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- Publisher Website: 10.3389/fonc.2024.1433073
- Scopus: eid_2-s2.0-85199561869
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Article: Case report: Treatment response of NF-1-associated bladder ganglioneuroma to trametinib
| Title | Case report: Treatment response of NF-1-associated bladder ganglioneuroma to trametinib |
|---|---|
| Authors | |
| Keywords | ganglioneuroma MAPK pathway MEK inhibitor neurofibromatosis type 1 (NF-1) targeted therapy |
| Issue Date | 12-Jul-2024 |
| Publisher | Frontiers Media |
| Citation | Frontiers in Oncology, 2024, v. 14 How to Cite? |
| Abstract | We present the clinical course of a 4-year-old girl with neurofibromatosis type 1-associated, unresectable, symptomatic urinary bladder ganglioneuroma. She was initially trialed on sirolimus without response and subsequently responded to MEK inhibitor trametinib, with improvement clinically and radiographically over 10 months. This report broadens the repertoire of therapeutic strategies for MEK inhibition in diseases related to the MAPK pathway. |
| Persistent Identifier | http://hdl.handle.net/10722/350490 |
| DC Field | Value | Language |
|---|---|---|
| dc.contributor.author | Chan, Marcus CY | - |
| dc.contributor.author | Fung, Kevin KF | - |
| dc.contributor.author | Ng, Wai Fu | - |
| dc.contributor.author | Luk, Ho Ming | - |
| dc.contributor.author | Ku, Dennis TL | - |
| dc.contributor.author | Liu, Anthony PY | - |
| dc.date.accessioned | 2024-10-29T00:31:52Z | - |
| dc.date.available | 2024-10-29T00:31:52Z | - |
| dc.date.issued | 2024-07-12 | - |
| dc.identifier.citation | Frontiers in Oncology, 2024, v. 14 | - |
| dc.identifier.uri | http://hdl.handle.net/10722/350490 | - |
| dc.description.abstract | We present the clinical course of a 4-year-old girl with neurofibromatosis type 1-associated, unresectable, symptomatic urinary bladder ganglioneuroma. She was initially trialed on sirolimus without response and subsequently responded to MEK inhibitor trametinib, with improvement clinically and radiographically over 10 months. This report broadens the repertoire of therapeutic strategies for MEK inhibition in diseases related to the MAPK pathway. | - |
| dc.language | eng | - |
| dc.publisher | Frontiers Media | - |
| dc.relation.ispartof | Frontiers in Oncology | - |
| dc.rights | This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License. | - |
| dc.subject | ganglioneuroma | - |
| dc.subject | MAPK pathway | - |
| dc.subject | MEK inhibitor | - |
| dc.subject | neurofibromatosis type 1 (NF-1) | - |
| dc.subject | targeted therapy | - |
| dc.title | Case report: Treatment response of NF-1-associated bladder ganglioneuroma to trametinib | - |
| dc.type | Article | - |
| dc.identifier.doi | 10.3389/fonc.2024.1433073 | - |
| dc.identifier.scopus | eid_2-s2.0-85199561869 | - |
| dc.identifier.volume | 14 | - |
| dc.identifier.eissn | 2234-943X | - |
| dc.identifier.issnl | 2234-943X | - |