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|Molecular Profile||Indication/Tumor Type||Response Type||Relevant Treatment Approaches||Therapy Name||Approval Status||Evidence Type||Efficacy Evidence||References|
|PIK3CA M1043V||Advanced Solid Tumor||sensitive||mTORC1 Inhibitor||Sirolimus||Preclinical||Actionable||In a preclinical study, Rapamune (sirolimus) inhibited proliferation in cultured cells expressing PIK3CA M1043V (PMID: 17376864).||17376864|
|PIK3CA M1043V||head and neck squamous cell carcinoma||sensitive||Celecoxib||Preclinical - Pdx||Actionable||In a preclinical study, Celebra (celecoxib) treatment reduced tumor growth rate in a patient-derived xenograft (PDX) model of head and neck squamous cell carcinoma harboring PIK3CA M1043V (PMID: 30683736).||30683736|
|PIK3CA M1043V||head and neck squamous cell carcinoma||sensitive||Sulindac||Preclinical - Pdx||Actionable||In a preclinical study, Clinoril (sulindac) treatment reduced tumor growth rate in patient-derived xenograft (PDX) models of head and neck squamous cell carcinoma harboring PIK3CA mutations, including PIK3CA M1043V (PMID: 30683736).||30683736|
|PIK3CA M1043V||head and neck squamous cell carcinoma||predicted - sensitive||PIK3CA inhibitor||Alpelisib||Preclinical - Pdx & cell culture||Actionable||In a preclinical study, treatment with Piqray (alpelisib) resulted in inhibition of proliferation and increased apoptosis in a head and neck squamous cell carcinoma (HNSCC) cell line expressing PIK3CA M1043V in culture and led to inhibition of tumor growth and decreased phosphorylated Akt levels in a patient-derived xenograft (PDX) model of HNSCC with PIK3CA M1043V (PMID: 34779417).||34779417|
|PIK3CA M1043V||histiocytosis||predicted - sensitive||PIK3CA inhibitor||Alpelisib||Case Reports/Case Series||Actionable||In a clinical case study, Piqray (alpelisib) treatment resulted in symptom improvement and a complete metabolic response lasting at least 9 months in a patient with Langerhans cell histiocytosis harboring PIK3CA M1043V (PMID: 37874915).||37874915|