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IEMbase 0653: TPI1-related triosephosphate isomerase deficiency

Scope

Field Value
IEMbase ID 653
Nosology 3.3.09.01
Nosology code IEM0382
Gene TPI1
External IDs OMIM:615512; ORPHA:868
Generated mapping UNMAPPED; weak candidate Hereditary_Intrinsic_Factor_Deficiency.yaml
Candidate DisMech targets No exact local target
Review date 2026-07-07

IEMbase phenotype signal

IEMbase represents autosomal recessive TPI1-related triosephosphate isomerase deficiency, also labeled hemolytic anemia due to triosephosphate isomerase deficiency and hereditary nonspherocytic hemolytic anemia due to triosephosphate isomerase deficiency. Treatability is marked no.

Biochemical rows include decreased red-cell triosephosphate isomerase activity and increased red-cell dihydroxyacetone phosphate. Clinical rows include recurrent infections and hemolytic anemia, with optional cardiomyopathy, dystonia, progressive muscle weakness, seizures, stroke, tremor, and intellectual disability.

DisMech phenotype coverage

Hereditary_Intrinsic_Factor_Deficiency.yaml is a false candidate based on anemia wording. It models CBLIF/GIF-related cobalamin absorption failure, methylmalonic aciduria, hyperhomocysteinemia, megaloblastic anemia, and vitamin B12 replacement. It does not model glycolysis, TPI1, red-cell TPI activity, dihydroxyacetone phosphate, nonspherocytic hemolysis, or the neuromuscular and cardiac complications of TPI deficiency.

Local hemolytic-anemia entries and modules provide broad anemia context, but no TPI1-specific disease anchor was found.

Concordance and completeness

Judgement: true local TPI1 / triosephosphate isomerase deficiency gap.

The generated candidate should be rejected because the anemia mechanism is different: cobalamin-dependent megaloblastic erythropoiesis versus a glycolytic enzyme defect causing hemolytic anemia with systemic neurologic and muscular features.

Curation actions

  • Keep this row unmapped until a TPI1 / triosephosphate isomerase deficiency target exists.
  • Do not map to Hereditary_Intrinsic_Factor_Deficiency.yaml.
  • Preserve red-cell TPI activity, dihydroxyacetone phosphate, hemolytic anemia, recurrent infections, cardiomyopathy, dystonia, progressive weakness, seizures, stroke, tremor, and intellectual-disability prompts.