- Center on Health Equity & Access
- Clinical
- Health Care Cost
- Health Care Delivery
- Insurance
- Policy
- Technology
- Value-Based Care
Examining Combination Therapy In NF1-PN
Panelists discuss the exploration of combination therapies in treating complex or refractory NF1-associated plexiform neurofibromas, including MEK inhibitors combined with PI3K inhibitors, anti-angiogenic agents, and chemotherapies, and review early clinical findings suggesting potential synergistic effects, while highlighting concerns about toxicity and the need for careful patient monitoring.
EP: 1.Genetic And Molecular Mechanisms of NF1
EP: 2.Diagnosis, Epidemiology, And Risk Factors Of NF1-PN
EP: 3.Hallmark Symptoms and Clinical Features of NF1-PN
EP: 4.Quality of Life in Patients With NF1-PN
EP: 5.Key Clinical Considerations in NF1-PN Management
EP: 6.Unmet Needs of Patients Living With NF1-PN
EP: 7.Main Goals in the Management of NF1-PN
EP: 8.Rationale for MEK Inhibitors and Surgery
EP: 9.Selumetinib as a Therapeutic Option for NF1-PN
EP: 10.Examining Combination Therapy In NF1-PN
Summary for Physicians
Combination Therapies in NF1-PN
For more complex or refractory neurofibromatosis type 1–associated plexiform neurofibromas (NF1-PN) cases, combination therapies are being explored to enhance therapeutic efficacy. Studies have investigated combinations of MEK inhibitors (eg, selumetinib) with other targeted agents, such as PI3K inhibitors, anti-angiogenic agents, and chemotherapies. The rationale behind these combinations is to target multiple pathways involved in tumor growth and progression, potentially improving response rates and reducing the risk of resistance.
Observed Results
Early clinical trials and preclinical studies suggest that combination therapies can result in synergistic effects, leading to better tumor shrinkage and symptom control compared to monotherapy. However, toxicity and adverse effects are a concern, especially with combinations that involve multiple agents, requiring careful patient monitoring.
