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Amyloid‐Related Imaging Abnormalities Summit Special Proceedings: Cardiovascular and Stroke Considerations for β‐Amyloid Immunotherapy in Alzheimer Disease
Greenberg, Steven M. , Iadecola, Costantino , Chou, Sherry , Cogswell, Petrice M, Elkind, Mitchell S.V. , II, William J. Hicks, Le, Caitlin, Levine, Deborah A. , Lipton, Richard B. , Massimo, Lauren, Mohl, Stephanie M. , Snyder, Heather M. , Yaffe, Kristine , Adams, Robert, Agronin, Marc, Atri, Alireza, Bauman, Mary Ann, Bernat, James L., Biffi, Alessandro , Blum, Sonja, Bussiere, Thierry, Carrillo, Maria C., Chiang, Gloria, Cato, Kenrick, Chaitoff, Alexander, Bakken, Suzanne, Schelke, Matthew W. , Cruz‐Flores, Salvador, Derdeyn, Colin, Dhand, Amar, Evans, David A., Gettel, Cameron, Goldstein, Larry B, Grill, Joshua, Kulshrestha, Ambar, Lam, Christine, Lazar, Ronald M. , Li, Hanlin , Madsen, Tracy, Ramanan, Vijay K., McCullough, Louise, Merino, José G., Orjuela, Karen, Guo, Michael, Messe, Steve, Tubiello, Maggie, Ruff, Christian T., Selim, Magdy, Sha, Sharon J., Sheth, Kevin, Stamm, Brian, Buckwalter, Marion, Skylar-Scott, Irina, Lavretsky, Helen, Saver, Jeffrey, Rabinovici, Gil D., VandeVrede, Lawren, Goyal, Manu S., Weisman, David C., Lipton, R.B. , Chou, Sherry H-Y , Seshadri, Sudha , Snyder, Heather M , Yaffe, Kristine , Zachrison, Kori S , Cogswell, Petrice M. , Hicks, William J., Massimo, Lauren , Seshadri, Sudha , Atri, Alireza, Blum, Sonja, Bussière, Thierry , Carlisle, Tara C. , Chaitoff, Alexander , Cruz-Flores, Salvador, Chyou, Janice Y. , Derdeyn, Colin P. , Gettel, Cameron J. , Goldstein, Larry B. , Grill, Joshua D. , Ishii, Makoto , Kulshreshtha, Ambar , Rabinstein, Alejandro A. , Ramanan, Vijay K. , McCullough, Louise D. , Merino, José G. , Messé, Steven R. , Ruff, Christian Thomas , Saklad, Stephen R. , Selim, Magdy H. , Shah, Sachin J. , Stamm, Brian , Buckwalter, Marion S. , Skylar-Scott, Irina A., Rabinovici, Gil D. , VandeVrede, Lawren , Chatterjee, Arindam R , Wunderink, Richard G.

Journal of the American Heart Association | August 20, 2026 | Volume: No volume available | Issue: No issue available | Pages: No page available

10.1161/jaha.125.048108 | PMID 42622140 Get It

Background New antiamyloid therapies (AAT) for treating patients with early Alzheimer disease (AD) have become available in the past 3 years. Although AAT can be beneficial in slowing progression of AD, they also come with potentially serious side effects, particularly for patients requiring anticoagulation or thrombolysis for cardiovascular conditions. Given that most patients with AD are older and likely to have cardiovascular risk factors, there is urgent need for additional guidance for clinicians about potential risks and benefits associated with AAT for patients with cardiovascular comorbidities, best practices for management of these patients, and special considerations with respect to emergency evaluation and treatment of patients on AAT who develop acute neurological symptoms. Methods The American Heart Association convened 75 multidisciplinary experts for the Amyloid‐Related Imaging Abnormalities Summit: Cardiovascular and Stroke Considerations for β‐Amyloid Immunotherapy in AD in July 2025. These experts were divided into 8 work groups to evaluate the current evidence related to AAT usage in patients with cardiovascular risk factors and conditions and develop consensus‐based guidance for clinicians. Results This statement outlines the outcomes of the Summit, including considerations for prescribers, primary care professionals, emergency personnel, and health care system stakeholders. These considerations are intended to improve identification of patients as appropriate candidates for AAT, management of patients with co‐occurring AD and cardiovascular conditions, and their emergency evaluation and treatment. The statement also includes guidance for shared decision‐making, implementation of system changes for improving dementia care, and recommendations for future research and data collection to add to the body of evidence for treating patients with concomitant AD and cardiovascular conditions. Conclusions Building on lessons learned from the establishment of stroke systems of care, the consensus‐based considerations outlined in this document can serve as a foundation for AD systems of care aimed at ensuring optimal use of these ground‐breaking therapies.
Acute Myeloid Leukemia Survivors Report Transplant-specific Burdens in Interviews Two Years after Receiving Allogeneic Hematopoietic Cell Transplantation During the Peak of the COVID-19 Pandemic: A Qualitative Study
McAndrew, Natalie S. , Patterson, S., Awoyinka, Iwalola, Costanzo, Erin S., Cole, Steve W. , Knight, Jennifer M., Patterson, Sam, Mohanraj, Lathika , Awoyinka, Iwalola , Knight, Jennifer M.

INQUIRY: The Journal of Health Care Organization, Provision, and Financing | August 18, 2026 | Volume: 63 | Issue: No issue available | Pages: No page available

10.1177/00469580261478062 | PMID 42610368 Get It

Introduction Allogeneic hematopoietic cell transplantation (HCT) for acute myeloid leukemia (AML) entails prolonged recovery, serious complications, functional limitations, psychological distress, and extended isolation to prevent infection. During the peak of the COVID-19 pandemic, treatment disruptions, restricted caregiver access, and intensified isolation compounded these transplant-specific burdens. The longer-term influence of the pandemic experience on HCT recovery and survivorship remains poorly understood. Methods In this qualitative descriptive study, adults with AML who underwent allogeneic HCT from March through September 2020 participated in virtual semi-structured interviews from July 2022 through April 2023, approximately two years post-transplant. Participants were identified through the Center for International Blood and Marrow Transplant Research registry. Zoom or phone interviews were recorded, transcribed verbatim, and analyzed using inductive qualitative content analysis to describe survivorship experiences and opportunities to improve supportive care. Results A total of 25 AML survivors (ages 24-78, 56% male) were interviewed approximately two years post-transplant. Three themes were found: (1) Transplant is an arduous journey; (2) The COVID-19 pandemic shaped the transplant experience in complex and far-reaching ways; (3) Transplant demands sustained personal and relational investment throughout the transplant journey. Conclusions This study enhances our understanding of the stressors associated with transplantation during periods of heightened adversity, while also identifying opportunities to strengthen healthcare systems and supportive care services for this population. Situating these experiences within the context of a global health emergency offers a rare lens into how external stressors can exacerbate the already demanding nature of HCT survivorship. By understanding survivors’ experiences, healthcare systems can better anticipate and address the evolving needs of transplant survivors.
Letter: FDA Approval of the HepQuant SHUNT Liver Diagnostic Test and Clarification of the SHUNT ‐V Anal
Shiffman, Mitchell, Reddy, K. Rajender , Leise, Michael D., Qureshi, Kamran , Smith, Alastair D., Helmke, Steve, Kittelson, John, Imperial, Joanne C. , Everson, Gregory T. , McRae, Michael P , Everson, Gregory T , Helmke, Steve M. , Kittelson, John , McRae, Michael P.

Alimentary Pharmacology & Therapeutics | August 11, 2026 | Volume: No volume available | Issue: No issue available | Pages: No page available

10.1111/apt.70925 | PMID 42576259 Get It

Beyond BMI: an interpretable integrated body composition index from low-dose chest CT for all-cause mortality risk stratification: a multicentre study
Patel, Krishna K., Miller, Robert J.H, Marcinkiewicz, Anna M , Kamagate, Assiata, Shanbhag, Aakash, Hijazi, Waseem , Lemley, Mark, Zhou, Jianhang, Liang, Joanna X. , Ramirez, Giselle, Mostafavi, Shiva , Urs, Meghana, Travin, Mark, Alexanderson, Erick, Caraval-Juarez, Isabel, Ruddy, Terrence D., Einstein, Andrew J., Feher, Attila, Le, Viet T, Mason, Steve, Calsavara, Vinicius F., Chareonthaitawee, Panithaya, Wopperer, Samuel, Wang, Lixia, Li, Debiao, Lopez-Ramirez, Felipe, Berman, Daniel S., Kwieciński, Jacek , Dey, Damini, Carli, Marcelo Di, Li, Debiao , Miller, Robert J.H. , Miller, Robert J H , Li, Debiao , Yi, Jirong , Lemley, Mark , Zhou, Jianhang , Urs, Meghana , Spielvogel, Clemens P. , Slipczuk, Leandro , Travin, Mark I. , Alexanderson, Erick , Packard, René R. Sevag , Al‐Mallah, Mouaz H. , Ruddy, Terrence D , Einstein, Andrew Jeffrey , Feher, Attila , Miller, Edward J. , Acampa, Wanda , Knight, Stacey , Le, Viet T. , Mason, Steve, Calsavara, Vinicius F , Chareonthaitawee, Panithaya , Kwan, Alan C. , Li, Debiao, Fishman, Elliot K. , Lopez-Ramirez, Felipe, Berman, Daniel S. , Dey, Damini , Carli, Marcelo F. Di , Slomka, Piotr J , Zhou, Jianhang , Slipczuk, Leandro , Packard, Rene R S , Al-Mallah, Mouaz H , Miller, Edward J , Fishman, Elliot K.

No container title available | August 10, 2026 | Volume: No volume available | Issue: No issue available | Pages: No page available

10.64898/2026.08.05.26359437 | PMID 42845593 Get It

Summary Background Body composition is recognized as a major determinant of health outcomes, but its multidimensional nature makes clinical adoption challenging. We sought to develop and validate a body composition index (BCI) for all-cause mortality risk assessment, integrating variables of six body composition tissues. Methods We analyzed 28509 consecutive patients undergoing myocardial perfusion imaging with routine low-dose chest CT attenuation correction (CTAC) scans acquired during myocardial perfusion imaging (MPI) at 12 centers across four countries. An artificial intelligence-based BCI was developed in a cohort of 15037 patients’ CTACs by integrating the CT-derived metrics of bone, skeletal muscle, and four adipose tissue compartments, coronary artery calcium score, and basic demographic variables (age, sex, BMI). The performance of BCI for mortality prediction was validated in an internal cohort of 6444 patients and an external cohort of 7028 patients by prognosis, calibration, net benefit, and explainability. Model-based simulation of tissue metrics modification was performed to evaluate estimated mortality risk reduction. Findings During a median of 3.5 (IQR [1.9, 5.1]) years, 4697 (16%) patients died. In the external testing cohort, the BCI demonstrated excellent discrimination for mortality (area under receiver operating characteristic curve 0.78 (95% CI [0.76, 0.79]) and Harrell’s concordance index 0.75 [0.73, 0.76]), calibration, and net benefit overall and across pre-specified subgroups stratified by patient characteristics and imaging protocols. Visceral adipose tissue attenuation was the most influential body composition measure, followed by skeletal muscle volume. Simulated improvement in body composition was associated with significant mortality risk reduction. Interpretation An index combining six body composition measures obtained opportunistically from routine chest CT provides robust mortality risk stratification. By converting complex body composition information into a single interpretable score, the BCI can facilitate clinical implementation of opportunistic CT biomarkers and guide individualized preventive strategies. Funding National Institute of Health Research in context Evidence before this study We searched PubMed and Google Scholar on July 7, 2026 for English language studies using the terms (“mortality prediction” OR “death prediction”) AND (“integrated body composition analysis” OR “body composition metrics integration”) AND (“predictive model” OR “model development”), and identified 41 publications describing predictive models incorporating body composition metrics. Recent advances in artificial intelligence (AI) have enabled increasingly granular quantification of body composition from CT, with multiple tissues and tissue-specific metrics showing independent prognostic value for all-cause mortality (ACM) prediction. However, reported associations of body composition metrics with mortality risk were highly inconsistent or even contradictory across studies. This heterogeneity likely reflected the differences in study design or the inherent complexity of body composition analysis, such as the interaction among variables derived from multiple tissues. Thus, it is difficult to interpret body composition results reliably in clinical practice. We hypothesized that integrating multiple body composition metrics could achieve comprehensive and reliable prediction of mortality across patient populations. Added value of this study In this large international, multicenter longitudinal cohort study of 28509 consecutive patients with suspected or known coronary artery disease (CAD) undergoing myocardial perfusion imaging across 12 sites in four countries, we developed and externally validated an artificial intelligence-based body composition index (BCI) for all-cause mortality prediction. The BCI integrated chest CT-derived metrics of six body composition tissue compartments – bone, skeletal muscle (SM), subcutaneous adipose tissue (SAT), intramuscular adipose tissue (IMAT), visceral adipose tissue (VAT), and epicardial adipose tissue (EAT), together with demographics (age, sex, BMI) and CT-derived coronary artery calcium (CAC) score, all derived automatically from routine low-dose attenuation correction CT scans without additional imaging or laboratory tests. In an external validation cohort of 7028 patients, the BCI achieved robust discrimination for mortality prediction, substantially outperforming a baseline model using demographics and calcium score alone, and exceeded the discriminative performance of prior body composition models. Performance was consistent across subgroups defined by sex, age, body mass index, cardiometabolic risk factors, imaging modality, and acquisition protocol, supporting generalizability across clinical settings. The BCI demonstrated excellent calibration and net clinical benefit. Explainability analysis identified visceral adipose tissue attenuation and skeletal muscle volume as the dominant contributors to mortality risk, offering tissue-specific therapeutic targets that complement generic weight-based metrics. Model-based simulations suggested that improvements in these specific compartments could reduce estimated mortality risk more effectively than equivalent changes in BMI alone, pointing toward precision therapeutic targets beyond weight reduction. Our study improved over previous literatures on body composition-based predictive models in one or more of the following ways: (1) we considered six types of body composition tissues while existing studies considered only three or less tissues, thus significantly higher granularity and improved ability to capture refined tissue interactions; (2) our model was rigorously validated in external sites, showing cross-site generalizability while most previous work lacked external validation; (3) our model achieved significant prediction performance improvement over previous body composition predictive models, offering better clinical outcomes prediction and facilitating the suitability for widespread clinical adoption; (4) our study was conducted with a large multi-center cohort across multiple countries, achieving statistical power and demonstrating reliability towards patients characteristics and imaging acquisition protocols while previous studies involved small single-center cohorts; (5) our model required only CT scans and basic demographics which were widely available in clinical routine while the existing models required dedicated laboratory tests or specialized imaging modalities, enhancing the applicability in routine clinical practice. Implications of all the available evidence BCI offers an explainable and clinically deployable tool for improved prediction of mortality risk from existing chest CT scans. Its use could inform updates to existing risk models, guide precision preventive strategies, and support development of therapeutics targeting specific body composition phenotypes.
Systemic Therapy in Patients With Metastatic Castration-Resistant Prostate Cancer: ASCO Living Guideline, Version 2026.1.4
Taplin, Mary‐Ellen , Rumble, R. Bryan , Naqvi, Syed Arsalan Ahmed , Nahhat, Fouad , Dias, Mariane Sousa Fontes , Hope, Thomas A. , He, Huan, Hotte, Sebastién J. , Emamekhoo, Hamid , Celano, Paul, Kungel, Terry M. , Hentzen, Steve, Marzalik, Jacob S. , Parikh, Rahul A , Rumble, R Bryan , Nahhat, Fouad , Dias, Mariane Fontes , Hope, Thomas A , Hotte, Sebastien J , Emamekhoo, Hamid , Murad, M Hassan , Hentzen, Steve , Marzalik, Jacob S , Serzan, Michael Thomas , Riaz, Irbaz B , Taplin, Mary-Ellen , He, Huan, Murad, M. Hassan , Celano, Paul , Serzan, Michael , Riaz, Irbaz Bin

Journal of Clinical Oncology | August 6, 2026 | Volume: 44 | Issue: 31 | Pages: No page available

10.1200/jco-26-01898 | PMID 42561262 Get It

This guideline has been updated. This ASCO Living Guideline is reviewed on an eight-week cycle. Please see the online dynamic version for the latest updates. Living guidelines are developed for selected topic areas with rapidly evolving evidence that drives frequent change in recommended clinical practice. Living guidelines are updated on a regular schedule by a standing expert panel that systematically reviews the health literature on a continuous basis, as described in the ASCO Guidelines Methodology Manual . ASCO Living Guidelines follow the ASCO Conflict of Interest Policy Implementation for Clinical Practice Guidelines . Living Guidelines and updates are not intended to substitute for independent professional judgment of the treating clinician and do not account for individual variation among patients. See appendix for disclaimers and other important information ( Appendix 1 and Appendix 2 , online only). Updates are published regularly and can be found on the ASCO Publications website .
Beyond the Snapshot: Overcoming GES Limitations to Characterize Gastric Phenotypes and Therapeutic Responses Using a 6‐Day Ambulatory Wearable Monitor
Lacy, Brian E. , Cangemi, David J. , Axelrod, Lindsay , Axelrod, Steve , Navalgund, Anand , Cangemi, David

Neurogastroenterology & Motility | August 4, 2026 | Volume: 38 | Issue: 8 | Pages: No page available

10.1111/nmo.70413 | PMID 42547873 Get It

ABSTRACT Background Gastric emptying scintigraphy (GES) is considered the standard for differentiating gastroparesis from functional dyspepsia and other foregut disorders characterized by nausea and vomiting but provides only a brief physiologic snapshot that may not accurately reflect daily function. We utilized a non‐invasive wireless patch system (WPS) to characterize multiday gastric myoelectrical activity, evaluate its relationship with GES, and assess changes following a therapeutic intervention. Methods Adults referred for GES for symptoms thought to represent gastroparesis were enrolled in a prospective study. Patients wore a WPS for up to 6 days, starting on the day of scintigraphy. Gastric myoelectrical activity was analyzed and categorized into relatively weak, relatively moderate, and relatively strong activity phenotypes. A subset of patients underwent repeat monitoring following individualized therapeutic interventions (e.g., anti‐emetics, proton pump inhibitors, lifestyle changes). Results Forty‐eight patients (mean age 44; 79% female) completed the study. GES identified delayed gastric emptying in 18%, rapid in 22%, and normal in 60% of subjects. During the 4‐h concurrent GES window, gastric activity in patients with delayed emptying was lower, but this difference did not reach statistical significance ( p = 0.08). When compared to the full multiday recording period, GES results showed no correlation with the myoelectric readings. Significant day‐to‐day variability was observed, both random and systemic. In the latter category, a “Test‐Day” dip was observed with gastric activity lowest on Day 1 (GES test day) and increasing significantly on subsequent days, stabilizing by Day 4 ( p < 0.001). Healthy control subjects recorded during normal daily routines showed no such day‐to‐day variation. In the retesting cohort, anti‐emetics produced robust increases in gastric amplitude, whereas PPIs showed minimal impact. Conclusions The WPS effectively captures distinct gastric phenotypes and quantifies physiologic responses to therapy in both gastric and intestinal regions, demonstrating its clinical utility as a robust tool for evaluating and managing foregut symptoms in real‐world settings. Importantly, the multiday WPS highlights significant day‐to‐day physiologic variability in gastric activity that extends beyond the single‐day GES ‘snapshot’.
Systematic literature review of clinical outcomes, health-related quality of life, and healthcare costs and resource use in relapsed/refractory large B-cell lymphoma treated with axicabtagene ciloleucel
Lin, Yi, Palivela, Madhu, Lakzadeh, Pardis , Limbrick-Oldfield, Eve, Wade, Sally, Limbrick‐Oldfield, Eve H. , Hsu, Hil , Kanters, Steve , Sanderson, Robin

Leukemia & Lymphoma | July 27, 2026 | Volume: No volume available | Issue: No issue available | Pages: 1-16

10.1080/10428194.2026.2695323 | PMID 42507490 Get It

Progress report on new epilepsy treatments: A summary of the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices ( EILAT XVIII ). II . Trea
Bialer, Meir , Landmark, Cecilie Johannessen , Koepp, Matthias J. , Perucca, Emilio , Perucca, Piero , Tomson, Torbjörn , Wirrell, Elaine , Johannessen Landmark, Cecilie , White, H Steve , Wirrell, Elaine C , Perucca, Emilio

Epilepsia | July 18, 2026 | Volume: No volume available | Issue: No issue available | Pages: No page available

10.1002/epi.70346 | PMID 42470359 Get It

Abstract This article summarizes data for 13 investigational treatments for which at least preliminary seizure outcome data in patients with epilepsy were reported at the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices held in Madrid, Spain, on May 3–6, 2026. The treatments reviewed include bexicaserin, a selective 5‐hydroxytryptamine (5‐HT, serotonin) type 2C (5‐HT 2C ) receptor superagonist investigated as a treatment for developmental and epileptic encephalopathies (DEEs); BMB‐101, a selective 5‐HT 2C receptor agonist investigated for the treatment of absence seizures and DEEs; elsunersen, an antisense oligonucleotide designed for the treatment of early‐onset SCN2A ‐DEE; EPX‐100 (clemizole hydrochloride), an antihistamine endowed with agonist activity at 5‐HT 2A and 5‐HT 2B receptors, repurposed as a treatment for DEEs; ES‐481, an antagonist of α‐amino‐3‐hydroxy‐5‐methyl‐4‐isoxazolepropionic acid (AMPA) receptors containing the transmembrane AMPA receptor regulatory protein γ8 (TARP‐γ8), under investigation for the treatment of drug‐resistant epilepsy; ETX‐101, a gene therapy in development for the treatment of SCN1A ‐positive Dravet syndrome; PrevEp‐006 (intranasal seletracetam), a synaptic vesicle glycoprotein 2A (SV2A) ligand under investigation as an acute seizure rescue therapy; radiprodil, a negative allosteric modulator that binds to the GluN2B subunit of the N ‐methyl‐ D ‐aspartate (NMDA) receptor, in development for the treatment of seizures and other symptoms associated with GRIN‐related neurodevelopmental disorder, tuberous sclerosis complex, and focal cortical dysplasia type II; RAP‐219, a selective negative allosteric modulator of TARP‐γ8, in development for drug‐resistant focal epilepsy; relutrigine, a selective disease‐state sodium channel modulator investigated as a treatment for SCN2A ‐ and SCN8A ‐DEE; Staccato Alprazolam, a breath‐actuated device that delivers alprazolam to the lungs for rapid seizure termination; vormatrigine, a functionally selective sodium channel modulator in development for the treatment of focal seizures; and zorevunersen, an antisense oligonucleotide engineered to restore endogenous Nav1.1 protein expression, investigated for the treatment of Dravet syndrome. Overall, the evidence reviewed justifies expectations for improved health outcomes for the millions of children and adults with epilepsy who do not fully benefit from existing therapies.
Progress report on new epilepsy treatments: A summary of the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices ( EILAT XVIII ). I. Treatments in preclinical and early clinical development
Bialer, Meir , Landmark, Cecilie Johannessen , Koepp, Matthias J. , Perucca, Emilio , Perucca, Piero , Tomson, Torbjörn , Wirrell, Elaine , Johannessen Landmark, Cecilie , White, H Steve , Wirrell, Elaine C , Perucca, Emilio

Epilepsia | July 18, 2026 | Volume: No volume available | Issue: No issue available | Pages: No page available

10.1002/epi.70354 | PMID 42470358 Get It

Abstract Over the last 34 years, the Eilat Conference on New Antiepileptic Drugs and Devices has provided an interactive forum for stakeholders to discuss investigational and recently licensed treatments for seizures and epilepsy. The Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVIII) took place in Madrid, Spain, on May 3–6, 2026. This article provides summaries of eight treatments in preclinical and early clinical development that were presented at EILAT XVIII. These include AUT00206, a positive allosteric modulator of Kv3.1 and Kv3.2 voltage‐gated potassium channels in development for the treatment of rare epilepsy syndromes as well as neurodevelopmental and neuropsychiatric disorders; ION283, an antisense oligonucleotide designed to suppress the expression of glycogen synthase 1, under investigation for the treatment of Lafora disease; MSCA‐7136, a selective allosteric RAS/mitogen‐activated protein kinase—extracellular signal regulated kinase (MEK) inhibitor being developed for the treatment of focal seizures associated with mesial temporal lobe epilepsy and seizures associated with tuberous sclerosis complex; OV329, a selective inhibitor of γ‐aminobutyric acid (GABA) aminotransferase, in development for the treatment of drug‐resistant focal seizures; PTI5803 (probenecid), an uricosuric agent and a blocker of pannexin 1 channels, repurposed as a treatment for seizures associated with focal cortical dysplasia; simufilam, a filamin A modulator under investigation for the treatment of tuberous sclerosis complex‐related epilepsy; SN‐2000, a selective allosteric phosphodiesterase 4B inhibitor, in development for the treatment of focal seizures; and sodium selenate, a promoter of the dephosphorylation of pathological hyperphosphorylated tau in the brain, under investigation as a disease‐modifying agent in mesial temporal lobe epilepsy. Treatments in more advanced clinical development are discussed in an accompanying article.
Patterns of presentation and outcomes in stage IV Hodgkin lymphoma: A report from the Children's Oncology Group ( COG ) AHOD1331
Casey, Dana L. , Renfro, Lindsay A., Wu, Yue , McCarten, Kathleen, Pei, Qinglin , Milgrom, Sarah A., Cho, Steve, Keller, Frank G., Punnett, Angela, Kelly, Kara M., Castellino, Sharon M., Hoppe, Bradford S , Casey, Dana L , McCarten, Kathleen M. , Belsky, Jennifer A. , Keller, Frank G. , Punnett, Angela , Hodgson, David , Hoppe, Bradford S. , Kelly, Kara M. , Castellino, Sharon M.

British Journal of Haematology | July 9, 2026 | Volume: No volume available | Issue: No issue available | Pages: No page available

10.1111/bjh.70625 | PMID 42419755 Get It

Summary Outcomes for high‐risk paediatric classic Hodgkin lymphoma (cHL) improved with the addition of brentuximab vedotin (Bv) to doxorubicin, vincristine, etoposide, prednisone and cyclophosphamide (AVEPC) chemotherapy. We evaluated the prognostic implications of distant extra‐nodal sites contributing to stage IV disease. On AHOD1331 (NCT02166463), patients aged 2–21 years with high‐risk cHL (stage IIB with bulk/IIIB/IVA/IVB) were randomized to 5 cycles of doxorubicin, bleomycin, vincristine, etoposide, prednisone and cyclophosphamide (ABVE‐PC) versus Bv‐AVEPC. Patients with stage IV disease, determined by protocol‐defined and centrally reviewed Positron Emission Tomography ‐ Computed Tomography (PET‐CT), were identified. Baseline characteristics and progression‐free survival (PFS) were compared by pattern of stage IV involvement (lung, bone, bone marrow, multiple sites and others). A total of 340 patients (median age 15 years) with stage IV disease were included. Of these patients, 173 (51%) received ABVE‐PC and 167 (49%) Bv‐AVEPC. PFS was highest with lung‐only involvement (4‐year PFS 88.6%) and lowest with bone marrow‐only involvement (4‐year PFS 71.9%). Compared to ABVE‐PC, Bv‐AVEPC improved PFS among all stage IV patients (hazard ratio [HR] 0.53, 90% confidence interval (CI) 0.33–0.85) and in the lung‐only and bone‐only subgroups. However, no PFS benefit was observed in those with bone marrow‐only involvement. Among paediatric patients with stage IV cHL, Bv‐AVEPC improved PFS across most patterns of stage IV disease, except in those with bone marrow involvement. These results will guide efforts to improve outcomes among children with stage IV cHL.