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Table 5 Efficacy and safety data from phase Ib/II trials of alpelisib in HR+, HER2− aBC/mBC

From: Everolimus versus alpelisib in advanced hormone receptor-positive HER2-negative breast cancer: targeting different nodes of the PI3K/AKT/mTORC1 pathway with different clinical implications

StudyStudy designPopulationN° of pts.Previous CT allowedORRmPFS (mos)mOS (mos)Any grade AEs (%)G3/4 AEs (%)Discontinuation ratea
Juric et al. [30], phase Ib, open-label, single-armAlpelisib + Fulvb (300–350–400 mg/d)dPostmenopausal PIK3CA-mutated (60%) or PIK3CA-wt (38%) HR+, LABC/mBC progressing on/after prior ET87NSPIK3CA-mutated, 29%; PIK3CA-wt, 0%PIK3CA-mutated, 9.1; PIK3CA-wt, 4.7NADiarrhea 60%
Nausea 53%
Hyperglycemia 51%
Hyperglycemia 22%
Maculopapular rash 13%
Rash 8%
Mayer et al. [35], phase Ib, multicenter, open-labelAlpelisib + Let (300–350 + 2.5 mg/d)cPostmenopausal HR+, HER2− mBC progressing on/after prior ET26YesPIK3CA-mutated, 25%; PIK3CA-wt, 10%NANAAlpelisib 300 mg/d
Diarrhea 80%
Nausea 60%
Hyperglicemia 55%
Rash 45%
Fatigue 45%
Diarrhea 10%
Hyperglicemia 10%
AST/ALT elevation 5%
Rugo et al. [51], phase 2, open-label, non-comparative studyAlpelisib + Fulvbd (300 mg/d)c, Alpelisib + Letd (300 + 2.5 mg/d)Men and women with PIK3CA-mutated HR+, HER2− aBC whose disease progressed on/after CDK4/6i + ET21, 18Yes20%, 18%NANANAHyperglycemia 38.1% (Fulv)/27.8% (Let)
Rash 4.8% (Fulv)/27.8% (Let)
5%, 5%
  1. AEs adverse events, AST aspartate aminotransferase, ALT alanine aminotransferase, CDKi cyclin-dependent kinase inhibitors, CT chemotherapy, d day, ET endocrine therapy, Fulv fulvestrant, G grade, mos months, HR hormone receptor, (L)ABC (locally) advanced breast cancer, mBC metastatic breast cancer, mg/d milligrams per day, mPFS median progression-free survival, mOS median overall survival, N° number, NA not available, NS not specified, ORR overall response rate, pts. patients, wt wild type
  2. aStudy treatment discontinuation due to AEs
  3. bFulv 500 mg intramuscular injection on day 1, followed by 500 mg doses on days 15 and 28, and then every 28 days
  4. cRefers to alpelisib
  5. dFulv cohort: patients treated with prior CDKi and aromatase inhibitors; Let cohort: patients treated with prior CDKi and Fulv