Therapeutic advances for the tumors associated with neurofibromatosis type 1, type 2, and schwannomatosis

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Therapeutic advances for the tumors associated with neurofibromatosis type 1, type 2, and schwannomatosis is …
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review articleQ7318358
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P356DOI10.1093/NEUONC/NOV200
P932PMC publication ID4827037
P698PubMed publication ID26851632

P2093author name stringJaishri O Blakeley
Scott R Plotkin
P2860cites workMerlin/NF2 loss-driven tumorigenesis linked to CRL4(DCAF1)-mediated inhibition of the hippo pathway kinases Lats1 and 2 in the nucleusQ24300770
The protein product of the neurofibromatosis type 1 gene is expressed at highest abundance in neurons, Schwann cells, and oligodendrocytesQ24309158
Diversity and specialization of mammalian SWI/SNF complexesQ24322918
The NF1 locus encodes a protein functionally related to mammalian GAP and yeast IRA proteinsQ24324729
Mutation and cancer: statistical study of retinoblastomaQ24618185
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A RASopathy gene commonly mutated in cancer: the neurofibromatosis type 1 tumour suppressorQ27003534
Phase II study of mTORC1 inhibition by everolimus in neurofibromatosis type 2 patients with growing vestibular schwannomas.Q27853117
The BTB-kelch protein LZTR-1 is a novel Golgi protein that is degraded upon induction of apoptosisQ28116515
Diagnostic criteria for schwannomatosisQ28256490
Evidence of a four-hit mechanism involving SMARCB1 and NF2 in schwannomatosis-associated schwannomasQ28260652
The molecular pathology of cancerQ28278125
Merlin, a "magic" linker between extracellular cues and intracellular signaling pathways that regulate cell motility, proliferation, and survivalQ28283076
SWI/SNF chromatin remodeling complex is obligatory for BMP2-induced, Runx2-dependent skeletal gene expression that controls osteoblast differentiationQ28587715
Tumour predisposition in mice heterozygous for a targeted mutation in Nf1Q28594951
The GAP-related domain of the neurofibromatosis type 1 gene product interacts with ras p21Q28609120
Cooperation between complexes that regulate chromatin structure and transcriptionQ29614769
Relationship between whole-body tumor burden, clinical phenotype, and quality of life in patients with neurofibromatosisQ60145699
Pediatric Plexiform Neurofibromas: Impact on Morbidity and Mortality in Neurofibromatosis Type 1Q60696729
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Allelic loss at the neurofibromatosis type 1 (NF1) gene locus is frequent in desmoplastic neurotropic melanomaQ73326046
Novel mutations of neurofibromatosis type 1 gene in small cell lung cancersQ73362762
Phase I trial of pirfenidone in children with neurofibromatosis 1 and plexiform neurofibromasQ80350602
Alterations in the SMARCB1 (INI1) tumor suppressor gene in familial schwannomatosisQ81684801
Internal tumor burden in neurofibromatosis Type I patients with large NF1 deletionsQ83372844
Gamma Knife radiosurgery for meningiomas in patients with neurofibromatosis Type 2Q86444215
Ketotifen suppression of NF1 neurofibroma growth over 30 yearsQ87309079
Hearing improvement after bevacizumab in patients with neurofibromatosis type 2Q30387415
Anti-vascular endothelial growth factor therapies as a novel therapeutic approach to treating neurofibromatosis-related tumors.Q30388655
Long-term natural history of neurofibromatosis Type 2-associated intracranial tumorsQ30391370
Mutations in LZTR1 add to the complex heterogeneity of schwannomatosisQ30394068
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Consensus recommendations to accelerate clinical trials for neurofibromatosis type 2.Q30406342
Phase II study of everolimus in children and adults with neurofibromatosis type 2 and progressive vestibular schwannomas.Q30422022
Recommendations for imaging tumor response in neurofibromatosis clinical trialsQ30425757
Hearing and facial function outcomes for neurofibromatosis 2 clinical trials.Q30425761
Optimizing biologically targeted clinical trials for neurofibromatosis.Q30438898
Phase II trial of lapatinib in adult and pediatric patients with neurofibromatosis type 2 and progressive vestibular schwannomas.Q30450038
AR42, a novel histone deacetylase inhibitor, as a potential therapy for vestibular schwannomas and meningiomasQ30464276
Glioblastoma multiforme after stereotactic radiotherapy for acoustic neuroma: case report and review of the literatureQ30493204
Gamma knife stereotactic radiosurgery for unilateral acoustic neuromasQ30495461
The NF2 tumor suppressor regulates microtubule-based vesicle trafficking via a novel Rac, MLK and p38(SAPK) pathwayQ30606515
Phase II study of carboplatin in children with progressive low-grade gliomasQ33343014
Longitudinal evaluation of quality of life in 288 patients with neurofibromatosis 2.Q33561323
Whole Body MRI at 3T with Quantitative Diffusion Weighted Imaging and Contrast-Enhanced Sequences for the Characterization of Peripheral Lesions in Patients with Neurofibromatosis Type 2 and Schwannomatosis.Q33711291
Neurofibromatosis type 1: A model condition for the study of the molecular basis of variable expressivity in human disordersQ33723849
Hyperactive Ras as a therapeutic target in neurofibromatosis type 1.Q33723856
SMARCB1/INI1 germline mutations contribute to 10% of sporadic schwannomatosisQ33800041
Quantitative analysis of mammalian GIRK2 channel regulation by G proteins, the signaling lipid PIP2 and Na+ in a reconstituted systemQ34055491
Malignancy in neurofibromatosis type 1.Q34100904
Meningiomas and neurofibromatosis.Q34131583
A mutant allele of the Swi/Snf member BAF250a determines the pool size of fetal liver hemopoietic stem cell populationsQ34162999
Merlin deficiency predicts FAK inhibitor sensitivity: a synthetic lethal relationshipQ34193347
Malignant peripheral nerve sheath tumors in children with neurofibromatosis type 1Q34275638
Bevacizumab for progressive vestibular schwannoma in neurofibromatosis type 2: a retrospective review of 31 patientsQ34288675
What is the real incidence of vestibular schwannoma?Q34299160
Proteomic and bioinformatic analysis of mammalian SWI/SNF complexes identifies extensive roles in human malignancy.Q34342958
Incidence of vestibular schwannoma and neurofibromatosis 2 in the North West of England over a 10-year period: higher incidence than previously thoughtQ34392256
Germline loss-of-function mutations in LZTR1 predispose to an inherited disorder of multiple schwannomasQ34393793
The nature and frequency of cognitive deficits in children with neurofibromatosis type 1.Q34458646
E-cadherin-mediated adhesion inhibits ligand-dependent activation of diverse receptor tyrosine kinases.Q37616951
Phase 2 randomized, flexible crossover, double-blinded, placebo-controlled trial of the farnesyltransferase inhibitor tipifarnib in children and young adults with neurofibromatosis type 1 and progressive plexiform neurofibromas.Q37699439
Clinicopathologic study of glioblastoma in children with neurofibromatosis type 1.Q37716733
Imatinib mesylate for plexiform neurofibromas in patients with neurofibromatosis type 1: a phase 2 trial.Q37737382
SWI/SNF complexes, chromatin remodeling and skeletal myogenesis: it's time to exchange!Q37765666
The Diagnostic and Clinical Significance of Café-au-lait MaculesQ37796203
Approaches to treating NF1 tibial pseudarthrosis: consensus from the Children's Tumor Foundation NF1 Bone Abnormalities ConsortiumQ38088574
Li-Fraumeni syndrome: cancer risk assessment and clinical managementQ38197030
Regulation of mTOR complex 2 signaling in neurofibromatosis 2-deficient target cell typesQ39377991
Nilotinib alone or in combination with selumetinib is a drug candidate for neurofibromatosis type 2.Q39512836
Phase I trial and pharmacokinetic study of sorafenib in children with neurofibromatosis type I and plexiform neurofibromas.Q39555847
Variable expressivity of neurofibromatosis-1 in identical twinsQ39611043
CXCL12 alone is insufficient for gliomagenesis in Nf1 mutant mice.Q39692148
The requirement for SNF5/INI1 in adipocyte differentiation highlights new features of malignant rhabdoid tumorsQ40070769
Merlin/neurofibromatosis type 2 suppresses growth by inhibiting the activation of Ras and Rac.Q40181731
Loss of neurofibromatosis type I (NF1) gene expression in pheochromocytomas from patients without NF1.Q41342455
Genomic sequencing of meningiomas identifies oncogenic SMO and AKT1 mutationsQ42548316
A novel moesin-, ezrin-, radixin-like gene is a candidate for the neurofibromatosis 2 tumor suppressor.Q42609349
Merlin suppresses the SRE-dependent transcription by inhibiting the activation of Ras-ERK pathwayQ42804824
Recurrent mutations in the NF1 gene are common among neurofibromatosis type 1 patientsQ43074091
A negative feedback signaling network underlies oncogene-induced senescenceQ43116108
Neurofibromatosis 2, radiosurgery and malignant nervous system tumoursQ43216302
Phase I study of thalidomide for the treatment of plexiform neurofibroma in neurofibromatosis 1.Q44276749
Germline SMARCB1 mutation predisposes to multiple meningiomas and schwannomas with preferential location of cranial meningiomas at the falx cerebriQ44718402
Three new datasets supporting use of the Numerical Rating Scale (NRS-11) for children's self-reports of pain intensityQ45349874
Natural history of vestibular schwannoma growth and hearing decline in newly diagnosed neurofibromatosis type 2 patientsQ46082677
Mutations of the NF1 gene in children with juvenile myelomonocytic leukemia without clinical evidence of neurofibromatosis, type 1.Q46138119
Vestibular schwannoma growth rates in neurofibromatosis type 2 natural history consortium subjectsQ46768033
The tumor suppressors Merlin and Expanded function cooperatively to modulate receptor endocytosis and signalingQ47071805
NF1 plexiform neurofibroma growth rate by volumetric MRI: relationship to age and body weight.Q47306235
Age at symptom onset and long-term survival in patients with neurofibromatosis Type 2.Q47642138
Mutational and functional analysis of the neurofibromatosis type 1 (NF1) geneQ48056312
Phase II trial of pirfenidone in children and young adults with neurofibromatosis type 1 and progressive plexiform neurofibromas.Q48079152
Prognostic factors of CNS tumours in Neurofibromatosis 1 (NF1): a retrospective study of 104 patientsQ48428414
Prolonged survival in adult neurofibromatosis type I patients with recurrent high-grade gliomas treated with bevacizumabQ48769865
Differentiation of cerebral radiation necrosis from tumor recurrence by proton magnetic resonance spectroscopyQ48784484
Three-dimensional volumetrics for tracking vestibular schwannoma growth in neurofibromatosis type II.Q48872525
Marked recovery of vision in children with optic pathway gliomas treated with bevacizumabQ48872539
Earliest clinical manifestations and natural history of neurofibromatosis type 2 (NF2) in childhood: a study of 24 patientsQ48961990
Conservative management of bilateral vestibular schwannomas in neurofibromatosis type 2 patients: hearing and tumor growth results.Q50357611
Clinical course of vestibular schwannoma in pediatric neurofibromatosis Type 2.Q50425679
Concordance of bilateral vestibular schwannoma growth and hearing changes in neurofibromatosis 2: neurofibromatosis 2 natural history consortiumQ50443009
Radiosurgery for type II neurofibromatosisQ50449685
Clinical experience with radiation therapy in the management of neurofibromatosis-associated central nervous system tumors.Q50450347
The neuroimaging and clinical spectrum of neurofibromatosis 2.Q50514920
Increasing annual incidence of vestibular schwannoma and age at diagnosis.Q50749724
Pulmonary hypertension in patients with neurofibromatosis type I.Q51472493
Von Recklinghausen neurofibromatosis. A clinical and population study in south-east Wales.Q52068358
Update from the 2011 International Schwannomatosis Workshop: From genetics to diagnostic criteria.Q53116195
Topical nerve growth factor as a visual rescue strategy in pediatric optic gliomas: a pilot study including electrophysiology.Q55463289
Second primary tumors in neurofibromatosis 1 patients treated for optic glioma: substantial risks after radiotherapy.Q55470115
Natural history of optic pathway tumors in children with neurofibromatosis type 1: a longitudinal study.Q55481914
SMARCB1 mutations in schwannomatosis and genotype correlations with rhabdoid tumorsQ57266497
Genetic testing and screening of individuals at risk of NF2Q57266536
Frequency of SMARCB1 mutations in familial and sporadic schwannomatosisQ57266550
Life expectancy in hereditary cancer predisposing diseases: an observational studyQ57266560
Vestibular schwannomas occur in schwannomatosis and should not be considered an exclusion criterion for clinical diagnosisQ57266581
SMARCB1 mutations are not a common cause of multiple meningiomasQ57266630
Birth incidence and prevalence of tumor-prone syndromes: Estimates from a UK family genetic register serviceQ57266646
Increasing the specificity of diagnostic criteria for schwannomatosisQ57266735
Merlin/NF2 Functions Upstream of the Nuclear E3 Ubiquitin Ligase CRL4DCAF1 to Suppress Oncogenic Gene ExpressionQ57275665
Longitudinal study of neurofibromatosis 1 associated plexiform neurofibromasQ57386667
Schwannomatosis associated with multiple meningiomas due to a familial SMARCB1 mutationQ57737866
Phenotypic variations in NF1-associated low grade astrocytomas: possible role for increased mTOR activation in a subsetQ34467227
Phase I trial of pegylated interferon-alpha-2b in young patients with plexiform neurofibromasQ34555308
Clinical response to bevacizumab in schwannomatosisQ34591266
Carboplatin and vincristine chemotherapy for children with newly diagnosed progressive low-grade gliomas.Q34739729
The diagnostic evaluation and multidisciplinary management of neurofibromatosis 1 and neurofibromatosis 2.Q34741372
Combined MEK inhibition and BMP2 treatment promotes osteoblast differentiation and bone healing in Nf1Osx -/- miceQ34791274
Dose-dependent effects of focal fractionated irradiation on secondary malignant neoplasms in Nf1 mutant miceQ34799331
Transient inhibition of the ERK pathway prevents cerebellar developmental defects and improves long-term motor functions in murine models of neurofibromatosis type 1.Q34980977
Conclusions and future directions for the REiNS International Collaboration.Q35046441
Susceptible stages in Schwann cells for NF1-associated plexiform neurofibroma developmentQ35133489
Handheld optical coherence tomography during sedation in young children with optic pathway gliomas.Q35652582
Defining key signaling nodes and therapeutic biomarkers in NF1-mutant cancersQ35657827
Expanding the mutational spectrum of LZTR1 in schwannomatosisQ35722195
Sirolimus for progressive neurofibromatosis type 1-associated plexiform neurofibromas: a neurofibromatosis Clinical Trials Consortium phase II study.Q35788825
Akt- or MEK-mediated mTOR inhibition suppresses Nf1 optic glioma growthQ35788976
A phase 2 study of pegylated interferon α-2b (PEG-Intron(®)) in children with diffuse intrinsic pontine gliomaQ35791274
Visual outcomes in children with neurofibromatosis type 1-associated optic pathway glioma following chemotherapy: a multicenter retrospective analysisQ36010097
Contact-dependent inhibition of EGFR signaling by Nf2/MerlinQ36118464
Management of the patient and family with neurofibromatosis 2: a consensus conference statementQ36251326
Malignant transformation and new primary tumours after therapeutic radiation for benign disease: substantial risks in certain tumour prone syndromesQ36254710
Comparative oncogenomics implicates the neurofibromin 1 gene (NF1) as a breast cancer driver.Q36268076
Clinical features of schwannomatosis: a retrospective analysis of 87 patientsQ36349611
The molecular biology of vestibular schwannomas: dissecting the pathogenic process at the molecular levelQ36377651
Spectrum and prevalence of vasculopathy in pediatric neurofibromatosis type 1.Q36395077
Neurofibromatosis type 1: new insights into neurocognitive issuesQ36415807
Genotype-phenotype associations in neurofibromatosis type 1 (NF1): an increased risk of tumor complications in patients with NF1 splice-site mutations?Q36483309
A prospective study of neurofibromatosis type 1 cancer incidence in the UK.Q36612055
Optic pathway gliomas in neurofibromatosis-1: controversies and recommendations.Q36771546
Pathogenesis of plexiform neurofibroma: tumor-stromal/hematopoietic interactions in tumor progressionQ36960893
Magic but treatable? Tumours due to loss of merlin.Q36972789
Neurofibromin 1 (NF1) defects are common in human ovarian serous carcinomas and co-occur with TP53 mutationsQ36982106
Gliomas in patients with neurofibromatosis type 1.Q37432196
ATP-dependent chromatin remodeling in neural development.Q37482388
Functional outcome measures for NF1-associated optic pathway glioma clinical trialsQ37536287
P433issue5
P304page(s)624-638
P577publication date2016-02-06
P1433published inNeuro-OncologyQ15724471
P1476titleTherapeutic advances for the tumors associated with neurofibromatosis type 1, type 2, and schwannomatosis
P478volume18