Cargando…

A randomized double-blind controlled trial of everolimus in individuals with PTEN mutations: Study design and statistical considerations

This randomized, double-blind controlled trial of everolimus in individuals with germline phosphatase and tensin homolog mutations (PTEN) was designed to evaluate the safety of everolimus compared with placebo and to evaluate the efficacy of everolimus on neurocognition and behavior compared to plac...

Descripción completa

Detalles Bibliográficos
Autores principales:	Hardan, Antonio Y., Jo, Booil, Frazier, Thomas W., Klaas, Patricia, Busch, Robyn M., Dies, Kira A., Filip-Dhima, Rajna, Snow, Anne V., Eng, Charis, Hanna, Rabi, Zhang, Bo, Sahin, Mustafa
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7887633/ https://www.ncbi.nlm.nih.gov/pubmed/33644493 http://dx.doi.org/10.1016/j.conctc.2021.100733

Ejemplares similares

A randomized controlled trial of everolimus for neurocognitive symptoms in PTEN hamartoma tumor syndrome
por: Srivastava, Siddharth, et al.
Publicado: (2022)

Neurobehavioral phenotype of autism spectrum disorder associated with germline heterozygous mutations in PTEN
por: Busch, Robyn M., et al.
Publicado: (2019)

Cross-level analysis of molecular and neurobehavioral function in a prospective series of patients with germline heterozygous PTEN mutations with and without autism
por: Frazier, Thomas W., et al.
Publicado: (2021)

Longitudinal neurobehavioral profiles in children and young adults with PTEN hamartoma tumor syndrome and reliable methods for assessing neurobehavioral change
por: Busch, Robyn M., et al.
Publicado: (2023)

Psychiatric Characteristics Across Individuals With PTEN Mutations
por: Steele, Morgan, et al.
Publicado: (2021)

Everolimus for treatment of tuberous sclerosis complex‐associated neuropsychiatric disorders
por: Krueger, Darcy A., et al.
Publicado: (2017)

Molecular and Phenotypic Abnormalities in Individuals with Germline Heterozygous PTEN Mutations and Autism
por: Frazier, Thomas W., et al.
Publicado: (2014)

Distinct metabolic profiles associated with autism spectrum disorder versus cancer in individuals with germline PTEN mutations
por: Yehia, Lamis, et al.
Publicado: (2022)

Copy Number Variation and Clinical Outcomes in Patients With Germline PTEN Mutations
por: Yehia, Lamis, et al.
Publicado: (2020)

Using the big data approach to clarify the structure of restricted and repetitive behaviors across the most commonly used autism spectrum disorder measures
por: Uljarević, Mirko, et al.
Publicado: (2021)

Neural transcriptome of constitutional Pten dysfunction in mice and its relevance to human idiopathic Autism Spectrum Disorder
por: Tilot, Amanda K, et al.
Publicado: (2015)

A reinvestigation of somatic hypermethylation at the PTEN CpG island in cancer cell lines
por: Hesson, Luke B, et al.
Publicado: (2012)

The microbiome in PTEN hamartoma tumor syndrome
por: Byrd, Victoria, et al.
Publicado: (2017)

Cytoplasm-predominant Pten associates with increased region-specific brain tyrosine hydroxylase and dopamine D2 receptors in mouse model with autistic traits
por: He, Xin, et al.
Publicado: (2015)

Gastrointestinal polyposis and PTEN mutations: an under-acknowledged diagnostic criterion
por: Heald, Brandie, et al.
Publicado: (2010)

Erratum to: Cytoplasm-predominant Pten associates with increased region-specific brain tyrosine hydroxylase and dopamine D2 receptors in mouse model with autistic traits
por: He, Xin, et al.
Publicado: (2016)

Cowden syndrome-associated germline SDHD variants alter PTEN nuclear translocation through SRC-induced PTEN oxidation
por: Yu, Wanfeng, et al.
Publicado: (2015)

A Longitudinal Pilot Study of Behavioral Abnormalities in Children with Autism
por: Libove, Robin A., et al.
Publicado: (2017)

Quantitative autism symptom patterns recapitulate differential mechanisms of genetic transmission in single and multiple incidence families
por: Frazier, Thomas W., et al.
Publicado: (2015)

Germline TTN variants are enriched in PTEN-wildtype Bannayan–Riley–Ruvalcaba syndrome
por: Yehia, Lamis, et al.
Publicado: (2017)

Relationship Between Social Motivation in Children With Autism Spectrum Disorder and Their Parents
por: Uljarević, Mirko, et al.
Publicado: (2021)

PTEN interacts with RNA polymerase II to dephosphorylate polymerase II C-terminal domain
por: Abbas, Ata, et al.
Publicado: (2019)

Characterization of cryptic splicing in germline PTEN intronic variants in Cowden syndrome
por: Chen, Hannah Jinlian, et al.
Publicado: (2017)

The mechanism of full activation of tumor suppressor PTEN at the phosphoinositide-enriched membrane
por: Jang, Hyunbum, et al.
Publicado: (2021)

Transcriptome-(phospho)proteome characterization of brain of a germline model of cytoplasmic-predominant Pten expression with autism-like phenotypes
por: Thacker, Stetson, et al.
Publicado: (2021)

PTEN modulates gene transcription by redistributing genome-wide RNA polymerase II occupancy
por: Abbas, Ata, et al.
Publicado: (2019)

Validation of a computational phenotype for finding patients eligible for genetic testing for pathogenic PTEN variants across three centers
por: Kothari, Cartik, et al.
Publicado: (2022)

The mitochondrial genome as a modifier of autism versus cancer phenotypes in PTEN hamartoma tumor syndrome
por: Wei, Ruipeng, et al.
Publicado: (2023)

Alternative splicing landscape of the neural transcriptome in a cytoplasmic-predominant Pten expression murine model of autism-like Behavior
por: Thacker, Stetson, et al.
Publicado: (2020)

Parent-reported measure of repetitive behavior in Phelan-McDermid syndrome
por: Srivastava, Siddharth, et al.
Publicado: (2021)

Structural and Dynamic Effects of PTEN C-Terminal Tail Phosphorylation
por: Smith, Iris N., et al.
Publicado: (2022)

Integrating somatic CNV and gene expression in breast cancers from women with PTEN hamartoma tumor syndrome
por: Brewer, Takae, et al.
Publicado: (2023)

Constitutional mislocalization of Pten drives precocious maturation in oligodendrocytes and aberrant myelination in model of autism spectrum disorder
por: Lee, Hyunpil, et al.
Publicado: (2019)

Germline nuclear-predominant Pten murine model exhibits impaired social and perseverative behavior, microglial activation, and increased oxytocinergic activity
por: Sarn, Nick, et al.
Publicado: (2021)

ATP modulates PTEN subcellular localization in multiple cancer cell lines
por: Lobo, Glenn P., et al.
Publicado: (2008)

Decreased nuclear Pten in neural stem cells contributes to deficits in neuronal maturation
por: Kang, Shin Chung, et al.
Publicado: (2020)

Early white matter development is abnormal in tuberous sclerosis complex patients who develop autism spectrum disorder
por: Prohl, Anna K., et al.
Publicado: (2019)

Shape shifting: The multiple conformational substates of the PTEN N‐terminal PIP(2) ‐binding domain
por: Dawson, Jennifer E., et al.
Publicado: (2022)

Exceptional Response to Everolimus in a Patient with Metastatic Castrate-Resistant Prostate Cancer Harboring a PTEN Inactivating Mutation
por: Kmak, Jamie A., et al.
Publicado: (2020)

Early-onset renal cell carcinoma in PTEN harmatoma tumour syndrome
por: Kim, Raymond H., et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_4pj1moulh9h82c19qcf8ra9did