Updating probability of pathogenicity for RYR1 and CACNA1S exon variants in individuals without malignant hyperthermia after exposure to triggering anesthetics

Pharmacogenet Genomics. 2025 Feb 1;35(2):65-72. doi: 10.1097/FPC.0000000000000551. Epub 2025 Jan 2.

Abstract

Objectives: We aimed to classify genetic variants in RYR1 and CACNA1S associated with malignant hyperthermia using biobank genotyping data in patients exposed to triggering anesthetics without malignant hyperthermia phenotype.

Methods: We identified individuals who underwent surgery and were exposed to triggering anesthetics without malignant hyperthermia phenotype and who had RYR1 or CACNA1S genotyping data available in our biobank. We classified all variants in the cohort using a Bayesian framework of the American College of Medical Genetics and Genomics and the Association of Molecular Pathologists guidelines for variant classification and updated the posterior probabilities from this model with the new information from our biobank cohort.

Results: We identified 253 patients with 95 RYR1 variants and 12 CACNA1S variants. After applying a Bayesian framework, we classified 17 variants as benign (B), 31 as likely benign (LB), 57 as uncertain (VUS), and 2 as likely pathogenic (LP). When we incorporated evidence about unique exposures to malignant hyperthermia triggering anesthetic agents, 48 of 107 (45%) variants were downgraded (9 to B, 37 to LB, and 2 to VUS). Notably, 41 (72%) of 57 VUSs were downgraded to B or LB. When repeat anesthetics in the same individual were counted as one exposure, 42 of 107 (39%) of variants were downgraded (5 to B, 35 to LB, and 2 to VUS). Specifically, 37 (65%) of 57 VUSs were downgraded to LB.

Conclusion: Deidentified biorepositories linked with anesthetic data offer a new method of integrating clinical evidence into the assessment of variant probability of pathogenicity.

MeSH terms

  • Adult
  • Aged
  • Anesthetics* / adverse effects
  • Bayes Theorem*
  • Calcium Channels, L-Type* / genetics
  • Exons* / genetics
  • Female
  • Genetic Variation
  • Genotype
  • Humans
  • Male
  • Malignant Hyperthermia* / genetics
  • Middle Aged
  • Ryanodine Receptor Calcium Release Channel* / genetics

Substances

  • Ryanodine Receptor Calcium Release Channel
  • CACNA1S protein, human
  • RYR1 protein, human
  • Calcium Channels, L-Type
  • Anesthetics