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IEMbase 0048: SLC3A1-related cystinuria type A

Scope

Field Value
IEMbase ID 48
Nosology 1.11.04.01
Gene SLC3A1
External IDs OMIM:220100
Generated mapping MAPPED by identifier:OMIM:220100
Candidate DisMech targets Cystinuria.yaml
Review date 2026-07-07

IEMbase phenotype signal

IEMbase represents this as autosomal recessive SLC3A1-related cystinuria type A. The listed prevalence is 1:7,000 and treatability is marked yes, although no specific treatment rows are present in the cached record.

The biochemical pattern is the expected COLA transporter profile: markedly increased urinary arginine, cystine, lysine, and ornithine from infancy onward, with low-to-normal plasma arginine, cystine, lysine, and ornithine. The characteristic clinical feature is cystine urolithiasis, strongest in adulthood. Additional clinical features include hematuria, obstructive uropathy, chronic renal failure, and urinary infections.

DisMech phenotype coverage

The generated mapping to Cystinuria.yaml is correct. The local entry models cystinuria as an SLC3A1 or SLC7A9 proximal tubular cystine/dibasic amino acid transport disorder and explicitly includes cystinuria type A as the SLC3A1/rBAT subtype.

DisMech covers the SLC3A1 and SLC7A9 genetic causes, impaired b(0,+) cystine and dibasic amino acid transport, increased urinary cystine, arginine, lysine, and ornithine, cystine supersaturation, crystalluria, recurrent cystine nephrolithiasis, hematuria, recurrent urinary tract infections, renal insufficiency, hypertension, flank pain, nausea/vomiting, abnormal urinary odor, hypocitraturia, hypercalciuria, hyperuricosuria, stone composition and urinary cystine evaluation, and SLC3A1/SLC7A9 genetic testing.

DisMech is also much richer therapeutically: high fluid intake and dietary sodium/protein moderation, urinary alkalinization with potassium citrate, cystine-binding thiol drugs such as tiopronin or D-penicillamine, calculi removal for symptomatic or large stones, and investigational alpha-lipoic acid.

Concordance and completeness

Judgement: correct mapping and high concordance. If a subtype-level target is available in downstream tooling, the best target is Cystinuria.yaml#Cystinuria type A; otherwise the file-level mapping is acceptable because type A is already represented inside the file.

IEMbase adds useful subtype-specific framing and plasma low-to-normal COLA amino acid values. DisMech adds the broader type A/type B structure, stone chemistry modifiers, diagnostic detail, chronic complication modeling, and treatment coverage.

Curation actions

  • Keep the generated mapping to Cystinuria.yaml.
  • Prefer subtype placement at Cystinuria.yaml#Cystinuria type A if the crosswalk later supports entity-level subtype anchors.
  • Consider adding IEMbase's plasma low-to-normal arginine/cystine/lysine/ ornithine detail if supported by citable evidence.
  • Do not import an empty IEMbase treatment block over the richer DisMech management content.