Main Research Findings

Alpelisib is a PI3K/AKT/mTOR pathway inhibitor that is used to treat a variety of diseases, including vascular anomalies, PIK3CA-related overgrowth spectrum (PROS), and certain types of cancer. 6 9 1 4 3 Alpelisib has shown to be effective in treating PROS, which is caused by activating mutations in the PIK3CA gene. 5 9 1 PROS affects many different tissues, including skin, bone, blood vessels, fat tissue, and connective tissue, so its symptoms can vary widely. 1 Alpelisib has been shown to reduce the frequency and severity of hypoglycemia associated with PROS. 5 In combination with fulvestrant, alpelisib has shown to improve progression-free survival in patients with metastatic hormone receptor-positive, HER2-negative breast cancer who harbor an activating PIK3CA mutation. 4 2 However, alpelisib can cause significant side effects, including hyperglycemia, rash, diarrhea, and vitiligo. 7 3 2

Benefits and Risks

Benefits Summary

Alpelisib has shown to be effective in treating PROS, which is caused by activating mutations in the PIK3CA gene. 5 9 1 Alpelisib has been shown to reduce the frequency and severity of hypoglycemia associated with PROS. 5 In combination with fulvestrant, alpelisib has shown to improve progression-free survival in patients with metastatic hormone receptor-positive, HER2-negative breast cancer who harbor an activating PIK3CA mutation. 4 2

Risks Summary

Alpelisib can cause significant side effects, including hyperglycemia, rash, diarrhea, and vitiligo. 7 3 2 Alpelisib interacts with CYP3A4 inducers and CYP2C9 substrates, so caution should be exercised when used in combination with these medications. 1

Comparison Between Studies

Similarities

Multiple studies have shown that alpelisib is effective in treating PROS. 5 9 1 Alpelisib has also been shown to improve progression-free survival in patients with metastatic hormone receptor-positive, HER2-negative breast cancer who harbor an activating PIK3CA mutation. 4 2

Differences

Each study has different dosages, treatment durations, and evaluation criteria. 5 9 1 4 2

Consistency and Contradictions

Multiple studies have shown that alpelisib is effective in treating PROS. 5 9 1 However, alpelisib can cause significant side effects, including hyperglycemia, rash, diarrhea, and vitiligo. 7 3 2 The severity of these side effects can vary from patient to patient. 2

Considerations for Real-World Application

Alpelisib is an effective drug for treating PROS, metastatic hormone receptor-positive, HER2-negative breast cancer, and other diseases. 5 9 1 4 2 However, it is important to consider the potential risks of alpelisib, including hyperglycemia, rash, diarrhea, and vitiligo. 7 3 2 Alpelisib interacts with CYP3A4 inducers and CYP2C9 substrates, so caution should be exercised when used in combination with these medications. 1

Limitations of Current Research

There is still limited research on alpelisib, and further research is needed to fully understand its long-term safety and effectiveness. 5 9 1 4 2 The severity of alpelisib's side effects can vary from patient to patient. 2

Future Research Directions

Further research is needed to understand the long-term safety and effectiveness of alpelisib, the mechanisms of its side effects, and its optimal dosage. 5 9 1 4 2 Research is also needed to develop strategies to maximize the benefits of alpelisib while minimizing its side effects. 7

Conclusion

Alpelisib is an effective drug for treating PROS, metastatic hormone receptor-positive, HER2-negative breast cancer, and other diseases. 5 9 1 4 2 However, it is important to consider the potential risks of alpelisib, including hyperglycemia, rash, diarrhea, and vitiligo. 7 3 2 It is essential to discuss the benefits and risks of alpelisib with your doctor and to be aware of the signs and symptoms of potential side effects. 7 3 2