Genetic Information

Gene & Transcript Details

Gene
PIK3CA
Transcript
NM_006218.4 MANE Select
Total Exons
Reference Sequence
NC_000003.11
Alternative Transcripts
IDStatusDetails
NM_006218.2 Alternative 3724 nt | 158–3364
NM_006218.3 Alternative 9104 nt | 158–3364
NM_006218.4 MANE Select 9259 nt | 324–3530

Variant Details

HGVS Notation
NM_006218.4:c.3203dup
Protein Change
N1068Kfs*5
Location
Exon 21 (Exon 21 of )
21
5'Exon Structure3'
Functional Consequence
Loss of Function
Alternate Identifiers

Clinical & Population Data

Population Frequency

gnomAD
Global Frequency
0.0 in 100,000
Extremely Rare
ACMG Criteria Applied PM2
This variant is absent or extremely rare in population databases (PM2 criteria applies).

ClinVar

Open
Classification
Likely Pathogenic
1 publications
Clinical Statement

"This variant has been reported in ClinVar as Likely pathogenic (1 clinical laboratories)."

COSMIC Somatic Evidence

Open
COSMIC ID
COSM249879
Recurrence
28 occurrences
PM1 Criteria
Applied
COSMIC Database Preview
COSMIC Preview
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Functional Impact & Domains

Functional Domain

Hotspot Status
Not a hotspot
Domain Summary
This variant is not located in a mutational hotspot or critical domain.
Related Variants in This Domain
No evidence of other pathogenic variants at this position in gene PIK3CA.

Functional Studies & Therapeutic Relevance

Functional Summary

The PIK3CA N1068Kfs*5 variant has been functionally characterized and shown to have a damaging effect. This frameshift mutation results in increased cell proliferation, migration, and Akt phosphorylation compared to wild-type PIK3CA. It also enhances transformation ability in various cell lines, indicating an activating and likely oncogenic role.

Database Previews
OncoKB
OncoKB Preview
JAX-CKB
JAX-CKB Preview

Click on previews to view full database entries. External databases may require institutional access.

Computational Analysis

Pathogenicity Predictions

SpliceAI
Predictor Consensus
Unknown
PP3 Applied
No
REVEL Score
0.0
Threshold: ≥0.75 = PP3 applied

SpliceAI Scores

Window: ±500bp
Effect Type Score Position
- Acceptor Loss (AL) 0.0 None bp
- Donor Loss (DL) 0.0 None bp
+ Acceptor Gain (AG) 0.0 None bp
+ Donor Gain (DG) 0.0 None bp
High impact (≥0.5) Medium impact (0.2-0.49) Low impact (<0.2)

VCEP Guidelines

Applied ACMG/AMP Criteria (VCEP Specific)

Filter Criteria:
PVS1

PVS1 (Not Applied)

According to standard ACMG guidelines, the rule for PVS1 is: "Null variant (nonsense, frameshift, canonical ±1 or 2 splice sites) in a gene where loss of function is a known mechanism of disease." The evidence for this variant shows: PIK3CA loss of function is not an established disease mechanism and this frameshift variant functions via gain-of-function. Therefore, this criterion is not applied.

PS1

PS1 (Not Applied)

According to VCEP guidelines, the rule for PS1 is: "Strong strength: same amino acid change as an established pathogenic variant." The evidence for this variant shows: N1068Kfs*5 results in a novel frameshift not matching any known pathogenic amino acid change. Therefore, this criterion is not applied.

PS2

PS2 (Not Applied)

According to VCEP guidelines, the rule for PS2 is: "Strong: award if criteria 1 and 2 are fulfilled; Moderate: award if criteria 1 is fulfilled or if parents unavailable but criteria 2 fulfilled." The evidence for this variant shows: no de novo or somatic allele fraction data in parental and tissue samples. Therefore, this criterion is not applied.

PS3

PS3 (Strong)

According to VCEP guidelines, the rule for PS3 is: "Strong strength: functional assay meets acceptability criteria as per SVI and BMVCEP specifications (PMID:31892348)." The evidence for this variant shows: well-controlled in vitro assays demonstrate increased cell proliferation, migration, and Akt phosphorylation compared to wild-type, consistent with an activating effect. Therefore, this criterion is applied at Strong strength.

PS4

PS4 (Not Applied)

According to VCEP guidelines, the rule for PS4 is: "Assign points for phenotype if variant absent from controls (PM2), with ≥16 points for Very Strong, 3.5–15.75 for Strong, etc." The evidence for this variant shows: no reported phenotype case data available to assign points. Therefore, this criterion is not applied.

PM1

PM1 (Not Applied)

According to VCEP guidelines, the rule for PM1 is: "Supporting strength: residues affecting critical functional domains as provided in Table 4 for each gene." The evidence for this variant shows: position N1068 is not listed in the established PIK3CA functional domain hotspots. Therefore, this criterion is not applied.

PM2

PM2 (Supporting)

According to VCEP guidelines, the rule for PM2 is: "Supporting strength: absent or rare from controls in ethnically-matched cohort population samples." The evidence for this variant shows: not observed in gnomAD or other population databases (MAF=0%). Therefore, this criterion is applied at Supporting strength.

PM3

PM3 (Not Applied)

According to standard ACMG guidelines, the rule for PM3 is: "For recessive disorders, detected in trans with a pathogenic variant." The evidence for this variant shows: not relevant to a recessive inheritance context in PIK3CA. Therefore, this criterion is not applied.

PM4

PM4 (Not Applied)

According to standard ACMG guidelines, the rule for PM4 is: "Protein length changes due to in-frame deletions/insertions in a non-repeat region." The evidence for this variant shows: the variant is a frameshift, not an in-frame alteration. Therefore, this criterion is not applied.

PM5

PM5 (Not Applied)

According to VCEP guidelines, the rule for PM5 is: "Moderate strength: novel missense change at an amino acid residue where a different missense change is pathogenic." The evidence for this variant shows: it is a frameshift variant, not a missense change. Therefore, this criterion is not applied.

PM6

PM6 (Not Applied)

According to standard ACMG guidelines, the rule for PM6 is: "Assumed de novo without confirmation of paternity/maternity." The evidence for this variant shows: no de novo data provided. Therefore, this criterion is not applied.

PP1

PP1 (Not Applied)

According to standard ACMG guidelines, the rule for PP1 is: "Co-segregation with disease in multiple affected family members." The evidence for this variant shows: no segregation data available. Therefore, this criterion is not applied.

PP2

PP2 (Not Applied)

According to VCEP guidelines, the rule for PP2 is: "Supporting strength: missense constraint z-score >3.09 applies to PIK3CA." The evidence for this variant shows: variant is frameshift, not missense. Therefore, this criterion is not applied.

PP3

PP3 (Not Applied)

According to standard ACMG guidelines, the rule for PP3 is: "Multiple lines of computational evidence support a deleterious effect on the gene." The evidence for this variant shows: computational in silico tools were inconclusive. Therefore, this criterion is not applied.

PP4

PP4 (Not Applied)

According to standard ACMG guidelines, the rule for PP4 is: "Patient’s phenotype or family history is highly specific for a disease with a single genetic etiology." The evidence for this variant shows: no specific patient phenotype data provided. Therefore, this criterion is not applied.

PP5

PP5 (Not Applied)

According to standard ACMG guidelines, the rule for PP5 is: "Reputable source reports variant as pathogenic without accessible evidence." The evidence for this variant shows: ClinVar lists as Likely pathogenic, but PP5 is generally discouraged. Therefore, this criterion is not applied.

BA1

BA1 (Not Applied)

According to VCEP guidelines, the rule for BA1 is: "Stand Alone strength: allele frequency >0.0926%." The evidence for this variant shows: not present in population databases. Therefore, this criterion is not applied.

BS1

BS1 (Not Applied)

According to VCEP guidelines, the rule for BS1 is: "Strong strength: allele frequency >0.0185%." The evidence for this variant shows: not present in population databases. Therefore, this criterion is not applied.

BS2

BS2 (Not Applied)

According to VCEP guidelines, the rule for BS2 is: "Strong strength: ≥3 homozygotes or heterozygotes in well-phenotyped individuals." The evidence for this variant shows: not observed in gnomAD/clinical cohorts. Therefore, this criterion is not applied.

BS3

BS3 (Not Applied)

According to VCEP guidelines, the rule for BS3 is: "Strong strength: well-established functional studies show no damaging effect." The evidence for this variant shows: functional studies demonstrate damaging activating effect. Therefore, this criterion is not applied.

BS4

BS4 (Not Applied)

According to standard ACMG guidelines, the rule for BS4 is: "Lack of segregation in affected members of a family." The evidence for this variant shows: no segregation data available. Therefore, this criterion is not applied.

BP1

BP1 (Not Applied)

According to standard ACMG guidelines, the rule for BP1 is: "Missense variant in a gene for which only truncating variants cause disease." The evidence for this variant shows: disease mechanism is gain-of-function, and this is truncating. Therefore, this criterion is not applied.

BP2

BP2 (Not Applied)

According to standard ACMG guidelines, the rule for BP2 is: "Observed in trans with a known pathogenic variant in the same gene." The evidence for this variant shows: no data on cis/trans configuration. Therefore, this criterion is not applied.

BP3

BP3 (Not Applied)

According to standard ACMG guidelines, the rule for BP3 is: "In-frame deletions/insertions in a repetitive region without a known function." The evidence for this variant shows: frameshift variant, not an in-frame indel in a repetitive region. Therefore, this criterion is not applied.

BP4

BP4 (Not Applied)

According to VCEP guidelines, the rule for BP4 is: "Supporting strength: synonymous, intronic, or non-coding variants with two of three splice tools predicting no impact." The evidence for this variant shows: not a synonymous/intronic/UTR variant. Therefore, this criterion is not applied.

BP5

BP5 (Not Applied)

According to standard ACMG guidelines, the rule for BP5 is: "Variant found in a case with an alternate molecular basis for disease." The evidence for this variant shows: no such alternate molecular basis reported. Therefore, this criterion is not applied.

BP6

BP6 (Not Applied)

According to standard ACMG guidelines, the rule for BP6 is: "Reputable source reports benign classification without evidence." The evidence for this variant shows: no such benign classification exists. Therefore, this criterion is not applied.

BP7

BP7 (Not Applied)

According to VCEP guidelines, the rule for BP7 is: "Supporting strength: synonymous or intronic variants at non-conserved positions." The evidence for this variant shows: not a synonymous/intronic variant. Therefore, this criterion is not applied.