Reversibility of cirrhotic regenerative liver nodules upon NTBC treatment in a child with tyrosinaemia type I
Abstract
In a male patient with hereditary tyrosinaemia type I (HTI), NTBC [2‐(2‐nitro‐4‐trifluoro‐methylbenzoyl)‐1,3‐cyclohexandion] treatment and a diet low in phenylalanine and tyrosine were started at the age of 4 wk. At the recommended average dosage (1 mg kg−1), liver failure improved transiently. After 4 mo of treatment, with increased body weight, the dose had decreased to 0.7 mg kg−1, and diffuse cirrhotic changes in liver parenchyma and multiple nodules were visualized by ultrasonography. Multiple nodules in the liver parenchyma were differentiated from hepatocellular carcinoma by magnetic resonance imaging (MRI) using mangafodipir trisodium as a paramagnetic liver‐specific contrast agent. Augmentation of NTBC dosage resulted in a decrease in serum α‐fetoprotein levels and in significant regression of liver nodules on MRI. Conclusion: In HTI patients with a poor response to NTBC treatment and/or development of cirrhotic changes of liver parenchyma, augmentation of the recommended NTBC dosage may result in significant improvement of symptoms.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
Occasionally nodules are present before nitisinone treatment but regress on repeated imaging following treatment.
“Occasionally nodules are present before nitisinone treatment but regress on repeated imaging following treatment [38].”
From Recommendations for the management of tyrosinaemia type 1 (2013) · cites this paper as [38]
Cites
Treatment of hereditary tyrosinaemia type I by inhibition of 4-hydroxyphenylpyruvate dioxygenase
LINDSTEDT, S. (1992) · The Lancet · doi:10.1016/0140-6736(92)92685-9 · Cited by 631 · see citing works
Tyrosinaemia type I and NTBC (2‐(2‐nitro‐4‐trifluoromethylbenzoyl)‐1,3‐cyclohexanedione)
Holme, E., & Lindstedt, S. (1998) · Journal of Inherited Metabolic Disease · doi:10.1023/a:1005410820201 · Cited by 264 · see citing works
Self-citation
Flagged first, then by citations
Cited by
Sorted by citations
Recommendations for the management of tyrosinaemia type 1
de Laet, C., Dionisi-Vici, C., Leonard, J. V., McKiernan, P., Mitchell, G., Monti, L., de Baulny, H. O., Pintos-Morell, G., & Spiekerkötter, U. (2013) · Orphanet Journal of Rare Diseases · doi:10.1186/1750-1172-8-8 · Cited by 227 · see citing works
Experience of nitisinone for the pharmacological treatment of hereditary tyrosinaemia type 1
Santra, S., & Baumann, U. (2008) · Expert Opinion on Pharmacotherapy · doi:10.1517/14656566.9.7.1229 · Cited by 68 · see citing works
- Authors
- Crone, J., Möslinger, D., Bodamer, O., Schima, W., Huber, W., Holme, E., & Ipsiroglu, S. S.
- Affiliations
- University of Gothenburg (SE)
- University of Vienna (AT)
- Corresponding author
- Ipsiroglu, S Stöckler
- ORCID
- Schima, W
- Venue
- Acta Paediatrica
- Publisher
- Wiley
- Published
- 2003-05
- Volume
- 92
- Issue
- 5
- Pages
- 625-628
- ISSN
- 0365-1436 (ISSN-L)
- 0803-5253
- 1651-2227
- OpenAlex
- W2020253627
- Cited by
- 34
- References
- 2
- Citations by year
- 2026: 1 · 2022: 1 · 2021: 1 · 2020: 1 · 2019: 1 · 2018: 3 · 2015: 2 · 2014: 2 · 2013: 2 · 2012: 1
- FWCI
- 2.26
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Acta Paediatrica (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Health Sciences › Medicine › Surgery › Pediatric Hepatobiliary Diseases and Treatments
- Health Sciences › Medicine › Hepatology › Liver Disease and Transplantation
- Keywords
- NTBC, liver cirrhosis, alpha-fetoprotein, mangafodipir trisodium, liver MRI, phenylalanine-restricted diet, pediatric inborn errors of metabolism
- MeSH terms
- Cyclohexanones, Enzyme Inhibitors, Humans, Infant, Infant, Newborn, Liver Cirrhosis, Liver Regeneration, Male, Nitrobenzoates, Remission Induction, Tyrosinemias
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
