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Acute intermittent porphyria

Gene: HMBS

Green List (high evidence)

HMBS (hydroxymethylbilane synthase)
EnsemblGeneIds (GRCh38): ENSG00000256269
EnsemblGeneIds (GRCh37): ENSG00000256269
OMIM: 609806, Gene2Phenotype
HMBS is in 13 panels

4 reviews

Achchuthan Shanmugasundram (Genomics England Curator)

The mode of inheritance of this gene has been updated to BOTH monoallelic and biallelic (but BIALLELIC mutations cause a more SEVERE disease form), autosomal or pseudoautosomal following NHS Genomic Medicine Service approval.
Created: 10 Dec 2025, 1:56 p.m. | Last Modified: 10 Dec 2025, 1:56 p.m.
Panel Version: 1.5

Sarah Leigh (Genomics England Curator)

Based on review by Sharon Whatley (International Porphyria Network), should the mode of inheritance for this gene on this panel be changed from MONOALLELIC, autosomal or pseudoautosomal, imprinted status unknown to BOTH monoallelic and biallelic (but BIALLELIC mutations cause a more SEVERE disease form), autosomal or pseudoautosomal?
Created: 24 Apr 2025, 11:52 a.m. | Last Modified: 24 Apr 2025, 11:52 a.m.
Panel Version: 1.3

Mode of inheritance
BOTH monoallelic and biallelic (but BIALLELIC mutations cause a more SEVERE disease form), autosomal or pseudoautosomal

Sharon Whatley (International Porphyria Network)

Green List (high evidence)

Relevant metabolic investigation: urine porphobilinogen (to be completed before genetic testing for diagnosis of an acute porphyric attack)
PMID: 27539938 Chen reports that pathogenic HMBS allele frequency is high in the general population (1/1,782) and that the HMBS gene has very low penetrance (<1%) so that genetic testing alone may be misleading and cause misdiagnosis
PMID: 38940544 Aarsand IPNET advises that an acute attack of acute intermittent porphyria is diagnosed using a biochemical test for urine porphobilinogen as the penetrance of acute intermittent porphyria is so low. Genetic testing of the HMBS gene should only be used for family testing where penetrance is higher (10-20%) so that the patients can be advised against precipitating factors such as alcohol and certain drugs.
Careful consideration should be given to the reporting of a single pathogenic variant as an incidental finding in the HMBS gene, due to its low clinical penetrance.
PMID: 14262853 De Villeneuve, 1577472 Llewellyn, 15534187 Solis, 14970743 Hessels, 27558376 Kevelam, 31153822 Dixon and 34089223 Stutterd, reported the very rare finding of biallelic variants in the HMBS gene in six children (5 families) five children with severe progressive neurological disease and in six adults (3 families) with leukoencephalopathy but without confirmed attacks of porphyria.
Created: 4 Apr 2025, 11:40 a.m. | Last Modified: 4 Apr 2025, 11:40 a.m.
Panel Version: 1.1

Mode of inheritance
BOTH monoallelic and biallelic (but BIALLELIC mutations cause a more SEVERE disease form), autosomal or pseudoautosomal

Phenotypes
176000

Publications

Eleanor Williams (Genomics England Curator)

HMBS has been added to the panel for R169 Acute intermittent porphyria with a green rating as agreed with the NHS Genomic Medicine Service.
Created: 14 Jan 2023, 5:58 p.m. | Last Modified: 14 Jan 2023, 6:30 p.m.
Panel Version: 0.4

Mode of inheritance
MONOALLELIC, autosomal or pseudoautosomal, imprinted status unknown

Details

Mode of Inheritance
BOTH monoallelic and biallelic (but BIALLELIC mutations cause a more SEVERE disease form), autosomal or pseudoautosomal
Sources
  • NHS GMS
  • Expert Review Green
Phenotypes
  • Porphyria, acute intermittent, nonerythroid variant, OMIM:176000
  • Porphyria, acute intermittent, OMIM:176000
  • acute intermittent porphyria, MONDO:0008294
  • Encephalopathy, porphyria-related, OMIM:620704
  • encephalopathy, porphyria-related, MONDO:0958224
  • Leukoencephalopathy, porphyria-related, OMIM: 620711
OMIM
609806
Clinvar variants
Variants in HMBS
Penetrance
None
Publications
Panels with this gene

History Filter Activity

10 Dec 2025, Gel status: 3

Removed Tag

Achchuthan Shanmugasundram (Genomics England Curator)

Tag Q2_25_ MOI was removed from gene: HMBS.

10 Dec 2025, Gel status: 3

Set mode of inheritance

Achchuthan Shanmugasundram (Genomics England Curator)

Mode of inheritance for gene HMBS was changed from MONOALLELIC, autosomal or pseudoautosomal, imprinted status unknown to BOTH monoallelic and biallelic (but BIALLELIC mutations cause a more SEVERE disease form), autosomal or pseudoautosomal

2 Jul 2025, Gel status: 3

Removed Tag

Arina Puzriakova (Genomics England Curator)

Tag Q2_25_expert_review was removed from gene: HMBS.

24 Apr 2025, Gel status: 3

Added Tag, Added Tag

Sarah Leigh (Genomics England Curator)

Tag Q2_25_ MOI tag was added to gene: HMBS. Tag Q2_25_expert_review tag was added to gene: HMBS.

24 Apr 2025, Gel status: 3

Set Phenotypes

Sarah Leigh (Genomics England Curator)

Phenotypes for gene: HMBS were changed from to Porphyria, acute intermittent, nonerythroid variant, OMIM:176000; Porphyria, acute intermittent, OMIM:176000; acute intermittent porphyria, MONDO:0008294; Encephalopathy, porphyria-related, OMIM:620704; encephalopathy, porphyria-related, MONDO:0958224; Leukoencephalopathy, porphyria-related, OMIM: 620711

24 Apr 2025, Gel status: 3

Set publications

Sarah Leigh (Genomics England Curator)

Publications for gene: HMBS were set to

14 Jan 2023, Gel status: 3

Created, Added New Source, Set mode of inheritance

Eleanor Williams (Genomics England Curator)

gene: HMBS was added gene: HMBS was added to Acute intermittent porphyria. Sources: Expert Review Green,NHS GMS Mode of inheritance for gene: HMBS was set to MONOALLELIC, autosomal or pseudoautosomal, imprinted status unknown