bluuebunny/crossref_metadata_2025_split huggingface dataset:
SURG-07. A PHASE I STUDY EXAMINING THE FEASIBILITY OF INTERMITTENT CONVECTION-ENHANCED DELIVERY (CED) OF MTX110 FOR THE TREATMENT OF CHILDREN WITH NEWLY DIAGNOSED DIFFUSE MIDLINE GLIOMAS
Authors: Szalontay Luca , CreveCoeur Travis , Neira Justin , Englander Zachary , Spinazzi Eleonora , Canoll Peter , Garvin James , Fino Jessica , Zamoryakhin Dmitry , Maddocks Alexis , Feldstein Neil , Bruce Jeffrey , Wu Cheng-Chia , Zacharoulis Stergios 
Published: June 2024

Abstract

BACKGROUND
Histone deacetylase inhibitors have been found preclinically to be among the most active agents against Diffuse Midline Gliomas (DMGs), however, they are clinically ineffective with systemic delivery due to low blood brain barrier penetration and systemic toxicity. Using an implantable subcutaneous pump connected with a catheter directly implanted into the pons we have conducted a phase I, 3 + 3 dose escalation study to investigate the safety and feasibility of repeated infusions of MTX110 (Biodexa Ltd), a water-soluble formulation of panobinostat, via CED.

METHODS
Eligible patents were between 3 and 18 years of age with newly diagnosed DMG following radiation therapy, without evidence of hemorrhage or cyst in the tumor, and having normal organ function. First, patients underwent tumor biopsy and device implantation, then received two 48-hour-infusion pulses 7 days apart. Three dose levels of MTX110 infusions (30, 60, 90 mcM) were studied. The infusion pump was prefilled with MTX110 (and gadolinium for co-infusion as a contrast agent), which was then administered using the wireless N’Vision clinical programmer at a rate of 0.2 mL/hr.

RESULTS
Nine patients were treated in the study (30 mcM group, n=3; 60 mcM group, n=4; 90 mcM group, n=2). All patients had an H3K27M mutated tumor. All but one patient had adequate tumor coverage with the infusate. One patient suffered a severe adverse event related to the infusion and tumor anatomy. Four patients had Grade 2 transient neurological deficits related to biopsy (n=1) or infusion (n=3). We observed one objective response. Median PFS was 10 months from diagnosis (range 8 to 20 months) while median OS was 16.5 months (range 12 to 35 months).

CONCLUSIONS
The study has demonstrated safety and feasibility of repeated infusions of MTX110 via CED in DMG patients with a favorable trend in overall survival warranting further trials with higher number of infusion pulses.
NURS-06. MUSIC THERAPY: AN INTEGRAL PART OF REHABILITATION FOR CEREBELLAR MUTISM SYNDROME
Authors: Matson Kathryn , Corr Gwendolyn 
Published: June 2024

Abstract

BACKGROUND
Cerebellar mutism syndrome (CMS) also known as posterior fossa syndrome (PFS), can occur in up to 25% of pediatric patients following surgical resection of posterior fossa brain tumors, particularly medulloblastoma. It is well recognized that intensive rehabilitation with physical therapy, occupational therapy and speech therapy are essential to recovery. Music therapy can be immensely helpful during rehabilitation to stimulate brain functions involved in movement, cognition, speech, emotions, and sensory perceptions. This case-based review aims at analyzing the available evidence to support our recommendation to include music therapy in the rehabilitation plan for all patients.

METHODS
We report on the case of a 4-year-old male with medulloblastoma and cerebellar mutism syndrome who began talking after just a few sessions with music therapy. We include an extensive review of the literature where multiple papers were found describing the positive effects of music therapy for management of neurologic diseases.

RESULTS
The pathophysiology of CMS is still poorly understood however, the most likely mechanism is injury along proximal components of the efferent cerebellar pathway. Intensive inpatient rehabilitation remains the standard of care. Music therapy uses and activates many different parts of the brain at the same time, promoting neuron repair and regeneration. Some of the advantages of music therapy are: it promotes plasticity in the brain and facilitates processing of sensory information and motor function; improves stress and helps patients adapt to stressful environments; facilitates more normative speech production for patients with dysarthria.

CONCLUSIONS
Despite efforts to identify preventative measures for CMS, it still occurs. Intensive rehabilitation remains the cornerstone of treatment. The case study as well as the review of the literature support our recommendation to include music therapy in the rehabilitation plan for patients with CMS. More studies are necessary to explore the therapeutic efficacy of music on patients with CMS.
NURS-07. A NEEDS ASSESSMENT OF THE BRAIN TUMOR EDUCATION AND THERAPY (BEAT) GROUP AT WASHINGTON UNIVERSITY SCHOOL OF MEDICINE IN ST. LOUIS
Authors: McHugh Michele , Meyer Ashley , Ogle Andrea , Beck Mary , Lampe Ashley , Brossier Nicole M , Cluster Andrew , Rocco Abbey , Shatara Margaret , Abdelbaki Mohamed 
Published: June 2024

Abstract

BACKGROUND
The Brain Tumor Education and Therapy (BEAT) group extends support to patients and families with pediatric brain tumor diagnoses. Social Work and healthcare providers typically meet with families quarterly to provide psychosocial support and address their educational needs. To better understand the factors that may impact participation and engagement, we sought to evaluate the opinions of current patients and their families.

METHODS
Both caregivers and adolescent patients, aged 16 years or older, were invited to participate in a 25-question survey. The survey assessed current sources of support, preferred meeting format, as well as educational and psychosocial needs. Additionally, the survey included questions about the patient’s diagnosis, treatment, and side effects experienced.

RESULTS
Over six weeks, 28 surveys were completed. Almost all respondents reported receiving support from family (93%) and friends (78%), while a smaller percentage (21%) sought support from families with similar diagnoses. Nearly half (46%) of respondents follow online or social media groups. When it comes to receiving support, 47% preferred online sessions (social media or virtual meetings), compared to in-person meetings (16%) or a combination of online and in-person (24%). Most respondents (85%) reported educational needs related to the late effects of therapy, while 69% cited concerns about friendships and socialization, and 57% on transitioning to young adulthood. Sixty-two percent request resources for emotional and psychosocial support. Respondents prefer (87%) healthcare professional-led sessions.

CONCLUSION
Our findings highlight an ongoing demand for healthcare professionals to provide psychosocial support and education to families of brain tumor patients. However, the preference is shifting towards virtual attendance of these meetings. In response, we intend to introduce a virtual support group and educational sessions, complemented by informative videos and handouts available on our hospital website or social media platforms. Subsequent studies will focus on family satisfaction with changes to the program.
NURS-08. CLINICAL MANAGEMENT OF PATIENTS RECEIVING CAR T CELL THERAPY FOR CNS TUMORS: FIVE YEAR UPDATE
Authors: Holtzclaw, ARNP Susan , Hoeppner, ARNP Cory 
Published: June 2024

Abstract

BACKGROUND
Chimeric antigen receptor (CAR) T cells are an innovative new therapy with proven efficacy in some pediatric cancers such as leukemia and lymphoma, but there has been less experience in solid tumors, especially tumors of the central nervous system (CNS). Seattle Children’s Hospital (SCH) has opened a total of four phase 1 CAR T cell studies for recurrent/refractory CNS tumors and DIPG. These include BrainChild-01; targeting HER2, BrainChild-02; targeting EGFR, BrainChild-03; targeting B7-H3, and BrainChild-04; targeting B7H3, EGFR, HER2, and IL13-zetakine (QUAD). As of February 2024, 105 patients have been treated at SCH with CAR T cells infused on a weekly to bi-weekly schedule through indwelling catheters into the tumor resection cavity or ventricular system. APPs provide much of the day-to-day care of these patients and have learned best management practices for these patients.

METHOD
Given the scrutiny of clinical care needed for Phase 1 studies, we are now able to report detailed clinical information around management of patients receiving CNS immunotherapy that we have learned during the treatment of these patients over the past five years.

RESULTS
Clinical care includes the judicious use of steroids, clinical support of each patient’s symptoms, and management of Ommaya catheters and reservoirs. Additionally, providing psychosocial support is essential for families who travel long distances to receive this therapy compounded by the many emotional components of being enrolled on any Phase 1 trial.

CONCLUSION
Case studies will be provided and discussed to provide the most up-to-date management of patients receiving CNS Immunotherapy. With now over 550 CAR T cell infusions completed, our discussion will provide recommendations for management of side effects from CAR T cell therapy and discuss clinical standard around accessing Ommaya catheters and shunt reservoirs for immunotherapy infusions.
NURS-14. DEVELOPMENT OF A CLINICAL PRACTICE INTERVENTION TO SUPPORT NURSES CARING FOR PATIENTS WITH DIABETES INSIPIDUS (DI) DURING CHEMOTHERAPY HYDRATION
Authors: Schmus Cynthia , DiDomenico Concetta , McWhorter Jessica 
Published: June 2024

Abstract

BACKGROUND
Pediatric neuro-oncology patients with germ cell tumors (GCT) and diabetes insipidus (DI) often require management with vasopressin (DDAVP). Fluid and sodium balance, ordinarily managed successfully with DDAVP, can be disrupted during chemotherapy and associated hydration. Patients with an intact thirst mechanism may be able to self-regulate fluid and salt balance under the right conditions, whereas patients without an intact thirst may experience sodium fluctuations if intravenous hydration exceeds the amount they drink independently. Patients may be unable to maintain adequate hydration and sodium levels due to nausea and/or sedating antiemetics. These factors create a unique clinical challenge for nurses. Nurses sought guidance in understanding DI and its impact on hydration during chemotherapy.

METHODS
Two nursing education sessions were held and focused on DI management. Nurses were surveyed pre and post education. The goals of the intervention were that the oncology nurse will be to describe the pathophysiology of diabetes insipidus, the role of desmopressin, the ability to differentiate between management of patients with and without an intact thirst mechanism, and the special care of a DI patient receiving chemotherapy.

RESULTS
Nurses reported an increased awareness post education of the need to closely assess patients and manage their DI in collaboration with clinicians. Routine chemotherapy management does not apply to this population, because drastic fluctuations in sodium and water balance put them at risk of other complications. Nurses identified the need to closely manage hydration, DDAVP, recording strict intake/output, monitoring sodium levels, assessing for nausea/vomiting and the degree of sedation from antiemetics.

CONCLUSION
Outcomes from this educational intervention were that nurses had an increased understanding of DI management in patients with GCT receiving chemotherapy. Future goals would be to implement a standard approach to hydration, DDAVP administration, and monitoring sodium levels in patients with GCT and DI.
OTHR-05. ROLES OF GEMCITABINE-OXALIPLATIN (GEMOX) ADD-ON THERAPY IN INFANT-TYPE BRAIN TUMORS: A SINGLE CENTER EXPERIENCE
Authors: Castelli Barbara , Fonte Carla , Guidi MIlena , Nicola Marco Di , Giunti Laura , Buccoliero Anna Maria , Caporalini Chiara , Giordano Flavio , Scagnet Mirko , Tintori Veronica , D’Incerti Ludovico , Genitori Lorenzo , Tellini Marco , Sardi Iacopo 
Published: June 2024

Abstract

BACKGROUND
Combined gemcitabine and oxaliplatin (GemOx) was evaluated in recurrent pediatric solid tumors, showing safety but limited activity. In neuro-oncological infant patients, chemotherapy intensified to high doses (HDC) represents the cornerstone, avoiding radiotherapy toxicities. We describe the roles of GemOx towards autologous transplantation in infant-type brain tumors.

METHODS
From May 2017 to January 2024, at the Meyer Children’s Hospital IRCCS in Florence, six children (median age 19 months, sex equally distributed, three high-grade gliomas, a metastatic medulloblastoma MM, a pineoblastoma, a choroid plexus carcinoma CPC) completed GemOx after induction chemotherapy delivered according to Italian program for malignant brain tumors under 3 years. GemOx was administered to achieve clinical and neuroradiological permissiveness to HDC and to raise the possibility of second look neurosurgery.

RESULTS
Gemcitabine 1000 mg/m2 and Oxaliplatin 100 mg/m2 were given every 14-28 days for 1-6 cycles. At re-assessment, maximum every four cycles, disease control was optimal (five stable diseases and one partial response according to RECIST criteria). After GemOx, one patient was also subjected to further neurosurgery. The treatment was overall well tolerated, except for Common Terminology Criteria for Adverse Events (CTCAE) grade 4 platelet toxicity in two patients (one of whom also developed hydrocephalus CTCAE 3 and hyponatremia CTCAE 4 during treatment). All patients were safely exposed to HDC (1-2 courses). MM and CPC patients progressed respectively 4 ad 19 months after the end of GemOx. The latter underwent second look neurosurgery, and a radiotherapy course was delivered to both, considering the achievement of permissive age. To date all patients are alive.

CONCLUSIONS
In brain infant-type malignant tumors strategies are limited due to the extremely vulnerable age. GemOx treatment should be considered a reasonable and safe option waiting for intensified treatment, especially in radiotherapy delaying programs, and it should result incisive in the therapeutic or palliative choice.
OTHR-09. HEALTHCARE PROVIDERS’ PERSPECTIVES ON MALAYSIA-SINGAPORE VIRTUAL PEDIATRIC NEURO-ONCOLOGY TUMOR BOARD MEETING
Authors: Rajagopal Revathi , Foo Jen Chun , Teo Jillian Chui Lin , Sawal Rafikah , Jawin Vida , Alcasabas Ana Patricia , Ho Thi Kim Nguyen , Loh C-Khai , Bansal Shweta , Teh Kok Hoi , Low Sharon , Ganesan Dharmendra , Chang Kenneth , Soh Shui Yen , Abdelbaki Mohamed S , Bouffet Eric , Gottardo Nicholas G , Tan Enrica 
Published: June 2024

Abstract

BACKGROUND
Virtual pediatric neuro-oncology tumor board meetings have been reported to be highly beneficial in improving the delivery of high-quality care for childhood central nervous system (CNS) tumors. However, such meetings are lacking in the Asia Pacific region. Hence, we initiated the first pediatric neuro-oncology online platform in the Asia Pacific region in January 2019, and we aimed to describe the participants’ perception of the virtual tumor board meetings.

METHODS
We retrospectively reviewed all cases from January 2019 to December 2023 and conducted a web-based survey to all participants in December 2023. Centers that presented more than five cases were invited to assess the impact of virtual meetings.

RESULTS
Fifty-four meetings have been conducted over five years. A total of 160 cases were presented by 20 institutions from six Asian countries, which include Malaysia, Singapore, Philippines, Vietnam, India, and Taiwan. Thirty participants responded to the survey. Majority of them attended the meetings to enhance their knowledge in pediatric neuro-oncology (96.7%), to learn evidence-based management (83.3%), and to build connections with regional colleagues and international experts (53.3%). Almost all concluded that tumor board meetings improved treatment decisions, addressed challenges of complex CNS tumor cases, and promoted continuity of care with appropriate post-surgical chemotherapy administration and surveillance investigations after completion of treatment. The main reason for case discussion was diagnostic difficulties followed by multimodal treatment advice (85.7%). More than 25% of management changes were observed in pathological diagnosis and treatment approaches. All the hospitals reported that the online case discussions improved pediatric neuro-oncology service delivery capacity and increased local team competence.

CONCLUSION
Virtual pediatric neuro-oncology tumor board meetings facilitated improvement in pathological diagnosis and multimodal treatment approach. The establishment of virtual CNS tumor board meetings is an effective way to seek international expert advice and promote pediatric neuro-oncology education in our region.
OTHR-11. TITLE: ELECTROGRAPHIC SEIZURES AND EEG BACKGROUND FEATURES IN PEDIATRIC NEURO-ONCOLOGY PATIENTS ADMITTED TO THE PEDIATRIC INTENSIVE CARE UNIT
Authors: Pickup Elizabeth E S , Keenan Julia S , Packer Roger J , Wells Elizabeth M , Conley Caroline , Staso Katelyn , Harrar Dana B , Sansevere Arnold J 
Published: June 2024

Abstract

BACKGROUND
Electrographic seizures (ES) are common in critically ill children with the incidence of ES ranging from 10-50%. Continuous EEG (cEEG) is frequently obtained in children with brain tumors due to encephalopathy or concern for seizures. Despite this, little is known about the incidence of ES, EEG background features, or the impact of EEG on clinical management in this population.

METHODS
This is a prospective single center study of critically ill children with brain tumors admitted to the pediatric intensive care unit (PICU) from July 2021 to July 2023. Patients with new or known primary brain tumors and cEEG completed during their admission were included. We excluded patients with planned admissions for EEG. Clinical variables included age, sex, oncologic diagnosis, tumor grade and location, indication for EEG, EEG features, and changes in management.

RESULTS
Thirty-six patients were identified who met the inclusion criteria. 21 patients were female. The median age was 11.0 years (IQR 6.6– 15.4 years). The most common indication for EEG was encephalopathy in 42% followed by clinical seizure in 31%. ES were captured in 19% (7/36) with the majority being subclinical; in patients with acute encephalopathy, ES were captured in 20% (3/15). The EEG background was abnormal in 97% of cases. The most common EEG background was slow disorganized with background asymmetry seen in in 74%. EEG background features met criteria for the ictal-interictal continuum in 14%. A higher rate of ES was seen in patients with disseminated tumors (p=0.03) and severe encephalopathy on EEG (p=0.006). EEG led to a change in management in 31% of patients.

CONCLUSIONS
ES are common in critically ill children with brain tumors and would go undetected without cEEG. EEG findings often led to a change in clinical management and is a vital tool for the evaluation of this population.
PALC-03. MEDICAL AID IN DYING: EXPERIENCE IN THE CENTER FOR CANCER AND BLOOD DISORDERS AT CHILDREN’S HOSPITAL COLORADO
Authors: Winzent-Oonk Shelby , Vallejos Chelsea , Hemenway Molly , Lindsay Holly B , Green Adam L 
Published: June 2024

Abstract

BACKGROUND
Medical Aid in Dying (MAiD) is now legal in 10 US states and Washington, DC, allowing terminally ill patients to self-administer medications to peacefully end their life. There is little published on pediatric hospitals’ approach to young adult patients requesting MAiD.

METHODS
We review the two patients who have participated in MAiD at the Center for Cancer and Blood Disorders at Children’s Hospital Colorado (CHCO). We also explore publicly available Colorado MAiD statistics.

RESULTS
Between 2017 and 2022, there have been 1,090 prescriptions written for MAiD medication in Colorado, 5 of which were for patients aged 18-34 (0.5%). Two of these patients were treated at CHCO. Patient A was a 26 year old male with a CNS non-germinomatous germ cell tumor whose treatment was complicated by mucormycosis and severe engraftment syndrome. He relapsed 23 months after completing therapy. He had neurologic decline including ataxia and blindness, at which time he requested MAiD. Patient B was a 24 year old male with multiply recurrent IDH-mutant medullary high grade glioma. With metastatic progression, he had many tumor-related symptoms including dysphagia, dysarthria, and weakness impacting his activities of daily living, so he requested MAiD. Both patients had multidisciplinary care conferences with neuro-oncology, social work, ethics, psychology, and palliative care. After completing psychological evaluation and all steps required by law, they were prescribed DDAMP2 (digoxin 50 mg, diazepam 1 gram, morphine 15 grams, and propranolol 2 grams). Both patients took the medications and died peacefully. Barriers encountered included infrequency of MAiD requests leading to unclear policies and processes.

CONCLUSIONS
Young adults represent a small but important subset of those seeking MAiD prescriptions. Despite challenges, patients were able to receive MAiD without delay in a compassionate manner. Our experience may be applicable to other pediatric hospitals facing these requests.
PATH-14. A CLINICAL CONUNDRUM - INFANTILE POSTERIOR FOSSA EPENDYMOMA WITH YAP1:MAML FUSION?
Authors: Govender Dinisha , Goetti Robert , Kim Young-Jun , Graf Nicole 
Published: June 2024

Abstract

BACKGROUND
Ependymomas of infancy (0-18months) have been reported to comprise distinct clinical, pathological and genetic entities. Young age is typically associated with posterior fossa group A (PF-A) tumours and therefore a poor prognosis. Treatment of these infants is challenging with limited therapeutic options that have significant long-term sequelae.

METHOD
We report a single case of a 4-month old baby girl who presented with obstructive hydrocephalus and a presumed posterior fossa mass.

RESULTS
Imaging demonstrated a large fairly well-defined solid lesion with multiple T1 high and corresponding T2 intermediate signal intensity foci scattered throughout the lesion and marked mass effect on the cerebellum and fourth ventricle. The lesion was inseparable from the pineal gland, habenula and tectum and only mild diffusion restriction. Whilst initially considered to be a posterior fossa tumour, the presence of a claw sign with the tectal plate, favoured a tectal origin. Histological appearances, together with immunohistochemistry findings (GFAP positive and EMA patchy perinuclear dot expression) were consistent with classic ependymoma. Grading was difficult as mitotic activity was relatively low, although focal areas of high ki67 proliferation index were noted suggesting at least focal grade 3 areas. Retained expression of tumour cells for H3K27me3 on immunohistochemistry suggested the likely posterior fossa group B (PF-B) ependymoma. Local SNP chromosome microarray found no acquired clonal abnormality; with no detection of gain chromosome 1q; loss/LOH 6q; Loss/LOH 9p21.3 [CDKN2A, CDKN2B], chromothripsis 11q [ZFTA::RELA], Loss/LOH 17p13 [TP53] or Loss/LOH 22q [NF2]. Additional NGS sequencing identified a YAP1::MAML2 fusion (confirmed with targeted RT-PCR followed by subsequent Sanger sequencing).

CONCLUSION
This case highlights the challenges with overlapping clinical, pathological and radiological features in an infant with ependymoma, necessitating a molecular diagnosis. After 21months, the patient remains in complete remission following surgery alone.