Trial reportGastroenterology2016
In Children With Nonalcoholic Fatty Liver Disease, Cysteamine Bitartrate Delayed Release Improves Liver Enzymes but Does Not Reduce Disease Activity Scores.
Trial report in Gastroenterology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01529268 (Cysteamine Bitartrate Delayed-Release for the Treatment of Nonalcoholic Fatty Liver Disease), which is not on this map. Cited by 65 papers, 5 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Cysteamine Bitartrate Delayed-Release for the Treatment of Nonalcoholic Fatty Liver Disease (NAFLD) in Children (CyNCh)
Who cites it
65 citing papers in PubMed, 5 syntheses or guidelines pooled it, 124 citations in OpenAlex.
- Effect of weight loss interventions on liver-related health in children and adolescents with obesity: a systematic review and meta-analyses.International journal of obesity (2005) · 2026Pooled it
- Comparative efficacy of drugs with different mechanistic pathways/targets in the treatment of pediatric NAFLD: evidence from a Bayesian network meta-analysis.Frontiers in pharmacology · 2026Pooled it
- Efficacy of pharmacotherapies on pediatric patients with metabolic dysfunction-associated steatotic liver disease: a systematic review and network meta-analysis.BMC gastroenterology · 2025Pooled it
- Systematic review of exercise for the treatment of pediatric metabolic dysfunction-associated steatotic liver disease.PloS one · 2024Pooled it
- Childhood obesity, cardiovascular and liver health: a growing epidemic with age.World journal of pediatrics : WJP · 2020Pooled it
- An Open Label, Randomized, Multicenter Study of Elafibranor in Children With Nonalcoholic Steatohepatitis.Journal of pediatric gastroenterology and nutrition · 2023Trial
- Shorter leukocyte telomere length protects against NAFLD progression in children.Scientific reports · 2023Trial
- Randomized placebo-controlled trial of losartan for pediatric NAFLD.Hepatology (Baltimore, Md.) · 2022Trial
- Trial
- Diagnostic accuracy of magnetic resonance imaging hepatic proton density fat fraction in pediatric nonalcoholic fatty liver disease.Hepatology (Baltimore, Md.) · 2018Trial
- Metabolic dysfunction-associated steatotic liver disease-a review of the cardiometabolic perspectives and treatments in children.International journal of obesity (2005) · 2026Review
- European Society for Pediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) steatotic liver disease special interest group position paper on screening, diagnosis and investigation of paediatric metabolic dysfunction-associated steatotic liver disease.Journal of pediatric gastroenterology and nutrition · 2026Article
- Lifespan approaches for the prevention and management of steatotic liver disease.Nature reviews. Gastroenterology & hepatology · 2026Review
- MASLD In Children - A Distinct Phenotype?Current obesity reports · 2026Review
- Genetic and non-genetic drivers of histological progression and regression in MASLD.Journal of hepatology · 2026Article
- Hepatocyte-hepatic stellate cell interactions in liver fibrosis: Mechanisms and therapeutic implications.Hepatology communications · 2026Review
- Pediatric metabolic dysfunction-associated steatotic liver disease and the gut microbiome: from research landscape to targeted modulation.Clinical and molecular hepatology · 2026Review
- Longitudinal response to standard of care in pediatric metabolic dysfunction-associated steatotic liver disease: Rates of improvement and worsening, and factors associated with outcomes.Hepatology (Baltimore, Md.) · 2025Article
- Call to action-Pediatric MASLD requires immediate attention to curb health crisis.Hepatology (Baltimore, Md.) · 2025Review
- MASLD in children: integrating epidemiological trends with mechanistic and translational advances.The Journal of clinical investigation · 2025Review
5 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
21 authors at 14 institutions in 1 country.
Funding
Abstract
BACKGROUND &
aimsNo treatment for nonalcoholic fatty liver disease (NAFLD) has been approved by regulatory agencies. We performed a randomized controlled trial to determine whether 52 weeks of cysteamine bitartrate delayed release (CBDR) reduces the severity of liver disease in children with NAFLD.
methodsWe performed a double-masked trial of 169 children with NAFLD activity scores of 4 or higher at 10 centers. From June 2012 to January 2014, the patients were assigned randomly to receive CBDR or placebo twice daily (300 mg for patients weighing ≤65 kg, 375 mg for patients weighing >65 to 80 kg, and 450 mg for patients weighing >80 kg) for 52 weeks. The primary outcome from the intention-to-treat analysis was improvement in liver histology over 52 weeks, defined as a decrease in the NAFLD activity score of 2 points or more without worsening fibrosis; patients without biopsy specimens from week 52 (17 in the CBDR group and 6 in the placebo group) were considered nonresponders. We calculated the relative risks (RR) of improvement using a stratified Cochran-Mantel-Haenszel analysis.
resultsThere was no significant difference between groups in the primary outcome (28% of children in the CBDR group vs 22% in the placebo group; RR, 1.3; 95% confidence interval [CI], 0.8-2.1; P = .34). However, children receiving CBDR had significant changes in prespecified secondary outcomes: reduced mean levels of alanine aminotransferase (reduction, 53 ± 88 U/L vs 8 ± 77 U/L in the placebo group; P = .02) and aspartate aminotransferase (reduction, 31 ± 52 vs 4 ± 36 U/L in the placebo group; P = .008), and a larger proportion had reduced lobular inflammation (36% in the CBDR group vs 21% in the placebo group; RR, 1.8; 95% CI, 1.1-2.9; P = .03). In a post hoc analysis of children weighing 65 kg or less, those taking CBDR had a 4-fold better chance of histologic improvement (observed in 50% of children in the CBDR group vs 13% in the placebo group; RR, 4.0; 95% CI, 1.3-12.3; P = .005).
conclusionsIn a randomized trial, we found that 1 year of CBDR did not reduce overall histologic markers of NAFLD compared with placebo in children. Children receiving CBDR, however, had significant reductions in serum aminotransferase levels and lobular inflammation. ClinicalTrials.gov no: NCT01529268.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.