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IEMbase 0023: HGD-related homogentisic acid oxidase deficiency

Scope

Field Value
IEMbase ID 23
Nosology 1.4.05.01
Gene HGD
External IDs OMIM:203500
Generated mapping MAPPED by alias_exact:aku
Candidate DisMech targets Alkaptonuria.yaml
Review date 2026-07-07

IEMbase phenotype signal

IEMbase represents classic alkaptonuria. The characteristic clinical triad is adult-onset arthritis, ochronosis, and urine darkening on standing, with urine darkening marked across all age bands. The defining biochemical signal is markedly elevated urinary homogentisate.

Additional IEMbase clinical signals include lumbosacral disc degeneration, scleral pigmentation, general pigmentation, intrinsic dental staining, and adult aortic and mitral valvulitis. No specific treatment rows are present in this JSON record despite the disease-level treatability value being yes.

DisMech phenotype coverage

The generated mapping to Alkaptonuria.yaml is correct. DisMech covers HGD loss of function, homogentisic acid accumulation and oxidation, ochronotic connective tissue degeneration, dark urine, ochronosis, osteoarthritis/arthritis, arthralgia, joint stiffness/swelling/dislocation, back pain, intervertebral disc calcification, cartilage calcification/destruction, tendon rupture, Achilles tendon thickening, aortic and mitral valve calcification/stenosis, coronary calcification, nephrolithiasis, hearing abnormalities, visual/ocular involvement, scleral and corneal-limbal pigmentation, skin pigmentation, elevated urinary homogentisic acid, and reduced HGD activity. It also includes nitisinone therapy.

Concordance and completeness

Judgement: correct mapping and very high concordance. DisMech is substantially richer than IEMbase for musculoskeletal, cardiovascular, renal, ocular, and treatment coverage.

IEMbase contributes a few useful granularity checks: intrinsic dental staining, the explicit lumbosacral-disc wording, and inflammatory valvulitis labels. DisMech already covers the same major organ systems, but mostly as calcific or structural valve disease rather than valvulitis.

Curation actions

  • Keep the generated mapping.
  • Consider whether intrinsic dental staining merits a phenotype entry if supported by alkaptonuria evidence.
  • Review whether the adult valve phenotype should stay framed as calcification or whether inflammatory valvulitis wording is evidence-supported.