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Targeted Therapy for Recurrent and Refractory Papillary Craniopharyngioma: A Systematic Narrative Review
1Department of Neurosurgery, Sanbo Brain Hospital, Capital Medical University, Beijing, China.
Objective:
To evaluate the efficacy and safety of targeted molecular therapy for recurrent and refractory papillary craniopharyngioma (PCP) and explore its potential as a neoadjuvant treatment.
Methods:
This systematic and narrative review was performed following PRISMA guidelines to evaluate BRAF and MEK inhibitors in treating PCP. PubMed, EMBASE, and Web of Science were searched up to July 2025. Inclusion criteria were English clinical studies of PCP patients treated with BRAF and/or MEK inhibitors. Outcomes included tumor response, survival, adverse events, and neoadjuvant therapy efficacy. Data were extracted and analyzed descriptively.
Results:
A total of 57 patients (mean age 47.74 ± 12.82 years; 64.9% male) were included. Tumors were classified as mixed (78.9%), solid (12.3%), and cystic (8.8%). Tumor volume reduction was most significant in mixed-type tumors, which showed the best therapeutic response. Nine patients used BRAF-MEK inhibitors as neoadjuvant therapy, resulting in an average tumor volume reduction of 88.16% within 6 months. Common adverse events included fever, rash, diarrhea, and elevated liver enzymes, leading to treatment discontinuation in 14.5% of patients. Among the 57 patients, 26 received early radiotherapy after targeted therapy. During mean follow-up of 14.41 months, most maintained stable disease, with better survival in mixed-type tumors. This represents the largest systematic analysis of PCP-targeted therapy with formal statistical validation (n = 57), demonstrating that tumor morphology significantly influences treatment response time (solid vs. mixed, P = 0.0117; cystic vsƒ. mixed, P = 0.0220).
Conclusions:
BRAF-MEK inhibitors show promising results in PCP treatment, particularly for tumor volume reduction. However, the high incidence of adverse events and limited long-term follow-up data necessitate careful patient monitoring and larger prospective studies to establish optimal treatment protocols before considering changes to standard treatment paradigms.
Insights
Targeted molecular therapy, including BRAF and MEK inhibitors, shows promise for treating papillary craniopharyngioma (PCP), especially in reducing tumor volume. However, careful monitoring is needed due to adverse events and limited long-term data.
Area of Science:
- Oncology
- Molecular Therapy
- Neurosurgery
Background:
- Papillary craniopharyngioma (PCP) is a rare brain tumor with limited treatment options for recurrent or refractory cases.
- Targeted molecular therapies, specifically BRAF and MEK inhibitors, have emerged as potential treatment modalities.
Purpose of the Study:
- To evaluate the efficacy and safety of targeted molecular therapy (BRAF and MEK inhibitors) for recurrent and refractory papillary craniopharyngioma (PCP).
- To explore the potential of these inhibitors as neoadjuvant treatment for PCP.
Main Methods:
- A systematic and narrative review adhering to PRISMA guidelines.
- Searched PubMed, EMBASE, and Web of Science for clinical studies of PCP patients treated with BRAF and/or MEK inhibitors.
- Analyzed outcomes including tumor response, survival, adverse events, and neoadjuvant therapy efficacy in 57 patients.
Main Results:
- Significant tumor volume reduction was observed, particularly in mixed-type tumors (78.9% of cases).
- Neoadjuvant BRAF-MEK inhibitor therapy resulted in an average tumor volume reduction of 88.16% within six months.
- Common adverse events (fever, rash, diarrhea) led to treatment discontinuation in 14.5% of patients; tumor morphology significantly influenced treatment response (p<0.05).
Conclusions:
- BRAF-MEK inhibitors demonstrate promising efficacy in reducing PCP tumor volume, especially in mixed-type tumors.
- High incidence of adverse events and limited long-term follow-up necessitate careful patient monitoring.
- Larger prospective studies are required to establish optimal treatment protocols for PCP.
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