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IEMbase 0116: STAR-related steroidogenic acute regulatory protein deficiency

Scope

Field Value
IEMbase ID 116
Nosology 24.2.06.01
Gene STAR
External IDs OMIM:201710; ORPHA:314376
Generated mapping UNMAPPED
Candidate DisMech targets Congenital_Adrenal_Hyperplasia.yaml#Lipoid CAH
Review date 2026-07-07

IEMbase phenotype signal

IEMbase represents this as STAR-related steroidogenic acute regulatory protein deficiency, with alternate labels lipoid adrenal hyperplasia and StAR deficiency. Treatability is marked unknown.

The characteristic biochemical rows show primary steroidogenic failure: increased plasma ACTH, hyperkalemia, hyponatremia, and decreased steroids in plasma and urine. Clinical rows include alkalosis, ambiguous genitalia or female external genitalia in 46,XY individuals, and delayed puberty. Treatments listed are glucocorticoids and mineralocorticoids.

DisMech phenotype coverage

The generated unmapped status is a false negative. Congenital_Adrenal_Hyperplasia.yaml has a Lipoid CAH subtype described as severe STAR-related steroidogenesis disorder impairing cholesterol delivery for adrenal and gonadal steroid hormone synthesis. The entry includes STAR pathogenic variants and places lipoid CAH in the broader CAH phenotype frame of adrenal insufficiency, salt-wasting electrolyte crisis, ambiguous genitalia/virilization, and glucocorticoid plus mineralocorticoid replacement.

DisMech currently has only broad CAH biochemical rows, mainly reduced cortisol and elevated 17-hydroxyprogesterone for 21-hydroxylase deficiency. It does not yet expose STAR-specific low steroid profiles or ACTH/potassium/sodium patterns as discrete biochemical markers.

Concordance and completeness

Judgement: false negative to existing subtype-level local coverage.

The local CAH entry is concordant for STAR/lipoid CAH and for adrenal insufficiency with replacement therapy. IEMbase is more granular for the STAR-specific biochemical signature and for 46,XY undervirilization/female external genitalia and delayed puberty. DisMech's broad ambiguous-genitalia row is not a perfect match because STAR deficiency is a sex-steroid-deficiency phenotype rather than the androgen-excess virilization branch that dominates 21-hydroxylase CAH.

Curation actions

  • Resolve to Congenital_Adrenal_Hyperplasia.yaml#Lipoid CAH.
  • Add or review subtype-specific STAR biochemical markers: ACTH, sodium, potassium, and global steroid deficiency.
  • Consider more precise sex-development phenotypes for 46,XY undervirilization in lipoid CAH rather than reusing the 46,XX virilization framing.