A NIS of Alpelisib in Combination With Fulvestrant in Postmenopausal Women, and Men, With HR+,HER2- , Locally Advanced or Metastatic Breast Cancer With a PIK3CA Mutation, After Disease Progression Following Endocrine Therapy as Monotherapy, in the Real-world Setting

Novartis Pharmaceuticals4 enrolled

Overview

This was a prospective, multi-national, non-interventional study (NIS) collecting data from postmenopausal women, and adult men, with HR+, HER2- locally advanced or metastatic breast cancer whose tumor harbors a PIK3CA mutation, and who were to be treated with alpelisib in combination with fulvestrant after disease progression following endocrine therapy as monotherapy, in the real-world setting.

Once the patient provided informed consent, he or she was enrolled in the study. Patients were planned to be followed from enrollment until 1) 30 days after alpelisib treatment discontinuation, or 2) death, or 3) lost to follow-up, or 4) patient withdrawal, or 5) physician decision to end treatment/study, or 6) end of the study, whichever occured first. The end of the study was defined as a maximum of 12 months after the date the last patient was enrolled (LPFV); if the last patient was still on treatment on that date, they were not be followed up any further. Due to very low patient numbers (4 patients, including 2 eligible patients) no statistical analyses were performed. Database lock was achieved without all queries resolved.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Hormone Receptor Positive HER2 Negative Breast Cancer With a PIK3CA Mutation

Interventions

Eligibility

Sex: ALLMin age: 18 YearsMax age: 100 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Signed informed consent from the patient or a legally acceptable representative, obtained before any study-related activities are undertaken * Patients diagnosed with HR+, HER2- locally advanced or metastatic breast cancer with a PIK3CA mutation * Patients who have disease progression following endocrine therapy as monotherapy * Patients must be postmenopausal women, or men, ≥18 years of age * Patients recruited on or before their first prescribed dose of alpelisib in combination with fulvestrant Exclusion Criteria: * Use of alpelisib prior to signing the informed consent form for this study * Participation in an interventional study within 30 days prior to the initiation of alpelisib

Outcomes

Primary Outcomes

Incidence proportion of hyperglycemia

To assess the incidence of hyperglycemia (Adverse Event of Special Iinterest) observed during follow-up of patients treated with alpelisib in combination with fulvestrant.

Time frame: Up to 53 months

Secondary Outcomes

Calculated BMI

Calculated BMI will be collected

Time frame: Baseline

Medical history

Number of patients with diabetes mellitus (including gestational diabetes), tobacco use, baseline diabetic status per laboratory values for HbA1c and FPG

Time frame: Baseline

Family history of diabetes mellitus

Yes/ No variable

Time frame: Baseline

Number of patients with concomitant medications known to affect blood glucose levels

Number of patients with concomitant medications known to affect blood glucose levels will be measured

Time frame: Baseline

Number of participants with incidence proportion of ketoacidosis and Hyperglycemic Hyperosmolar Non-Ketotic Syndrome (HHNKS)

Number of participants with incidence proportion of ketoacidosis and HHNKS based on AE data

Time frame: Up to 53 months

Incidence of the Adverse Events of Special Interest (AESI) of Osteonecrosis of the Jaw (ONJ)

Incidence of AESI of Osteonecrosis of the Jaw (ONJ) will be provided

Time frame: Up to 53 months

Number of participants with risk factors for Osteonecrosis of the Jaw (ONJ) observed

Number of participants with risk factors for ONJ will be collected. Risk factors for ONJ include: * Patient characteristics: age, calculated BMI, sex * Prior and/or concomitant use of bisphosphonates (e.g. zoledronic acid). * Prior and/or concomitant use of RANKligand inhibitors (e.g. denosumab).

Time frame: Baseline

Incidence proportion of AESIs

The incidence proportion of AESIs: * GI toxicity (nausea, vomiting and diarrhea) * Rash * Hypersensitivity (e.g. anaphylactic reaction) * Pancreatitis * Pneumonitis * SCARs

Time frame: Up to 53 months

Other safety and tolerability events

The incidence proportion of: * AEs * AEs leading to dose interruptions * AEs leading to dose reductions * AEs leading to permanent discontinuation of alpelisib in combination with fulvestrant * SAEs

Time frame: Up to 53 months

Number of patients with hematological and biochemical laboratory abnormalities

Number of patients with hematological and biochemical laboratory abnormalities will be provided

Time frame: Up to 53 months