International Group Of Experts Research Articles

Management of Metabolic-Associated Fatty Liver Disease/Metabolic Dysfunction-Associated Steatotic Liver Disease: From Medication Therapy to Nutritional Interventions.

Non-alcoholic fatty liver disease (NAFLD) is a common long-lasting liver disease that affects millions of people around the world. It is best identified with a hepatic fat build-up that ultimately leads to inflammation and damage. The classification and nomenclature of NAFLD have long been a controversial topic, until 2020 when a group of international experts recommended substituting NAFLD with MAFLD (metabolic dysfunction-associated FLD). MAFLD was then terminologically complemented in 2023 by altering it to MASLD, i.e., metabolic dysfunction-associated steatotic liver disease (MASLD). Both the MAFLD and the MASLD terminologies comprise the metabolic element of the disorder, as they offer diagnostic benchmarks that are embedded in the metabolic risk factors that underlie the disease. MASLD (as a multisystemic disease) provides a comprehensive definition that includes a larger population of patients who are at risk of liver morbidity and mortality, as well as adverse cardiovascular and diabetes outcomes. MASLD highlights metabolic risks in lean or normal weight individuals, a factor that has not been accentuated or discussed in previous guidelines. Novel antihyperglycemic agents, anti-hyperlipidemic drugs, lifestyle modifications, nutritional interventions, and exercise therapies have not been extensively studied in MAFLD and MASLD. Nutrition plays a vital role in managing both conditions, where centralizing on a diet rich in whole vegetables, fruits, foods, healthy fats, lean proteins, and specific nutrients (e.g., omega-3 fatty acids and fibers) can improve insulin resistance and reduce inflammation. Thus, it is essential to understand the role of nutrition in managing these conditions and to work with patients to develop an individualized plan for optimal health. This review discusses prevention strategies for NAFLD/MAFLD/MASLD management, with particular attention to nutrition and lifestyle correction.

A standard to report biological variation data studies- based on an expert opinion.

There is a need for standards for generation and reporting of Biological Variation (BV) reference data. The absence of standards affects the quality and transportability of BV data, compromising important clinical applications. To address this issue, international expert groups under the European Federation of Clinical Chemistry and Laboratory Medicine (EFLM) have developed an online resource (https://tinyurl.com/bvmindmap) in the form of an interactive mind map that serves as a guideline for researchers planning, performing and reporting BV studies. The mind map addresses study design, data analysis, and reporting criteria, providing embedded links to relevant references and resources. It also incorporates a checklist approach, identifying a Minimum Data Set (MDS) to enable the transportability of BV data and incorporates the Biological Variation Data Critical Appraisal Checklist (BIVAC) to assess study quality. The mind map is open to access and is disseminated through the EFLM BV Database website, promoting accessibility and compliance to areporting standard, thereby providing a tool to be used toensure data quality, consistency, and comparability of BV data. Thus, comparable to the STARD initiative for diagnostic accuracy studies, the mind map introduces a Standard for Reporting Biological Variation Data Studies (STARBIV), which can enhance the reporting quality of BV studies, foster user confidence, provide better decision support, and be used as a tool for critical appraisal. Ongoing refinement is expected toadapt to emerging methodologies, ensuring a positive trajectory toward improving the validity and applicability of BV data in clinical practice.

Leptomeningeal metastases from solid tumors: A Society for Neuro-Oncology and American Society of Clinical Oncology consensus review on clinical management and future directions.

Leptomeningeal metastases (LM) are increasingly becoming recognized as a treatable, yet generally incurable, complication of advanced cancer. As modern cancer therapeutics have prolonged the lives of patients with metastatic cancer, specifically in patients with parenchymal brain metastases, treatment options, and clinical research protocols for patients with LM from solid tumors have similarly evolved to improve survival within specific populations. Recent expansions in clinical investigation, early diagnosis, and drug development have given rise to new unanswered questions. These include leptomeningeal metastasis biology and preferred animal modeling, epidemiology in the modern cancer population, ensuring validation and accessibility of newer leptomeningeal metastasis diagnostics, best clinical practices with multimodality treatment options, clinical trial design and standardization of response assessments, and avenues worthy of further research. An international group of multi-disciplinary experts in the research and management of LM, supported by the Society for Neuro-Oncology and American Society of Clinical Oncology, were assembled to reach a consensus opinion on these pressing topics and provide a roadmap for future directions. Our hope is that these recommendations will accelerate collaboration and progress in the field of LM and serve as a platform for further discussion and patient advocacy.

A non-inferiority study of MRI versus CT for staging and image-defined risk factor assessment in the preoperative work-up of abdominopelvic neuroblastoma

BackgroundNeuroblastoma accounts for 15 % of cancer deaths in children. Complete surgical resection is associated with a higher overall survival rate but also a higher morbidity rate. An international group of experts has defined a nomenclature of image-defined risk factors (IDRFs) for the determination of operability and the anticipation of reasonably foreseeable complications of surgery. However, there is no consensus on the optimal imaging modality (CT or MRI) for the assessment of IDRFs. The objective of the present study was to determine the non-inferiority of MRI vs. CT in the preoperative assessment of abdominopelvic neuroblastoma. The secondary objective was to assess the contribution of gadolinium contrast enhancement. MethodsAll children diagnosed with abdominopelvic neuroblastoma and whose preoperative work-up included a contrast-enhanced CT or MRI scan of the abdomen and pelvis between January 2014 and January 2023 were included. To evaluate the IDRFs, all the images were reviewed in three steps: (i) non-contrast MRI scans, (ii) both non-contrast and contrast-enhanced MRI scans, and (iii) contrast-enhanced CT scans. ResultsTwenty-five patients were found to be eligible, and fifteen were included. The mean time interval between MRI and preoperative CT was 23 days. In all patients, the identified IDRFs were similar for all three imaging modalities. Fourteen patients underwent full resection of the tumour. The surgical reports were fully consistent with the IDRFs described on CT and/or MRI. ConclusionA high-resolution three-dimensional T2 MRI sequence agreed fully with contrast-enhanced CT for the detection of IDRFs. Contrast-enhanced MRI did not add value. However, surgeons will need time to adapt to this MRI-based approach and learn how to interpret the results with confidence.

Recurrence of autoimmune liver diseases after liver transplantation: Review and expert opinion statement.

Autoimmune liver diseases (AILDs) constitute the fourth most common indication for liver transplantation (LT) across the world. In general, the outcomes after LT are acceptable; however, disease recurrence after LT is common for all AILD, which can negatively affect graft and overall survival. Several questions persist, including the risk factors associated with recurrent disease, optimal antirejection medications, strategies to reduce the risk of recurrence, and how to best incorporate these strategies into clinical practice. For that reason, we assembled an international group of experts to review evidence to address these outstanding questions regarding LT for AILD. Survival rates after LT are ~90% and 70% at 1 and 5 years, and recurrent disease occurs in 10%-50% of patients with AILD. In patients with disease recurrence, graft survival decreased by 18% and 28% and overall survival by 8% and 12% at 5 and 10 years after LT, respectively. Recurrent autoimmune hepatitis is associated with high aminotransferases and immunoglobulin G (IgG) before LT, lymphoplasmacytic infiltrates in the explants, and may be associated with the absence of steroids after LT. However, the efficiency and safety of triple immunosuppressive maintenance therapy is still debatable. Younger age at diagnosis with primary biliary cholangitis or LT is associated with primary biliary cholangitis recurrence. Preventive use of ursodeoxycholic acid reduces the risk of recurrence and has a benefit in graft and patient survival. Episodes of systemic inflammation, including T-cell-mediated rejection, active ulcerative colitis, and episodes of cholangitis, are associated with recurrent PSC. Recurrent disease for AILD is associated with worse graft and patient survival. Patients with autoimmune hepatitis could be considered for long-term low-dose predniso(lo)ne, whereas patients with primary biliary cholangitis should be placed on preventive ursodeoxycholic acid after LT. There are no specific treatments for PSC recurrence; however, adequate control of inflammatory bowel disease and optimal immunosuppression to avoid T-cell-mediated rejection should be encouraged.

International consensus panel for transurethral resection of bladder tumours metrics: assessment of face and content validity.

To develop performance metrics that objectively define a reference approach to a transurethral resection of bladder tumours (TURBT) procedure, seek consensus on the performance metrics from a group of international experts. The characterisation of a reference approach to a TURBT procedure was performed by identifying phases and explicitly defined procedure events (i.e., steps, errors, and critical errors). An international panel of experienced urologists (i.e., Delphi panel) was then assembled to scrutinise the metrics using a modified Delphi process. Based on the panel's feedback, the proposed metrics could be edited, supplemented, or deleted. A voting process was conducted to establish the consensus level on the metrics. Consensus was defined as the panel majority (i.e., >80%) agreeing that the metric definitions were accurate and acceptable. The number of metric units before and after the Delphi meeting were presented. A core metrics group (i.e., characterisation group) deconstructed the TURBT procedure. The reference case was identified as an elective TURBT on a male patient, diagnosed after full diagnostic evaluation with three or fewer bladder tumours of ≤3 cm. The characterisation group identified six procedure phases, 60 procedure steps, 43 errors, and 40 critical errors. The metrics were presented to the Delphi panel which included 15 experts from six countries. After the Delphi, six procedure phases, 63 procedure steps, 47 errors, and 41 critical errors were identified. The Delphi panel achieved a 100% consensus. Performance metrics to characterise a reference approach to TURBT were developed and an international panel of experts reached 100% consensus on them. This consensus supports their face and content validity. The metrics can now be used for a proficiency-based progression training curriculum for TURBT.

A comprehensive literature review and an international expert consensus on the management of scalp seborrheic dermatitis in adults.

Seborrheic Dermatitis of the scalp (SSD) is a chronic and relapsing inflammatory skin condition. Current SSD treatments mainly consist of topical applications of anti-fungals and anti-inflammatory agents. to review information about SSD and to provide dermatologists with practical recommendations for managing adult SSD. Material and methods: Between September and December 2023, an international group of experts in dermatology and hair and scalp disorders met to discuss published data about SD, SSD, dandruff, and management options. A total of 131 manuscripts available from PubMed were analysed, discussed and used for the present consensus. Each author was asked to complete a table listing currently used treatments to treat SSD according to the literature and to their own experience. The authors confirmed their use and regimen and commented on local treatment exceptions. They then agreed on prescription practices and proposed a general treatment approach. Currently, approved therapies to manage moderate and severe forms of SSD do not exist and there is a need for adapted and approved medications that treat efficiently and safely the disease. We propose a treatment algorithm that allows for the treatment of all severity grades of SSD. This algorithm may be completed with local treatment specifications. Despite the lack of approved therapies to manage moderate forms of SSD, a treatment algorithm is proposed and may help prescribers to manage SSD more efficiently.

Регулирование цифровых платформ в БРИКС: приоритеты и опыт ЮАР

South Africa represents an actively developing digital services market that ranks among first in Africa. The local marketattracts major international companies that endangers local suppliers threaten with prospects of loosing competitive marketpower and overall monopolization of the segment. Loose of competitive power by local suppliers threatens the country’s datasovereignty and users data safety. Digital platform services suppliers’ regulation issues are among the most demanded researchtopics that is especially important for the BRICS bringing together digital growth leaders from the countries of globalmajority.The article presents results of South Africa’s digital platform services regulation framework analysis. The paper reliesupon the author’s personal attempt to examine the country’s existing regulatory framework as well as upon reports presentedby competent international expert groups and the national regulator. The analysis indicates existence of systemic constrainsreferring to institutional arrangements within the digital platform services provision regulatory framework along with imperfectionof the local legislation. The author claims that South Africa’s experience in promotion of the local businesses might beof interest for the competent governmental bodies of Russia. For South Africa deepening of cooperation within the BRICS isone of the top priorities as its realization would provide firm ground for implementation of the country’s development goals.

#2422 Iohexol plasma clearance measurement protocol standardization—a consensus of the European Kidney Function Consortium

Abstract Background and Aims International consensus supports the development of standardized protocols for measured glomerular filtration rate (mGFR) to facilitate the adoption of mGFR determination in both clinical and research settings. Method The European Kidney Function Consortium (EKFC) convened an international group of mGFR experts. The working group performed an extensive literature search to inform the development of recommendations for mGFR determination using one-compartment plasma clearance models and iohexol as the exogenous filtration marker. Iohexol was selected as it is non-radio labeled, inexpensive, widely available, and can be assayed at a central laboratory. Other commonly used non-radio labeled tracers have been (inulin) or are soon to be (iothalamate) discontinued. A plasma clearance model was selected as it requires no timed urine collections. A one-compartment model was preferred to a two compartment model as it requires fewer samples. The resulting recommendations were based on published evidence complemented by expert opinion. Results Recommendations include practical advice for patients and health professionals, preparation, administration and safety aspects of iohexol, laboratory analysis, blood sample collection and sample timing for both multiple and single sample protocols, description of the mGFR mathematical calculations as well as implementation strategies (Fig.). EKFC will provide materials such as patient information sheets, standard operating procedures, a study protocol template, and support for mGFR calculation. Conclusion Members of the EKFC have evaluated the evidence and formulated recommendations that include all aspects of iohexol plasma clearance measurement using a one compartment model. *Author list on behalf of the European Kidney Function Consortium (EKFC).

Application of the Milan system for reporting salivary gland cytopathology to core needle biopsies of the parotid gland.

The Milan system for reporting salivary gland cytopathology was developed by an international group of experts and first published in 2018 with the goal to standardise reporting of salivary gland aspirates. Seven categories with distinct risks of malignancy were proposed. Core needle biopsies (CNB) of salivary glands are also common, but reporting lacks standardisation. Here we explore the feasibility of a Milan-like reporting system on CNB of the parotid gland. Our laboratory information system was searched for parotid gland CNBs from 2010 to 2021. Reports were translated into a Milan-like reporting system. When available, CNB findings were correlated with cytology and resection specimens. In order to compare the performance of CNB with fine-needle aspirations (FNA), we established a second cohort of cases consisting of parotid FNA with surgical follow-up. The risk of neoplasia (RON) and risk of malignancy (ROM) was calculated for FNA and CNB Milan categories using cases with follow-up resection. We analysed 100 cases of parotid gland CNB. Of these cases, 32 underwent subsequent resection, while 52 had concurrent FNA. A total of 20 cases had concurrent FNA and underwent follow-up resection. In 63 (63%) cases, a specific diagnosis was provided on CNB, with 18 cases undergoing follow-up resection having an accuracy rate of 94%. This study confirms the feasible of using a Milan-like system in the setting of parotid gland CNB with differentiation in RON and ROM. CNB allows assessment of architectural features that may allow more specific diagnoses in some cases.

Detection of antinuclear antibodies: recommendations from EFLM, EASI and ICAPEFLM Paper

<br><b>Aim:</b> Antinuclear antibodies (ANA) are important for the diagnosis of various autoimmune diseases. ANA are usually detected by indirect immunofluorescence assay (IFA) using HEp-2 cells (HEp-2 IFA). There are many variables influencing HEp-2 IFA results, such as subjective visual reading, serum screening dilution, substrate manufacturing, microscope components and conjugate. Newer developments on ANA testing that offer novel features adopted by some clinical laboratories include automated computer-assisted diagnosis (CAD) systems and solid phase assays (SPA).</br> <br><b>Methods:</b> A group of experts reviewed current literature and established recommendations on methodological aspects of ANA testing. This process was supported by a two round Delphi exercise. International expert groups that participated in this initiative included (i) the European Federation of Clinical Chemistry and Laboratory Medicine (EFLM) Working Group "Autoimmunity Testing"; (ii) the European Autoimmune Standardization Initiative (EASI); and (iii) the International Consensus on ANA Patterns (ICAP).</br> <br><b>Results:</b> In total, 35 recommendations/statements related to (i) ANA testing and reporting by HEp-2 IFA; (ii) HEp-2 IFA methodological aspects including substrate/conjugate selection and the application of CAD systems; (iii) quality assurance; (iv) HEp-2 IFA validation/verification approaches and (v) SPA were formulated. Globally, 95% of all submitted scores in the final Delphi round were above 6 (moderately agree, agree or strongly agree) and 85% above 7 (agree and strongly agree), indicating strong international support for the proposed recommendations.</br> <br><b>Conclusions:</b> These recommendations are an important step to achieve high quality ANA testing.</br>

Development of an International Standard Set of Outcomes and Measurement Methods for Routine Practice for Infants, Children, and Adolescents with Epilepsy: The International Consortium for Health Outcomes Measurement Consensus Recommendations.

At present, there is no internationally accepted set of core outcomes or measurement methods for epilepsy clinical practice. The International Consortium for Health Outcomes Measurement (ICHOM) convened an international working group of experts in epilepsy, people with epilepsy, and their representatives to develop minimum sets of standardized outcomes and outcome measurement methods for clinical practice. Using modified Delphi consensus methods with consecutive rounds of online voting over 12 months, a core set of outcomes and corresponding measurement tool packages to capture the outcomes were identified for infants, children, and adolescents with epilepsy. Consensus methods identified 20 core outcomes. In addition to the outcomes identified for the ICHOM Epilepsy adult standard set, behavioral, motor, and cognitive/language development outcomes were voted as essential for all infants and children with epilepsy. The proposed set of outcomes and measurement methods will facilitate the implementation of the use of patient-centered outcomes in daily practice.

Development of an International Standard Set of Outcomes and Measurement Methods for Routine Practice for Adults with Epilepsy: The International Consortium for Health Outcomes Measurement Consensus Recommendations.

At present, there is no internationally accepted set of core outcomes or measurement methods for epilepsy clinical practice. Therefore, the International Consortium for Health Outcomes Measurement (ICHOM) convened an international working group of experts in epilepsy, people with epilepsy and their representatives to develop minimum sets of standardized outcomes and outcomes measurement methods for clinical practice that support patient-clinician decision-making and quality improvement. Consensus methods identified 20 core outcomes. Measurement tools were recommended based on their evidence of strong clinical measurement properties, feasibility, and cross-cultural applicability. The essential outcomes included many non-seizure outcomes: anxiety, depression, suicidality, memory and attention, sleep quality, functional status, and the social impact of epilepsy. The proposed set will facilitate the implementation of the use of patient-centered outcomes in daily practice, ensuring holistic care. They also encourage harmonization of outcome measurement, and if widely implemented should reduce the heterogeneity of outcome measurement, accelerate comparative research, and facilitate quality improvement efforts.

Towards Re-Architecting Today's Internet for Survivability: NSF Workshop Report

On November 28--29, 2023, Northwestern University hosted a workshop titled "Towards Re-architecting Today's Internet for Survivability" in Evanston, Illinois, US. The goal of the workshop was to bring together a group of national and international experts to sketch and start implementing a transformative research agenda for solving one of our community's most challenging yet important tasks: the re-architecting of tomorrow's Internet for "survivability", ensuring that the network is able to fulfill its mission even in the presence of large-scale catastrophic events. This report provides a necessarily brief overview of two full days of active discussions.

Intervention for critical aortic stenosis in Hutchinson-Gilford progeria syndrome.

Hutchinson-Gilford Progeria Syndrome (HGPS) is an ultra-rare genetic premature aging disease that is historically fatal in teenage years, secondary to severe accelerated atherosclerosis. The only approved treatment is the farnesyltransferase inhibitor lonafarnib, which improves vascular structure and function, extending average untreated lifespan of 14.5 years by 4.3 years (30%). With this longer lifespan, calcific aortic stenosis (AS) was identified as an emerging critical risk factor for cardiac death in older patients. Intervention to relieve critical AS has the potential for immediate improvement in healthspan and lifespan. However, HGPS patient-device size mismatch, pervasive peripheral arterial disease, skin and bone abnormalities, and lifelong failure to thrive present unique challenges to intervention. An international group of experts in HGPS, pediatric and adult cardiology, cardiac surgery, and pediatric critical care convened to identify strategies for successful treatment. Candidate procedures were evaluated by in-depth examination of 4 cases that typify HGPS clinical pathology. Modified transcatheter aortic valve replacement (TAVR) and left ventricular Apico-Aortic Conduit (AAC) placement were deemed high risk but viable options. Two cases received TAVR and 2 received AAC post-summit. Three were successful and 1 patient died perioperatively due to cardiovascular disease severity, highlighting the importance of intervention timing and comparative risk stratification. These breakthrough interventions for treating critical aortic stenosis in HGPS patients could rewrite the current clinical perspective on disease course by greatly improving late-stage quality of life and increasing lifespan. Expanding worldwide medical and surgical competency for this ultra-rare disease through expert information-sharing could have high impact on treatment success.

Meniscal extrusion consensus statement: Acollaborative survey within the Meniscus International Network (MenIN) Study Group.

The purpose of the present study was to perform a survey administered to members of the Meniscus International Network (MenIN) Study Group, seeking to delineate the most contentious aspects of meniscal extrusion classification and provide a foundation for new, more comprehensive definitions and treatments for these pathologies. MenIN Study Group is a group of international experts treating and performing research on meniscus pathology and treatment. All MenIN Study Group members were asked to complete a survey aimed at establishing criteria for the optimal classification system for meniscal extrusion. Data obtained from the completed questionnaires were transferred into a spreadsheet and then analysed. All responses are presented as counts, percentagesor means. Forty-seven (85.5%) MenIN Study Group members completed the survey and were included in this analysis. Key aspects recommended for inclusion in a comprehensive classification system for meniscal extrusion included laterality (93.6%), anatomical location (76.6%), patient age (76.6%), body mass index (BMI) (68.1%)and aetiology (68.1%). For classifying meniscal extrusion, 53.2% considered the distance in millimetres from the tibial plateau's outer margin as the most reliable measurement technique on imaging. Preferences for imaging modalities varied, with 44.7% favouring weight-bearing magnetic resonance imaging (MRI) and 36.2% opting for weight-bearing ultrasound due to its greater availability. Respondents advocated for a classification system addressing stability or progression of meniscal extrusion (66%), reducibility (53.2%), potential progression of knee osteoarthritis (OA) (83%), influencing treatment approaches (83%), a gradation system (83%), consideration of dynamic factors (66%), association with clinical outcomes and prognosis (76.6%)and investigation around centralization procedures (57.4%). In conclusion, the findings of this survey shed light on the global perspectives regarding meniscal extrusion classification. It was generally felt that a new classification of extrusion measured on MRI scans at the mid-tibial plateau should be developed, which considers factors such as laterality, anatomical location, age, BMIand aetiology. Additionally, the results support the integration of dynamic factors and clinical outcomes in MRI-based classifications to inform treatment approaches. Level IV.

A Systematic Guideline by the ASPN Workgroup on the Evidence, Education, and Treatment Algorithm for Painful Diabetic Neuropathy: SWEET.

Painful diabetic neuropathy (PDN) is a leading cause of pain and disability globally with a lack of consensus on the appropriate treatment of those suffering from this condition. Recent advancements in both pharmacotherapy and interventional approaches have broadened the treatment options for PDN. There exists a need for a comprehensive guideline for the safe and effective treatment of patients suffering from PDN. The SWEET Guideline was developed to provide clinicians with the most comprehensive guideline for the safe and appropriate treatment of patients suffering from PDN. The American Society of Pain and Neuroscience (ASPN) identified an educational need for a comprehensive clinical guideline to provide evidence-based recommendations for PDN. A multidisciplinary group of international experts developed the SWEET guideline. The world literature in English was searched using Medline, EMBASE, Cochrane CENTRAL, BioMed Central, Web of Science, Google Scholar, PubMed, Current Contents Connect, Meeting Abstracts, and Scopus to identify and compile the evidence for diabetic neuropathy pain treatments (per section as listed in the manuscript) for the treatment of pain. Manuscripts from 2000-present were included in the search process. After a comprehensive review and analysis of the available evidence, the ASPN SWEET guideline was able to rate the literature and provide therapy grades for most available treatments for PDN utilizing the United States Preventive Services Task Force criteria. The ASPN SWEET Guideline represents the most comprehensive review of the available treatments for PDN and their appropriate and safe utilization.

THE FOOD CRISIS AND THE THREAT OF HUNGER ON EARTH

This article examines such a global problem as the food crisis and the threat of hunger on the planet. An analysis of the causes and composition of the global food problem is carried out. Attention is paid to the statistics and dynamics of changes in the problem of hungry people around the globe and the entire current situation at the moment. Recommendations and attempts to resolve the food crisis by international groups of experts, including scientists and politicians, are also considered in order to implement the proposals made on this issue and overcome the current problem

Endoscopic Surveillance of the Intestinal Allograft: Recommendations From the Intestinal Rehabilitation and Transplant Association Working Group.

Intestinal transplant (ITx) rejection lacks a reliable noninvasive biomarker and rejection surveillance relies on serial endoscopies and mucosal biopsies followed by histologic assessment. Endoscopic biopsies are also essential for identifying other ITx-related complications such as infectious, allergic, and inflammatory graft enteritis as well as post-transplant lymphoproliferative disease or graft versus host disease. In spite of its central role in ITx, published guidelines on endoscopy and biopsy are lacking and significant variability between centers in terms of timing and technical performance exists. Therefore, an international expert group convened and discussed several aspects related to the surveillance endoscopy after ITx with the aim to summarize and standardize its practice. This article summarizes these considerations on endoscopic ITx monitoring and highlights practices of surveillance and for-cause endoscopy, biopsy techniques, pathologic evaluation, potential risks and complications, outsourcing, and less-invasive monitoring techniques.

Abstract A034: A new international program to accelerate novel therapeutics for brain tumors: presenting the brain tumor research novel therapeutics accelerator

Abstract Each year, we see many journal manuscripts highlighting successful pre-clinical experiments for potential new brain tumor therapies. Only a few of these therapies move forward into successful clinical trials. Indeed, developing a comprehensive translational program covers many aspects, including but not limited to, medicinal chemistry, blood brain barrier penetration, tumor heterogeneity, delivery, commercialization, and navigating the regulatory environment. Because of this, bringing a successful translational project from bench to bedside requires access to a diverse set of cutting-edge expertise to give promising therapies the best chance of succeeding. Recognizing that this journey is not easy to navigate, the Tessa Jowell Brain Cancer Mission has launched an international Brain Tumor Research Novel Therapeutics Accelerator (BTR-NTA). This program is led by an international group of drug development and brain tumor experts who provide independent guidance to researchers to position their therapeutic along a realistic pathway to clinical trials. The BTR-NTA is open to any international academic and industry researchers developing a medicinal product, delivery technology or device for brain tumors. The BTR-NTA welcomes groups at any stage of pre-clinical or clinical work. Academics and smaller biotech’s could benefit from feedback on their pre-clinical studies and plans for commercialization, while larger pharmaceutical companies could benefit from access to brain tumor specific expertise and patient input into clinical trial design. Researchers can apply to BTR-NTA twice a year, and if accepted will go through the following process: 1. Complete an application form covering work to date and future plans under strict confidentiality agreements; 2. Attend an in-person meeting with a tailored group of experts to further discuss their work; and 3. Receive a comprehensive written report from the BTR-NTA committee with feedback on strengths and weaknesses and guidance on next steps. The expert committee dedicates half a day to reviewing the applicants’ work in-person, and extensive time pre- and post-meeting. Throughout the process, applicants receive up to 150 hours of expert input. The multidisciplinary nature of the review committee spans the entirety of the drug development pathway to support longitudinal collaboration and avoid a siloed approach to research. The BTR-NTA was designed after a similar program in another rare disease field yielded impressive results. Thanks to charitable funding, the BTR-NTA is free to academics and at low cost for industry. The BTR-NTA aims to help address barriers for researchers by highlighting potential pitfalls or risks, providing multi-disciplinary feedback on work to date, giving constructive guidance on next steps and strengthening international collaboration in the brain tumor field. The ultimate goal of the BTR-NTA is to enable researchers with therapy for brain tumors to navigate the drug development journey and make it to the clinic in a low-cost and timely way. Citation Format: Nicky Huskens, Charlotte Aitken, Katie Bushby. A new international program to accelerate novel therapeutics for brain tumors: presenting the brain tumor research novel therapeutics accelerator [abstract]. In: Proceedings of the AACR Special Conference on Brain Cancer; 2023 Oct 19-22; Minneapolis, Minnesota. Philadelphia (PA): AACR; Cancer Res 2024;84(5 Suppl_1):Abstract nr A034.