Recent advances in craniopharyngioma pathophysiology and emerging therapeutic approaches
Craniopharyngiomas are rare intracranial tumors originating from the Rathke’s pouch, affecting the sellar and parasellar regions. Despite their benign nature, they cause significant morbidity and mortality due to their proximity to vital structures such as the optic pathways and the hypothalamic-pit...
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Frontiers Media S.A.
2025-05-01
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| Series: | Frontiers in Endocrinology |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fendo.2025.1562942/full |
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| author | Clariano Pires de Oliveira Neto Clariano Pires de Oliveira Neto Clariano Pires de Oliveira Neto Clariano Pires de Oliveira Neto Gilvan Cortês Nascimento Gilvan Cortês Nascimento Gilvan Cortês Nascimento Gilvan Cortês Nascimento Sabrina da Silva Pereira Damianse Sabrina da Silva Pereira Damianse Sabrina da Silva Pereira Damianse Manuel dos Santos Faria Manuel dos Santos Faria Manuel dos Santos Faria Manuel dos Santos Faria |
| author_facet | Clariano Pires de Oliveira Neto Clariano Pires de Oliveira Neto Clariano Pires de Oliveira Neto Clariano Pires de Oliveira Neto Gilvan Cortês Nascimento Gilvan Cortês Nascimento Gilvan Cortês Nascimento Gilvan Cortês Nascimento Sabrina da Silva Pereira Damianse Sabrina da Silva Pereira Damianse Sabrina da Silva Pereira Damianse Manuel dos Santos Faria Manuel dos Santos Faria Manuel dos Santos Faria Manuel dos Santos Faria |
| author_sort | Clariano Pires de Oliveira Neto |
| collection | DOAJ |
| description | Craniopharyngiomas are rare intracranial tumors originating from the Rathke’s pouch, affecting the sellar and parasellar regions. Despite their benign nature, they cause significant morbidity and mortality due to their proximity to vital structures such as the optic pathways and the hypothalamic-pituitary axis, resulting in endocrine, visual, neurological impairment, and hypothalamic syndrome. Classified into adamantinomatous (ACP) and papillary (PCP), these tumors differ in epidemiology, histology, and pathophysiology. ACP, the most common type, presents a bimodal peak incidence between 5 and 15 years of age and 45 and 60 years of age, while PCP is more restricted to adults. Traditional treatments such as surgery and radiotherapy face significant challenges, including high recurrence rates. Intracystic chemotherapy is used in monocystic ACP but with limited efficacy and adverse effects related to toxicity. Recent advances in molecular biology have introduced targeted therapies, such as BRAF and MEK inhibitors, which show potential benefits in craniopharyngioma patients, particularly in the PCP. For ACP, however, therapeutic outcomes remain limited despite advances in molecular understanding, including mutations in the CTNNB1 gene and growth factors. Increasing investigation into the inflammatory microenvironment and immune response of these tumors presents new therapeutic possibilities and promising alternatives for tumor control, such as the use of anti-IL-6R, anti-VEGF agents and immune checkpoints inhibitors. This review aims to synthesize advancements in the pathophysiology of craniopharyngiomas and explore emerging therapeutic implications, focusing on precision medicine approaches for the management of this challenging disease. |
| format | Article |
| id | doaj-art-dfa76ec26d1f455e912d9dcc4157a8fd |
| institution | OA Journals |
| issn | 1664-2392 |
| language | English |
| publishDate | 2025-05-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Endocrinology |
| spelling | doaj-art-dfa76ec26d1f455e912d9dcc4157a8fd2025-08-20T01:50:23ZengFrontiers Media S.A.Frontiers in Endocrinology1664-23922025-05-011610.3389/fendo.2025.15629421562942Recent advances in craniopharyngioma pathophysiology and emerging therapeutic approachesClariano Pires de Oliveira Neto0Clariano Pires de Oliveira Neto1Clariano Pires de Oliveira Neto2Clariano Pires de Oliveira Neto3Gilvan Cortês Nascimento4Gilvan Cortês Nascimento5Gilvan Cortês Nascimento6Gilvan Cortês Nascimento7Sabrina da Silva Pereira Damianse8Sabrina da Silva Pereira Damianse9Sabrina da Silva Pereira Damianse10Manuel dos Santos Faria11Manuel dos Santos Faria12Manuel dos Santos Faria13Manuel dos Santos Faria14Post-Graduate Program in Health Sciences (PPGCS), Federal University of Maranhão (UFMA), São Luis, BrazilService of Endocrinology, University Hospital of the Federal University of Maranhao (HUUFMA), São Luis, BrazilResearch Group in Clinical and Molecular Endocrinology and Metabology (ENDOCLIM), Federal University of Maranhão (UFMA), São Luis, BrazilClinical Research Center (CEPEC), University Hospital of the Federal University of Maranhao (HUUFMA), São Luís, BrazilService of Endocrinology, University Hospital of the Federal University of Maranhao (HUUFMA), São Luis, BrazilResearch Group in Clinical and Molecular Endocrinology and Metabology (ENDOCLIM), Federal University of Maranhão (UFMA), São Luis, BrazilClinical Research Center (CEPEC), University Hospital of the Federal University of Maranhao (HUUFMA), São Luís, BrazilDepartment of Medicine I, Federal University of Maranhao (UFMA), São Luis, BrazilService of Endocrinology, University Hospital of the Federal University of Maranhao (HUUFMA), São Luis, BrazilResearch Group in Clinical and Molecular Endocrinology and Metabology (ENDOCLIM), Federal University of Maranhão (UFMA), São Luis, BrazilClinical Research Center (CEPEC), University Hospital of the Federal University of Maranhao (HUUFMA), São Luís, BrazilPost-Graduate Program in Health Sciences (PPGCS), Federal University of Maranhão (UFMA), São Luis, BrazilService of Endocrinology, University Hospital of the Federal University of Maranhao (HUUFMA), São Luis, BrazilClinical Research Center (CEPEC), University Hospital of the Federal University of Maranhao (HUUFMA), São Luís, BrazilDepartment of Medicine I, Federal University of Maranhao (UFMA), São Luis, BrazilCraniopharyngiomas are rare intracranial tumors originating from the Rathke’s pouch, affecting the sellar and parasellar regions. Despite their benign nature, they cause significant morbidity and mortality due to their proximity to vital structures such as the optic pathways and the hypothalamic-pituitary axis, resulting in endocrine, visual, neurological impairment, and hypothalamic syndrome. Classified into adamantinomatous (ACP) and papillary (PCP), these tumors differ in epidemiology, histology, and pathophysiology. ACP, the most common type, presents a bimodal peak incidence between 5 and 15 years of age and 45 and 60 years of age, while PCP is more restricted to adults. Traditional treatments such as surgery and radiotherapy face significant challenges, including high recurrence rates. Intracystic chemotherapy is used in monocystic ACP but with limited efficacy and adverse effects related to toxicity. Recent advances in molecular biology have introduced targeted therapies, such as BRAF and MEK inhibitors, which show potential benefits in craniopharyngioma patients, particularly in the PCP. For ACP, however, therapeutic outcomes remain limited despite advances in molecular understanding, including mutations in the CTNNB1 gene and growth factors. Increasing investigation into the inflammatory microenvironment and immune response of these tumors presents new therapeutic possibilities and promising alternatives for tumor control, such as the use of anti-IL-6R, anti-VEGF agents and immune checkpoints inhibitors. This review aims to synthesize advancements in the pathophysiology of craniopharyngiomas and explore emerging therapeutic implications, focusing on precision medicine approaches for the management of this challenging disease.https://www.frontiersin.org/articles/10.3389/fendo.2025.1562942/fullcraniopharyngiomaadamantinomatous craniopharyngiomapapillary craniopharyngiomatarget therapiesprecision medicine |
| spellingShingle | Clariano Pires de Oliveira Neto Clariano Pires de Oliveira Neto Clariano Pires de Oliveira Neto Clariano Pires de Oliveira Neto Gilvan Cortês Nascimento Gilvan Cortês Nascimento Gilvan Cortês Nascimento Gilvan Cortês Nascimento Sabrina da Silva Pereira Damianse Sabrina da Silva Pereira Damianse Sabrina da Silva Pereira Damianse Manuel dos Santos Faria Manuel dos Santos Faria Manuel dos Santos Faria Manuel dos Santos Faria Recent advances in craniopharyngioma pathophysiology and emerging therapeutic approaches Frontiers in Endocrinology craniopharyngioma adamantinomatous craniopharyngioma papillary craniopharyngioma target therapies precision medicine |
| title | Recent advances in craniopharyngioma pathophysiology and emerging therapeutic approaches |
| title_full | Recent advances in craniopharyngioma pathophysiology and emerging therapeutic approaches |
| title_fullStr | Recent advances in craniopharyngioma pathophysiology and emerging therapeutic approaches |
| title_full_unstemmed | Recent advances in craniopharyngioma pathophysiology and emerging therapeutic approaches |
| title_short | Recent advances in craniopharyngioma pathophysiology and emerging therapeutic approaches |
| title_sort | recent advances in craniopharyngioma pathophysiology and emerging therapeutic approaches |
| topic | craniopharyngioma adamantinomatous craniopharyngioma papillary craniopharyngioma target therapies precision medicine |
| url | https://www.frontiersin.org/articles/10.3389/fendo.2025.1562942/full |
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