostomy in Adults04:14

Laparoscopic Choledochal Cyst Excision and Roux-en-Y Choledochojejunostomy in Adults

Published on: February 28, 2025

592

Related Concept Videos

Drug Dosing: Infants and Children01:29

Drug Dosing: Infants and Children

Pediatric patient dosages diverge from adults due to disparities in body surface area, total body water, and extracellular fluid per kilogram of body weight. The dosing regimen considers the variations in pharmacokinetics and pharmacology across distinct age groups, encompassing preterm newborns, infants, young children, older children, and adolescents. Calculation of pediatric patient doses is predicated on determining body surface area, which exhibits a superior correlation with the child's...
190
Drugs for Treatment of Constipation-Predominant IBS01:21

Drugs for Treatment of Constipation-Predominant IBS

Pharmacological therapies for IBS-C are designed to alleviate abdominal discomfort and enhance bowel function. In patients with IBS-C, fiber supplements may help soften stools and decrease straining, but may also lead to increased gas production and bloating. Osmotic laxatives like milk of magnesia are frequently used to soften stools and increase stool frequency in IBS-C patients. In addition, two drugs approved for use in severe IBS-C adult cases are linaclotide (Linzess) and lubiprostone...
627
Drugs for Treatment of Diarrhea-Predominant IBS01:17

Drugs for Treatment of Diarrhea-Predominant IBS

Diarrhea-predominant irritable bowel syndrome (IBS-D) is a subtype of IBS characterized primarily by frequent, loose, or watery stools, abdominal pain, and abdominal discomfort. Therapeutic approaches to managing IBS-D include dietary changes, stress management techniques, and pharmaceutical interventions.
Two specific drugs used in the treatment are alosetron (Lotronex) and eluxadoline (Viberzi). Alosetron, a 5-HT3 antagonist, works by slowing the movement of stools in the gut, reducing bowel...
549
Drugs Affecting GI Tract Motility: Opioids as Antidiarrheal Agents01:17

Drugs Affecting GI Tract Motility: Opioids as Antidiarrheal Agents

Diarrhea, a condition marked by frequent loose or watery bowel movements, can be triggered by multiple factors such as viral or bacterial infections, food intolerances, anxiety, medications, and digestive disorders. Symptoms may include abdominal pain, bloating, nausea, and cramping. Severe or prolonged diarrhea can lead to complications like electrolyte imbalances, malnutrition, and dehydration if left untreated.
Opioids, widely used antidiarrheal agents, mitigate diarrhea by slowing down...
558
Drugs for Treatment of Ulcerative Colitis in IBD01:29

Drugs for Treatment of Ulcerative Colitis in IBD

Ulcerative colitis is a chronic inflammatory condition primarily affecting the colon and rectum. The primary drugs used in the treatment of ulcerative colitis are aminosalicylates. They exhibit anti-inflammatory and immunosuppressive properties. They modulate inflammatory mediators and inhibit the activity of nuclear factor κB (NF-κB). Aminosalicylates also reduce inflammation by inhibiting prostaglandin and leukotriene production and decreasing neutrophil chemotaxis and superoxide...
384
Chronic Bowel Disorders: Introduction01:17

Chronic Bowel Disorders: Introduction

Chronic bowel diseases are a group of long-term conditions affecting the digestive tract, characterized by inflammation and damage to the gut lining. These conditions primarily include irritable bowel syndrome and inflammatory bowel disease.
Irritable Bowel Syndrome (IBS) is a common disorder affecting the gastrointestinal tract. The distinctive feature is recurrent abdominal pain associated with altered bowel movements, manifesting as constipation, diarrhea, or fluctuating between both. The...
699