Landscape of TPMT and NUDT15 Pharmacogenetic Variation in a Cohort of Canadian Pediatric Inflammatory Bowel Disease Patients

Inflamm Bowel Dis. 2024 Dec 5;30(12):2418-2427. doi: 10.1093/ibd/izae109.

Abstract

Background: Patients with inflammatory bowel disease (IBD) exhibit considerable interindividual variability in medication response, highlighting the need for precision medicine approaches to optimize and tailor treatment. Pharmacogenetics (PGx) offers the ability to individualize dosing by examining genetic factors underlying the metabolism of medications such as thiopurines. Pharmacogenetic testing can identify individuals who may be at risk for thiopurine dose-dependent adverse reactions including myelosuppression. We aimed to evaluate PGx variation in genes supported by clinical guidelines that inform dosing of thiopurines and characterize differences in the distribution of actionable PGx variation among diverse ancestral groups.

Methods: Pharmacogenetic variation in TPMT and NUDT15 was captured by genome-wide genotyping of 1083 pediatric IBD patients from a diverse Canadian cohort. Genetic ancestry was inferred using principal component analysis. The proportion of PGx variation and associated metabolizer status phenotypes was compared across 5 genetic ancestral groups within the cohort (Admixed American, African, East Asian, European, and South Asian) and to prior global estimates from corresponding populations.

Results: Collectively, 11% of the cohort was categorized as intermediate or poor metabolizers of thiopurines, which would warrant a significant dose reduction or selection of alternate therapy. Clinically actionable variation in TPMT was more prevalent in participants of European and Admixed American/Latino ancestry (8.7% and 7.5%, respectively), whereas variation in NUDT15 was more prevalent in participants of East Asian and Admixed American/Latino ancestry (16% and 15% respectively).

Conclusions: These findings demonstrate the considerable interpopulation variability in PGx variation underlying thiopurine metabolism, which should be factored into testing diverse patient populations.

Keywords: NUDT15; TPMT; genetic ancestry; pharmacogenetics; thiopurines.

Plain language summary

In a large, pediatric inflammatory bowel disease cohort comprised of 5 genetic ancestry groups, we evaluated the distribution of loss-of-function pharmacogenetic variants in TPMT and NUDT15 and predicted phenotypes (impact on thiopurine metabolism).

MeSH terms

  • Adolescent
  • Azathioprine / adverse effects
  • Azathioprine / therapeutic use
  • Canada
  • Child
  • Child, Preschool
  • Cohort Studies
  • Colitis, Ulcerative / drug therapy
  • Colitis, Ulcerative / genetics
  • Crohn Disease / drug therapy
  • Crohn Disease / genetics
  • Female
  • Genotype
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Inflammatory Bowel Diseases / drug therapy
  • Inflammatory Bowel Diseases / genetics
  • Male
  • Mercaptopurine / therapeutic use
  • Methyltransferases* / genetics
  • Nudix Hydrolases
  • Pharmacogenetics
  • Pharmacogenomic Testing
  • Pharmacogenomic Variants
  • Pyrophosphatases* / genetics

Substances

  • NUDT15 protein, human
  • Pyrophosphatases
  • TPMT protein, human
  • Methyltransferases
  • Azathioprine
  • Mercaptopurine
  • Immunosuppressive Agents
  • thiopurine methyltransferase
  • Nudix Hydrolases

Supplementary concepts

  • Pediatric Crohn's disease