Sameer Farouk Sait
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View article: CTIM-22. WINDOW OF OPPORTUNITY STUDY OF NIVOLUMAB AND IPILIMUMAB IN PEOPLE WITH NEUROFIBROMATOSIS TYPE 1 AND NEWLY DIAGNOSED MALIGNANT AND PRE-MALIGNANT PERIPHERAL NERVE SHEATH TUMORS
CTIM-22. WINDOW OF OPPORTUNITY STUDY OF NIVOLUMAB AND IPILIMUMAB IN PEOPLE WITH NEUROFIBROMATOSIS TYPE 1 AND NEWLY DIAGNOSED MALIGNANT AND PRE-MALIGNANT PERIPHERAL NERVE SHEATH TUMORS Open
Neurofibromatosis type 1 (NF1)-associated malignant peripheral nerve sheath tumors (MPNST) are aggressive sarcomas with no curative medical therapies. Most clinical trials target recurrent or refractory disease, leaving a critical gap in e…
View article: Data from Improving Global Access to Genomic Profiling in Rare Pediatric Cancers
Data from Improving Global Access to Genomic Profiling in Rare Pediatric Cancers Open
Purpose:To address financial barriers that limit access to genomic profiling and precision medicine, philanthropy-supported clinical genomic testing was offered worldwide at no cost to patients with select rare cancers via the Make-an-IMPA…
View article: Supplementary Table 1 from Improving Global Access to Genomic Profiling in Rare Pediatric Cancers
Supplementary Table 1 from Improving Global Access to Genomic Profiling in Rare Pediatric Cancers Open
Supplementary Table 1. Table representing country of referral for patients enrolled on the Make An Impact program (n = 63).
View article: Supplementary Figure 1 from Improving Global Access to Genomic Profiling in Rare Pediatric Cancers
Supplementary Figure 1 from Improving Global Access to Genomic Profiling in Rare Pediatric Cancers Open
Supplementary Figure 1. Overview of the workflow involved from patient outreach, screening, enrollment, testing and return of results to local physician/patient.
View article: Supplementary Table 3 from Improving Global Access to Genomic Profiling in Rare Pediatric Cancers
Supplementary Table 3 from Improving Global Access to Genomic Profiling in Rare Pediatric Cancers Open
Supplementary Table 3. Clinical, demographic and molecular characteristics of pediatric CNS tumor patients enrolled on the Make an Impact program.
View article: Supplementary Figure 2 from Improving Global Access to Genomic Profiling in Rare Pediatric Cancers
Supplementary Figure 2 from Improving Global Access to Genomic Profiling in Rare Pediatric Cancers Open
Supplementary Figure 2. Overview of the analytical pipeline for interpretation of cerebrospinal fluid (CSF) circulating tumor DNA (ctDNA).
View article: Supplementary Table 2 from Improving Global Access to Genomic Profiling in Rare Pediatric Cancers
Supplementary Table 2 from Improving Global Access to Genomic Profiling in Rare Pediatric Cancers Open
Supplementary Table 2. Clinical, demographic and molecular characteristics of pediatric solid tumor patients enrolled on the Make an Impact program.
Hydroxychloroquine prevents resistance and potentiates the antitumor effect of SHP2 inhibition in NF1-associated malignant peripheral nerve sheath tumors Open
Malignant peripheral nerve sheath tumors (MPNSTs) are aggressive sarcomas and the primary cause of mortality in patients with neurofibromatosis type 1 (NF1). These malignancies develop within preexisting benign lesions called plexiform neu…
CTNI-77. PHASE 1 DOSE FINDING STUDY EVALUATING THE SAFETY, TOLERABILITY, AND EFFICACY OF IOPOFOSINE I 131 IN CHILDREN, ADOLESCENTS, AND YOUNG ADULTS WITH INOPERABLE RELAPSED OR REFRACTORY HIGH-GRADE GLIOMAS: A TRIAL-IN-PROGRESS Open
Iopofosine I 131, a radioconjugate therapy targeting tumor lipid rafts, is being assessed in CLOVER-2 [NCT05610891]–an ongoing dose-finding study evaluating iopofosine in children, adolescents, and young adults with relapsed, refractory, r…
View article: EPCO-32. PROSPECTIVE GERMLINE SEQUENCING OF PATIENTS WITH GLIOMAS, GLIONEURONAL OR NEURONAL TUMORS
EPCO-32. PROSPECTIVE GERMLINE SEQUENCING OF PATIENTS WITH GLIOMAS, GLIONEURONAL OR NEURONAL TUMORS Open
BACKGROUND Several genetic syndromes have been linked to the development of central nervous system (CNS) tumors; however, the prevalence and clinical significance of germline pathogenic variants in this population remain unclear. METHODS 2…
Craniospinal irradiation and/or intraventricular radioimmunotherapy after high‐dose chemotherapy and autologous stem cell rescue in patients with CNS retinoblastoma—Safety and outcomes Open
Background The prognosis for patients with central nervous system (CNS) retinoblastoma (RB) (trilateral or stage 4b metastatic RB) treated with high‐dose chemotherapy and autologous stem cell transplant (HDC‐ASCT) remains poor. The impact …
CNSC-02. PEDIATRIC BRAIN TUMOR CONSORTIUM (PBTC)-056: A PHASE 1 STUDY OF THE ADAM-10 INHIBITOR, INCB007839, IN CHILDREN WITH RECURRENT/PROGRESSIVE HIGH-GRADE GLIOMAS TO TARGET MICROENVIRONMENTAL NEUROLOGIN-3 Open
BACKGROUND Pediatric high-grade gliomas (pHGGs), including diffuse intrinsic pontine glioma (DIPG), are a leading cause of central nervous system tumor-related morbidity and mortality in children. Neuronal activity promotes growth of HGGs;…
View article: LGG-34. THE LANDSCAPE OF GLIOMAS IN CHILDREN, ADOLESCENTS AND YOUNG ADULTS REVEALS INSIGHTS ON GLIOMAGENESIS: A CANADIAN ADOLESCENTS AND YOUNG ADULTS NEURO-ONCOLOGY NETWORK (CANON) STUDY
LGG-34. THE LANDSCAPE OF GLIOMAS IN CHILDREN, ADOLESCENTS AND YOUNG ADULTS REVEALS INSIGHTS ON GLIOMAGENESIS: A CANADIAN ADOLESCENTS AND YOUNG ADULTS NEURO-ONCOLOGY NETWORK (CANON) STUDY Open
BACKGROUND Gliomas are a heterogenous and common cancer in children, adolescents and young adults (CAYA, ages 0-39 years). Little is known about the biologic and clinical implications of gliomas in AYA limiting our ability to appropriately…
View article: OTHR-26. MINIMALLY-INVASIVE MOLECULAR DIAGNOSIS OF DIFFUSE LEPTOMENINGEAL GLIONEURONAL TUMOR (DLGNT) USING CEREBROSPINAL FLUID CELL-FREE DNA SEQUENCING IN PEDIATRIC AND YOUNG ADULT PATIENTS
OTHR-26. MINIMALLY-INVASIVE MOLECULAR DIAGNOSIS OF DIFFUSE LEPTOMENINGEAL GLIONEURONAL TUMOR (DLGNT) USING CEREBROSPINAL FLUID CELL-FREE DNA SEQUENCING IN PEDIATRIC AND YOUNG ADULT PATIENTS Open
BACKGROUND Diffuse leptomeningeal glioneuronal tumor (DLGNT) presents with nodular leptomeningeal disease throughout the neuroaxis and is characterized by the presence of KIAA1549::BRAF fusion and chromosome arm 1p deletion. Concomitant ga…
View article: OTHR-20. MINIMALLY-INVASIVE MOLECULAR DIAGNOSIS OF INFILTRATING BRAINSTEM GLIOMAS THROUGH CEREBROSPINAL FLUID CELL-FREE DNA SEQUENCING
OTHR-20. MINIMALLY-INVASIVE MOLECULAR DIAGNOSIS OF INFILTRATING BRAINSTEM GLIOMAS THROUGH CEREBROSPINAL FLUID CELL-FREE DNA SEQUENCING Open
BACKGROUND Accurate molecular diagnosis of infiltrating brainstem tumors, including diffuse midline gliomas and IDH mutant astrocytomas, is essential for prognostication and optimal therapy. Surgical biopsy of the brainstem carries risk of…
View article: DEI-02. DIRECT PATIENT OUTREACH IN PEDIATRIC NEURO-ONCOLOGY TO IMPROVE ACCESS TO GENOMIC PROFILING – THE MAKE-AN-IMPACT PROGRAM
DEI-02. DIRECT PATIENT OUTREACH IN PEDIATRIC NEURO-ONCOLOGY TO IMPROVE ACCESS TO GENOMIC PROFILING – THE MAKE-AN-IMPACT PROGRAM Open
BACKGROUND Genomic profiling and precision medicine have revolutionized cancer care but are not accessible to all due to high costs, insurance issues, limited access to testing facilities or qualified personnel. We established a program pr…
View article: Hypofractionated re‐irradiation for diffuse intrinsic pontine glioma
Hypofractionated re‐irradiation for diffuse intrinsic pontine glioma Open
Background Re‐irradiation (reRT) increases survival in locally recurrent diffuse intrinsic pontine glioma (DIPG). There is no standard dose and fractionation for reRT, but conventional fractionation (CF) is typically used. We report our in…
View article: CTNI-64. EFFICACY AND SAFETY OF ERDAFITINIB IN PATIENTS WITH HIGH-GRADE AND LOW-GRADE GLIOMAS AND PRESPECIFIED FIBROBLAST GROWTH FACTOR RECEPTOR ALTERATIONS (FGFRALT) IN THE RAGNAR TRIAL
CTNI-64. EFFICACY AND SAFETY OF ERDAFITINIB IN PATIENTS WITH HIGH-GRADE AND LOW-GRADE GLIOMAS AND PRESPECIFIED FIBROBLAST GROWTH FACTOR RECEPTOR ALTERATIONS (FGFRALT) IN THE RAGNAR TRIAL Open
BACKGROUND Erda is an oral selective pan-FGFR tyrosine kinase inhibitor approved to treat adults with locally advanced/metastatic urothelial carcinoma with susceptible FGFR3/2alt who have progressed during/after ≥1 line of platinum-based c…
View article: METB-05. NON-INVASIVE DIAGNOSIS OF BRAINSTEM GLIOMAS IN PEDIATRIC, ADOLESCENT, AND YOUNG ADULT PATIENTS THROUGH CEREBROSPINAL FLUID CELL-FREE DNA SEQUENCING
METB-05. NON-INVASIVE DIAGNOSIS OF BRAINSTEM GLIOMAS IN PEDIATRIC, ADOLESCENT, AND YOUNG ADULT PATIENTS THROUGH CEREBROSPINAL FLUID CELL-FREE DNA SEQUENCING Open
Intrinsic brainstem tumors arising in pediatric, adolescent, and young adult patients comprise a spectrum of entities, predominantly diffuse midline gliomas (DMG) and IDH mutant astrocytomas. Accurate molecular diagnosis is essential for p…
View article: METB-07. MINIMALLY-INVASIVE DIAGNOSIS OF DIFFUSE LEPTOMENINGEAL GLIONEURONAL TUMOR (DLGNT) USING CEREBROSPINAL FLUID CELL-FREE DNA SEQUENCING IN PEDIATRIC AND YOUNG ADULT PATIENTS
METB-07. MINIMALLY-INVASIVE DIAGNOSIS OF DIFFUSE LEPTOMENINGEAL GLIONEURONAL TUMOR (DLGNT) USING CEREBROSPINAL FLUID CELL-FREE DNA SEQUENCING IN PEDIATRIC AND YOUNG ADULT PATIENTS Open
Diffuse leptomeningeal glioneuronal tumor (DLGNT) presents with nodular leptomeningeal disease throughout the neuroaxis. Histology shows variable presence of a low density intraparenchymal lesion containing OLIG2-expressing neoplastic cell…
View article: DIPG-44. DIFFUSE MIDLINE GLIOMA WITH EXTRACRANIAL METASTASIS
DIPG-44. DIFFUSE MIDLINE GLIOMA WITH EXTRACRANIAL METASTASIS Open
Diffuse midline gliomas (DMGs) are malignant infiltrative gliomas enriched in the pediatric population and characterized by loss of the H3 K27me3 epigenetic marker, most frequently via mutation of the H3F3A gene. Few cases of extracranial …
LGG-20. LANDSCAPE OF FGFR ALTERATIONS IN PEDIATRIC AND AYA GLIOMAS Open
FGFR alterations including single nucleotide variants (SNV) and rearrangements represent common oncogenic alterations found in pediatric low-grade gliomas. From a population-based cohort of over 1800 pediatric and AYA (adolescent and young…
Slipped capital femoral epiphyses: A major on‐target adverse event associated with FGFR tyrosine kinase inhibitors in pediatric patients Open
Fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitors (TKIs) are increasingly being used off label in pediatrics. Long‐term safety data are limited, and serious toxicities unique to pediatrics may emerge. In a retrospective a…
Progress in precision therapy in pediatric oncology Open
Purpose of review The fields of precision medicine and cancer genomics in pediatric oncology are rapidly evolving. Novel diagnostic tools are critical in refining cancer diagnoses, stratifying patient risk, and informing treatment decision…
NIMG-57. NON-INVASIVE DIAGNOSIS OF IDH-MUTANT BRAINSTEM GLIOMAS Open
INTRODUCTION Treatment of brainstem tumors is often initiated without a tissue diagnosis due to the risk of biopsy. A subset of brainstem gliomas harbor an isocitrate dehydrogenase 1/2 (IDH) mutation, which predicts response to alkylator c…
Dual inhibition of SHP2 and autophagy suppresses NF1-associated Malignant Peripheral Nerve Sheath Tumors Open
Malignant peripheral nerve sheath tumors (MPNSTs) are aggressive sarcomas and the primary cause of mortality in patients with neurofibromatosis type 1 (NF1). MPNSTs develop within pre-existing benign plexiform neurofibromas (PNs). PNs are …