IEMbase 0661: UGT1A1-related UDP-glucuronosyltransferase A1 deficiency
Scope
| Field | Value |
|---|---|
| IEMbase ID | 661 |
| Nosology | 17.2.01.01 |
| Nosology code | IEM0802 |
| Gene | UGT1A1 |
| External IDs | OMIM:218800; OMIM:606785; ORPHA:205 |
| Generated mapping | MAPPED to Gilberts_Syndrome.yaml |
| Candidate DisMech targets | Gilberts_Syndrome.yaml covers mild Gilbert physiology only |
| Review date | 2026-07-07 |
IEMbase phenotype signal
IEMbase represents autosomal recessive UGT1A1-related UDP-glucuronosyltransferase A1 deficiency with both severe Crigler-Najjar syndrome and milder Gilbert syndrome named as alternate labels.
Clinical rows include persistent jaundice across all ages, neonatal temperature instability, abnormal eye movements, convulsions, and hearing loss. The convulsion row increases from optional neonatal involvement to strong childhood/adolescent involvement, consistent with bilirubin neurotoxicity scope. The biochemical row includes increased plasma bilirubin.
DisMech phenotype coverage
Gilberts_Syndrome.yaml is a correct target for the mild Gilbert end of the
UGT1A1 spectrum. It models reduced UGT1A1 activity, impaired bilirubin
glucuronidation, mild unconjugated hyperbilirubinemia, intermittent jaundice,
UGT1A128 and UGT1A16 genetics, and pharmacogenomic irinotecan relevance. It
also references Crigler-Najjar type II versus Gilbert bilirubin concentrations
in supporting evidence.
However, the local entry is explicitly a benign Gilbert syndrome entry. It does not model Crigler-Najjar syndrome as a disease entity, profound neonatal unconjugated hyperbilirubinemia, bilirubin encephalopathy/kernicterus, abnormal eye movements, seizures/convulsions, hearing loss, temperature instability, or Crigler-Najjar-specific treatment intensity such as phototherapy or liver transplantation.
Concordance and completeness
Judgement: partial mapping only. Accept Gilberts_Syndrome.yaml for the mild
Gilbert component, but do not treat it as complete coverage for the IEMbase
UGT1A1 deficiency spectrum.
The generated high-confidence alias match is understandable because IEMbase lists Gilbert syndrome, but the same row also names severe Crigler-Najjar syndrome. A single Gilbert entry under-covers the severe bilirubin-neurotoxicity phenotype package.
Curation actions
- Keep
Gilberts_Syndrome.yamlas the mild-spectrum target. - Add or map a separate Crigler-Najjar syndrome target if DisMech wants full UGT1A1 deficiency spectrum coverage.
- Preserve bilirubin, neonatal temperature instability, abnormal eye movements, convulsions/seizures, hearing loss, and persistent jaundice prompts.
- Avoid claiming full row-level completeness from the Gilbert mapping alone.