Molecular Advances and Targeted Therapies for Pediatric Central Nervous System Tumors

2020 ◽  
Vol 36 (1) ◽  
pp. 5-29
Author(s):  
Danielle Gordon ◽  
Bernard L. Maria
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Targeted Therapies ◽  
Pediatric Population ◽  
Treatment Plan ◽  
Panel Discussion ◽  
Central Nervous System Tumors ◽  
Nervous System Tumors ◽  
Practice Experience

Central nervous system tumors are extremely rare in the pediatric population and molecularly heterogeneous. Growing scientific research and clinical practice experience are improving medical therapies to increase survival outcomes and quality of life and reduce side effects. The 2019 Neurobiology of Disease in Children Symposium, held in conjunction with the 48th annual meeting of the Child Neurology Society, aimed to (1) describe molecular advances in tumor classification, (2) better understand the evolution of targeted therapies, and (3) more clearly formulate a treatment plan for patients. The article summarizes the presentations and includes an edited transcript of a panel discussion.

Primary Central Nervous System Tumors

2017 ◽  
Author(s):  
Brett J. Theeler ◽  
Mark R. Gilbert
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Pediatric Population ◽  
Tumor Resection ◽  
Specific Pattern ◽  
Ventricular System ◽  
Ependymal Cells ◽  
Central Nervous System Tumors ◽  
Ongoing Research ◽  
Nervous System Tumors

Ependymomas are rare primary central nervous system (CNS) tumors that are thought to arise from ependymal cells lining the ventricular system located throughout the CNS. Ependymomas occur in all age groups but are more common in the pediatric population. Ependymomas typically present as mass lesions within the ventricular system, brain or spinal cord parenchyma. As with most central nervous system tumors, pathologic evaluation is required for definitive diagnosis. Ependymomas are typically treated with a combination of surgery and radiotherapy although this varies depending on tumor location, tumor grade, patient age, extent of tumor resection, and other pretreatment factors. Recent molecular studies demonstrate molecularly defined tumor heterogeneity that appears to have a region-specific pattern. Translating the emerging molecular profiles of ependymomas into improved treatment strategies is the primary goal of ongoing research efforts.

scholarly journals P08.01 Impact of cannabis use on quality of life in patients with central nervous system tumors

2019 ◽  
Vol 21 (Supplement_3) ◽  
pp. iii37-iii37
Author(s):  
J E Rodriguez Almaraz ◽  
N A Butowski ◽  
S M Chang ◽  
N A E Oberheim-Bush ◽  
J R Clarke ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Central Nervous System ◽  
Nervous System ◽  
Brain Tumors ◽  
Global Health ◽  
Cannabis Use ◽  
Central Nervous System Tumors ◽  
Nervous System Tumors ◽  
The Mean

Abstract BACKGROUND Nearly 80,000 new cases of primary brain tumors are expected to be diagnosed this year in the United States: 32% of CNS tumors are malignant. Anecdotally, patients who report use of cannabis, frequently describe higher quality of life scores (QOL) in standardized instruments. However, the lack of available tools that allow systematic documentation of cannabis use results in a barrier to accurately assess efficacy and potential benefits and risks. MATERIAL AND METHODS We conducted a single center, observational study: patients with primary brain tumors answered a previously validated instrument to explore cannabis use. QOL was assessed using the instruments from the European Organisation for Research and Treatment of Cancer(EORTC): QLQ-C30 and its complementary module BN-20 as well as the EuroQol group’s instrument EQ-5D-5L. Eligible participants were identified as cannabis users or non-users, completing the instruments in a self-administered fashion. RESULTS To date, 45 patients who signed informed consent were enrolled and answered the questionnaires, mean age was 51 (SD 13.5) years, 31 were male, 25 were considered active cannabis users (624% males and 36% females). At baseline, the mean Global Health score in the QLQ-C30 instrument was 68.6 (SD: 20.6) among cannabis users and 82 (SD: 18.05) among non-users. The mean difference in Global Health QOL scores between users and non-users was 13.35 (95%CI: 1.34, 25.35; p=0.03). In contrast the difference between cannabis users and non-users in QOL index in the EQ-5D-5L instrument was 0.13 (0.77 vs 0.91; p=0.002). Among cannabis users, patients perceive their symptoms as moderate before using cannabis and mild after using cannabis (p>0.001). CONCLUSION In our analysis, patients who use cannabis have, on average, lower QOL scores signaling that sicker patients resort to cannabis to improve their symptoms and ultimately their quality of life. Patients’ perception is one of improvement in the overall quality of life when using cannabis. Our findings provide background support to perform prospective studies in the impact of cannabis in quality of life of patients with central nervous system tumors.

scholarly journals LINC-40. VERY YOUNG PATIENTS AND CENTRAL NERVOUS SYSTEM TUMORS: A SINGLE-CENTER EXPERIENCE IN AN UPPER-MIDDLE-INCOME COUNTRY

2020 ◽  
Vol 22 (Supplement_3) ◽  
pp. iii386-iii386
Author(s):  
Claudia Madrigal-Avila ◽  
Alfonso Perez-Bañuelos ◽  
Martin Perez-Garcia ◽  
Rafael Ruvalcaba-Sanchez ◽  
Lourdes Vega-Vega ◽  
...  
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Pediatric Population ◽  
Young Patients ◽  
Cns Tumors ◽  
Central Nervous System Tumors ◽  
Mortality And Morbidity ◽  
Nervous System Tumors ◽  
Tertiary Center ◽  
The Impact

Abstract Tumors of the central nervous system comprise nearly a quarter of all childhood cancers and are the most frequent solid tumor in the pediatric population. Primary central nervous system tumors (PCNST) are a rare and heterogeneous group of tumors responsible for high mortality and morbidity. Around 10% of primary CNS tumors occur during the first year of life with almost half of them during the first six months. About 18% of these tumors appear before the age of two years. Very young children differ from older children and adolescents regarding the incidence and location of different histological entities of CNS tumors. We aimed at providing descriptive epidemiological data and report the outcome in a tertiary center from December 2013 to January 2020 for all histological subtypes of primary central nervous system tumors in very young patients, defined as patients younger than three years. We collect data from 19 patients treated in an oncology exclusive tertiary center in Mexico between 2013 and 2020. This study aims to relate factors such as age, radiotherapy, surgery, chemotherapy with Lansky Performance Scale and determine the impact, not only in the overall survival but also in the quality of life.

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