medulloblastoma IV

Background Central nervous system (CNS) tumors cause significant mortality and morbidity in all age groups. There was no data about the histological spectrum of all CNS tumors in the tertiary care center serving primarily the rural population of Uttar Pradesh. Aims and objectives The present study aimed to describe the histopathological spectrum of all CNS tumors reported in a rural tertiary care center at Saifai, Uttar Pradesh. It also aimed to provide an overview of the descriptive epidemiology of CNS tumors...

PURPOSE: Medulloblastoma (MB), the most common childhood malignant brain tumor, has a poor prognosis in about 30% of patients. The current standard of care, which includes surgery, radiation and chemotherapy, is often responsible for cognitive, neurologic and endocrine side effects. We investigated whether chimeric antigen receptor (CAR) T-cells directed towards the disialoganglioside GD2 can represent a potentially more effective treatment with reduced long-term side effects. EXPERIMENTAL DESIGN: GD2 expression was evaluated on primary tumor biopsies of MB children by flow-cytometry...

INTRODUCTION: Medulloblastoma, a highly malignant embryonal tumor predominantly found in the pediatric population, typically arises within the cerebellum. This case report holds particular importance due to the rarity of medulloblastoma within the cerebellopontine angle (CPA). The distinct anatomical challenge posed by the CPA complex neurovascular structures, along with the absence of pathognomonic clinical or radiographic features, highlights the unique diagnostic and management challenge of this case...

Recent studies suggest that long non-coding RNAs (lncRNAs) contribute to medulloblastoma (MB) formation and progression. We have identified an lncRNA, lnc-HLX-2-7, as a potential therapeutic target in group 3 (G3) MBs. lnc-HLX-2-7 RNA specifically accumulates in the promoter region of HLX, a sense-overlapping gene of lnc-HLX-2-7, which activates HLX expression by recruiting multiple factors, including enhancer elements. RNA sequencing and chromatin immunoprecipitation reveal that HLX binds to and activates the promoters of several oncogenes, including TBX2, LIN9, HOXM1, and MYC...

PURPOSE: AcSé-ESMART Arm C aimed to define the recommended dose and activity of the WEE1 inhibitor adavosertib in combination with carboplatin in children and young adults with molecularly enriched recurrent/refractory malignancies. PATIENTS AND METHODS: Adavosertib was administered orally, twice daily on Days 1 to 3 and carboplatin intravenously on Day 1 of a 21-day cycle, starting at 100 mg/m2/dose and AUC 5, respectively. Patients were enriched for molecular alterations in cell cycle and/or homologous recombination (HR)...

IMPORTANCE: Medulloblastoma recurrence in patients who have previously received irradiation has a dismal prognosis and lacks a standard salvage regimen. OBJECTIVE: To evaluate the response rate of pediatric patients with medulloblastoma recurrence using an antiangiogenic metronomic combinatorial approach (Medulloblastoma European Multitarget Metronomic Anti-Angiogenic Trial [MEMMAT]). DESIGN, SETTING, AND PARTICIPANTS: This phase 2, investigator-initiated, multicenter nonrandomized controlled trial assessed 40 patients with relapsed or refractory medulloblastoma without a ventriculoperitoneal shunt who were younger than 20 years at original diagnosis...

PURPOSE: To investigate the skeletal phenotype of adolescent girls with premature ovarian insufficiency (POI). METHODS: Data are presented from two adolescent girls who participated in a clinical research protocol to evaluate axial bone mineral density (BMD) (via dual-energy x-ray absorptiometry, DXA) and appendicular bone density, microarchitecture, and strength (via high-resolution peripheral quantitative computed tomography, HRpQCT). Anthropometric data were also obtained, and pubertal staging performed by a clinician...

Achieving adequate exposure of a therapeutic agent at the target is a critical determinant of efficacious chemotherapy. With this in mind, a major challenge in developing therapies for central nervous system (CNS) tumors is to overcome barriers to delivery, including the blood-brain barrier (BBB). Panobinostat is a nonselective pan-HDAC inhibitor that is being tested in pre-clinical and clinical studies, including for the treatment of pediatric medulloblastoma, which has a propensity for leptomeningeal spread, and diffuse midline glioma (DMG), which can infiltrate into supratentorial brain regions...

PURPOSE/OBJECTIVE(S): Craniospinal irradiation (CSI) is an essential part of curative treatment for several pediatric brain tumors. Proton CSI allows for sparing of the organs anterior to the vertebral bodies (VBs), but this technique still includes the entire VB in the target for growing children. providing no advantage in marrow sparing or adverse effects on growth. Advances in proton therapy including Proton Beam Scanning (PBS) allow for delivery of proton CSI with substantial vertebral body sparing (VBS)...

PURPOSE/OBJECTIVE(S): The rarity of medulloblastoma in the adult population limits information on optimal treatment and clinical outcomes. Adjuvant chemoradiation has been correlated to improved overall survival (OS) in a recent National Cancer Database (NCDB) analysis. We performed a retrospective safety and efficacy analysis of radiation (RT) +/- adjuvant chemotherapy (aCTH) in young adult patients with medulloblastoma to better understand outcomes, prognostic factors, and possibly optimal treatment strategies...

PURPOSE/OBJECTIVE(S): While memantine has become standard in certain adults receiving brain RT to decrease the cognitive impacts of RT, it is unknown whether pediatric patients can take and tolerate memantine or experience benefit. In this prospective single-arm feasibility study, we hypothesized pediatric patients receiving brain RT would tolerate memantine with good treatment adherence. MATERIALS/METHODS: Patients aged 4-18 years with a primary CNS malignancy (excluding WHO Grade IV astrocytoma and glioblastoma) receiving intracranial RT were eligible...

BACKGROUND: The Toronto Paediatric Cancer Stage Guidelines are a compendium of staging systems developed to facilitate collection of consistent and comparable data on stage at diagnosis for childhood cancers by cancer registries. MATERIAL AND METHODS: This retrospective, observational cohort study investigated changes in stage-specific incidence and survival for children diagnosed between 2000-2008 compared to 2009-2017 using the population-based Australian Childhood Cancer Registry...

BACKGROUND: Published literature on epidemiological profile of paediatric brain tumours in India is limited. Aim : To present a retrospective analysis of the histological spectrum of 158 paediatric age group central nervous system tumours operated in a single tertiary care hospital in Coastal South India between January 2015 and December 2021. MATERIAL AND METHODS: Retrospective analysis of the data regarding frequencies of various primary brain tumours among 158 paediatric patients (<18 years of age)...

Embryonal tumors (ETs) of the central nervous system (CNS) comprise a large heterogeneous group of highly malignant tumors that predominantly affect children and adolescents. Currently, the neoplasms classified as ET are the medulloblastoma (MB), embryonal tumors with multilayered rosettes (ETMR), medulloepithelioma (ME), CNS neuroblastoma (NB), CNS ganglioneuroblastoma (GNB), atypical teratoid/rhabdoid tumors (AT/RT), and CNS embryonal tumors with rhabdoid features. All these tumors are classified as malignant-grade IV neoplasms, and the prognosis of patients with these neoplasms is very poor...

BACKGROUND & AIMS: Medulloblastoma (MB) is a primary malignant tumor of the brain. They are categorized as WHO grade IV neoplasms, and mostly occur in children. The traditional therapy for MB is surgery, followed by radiation and chemotherapy, but the clinical outcome is still poor and has a high possibility of recurrence. The mechanism underlying the development of MB should be further investigated to develop novel therapeutic strategies. METHODS: Research has demonstrated that circRNAs contribute to tumorigenesis, but the functional mechanism of circRNAs in MB has not been fully explored and remains vague...

Medulloblastoma is the most common childhood brain tumor with an unfavorable prognosis and limited options of harmful treatments that are associated with devastating long-term side effects. Therefore, the development of safe, noninvasive, and effective therapeutic approaches is required to save the quality of life of young medulloblastoma survivors. We postulated that therapeutic targeting is a solution. Thus, we used a recently designed tumor-targeted bacteriophage (phage)-derived particle, named transmorphic phage/AAV, TPA, to deliver a transgene expressing the tumor necrosis factor-alpha (TNFα) for targeted systemic therapy of medulloblastoma...

Medulloblastoma (MB) is one of the most common malignant childhood brain tumors (WHO grade IV). Its high degree of malignancy leads to an unsatisfactory prognosis, requiring more precise and personalized treatment in the near future. Multi-omics and artificial intelligence have been playing a significant role in precise medical research, but their implementation needs a large amount of clinical information and biomaterials. For these reasons, it is urgent for current MB researchers to establish a large sample-size database of MB that contains complete clinical data and sufficient biomaterials such as blood, cerebrospinal fluid (CSF), cancer tissue, and urine...

BACKGROUND: The HIT-SKK protocol is used for low/standard-risk medulloblastomas in young children with the aim to eliminate cranial irradiation and its neuropsychological (NP) sequelae. This therapy includes IV and intraventricular (ITV) methotrexate (MTX) potentially responsible for leukoencephalopathy (LE) and neurocognitive disorders. This study describes the risk factors and course of LE, and investigates its correlation with neurocognitive impact. METHODS: A retrospective, multicenter study was conducted in 35 children under 5 years old, with a median follow-up of 72 months (range 14 to 130)...

The brain penetration of methotrexate (MTX) and its metabolite 7-hydroxymethotrexate (7OHMTX) was characterized in non-tumor bearing mice and mice bearing orthotopic Group 3 medulloblastoma. Plasma pharmacokinetic studies and cerebral and ventricular microdialysis studies were performed in animals dosed with 200 or 1000 mg/kg MTX by IV bolus. Plasma, brain/tumor extracellular fluid (ECF) and lateral ventricle cerebrospinal fluid (CSF) MTX and 7OHMTX concentration-time data were analyzed by validated LC-MS/MS methods and modeled using a population-based pharmacokinetic approach and a hybrid physiologically-based model structure for the brain compartments...

A 16-month-old girl was referred for tachycardia and upper respiratory tract infection. Echocardiographic examination revealed pericardial effusion, mild mitral regurgitation, and left ventricle systolic dysfunction. Patient was positive for Parainfluenza type 4 virus. Her laboratory tests revealed increased troponin I level. The patient was treated with intravenous immunoglobulin considering acute viral myopericarditis. Two weeks after treatment, midventricular hypertrophy was detected.