Biochemistry · Nucleotide Metabolism and Disorders (Purine/Pyrimidine, Gout, Lesch-Nyhan, ADA-SCID)

A 64-year-old woman with newly diagnosed colorectal cancer receives standard-dose fluorouracil and within days develops severe mucositis, diarrhea, neutropenia, and neurotoxicity far beyond expected toxicity. Genotyping shows homozygous loss-of-function variants in DPYD. Which enzyme activity is absent, and what does it normally do?

  • A Thymidylate synthase, which methylates dUMP to dTMP
  • B Dihydropyrimidine dehydrogenase, which reduces fluorouracil to dihydrofluorouracil in the first catabolic step
  • C Orotate phosphoribosyltransferase, which converts orotate to OMP
  • D Thymidine phosphorylase, which cleaves thymidine to thymine
Correct answer: B. Dihydropyrimidine dehydrogenase, which reduces fluorouracil to dihydrofluorouracil in the first catabolic step

Explanation

More than 80 percent of administered fluorouracil is degraded by dihydropyrimidine dehydrogenase, the rate-limiting first enzyme of pyrimidine catabolism. DPYD loss-of-function abolishes this clearance, causing profound drug exposure and life-threatening toxicity. Testing for DPYD variants before fluorouracil dosing is now standard practice.

Reference: Katzung's Basic and Clinical Pharmacology, 16th ed.

High-yield for: NEET PGINI-CETNExTFMGEUSMLEPLABMRCP

Written and medically reviewed by the StethoPrep medical team.

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