bluuebunny/crossref_metadata_2025_split huggingface dataset:
SURG-04. COMPARABLE SAFETY OF INTRATHECAL CHEMOTHERAPY GIVEN VIA LUMBAR PUNCTURE OR INTRAVENTRICULAR RESERVOIRS FOR PEDIATRIC CENTRAL NERVOUS SYSTEM (CNS) TUMORS: A SINGLE-INSTITUTION EXPERIENCE
Authors: Richmond Caleb , Zimmerman Mary Ann , Chordas Christine , Helt Jessica , Fitzgerald Caroline , Matson Kathryn , Wright Karen D , Yeo Kee Kiat , Chi Susan N , Rosenberg Tom 
Published: June 2024

Abstract

BACKGROUND
Intrathecal (IT) delivery of chemotherapy is utilized for the treatment of pediatric cancers, particularly leukemia and CNS tumors, either via lumbar puncture (LP) or intraventricular reservoir (IR). While the latter allows easier administration and more frequent IT dosing, data are lacking on the comparative safety and efficacy between these administration routes. We now present the IT chemotherapy experience from a single high-volume pediatric neuro-oncology center.

METHODS
We performed a retrospective chart review of patients with primary CNS tumors treated with IT chemotherapy between the years 2004-2022 at Dana-Farber/Boston Children’s Hospital. We collected demographic, clinical and outcome data. Safety evaluation included review of immediate (procedural) and short-term post-procedural assessments.

RESULTS
We identified 74 patients who received a total of 1,211 IT chemotherapy administrations. Excluding patients with insufficient data, we report on 38 patients who received 717 administrations (LP, n=207; IR, n=510). Median age was 26.5 months (range, 0.1-14.5), and diagnoses included medulloblastoma (n=14), ATRT (n=14) and others (n=10). Immediate events were reported in 11 (0.2%) IR and 1 (0.4%) LP administrations, i.e technical (n=5), high ICP (n=3), and immediate adverse side effects (n=3). Short-term events were reported in 16 (3%) IR and 12 (5.8%) LP administrations, i.e. fever (n=6), seizures (n=6), arachnoiditis (n=4), and others (n=10). No grade 4-5 adverse events were reported. No statistically significant difference was found between routes of administration and the rate of immediate or short-term events. Of note, 14/18 (78%) patients with CSF-positive disease achieved CSF clearance, with median time to response of 57 days (range, 27-446).

CONCLUSIONS
IT chemotherapy shows an excellent safety profile in children with malignant CNS tumors, with no differences between IR and LP administration. IT delivery can be efficacious in CFS clearance for patients with metastatic disease, lending support to inclusion of IT chemotherapy in current treatment regimens.
SURG-06. FRAMELESS STEREOTACTIC INTRACRANIAL CATHETER PLACEMENT FOR DELIVERY OF INTRATUMORAL CELLULAR THERAPIES
Authors: Murugesan Rakesh A , Tucker Alexander , Kline Cassie , Foster Jessica , Madsen Peter 
Published: June 2024

Abstract

BACKGROUND
As the leading disease-related cause of death in children, pediatric brain tumors, and in particular diffuse midline glioma, require novel treatment approaches. Cellular-based therapies offer promise, but challenges still exist in defining their optimal delivery route. Emerging data from our group suggest there may be a role for intratumoral injection in treatment algorithms. We here describe a surgical workflow for intratumoral injection of cellular therapies that utilizes existing and widely used neurosurgical technologies that can be incorporated into future clinical trials.

METHODS
Infusion target and catheter trajectory was planned using standard pre-operative MRI. Components from an FDA-approved frameless stereotactic intracranial biopsy system were chosen such that they would accept and guide an FDA-approved rigid intracranial catheter system intended for MR-guided procedures. Following catheter insertion, an intraoperative CT was used to determine accuracy of catheter placement by merging the image with the pre-operative MRI.

RESULTS
The proposed system was tested in a simulated fashion using a phantom cranial model registered to an accompanying brain MRI. The stereotactic biopsy mount was affixed to the skull at the planned entry site for a posterior fossa approach to the pons. Catheter insertion depth was determined using the stereotactic system and the catheter was inserted to this depth through the biopsy mount. Intraoperative CT confirmed the catheter tip location which correlated with the planned infusion target with sub-millimetric accuracy.

CONCLUSION
Our proposed intratumoral delivery method using frameless stereotaxy shows promise for use in future clinical trials that require intratumoral delivery of cellular therapies. Utilizing FDA-approved devices allows for quick integration into protocols and their wide-spread use makes the approach generalizable. This approach also eliminates the need for MR-guided procedures which can increase cost and require significant hospital resources to perform. Future work will be aimed at streamlining the intraoperative workflow.
NURS-02. THE DEVELOPMENT AND IMPLEMENTATION OF SKINCARE TREATMENT PLANS AND PRODUCT KITS FOR PEDIATRIC PATIENTS RECEIVING TARGETED THERAPIES THAT CAUSE SKIN TOXICITY
Authors: Meyer Ashley S , Ogle Andrea , Mchugh Michele , Beck Mary , Lampe Ashley , Cluster Andrew , Brossier Nicole M , Shatara Margaret , Rocco Abbey , Abdelbaki Mohamed S , Coughlin Carrie C 
Published: June 2024

Abstract

BACKGROUND
Mitogen-activated protein kinase (MAPK) pathway inhibitors are incorporated into the treatment regimens for several pediatric cancers. However, they commonly trigger dermatologic side effects, which can be challenging to manage and may have a significant impact on the quality of life.

METHODS
We previously performed a prospective study looking at the satisfaction and utilization of skincare kits and patient education regarding basic skincare practices. Based on these data, we developed revised skincare product kits and treatment plans that can be individualized for patients who are at risk of developing dermatologic side effects from targeted therapies.

RESULTS
Based on our prior study, 77% of patients thought the skincare kits and instructions were helpful. Seventy-seven percent of patients also recommended that they be distributed at the initiation of therapy. Through this study, we discovered that the patient reported medical history questions were inconsistent when compared with medical chart reviews. The most common discrepancies included information regarding patient’s diagnosis, prescribed medications, skin-related problems, and past medical history. From this data, we have developed a new skincare product kit and patient skincare plans which we distribute to every patient prior to starting their targeted therapy. The skincare kits consist of a cleanser, emollient, oral care products, sunscreen, pyrithione zinc containing shampoo, and facial moisturizers for adolescent patients. Our patient skincare plans consist of patient-specific information including diagnosis, medications, and their targeted agent. The plan provides details on how and when to use each product, application time, and order in which to apply products and topical medications.

CONCLUSION
Our project has highlighted the importance of patient education regarding dermatologic side effects in those starting targeted therapies. We have successfully implemented this by providing our patients with both skincare products and patient-specific care plans.
NURS-03. NEED FOR PROGRAMS FOCUSING ON CAREGIVERS FOR YOUNG ADULT SURVIVORS OF CHILDHOOD BRAIN TUMORS
Authors: Deatrick Janet A , Joseph Melanie , Herchline Emily , Hammer Shannon , Anil Ashley , Ramaswamy Nithya , Hobbie Wendy , Minturn Jane E , Barakat Lamia P 
Published: June 2024

Abstract

BACKGROUND
The physical, emotional, social, developmental, and economic risk experienced by survivors of childhood brain tumors and their families is well documented, as is the acceleration of those risks as survivors transition into young adulthood. Further, the impact of the lack of services in the community is compounded by insufficient investigation of interventions to support survivor functioning and their caregiver’s management of their medical and psychosocial needs.The purpose of this study was to describe maternal caregivers’ perspectives about key components of potential interventions to support maternal caregivers of young adult survivors of childhood brain tumors.

METHODS
Data were obtained from 21 at-risk maternal caregivers (based on the Family Management Style Measure) of young adult survivors aged 18-29 years old who were not partnered, were presently living at home, and who participated in a pilot randomized trial Training in Problem Solving (TIPS) for Caregiver of Young Adult Survivors of Childhood Brain Tumors. Caregivers were interviewed using a semi-structured qualitative interview guide constructed using the Consolidated Framework for Implementation Science. Codes, categories, then themes were identified by means of directed content analysis.

RESULTS
All caregivers agreed these services were absent and the most effective timing would be at the transition to survivorship with ongoing follow-up. Key components of potential interventions identified by maternal caregivers were skill-building, motivational interviewing to increase readiness to change long-standing patterns of family management, use of validation of their efforts as parents and caregivers to build confidence in trying new approaches and shifting their perspectives on ongoing caregiving for a childhood brain tumor survivor to find hope in even small changes.

CONCLUSIONS
Caregivers endorsed the importance of targeted psychosocial support throughout survivorship for caregivers within the context of their family, facilitating their roles as both parents and caregivers to young adult childhood brain tumor survivors.
NURS-10. DEVELOPMENT OF A NATIONAL PAEDIATRIC NEURO-ONCOLOGY NURSES GROUP (UNITED KINGDOM)
Authors: McAndrew Rachel , Laughlin Elizabeth , Page Joanne , Paisley Helen , Howat Angela , Coyle Paul 
Published: June 2024

Abstract

BACKGROUND
The purpose of developing this group is to provide neuro-oncology patients with a high standard of care from diagnosis, treatment and beyond that is research based and reflects individual needs. This forum allows nurses working within paediatric neuro-oncology to share knowledge, experience, collaborate on projects, benchmarking and research opportunities. This will allow paediatric neuro-oncology nurses to increase their expertise and to foster relationships and collaborate with the wider neuro-oncology field and multi-disciplinary team.

METHODS
Develop an affiliation with a national association or organization Develop links to ensure all neuro-oncology nurses within CCLG centres are aware of the group, have the opportunity to join, fostering collaboration and support. Develop awareness of the neuro-oncology nursing speciality Develop a mission statement, terms of reference, and rolling agenda Literature reviews to ensure group has similar operation in line with other national nurses’ groups. Developing accountability.

RESULTS
Developed database of national neuro oncology nurses with all CCLG UK and Ireland centres represented. Set frequency of meetings and agendas. Ensure it operates to the same standard as other nurses’ groups. Organised working groups for projects. Agreed role of chair.

CONCLUSION
These activities have continued the development of the group since it’s conception in 2015 providing more structure, continued support to its members and wider recognition of its work. A robust mission statement, terms of reference, organisation and structure will ensure continued provision of education and support to neuro-oncology nurses, recognition of the group to the wider neuro-oncology community and improve overall patient experience.
NURS-16. CREATING A STANDARDISED DOCUMENT FOR EDUCATION SETTINGS ACROSS THE UNITED KINGDOM FOR CHILDREN WITH CENTRAL NERVOUS SYSTEM TUMOURS
Authors: Hayes Melissa , Blount Nicola , Robinson Louise , Reynolds Gabrielle , Ryles Jade 
Published: June 2024

Abstract
There are currently nineteen Principle Treatment Centres (PTC) in the UK delivering care to children, aged 0-19, following diagnosis of a Central Nervous System (CNS) tumour. Every centre promotes inclusivity and the safe and successful return to education, however reintegration can be a challenging process for this complex group of patients and there is a vast amount of information available. At present each PTC provides their own personalised document, including a list of resources, to signpost the education setting to appropriate information. With the collaboration of participating PTC’s, our aim was to create a standardised document for education settings to utilise following a child’s CNS diagnosis. Each of the nineteen PTC’s were approached, requesting representation from a neuro oncology nurse specialist and the sharing of their bespoke information. Following this, benchmarking criteria were created and each document reviewed and evaluated. The review and evaluation process also included websites and information leaflets that centres currently signpost to, from charities and other relevant organisations. By working closely together as a national group of neuro oncology nurses, we have created a standardised document and list of suitable resources for education settings. The finalised document will be shared with each PTC, with the intention of promoting a smooth transition back into education and quality and equity of information for all children, irrespective of where they live in the UK. Auditing of the document will be part of the ongoing work of the group.
NURS-21. EXPLORATION OF CORRELATIVE STUDIES IN EARLY PHASE TRIALS FOR PEDIATRIC AND YOUNG ADULT CENTRAL NERVOUS SYSTEM (CNS) TUMORS AND THE POTENTIAL TRANSLATION TO CLINICAL CARE
Authors: Seidl Ken , Briamonte Courtney Baker , Higgins Micah , Hoffman Carly , Holtzlaw Susan , McHugh Michele , Meyer Ashley , Rangan Kasey , Schmus Cindy , Strachan Marylynne , Waters Kaaren , Yeh-Nayre Lanipua , Kline Cassie , Prados Michael , Mueller Sabine , Raber Shannon 
Published: June 2024

Abstract

BACKGROUND
Correlative studies are common in early phase clinical trials. In addition to primary aims establishing maximum tolerated dose or efficacy, supplementary information from biospecimens is frequently collected with patient consent. The Pediatric Neuro-Oncology Consortium (PNOC) has included several correlative studies with implications for understanding patterns in the development of tumors, response to treatment, and prognosis. A needs assessment survey of PNOC institutions by the Advanced Practice Provider and Investigational Nurse Committee (APPNIC) highlighted that nurses and advanced practice providers (APPs) desire further education regarding correlative studies.

METHODS
Blood, stool, cerebrospinal fluid (CSF) and hair strands are examples of correlative biospecimens incorporated in PNOC protocols. We will share a review of the literature and implications for practice.

RESULTS
Blood, drawn in conjunction with clinical labs, is commonly analyzed for circulating tumor DNA to conduct molecular profiling in blood compared to tissue, explore less invasive means of molecular evolution of tumor somatic mutations, and identify early markers of tumor response or resistance. Stool is collected to utilize the gut microbiome as a lens into treatment response in CNS tumors via the gut-brain axis. Stool samples are compared to normal controls and longitudinally within patients during treatment. CSF sampling provides less invasive disease monitoring, tumor molecular characterization, markers of tumor response or resistance, and drug pharmacokinetics. Hair sampling can be used to further quantify drug levels for pharmacokinetics and assess treatment compliance. The robustness of these correlative samples collected at various timepoints (diagnosis, pre-surgery and post-surgery, post-radiation and/or with recurrence) informs on prognosis, response to treatment; and ultimately, interventions to improve outcomes for pediatric and young adult CNS tumors.

CONCLUSION
Our assessment identified a need for APP and nursing-focused education on biospecimen collection to inform on emerging clinical and research advances while improving protocol compliance.
OTHR-01. ALK-DRIVEN INFANT-TYPE HEMISPHERIC HIGH-GRADE GLIOMA AND CNS HISTIOCYTOSIS SUCCESSFULLY TREATED WITH A SECOND GENERATION ALK-INHIBITOR: MOLECULAR CHARACTERIZATION SAVES THE DAY
Authors: Chamdine Omar , Alotaibi Rasees , Alharbi Qasim , Altarrah Hiba , Abduljabbar Lulwah , Alzaher Asrar , Taha Mahmoud , Alsaiari Sultan , Alisawi Wissam , AlSadeq Jalilah , Abdulkader Marwah , Alhaddad Fatima 
Published: June 2024

Abstract

BACKGROUND
Molecular characterization of brain tumors (BT) is increasingly becoming essential for diagnosing and managing central nervous system (CNS) tumors. Infant BT is a particular category of pediatric CNS tumors that poses a challenge to the neuro-oncologist.

METHODS
Here, we present two distinctive cases where molecular characterization significantly influenced diagnosis, management, and outcome.

RESULTS
The first case is a 2-year-old boy presented with generalized seizures. MRI of the brain showed multiple dural-based tumors. Biopsy was consistent with ALK-positive histiocytosis (KIF5B-ALK fusion). Additional lesions were detected in the right kidney and left femoral head. Despite three months of LCH-based therapy, the tumor progressed. Chemotherapy was halted, and treatment with alectinib was initiated. Follow-up MRIs after 3 and 6 months showed marked regression and a complete resolution of the lesions, respectively. The second case is a 12-month-old boy who exhibited developmental regression and increased head circumference over the past three months. MRI revealed a large complex right ventricle mass measuring 8.6x7.2x8 cm. Subtotal resection was complicated by severe intracranial bleeding, requiring prolonged intensive care stay. The initial pathological diagnosis was anaplastic ependymoma. He received two cycles of neoadjuvant chemotherapy followed by a second look surgery, which failed to achieve a gross total resection. Molecular testing by this time identified an ALK-CCDC85A fusion, altering the diagnosis to infant-type hemispheric glioma. He was started on alectinib. MRI after 3 and 6 months showed a favorable response with an overall improvement in his cognitive function. Both patients tolerated alectinib well, remaining on therapy for 6 and 12 months, respectively. No toxicities were reported.

CONCLUSION
These cases illustrate the transformative role of molecular characterization in managing infants with CNS tumors, highlighting its potential as a game-changer in pediatric neuro-oncology.
OTHR-03. DEVELOPMENT OF PANORAMA: AN ASSESSMENT TOOL FOR PEDIATRIC NEURO-ONCOLOGY SERVICE DELIVERY CAPACITY
Authors: Rajagopal Revathi , Diaz-Coronado Rosdali , Hamid Syed Ahmer , Campo Regina M Navarro-Martin del , Boop Frederick , Bag Asim , Reséndiz Alma Benito , Bhat Kalya Vasudeva , Campos Danny , Chang Kenneth T E , Cirt Ramona , Rahmartani Ludi Dhyani , Foo Jen Chun , Hoveyan Julieta , Lucas Jr. John T , Ngcana Thandeka , Ain Rahat Ul , Omran Nuha , Osorio Diana , Qureshi Bilal Mazhar , Sabin Noah , Schandorf Ernestina , Bankah Patrick , Dadzie Mary-Ann , Gbadamosi Hafisatu , Sharafeldin Hend , Somathilaka Mahendra , Yang Peiyi , Jean-Jacques Yao Atteby , Zhang Anan , Salman Zeena , Gonzalez Miriam , Friedrich Paola , Qaddoumi Ibrahim , Moreira Daniel C 
Published: June 2024

Abstract

BACKGROUND
To enhance the quality of care available for children with central nervous system (CNS) tumors across the world, a systematic evaluation of service delivery capacity is needed to identify gaps in pediatric neuro-oncology and prioritize interventions. To that end, we created the PediAtric Neuro-Oncology Resource AssesMent Aid (PANORAMA) tool.

METHODS
PANORAMA development consisted of three phases: operationalization, consensus building, and piloting. PANORAMA was designed to have domains that capture the elements of the pediatric neuro-oncology care continuum using assessments which were weighted according to their importance. Responses were ordinally scored to reflect adequacy of satisfaction. PANORAMA was revised based on feedback at various phases to improve its relevance, usability, and clarity.

RESULTS
The operationalization phase identified thirteen domains using 252 questions. The consensus phase included validating the identified domains, questions, and scoring by fifteen experts (six pediatric oncologists, three radiation oncologists, two neurosurgeons, two radiologists, and two pathologists). The validated PANORAMA domains were national context, hospital infrastructure, organization and service integration, human resources, financing, laboratory, neurosurgery, diagnostic imaging, pathology, chemotherapy, radiotherapy, supportive care, and patient outcomes. Seventy-six questions were added during the expert review. PANORAMA was piloted at thirteen institutions in twelve countries, representing diverse patient care contexts. Face validity was assessed using the correlation between the estimated score by respondents and calculated PANORAMA scores for each domain (r = 0.67, p &lt;0.0001). Piloting eliminated two questions.

CONCLUSIONS
PANORAMA was developed following a systematic approach, leading to a relevant tool to evaluate the core elements of pediatric neuro-oncology service capacity. PANORAMA will be distributed to facilitate quantitative evaluation of institutional services, allowing for prioritization and benchmarking with centers at the local, regional, and international levels.
OTHR-30. THE CLINICAL AND BIOLOGICAL LANDSCAPE OF CNS CANCERS IN CONSTITUTIONAL MISMATCH REPAIR DEFICIENCY: AN IRRDC COHORT STUDY
Authors: Ercan Ayse Bahar , Aronson Melyssa , Fernandez Nicholas R , Chang Yuan , Liu Zhihui Amy , Negm Logine , Edwards Melissa , Bianchi Vanessa , Villani Anita , Malkin David , Hawkins Cynthia , Bouffet Eric , Das Anirban , Tabori Uri 
Published: June 2024

Abstract

BACKGROUND
Constitutional mismatch repair deficiency (CMMRD) is an aggressive cancer predisposition syndrome. As a lack of data contributes to management challenges and inferior outcomes, especially in central nervous system (CNS) cancers, we aimed to determine the clinical spectrum, cancer biology, and impact of genetics on patient survival.

METHODS
To determine the cancer spectrum, biology, estimated incidence, and impact of genotype on cancer onset and survival, including following surveillance and immunotherapy, we collected cross-sectional, longitudinal, and genomic data on CMMRD patients and their cancers registered to an international consortium.

RESULTS
We report 201 patients enrolled between 2007-2022 (median follow-up: 7.2 years). Overall, 194 (97%) patients developed 339 cancers. Cumulative cancer and CNS tumor incidence were 93% and 82% by age 20, respectively. Median time between cancers was 2 years. Although neoplasms developed in 15 organs and included early-onset adult cancers, CNS tumors had the earliest median age of diagnosis (9.7 years), were the most common cancer type (51%), and the main cause of death (p&lt;0.0001). All cancers exhibited high mutation and microsatellite burden, and pathognomonic mutational signatures. CNS cancers were enriched for mutations in POLE/POLD1, TP53, IDH1, and RAS/MAPK compared to hematopoietic and other cancers. These mutations determined response to therapy. Germline MLH1/MSH2 variants caused earlier cancer and CNS tumor onset than PMS2/MSH6, and inferior survival (survival at 15 years: PMS2: 63%, MSH6: 49%, MLH1: 19%, MSH2: 0%, p&lt;0.0001). Strikingly, frameshift/truncating variants within the same gene caused earlier cancers and inferior outcomes (p&lt;0.0001). The deleterious impact of MLH1/MSH2 persisted despite overall improvement in survival following surveillance and immunotherapy.

CONCLUSION
The extreme cancer burden and unique genomic landscape of CMMRD highlight the benefit of comprehensive assays in timely diagnosis focusing on specific genotypes to perform precision approaches toward surveillance and immunotherapy. These data will guide the management of children and emerging adult survivors of CMMRD.