Reasoning
The VCEP makes PS4 applicable using point-based scoring for autosomal-dominant RASopathy cases, with full points for RASopathy phenotypes and reduced points for minimal phenotypic information. The available literature verifies variant observations in somatic lung cancer and melorheostosis, not an affected RASopathy cohort with a VCEP PS4 point assignment. PMID:29643386 reports p.K57N in affected bone from four melorheostosis patients and absence from matched unaffected bone and blood, but this is a somatic mosaic phenotype rather than the VCEP's RASopathy PS4 context; PMID:18632602 reports 2/93 and 2/207 lung tumors, and PMID:25351745 reports 20/26 MEK1-mutated lung cancers, without matched unaffected-control counts for this variant. These observations do not provide the VCEP-required PS4 point score, and the available counts cannot be converted into a qualifying VCEP PS4 assessment. The VCEP rule's case-control note states that OR/RR >5.0 with a confidence interval excluding 1.0 supports the original PS4 path, but no suitable matched case-control dataset for the VCEP disease context is available here.
VCEP rule
Strength adjustment using point-based scoring for autosomal dominant cases with phenotypic specifications: full points awarded with RASopathy phenotypes, reduced points applicable to cases with minimal phenotypic information (i.e. prenatal cases, cases with a clinical order of a RASopathy panel without clinical information, and cases with limited clinical information in other global tests (such as WES)). ... The prevalence of the variant in affected individuals is significantly increased compared to the prevalence in controls. Note 1: Relative risk (RR) or odds ratio (OR), as obtained from case-control studies, is >5.0 and the confidence interval around the estimate of RR or OR does not include 1.0. See manuscript for detailed guidance. Note 2: In instances of very rare variants where case-control studies may not reach statistical significance, the prior observation of the variant in multiple unrelated patients with the same phenotype, and its absence in controls, may be used as moderate level of evidence. ... ≥5 points. ... ≥3 points. ... ≥1 points.
Evidence considered
- The VCEP CSPEC specifies PS4 point-based scoring for autosomal-dominant cases with phenotypic specifications, full points for RASopathy phenotypes, and reduced points for minimal phenotypic information.
- PMID:18632602 reports the exact MEK1 c.171G>T/p.K57N somatic mutation in 2 of 93 initially analyzed lung tumors and 2 of 207 NSCLCs; no matched unaffected-control comparison for this variant is reported.
- PMID:25351745 reports K57N in 20 of 26 MEK1-mutated lung cancers (77%), with additional public-dataset and COSMIC observations; this is a cancer cohort denominator, not a matched RASopathy case-control comparison.
- PMID:29643386 reports p.K57N in affected bone from four melorheostosis patients at approximately 3–34% mutant allele frequency and absence from matched unaffected bone and blood; this is somatic mosaic disease evidence, not a VCEP RASopathy point assignment.
- The PS4 governing text states that relative risk or odds ratio from case-control studies is >5.0 with a confidence interval excluding 1.0, or that multiple unrelated patients with the same phenotype and absence in controls may support moderate evidence for very rare variants; the available evidence does not establish either qualifying VCEP path.
Still missing
- A VCEP PS4 point-based assessment for unrelated autosomal-dominant RASopathy cases with phenotype detail is unavailable.
- No suitable matched RASopathy case-control counts or OR/RR with a 95% confidence interval were identified.
Sources