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IEMbase 0129: AR-related Androgen receptor deficiency

Scope

Field Value
IEMbase ID 129
Nosology 24.2.23.01
Gene AR
External IDs OMIM:300068; ORPHA:754
Generated mapping MAPPED, high confidence
Candidate DisMech targets Complete_Androgen_Insensitivity_Syndrome.yaml
Review date 2026-07-07

IEMbase phenotype signal

IEMbase represents this as AR-related androgen receptor deficiency, with alternate labels testicular feminization, androgen insensitivity syndrome, and AIS. Treatability is marked unknown.

The characteristic biochemical row is normal DHT. Clinical rows include absent axillary hair, absent pubic hair, and absent uterus. No treatment rows are listed.

DisMech phenotype coverage

Complete_Androgen_Insensitivity_Syndrome.yaml is the generated local target. It describes X-linked AR variants in 46,XY individuals with age-appropriate androgen production but target-tissue androgen resistance, leading to female external genitalia, absent or sparse pubic/axillary hair, primary amenorrhea, absent Mullerian structures, and undescended testes.

The local phenotype coverage includes absent uterus, sparse or absent axillary/pubic hair, primary amenorrhea, blind vagina, cryptorchidism, female external genitalia in 46,XY individuals, elevated LH, increased testosterone, increased estradiol, increased AMH, and germ-cell tumor risk. Treatments include individualized gonadectomy, estrogen replacement, and vaginal dilation.

Concordance and completeness

Judgement: correct current mapping, with a label-scope caveat.

The DisMech CAIS entry covers the specific IEMbase clinical signals of absent uterus and absent terminal sexual hair well. It is also much richer for AR mechanism, 46,XY phenotype, hormone profile, and management. IEMbase's normal DHT row is a useful reminder that AIS is androgen-resistance physiology rather than impaired DHT synthesis.

The IEMbase label "androgen receptor deficiency" and alternate "AIS" are broad; if separate partial or mild AIS records are curated later, they should not be forced into the complete AIS target without subtype review.

Curation actions

  • Keep Complete_Androgen_Insensitivity_Syndrome.yaml as the current target for this IEMbase record.
  • Consider adding normal DHT as a differentiating biochemical row, especially relative to SRD5A2 deficiency.
  • Preserve the broader AIS label-scope caveat for future subtype handling.