Cargando…

Case report: rapid and durable response to PDGFR targeted therapy in a child with refractory multiple infantile myofibromatosis and a heterozygous germline mutation of the PDGFRB gene

BACKGROUND: Infantile myofibromatosis belongs to a family of soft tissue tumors. The majority of these tumors have benign behavior but resistant and malignant courses are known, namely in tumors with visceral involvement. The standard of care is surgical resection. Observations suggest that low dose...

Descripción completa

Detalles Bibliográficos
Autores principales:	Mudry, Peter, Slaby, Ondrej, Neradil, Jakub, Soukalova, Jana, Melicharkova, Kristyna, Rohleder, Ondrej, Jezova, Marta, Seehofnerova, Anna, Michu, Elleni, Veselska, Renata, Sterba, Jaroslav
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2017
Materias:	Case Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5301362/ https://www.ncbi.nlm.nih.gov/pubmed/28183292 http://dx.doi.org/10.1186/s12885-017-3115-x

Ejemplares similares

Effects of Sunitinib and Other Kinase Inhibitors on Cells Harboring a PDGFRB Mutation Associated with Infantile Myofibromatosis
por: Sramek, Martin, et al.
Publicado: (2018)

Novel PDGFRB rearrangement in multifocal infantile myofibromatosis is tumorigenic and sensitive to imatinib
por: Hassan, Mohammed, et al.
Publicado: (2019)

Infantile myofibromatosis
por: Larralde, Margarita, et al.
Publicado: (2017)

Improved osteosarcoma survival with addition of mifamurtide to conventional chemotherapy – Observational prospective single institution analysis
por: Múdry, Peter, et al.
Publicado: (2021)

Individualization of Treatment Improves the Survival of Children With High-Risk Solid Tumors: Comparative Patient Series Analysis in a Real-Life Scenario
por: Kyr, Michal, et al.
Publicado: (2019)

The infantile myofibromatosis NOTCH3 L1519P mutation leads to hyperactivated ligand-independent Notch signaling and increased PDGFRB expression
por: Wu, Dan, et al.
Publicado: (2021)

Assessment of Tumor Mutational Burden in Pediatric Tumors by Real-Life Whole-Exome Sequencing and In Silico Simulation of Targeted Gene Panels: How the Choice of Method Could Affect the Clinical Decision?
por: Noskova, Hana, et al.
Publicado: (2020)

Comprehensive Molecular Profiling for Relapsed/Refractory Pediatric Burkitt Lymphomas—Retrospective Analysis of Three Real-Life Clinical Cases—Addressing Issues on Randomization and Customization at the Bedside
por: Polaskova, Kristyna, et al.
Publicado: (2020)

Aggressive infantile myofibromatosis with intestinal involvement
por: Römer, Tristan, et al.
Publicado: (2021)

New Target for Precision Medicine Treatment of Giant-Cell Tumor of Bone: Sunitinib Is Effective in the Treatment of Neoplastic Stromal Cells with Activated PDGFRβ Signaling
por: Mahdal, Michal, et al.
Publicado: (2021)

Spontaneous Remission of Solitary-Type Infantile Myofibromatosis
por: Kikuchi, Kazuhiro, et al.
Publicado: (2011)

Targeted treatment of severe vascular malformations harboring PIK3CA and TEK mutations with alpelisib is highly effective with limited toxicity
por: Sterba, Martin, et al.
Publicado: (2023)

Solitary infantile myofibromatosis in the upper extremities: Case report
por: Najwa, Amsiguine, et al.
Publicado: (2023)

Minimal Residual Disease–Guided Intermittent Dosing in Patients With Cancer: Successful Treatment of Chemoresistant Anaplastic Large Cell Lymphoma Using Intermittent Lorlatinib Dosing
por: Pokorna, Petra, et al.
Publicado: (2022)

Non-DHFR-mediated effects of methotrexate in osteosarcoma cell lines: epigenetic alterations and enhanced cell differentiation
por: Sramek, Martin, et al.
Publicado: (2016)

Extracalvarial Composite Infantile Myofibromatosis: Case Report and Literature Review
por: Ivanov, Alexander, et al.
Publicado: (2016)

Phospho-Protein Arrays as Effective Tools for Screening Possible Targets for Kinase Inhibitors and Their Use in Precision Pediatric Oncology
por: Neradil, Jakub, et al.
Publicado: (2019)

NANOG/NANOGP8 Localizes at the Centrosome and is Spatiotemporally Associated with Centriole Maturation
por: Mikulenkova, Erika, et al.
Publicado: (2020)

Kidney transplantation in infantile myofibromatosis and fibromuscular dysplasia: a case report
por: Frezin, Julie, et al.
Publicado: (2015)

Diagnostic limitations and considerations in the imaging evaluation of advanced multicentric infantile myofibromatosis
por: Parikh, Abhinav, et al.
Publicado: (2020)

A Unique Case of Multicentric Infantile Myofibromatosis with Radiologic-Pathologic Correlation
por: Pušnik, Luka, et al.
Publicado: (2022)

Avoidance of surgery for head and neck infantile myofibromatosis using imatinib monotherapy
por: Pattisapu, Prasanth, et al.
Publicado: (2022)

Unusually Aggressive Myofibromatosis In A Neonate
por: Hazarey, Jitendra, et al.
Publicado: (2012)

CD133 Expression and Identification of CD133/nestin Positive Cells in Rhabdomyosarcomas and Rhabdomyosarcoma Cell Lines
por: Sana, Jiri, et al.
Publicado: (2011)

The ATRA-induced differentiation of medulloblastoma cells is enhanced with LOX/COX inhibitors: an analysis of gene expression
por: Chlapek, Petr, et al.
Publicado: (2014)

DHFR-mediated effects of methotrexate in medulloblastoma and osteosarcoma cells: The same outcome of treatment with different doses in sensitive cell lines
por: NERADIL, JAKUB, et al.
Publicado: (2015)

Prenatal genetic diagnosis of disseminated infantile myofibromatosis: a case report and literature review
por: Lü, Yan, et al.
Publicado: (2023)

Solitary infantile myofibromatosis in the bones of the upper extremities: Two rare cases and a review of the literature
por: WU, WEILIANG, et al.
Publicado: (2013)

Diagnosis of infantile myofibromatosis with pseudo-ulcerated plaque using prenatal ultrasound: A case report
por: ZHANG, FEIXUE, et al.
Publicado: (2014)

The power of natural phenolic compounds: caffeic acid is able to enhance the retinoid-induced differentiation of tumor cells
por: Veselska, Renata, et al.
Publicado: (2014)

Atypical nuclear localization of CD133 plasma membrane glycoprotein in rhabdomyosarcoma cell lines
por: NUNUKOVA, ALENA, et al.
Publicado: (2015)

Prediction of neuroblastoma cell response to treatment with natural or synthetic retinoids using selected protein biomarkers
por: Dobrotkova, Viera, et al.
Publicado: (2019)

Comparative Analysis of Putative Prognostic and Predictive Markers in Neuroblastomas: High Expression of PBX1 Is Associated With a Poor Response to Induction Therapy
por: Veselska, Renata, et al.
Publicado: (2019)

Spindle Cell Hemangioma and Atypically Localized Juxtaglomerular Cell Tumor in a Patient with Hereditary BRIP1 Mutation: A Case Report
por: Papez, Jan, et al.
Publicado: (2021)

Genetic testing and surveillance in infantile myofibromatosis: a report from the SIOPE Host Genome Working Group
por: Hettmer, Simone, et al.
Publicado: (2020)

Nestin as a marker of cancer stem cells
por: Neradil, Jakub, et al.
Publicado: (2015)

Colorectal infantile myofibromatosis: an unusual cause of rectal prolapse and sigmoid colo-colonic intussusception: a case report
por: Dhall, Deepti, et al.
Publicado: (2008)

Infantile myofibromatosis treated by mandibulectomy and staged reconstruction with submental flap and free fibula flap: a case report
por: Maby, Alexandra, et al.
Publicado: (2019)

Solitary infantile myofibromatosis of the petrous bone: a diagnostic pitfall in uncommon location illustrated by a case report
por: El Ouazzani, Hafsa, et al.
Publicado: (2023)

Novel Thiosemicarbazones Sensitize Pediatric Solid Tumor Cell-Types to Conventional Chemotherapeutics through Multiple Molecular Mechanisms
por: Paukovcekova, Silvia, et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_6kvf5kot4p9ts0125l3u547grs