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ELSO 2025 Narrative Guideline on ECMO Training and Continuing Education
Han, Peggy , Shekar, Kiran, Ramanathan, Kollengode, Brio, Jordi Riera del, Diaz, Rodrigo, Alfoudri, Huda , Ramanan, Raj , Sin, Simon W. C., Vercaemst, Leen , Friedrich, Tammy, Said, Ahmed, Zakhary, Bishoy, Riera, Jordi , Moore, Elizabeth A , Riera, Jordi , Moore, Elizabeth A. , Shekar, Kiran , Ramanathan, Kollengode R. , Puślecki, Mateusz , Sin, Wai Ching , Roeleveld, Peter Paul , Alinier, Guillaume , Zakhary, Bishoy

ASAIO Journal | March 30, 2026 | Volume: 72 | Issue: 4 | Pages: 274-283

10.1097/mat.0000000000002686 | PMID 41910607 Get It

Disclaimer: These guidelines are intended for educational use to build the knowledge of physicians and other health professionals in assessing the conditions and managing the treatment of patients undergoing extracorporeal life support (ECLS)/ECMO and describe what are believed to be useful and safe practice for ECLS and ECMO. The aim of clinical guidelines was to help clinicians to make informed decisions about their patients. However, adherence to a guideline does not guarantee a successful outcome. Healthcare professionals must make their own treatment decisions about care on a case-by-case basis, after consultation with their patients, using their clinical judgment, knowledge, and expertise. These guidelines do not take the place of physicians’ and other health professionals’ judgment in diagnosing and treatment of patients. These guidelines are not intended to and should not be interpreted as setting a standard of care or being deemed inclusive of all proper methods of care nor exclusive of other methods of care directed at obtaining the same results. The ultimate judgment must be made by the physician and other health professionals and the patient considering all the circumstances presented by the individual patient, and the known variability and biologic behavior of the clinical condition. These guidelines reflect the data at the time the guidelines were prepared; the results of subsequent studies or other information may cause revisions to the recommendations in these guidelines to be prudent to reflect new data, but ELSO is under no obligation to provide updates. In no event will ELSO be liable for any decision made or action taken in reliance upon the information provided through these guidelines.
Clinical, Genetic, and Pathologic Variability in Myelodysplastic Syndromes and Precursor Conditions Across Race, Ethnicity, and Sex
Colin‐Leitzinger, Christelle, Tang, Yi-Han , Sherman, Seth, Zhang, Ling, Walker, Mary Ellen, Painter, Jeffrey S. , Abel, Gregory A., Baghdadi, Tareq Al, Deeg, H. Joachim, Foran, James M., Gore, Steven D., Harrington, Alexandra M., Kroft, Steven H. , Liu, Jane Jijun, Saber, Wael, Virani, Shamsuddin, Bejar, Rafael, Padron, Eric , Komrokji, Rami, DeZern, Amy E., Sekeres, Mikkael A., Colin, Christelle , Foran, James M , Gillis, Nancy , Otterstatter, Michael , Zhang, Ling , Moscinski, Lynn C. , Walker, Mary E. , Abel, Gregory A. , Deeg, H. Joachim , Foran, James M. , Gore, Steven D. , Saber, Wael , Bejar, Rafael , Lindsley, R Coleman , Walter, Matthew J , Komrokji, Rami, Dezern, Amy Elizabeth , Sekeres, Mikkael A.

American Journal of Hematology | March 30, 2026 | Volume: 101 | Issue: 6 | Pages: 1407-1420

10.1002/ajh.70279 | PMID 41906220 Get It

ABSTRACT The epidemiology of myelodysplastic syndromes/neoplasms (MDS) is challenging to define due to inconsistent reporting, complex diagnostic procedures, and evolving diagnostic criteria. Using the National MDS Natural History Study—a prospective cohort with centrally adjudicated histopathology and genetic variant review—we characterized the landscape of MDS across the United States and identified differences across demographics. Among 2115 participants, 64% (1346) had an MDS spectrum condition, including MDS (24%), MDS/myeloproliferative neoplasm (5%) and precursor conditions—clonal cytopenia of undetermined significance (22%) and idiopathic cytopenia/dysplasia of undetermined significance (13%). The median age was 74 years, and participants were predominantly male (66%), White (91%), and Non‐Hispanic (92%). Myeloid‐associated variants were detected in 68% of participants, most commonly in TET2, DNMT3A, ASXL1, SF3B1, and SRSF2 . Black, compared to White, participants were younger at diagnosis (69 vs. 74 years, p = 0.01), had equal or increased prevalence of higher‐risk MDS, lower hemoglobin, and higher peripheral blood blasts, yet were less likely to receive MDS‐directed therapy (14% vs. 42%, p = 0.008). Black and Hispanic participants had fewer detectable gene mutations than White participants. Females had lower variant allele frequencies and fewer RNA splicing gene mutations than males. After multivariable adjustment, TP53 mutations, MDS diagnosis, and higher‐risk disease were associated with worse progression‐free and overall survival; age was also associated with overall survival. Black race trended toward improved progression‐free survival. These findings highlight the need for enhanced understanding of MDS pathogenesis across patient groups and refined prognostic tools to improve personalized management of MDS spectrum conditions. Trial Registration: ClinicalTrials.gov identifier: NCT02775383
Magnetic resonance–guided focused ultrasound for the management of tremor: update on the position statement of the American Society for Stereotactic and Functional Neurosurgery
Elias, W. Jeffrey, Baltuch, Gordon H., Machado, Andre G., Sweet, Jennifer A. , Englot, Dario J., Lipsman, Nir , Cosgrove, G. Rees, Ali, Rushna , Pilitsis, Julie G., Danish, Shabbar F. , Ali, Rushna , Hamani, Clement , Elias, W. Jeffrey , Gross, Robert E. , Rosenow, Joshua M. , Schwalb, Jason M. , Air, Ellen L. , Machado, André G. , Willie, Jon T , Rolston, John D. , Englot, Dario J. , Zemmar, Ajmal , Neimat, Joseph S. , Cosgrove, Garth Rees , Asaad, Wael F. , Pilitsis, Julie G.

Journal of Neurosurgery | March 28, 2026 | Volume: No volume available | Issue: No issue available | Pages: 1-9

10.3171/2025.11.jns251828 | PMID 41894813 Get It

OBJECTIVE Magnetic resonance–guided focused ultrasound (MRgFUS) thalamotomy has become a popular tool in the neurosurgical armamentarium for managing tremor in patients with essential tremor (ET) and Parkinson’s disease (PD). METHODS Given the recent exponential growth in the use of this technology, the American Society for Stereotactic and Functional Neurosurgery, which acts as the joint section representing the field of stereotactic and functional neurosurgery on behalf of the Congress of Neurological Surgeons and the American Association of Neurological Surgeons, provides an update on treatment indications and the efficacy and safety of MRgFUS for the treatment of tremor. RESULTS In ET, the efficacy of MRgFUS thalamotomy is supported by several open-label and 1 prospective, double-blind, sham-controlled randomized clinical trial (RCT) that showed a 47% improvement in hand tremor in the short term. Follow-up studies demonstrated that the benefits of MRgFUS for ET are sustained in the long term. Studies investigating the effects of bilateral staged MRgFUS thalamotomy in patients with ET have shown that tremor improvement on the second side was as significant as that observed after the first procedure. The efficacy of MRgFUS thalamotomy for PD tremor is supported by a systematic meta-analysis that consisted predominantly of smaller, nonrandomized studies; 1 RCT; and 1 prospective cohort study. Despite a significant short-term improvement in PD tremor, the long-term effects of this therapy remain unknown. Common early adverse effects of MRgFUS thalamotomy include gait disturbance and paresthesias. These, however, tend to subside over time. Other reported side effects are dysarthria, ataxia, taste disturbance, motor deficits, and speech difficulty. CONCLUSIONS MRgFUS thalamotomy is a safe, effective, and durable intervention for refractory ET in appropriately selected patients. The procedure appears to be safe and effective for PD tremor, although the long-term durability of the technique remains a concern. Early experience with bilateral staged lesions for ET suggests the procedure to be safe and effective.
Molecular-Based Ecosystem to Improve Personalized Medicine in Chronic Myelomonocytic Leukemia
Lanino, Luca , Hunter, Anthony M. , Gagelmann, Nico, Robin, M., Dall'Olio, Daniele , Flamigni, Alice, Gurnari, Carmelo, Wang, Yu‐Hung , Pleyer, Lisa , Xicoy, B., Montalban-Bravo, Guillermo, Shih, Lee-Yung, Alsugair, Ali, Gregorio, Caterina, Platzbecker, Anne Sophie, Sallman, David A., Fariselli, Piero, Garcia-Manero, Guillermo, Platzbecker, U, Solé, Francesc Gessé, Díez-Campelo, María, Maciejewski, Jaroslaw, Bejar, Rafael, Thol, Felicitas, Kroger, N., Fenaux, Pierre, Sanz, G. F., Kordasti, S., Santini, Valeria, Zeidan, Amer M., Komrokji, Rami S., Haferlach, Torsten, Germing, Ulrich, Castellani, Gastone, D'Amico, Saverio, Patnaik, M., Ieva, F., Tefferi, Ayalew, Padron, Eric , Porta, Matteo Giovanni Della, Alliance, for the iCPSS, Haque, Tamanna, Abdel-Wahab, Omar, Geissler, Klaus, Bataller, Alex, Bazinet, Alexandre , Meggendorfer, Manja , Quintavalla, Mirco, Bruseghini, Alessandro , Carota, Luciana , Dall'Olio, Lorenzo, Travaglino, Erica , Zamora, Lurdes López, Quintela, David, Jerez, Andrés , Cornejo, Elena, Martín, Paloma García, Díaz-Beyá, Marina, Pita, Alejandro Avendano, Roldán, Verónica , Suarez, Dolly Viviana Fiallo, Velasco, Estefania Cerezo, Calabuig, Marisa , Such, Esperanza, Castilla-Llorente, Cristina, Bulabois, Claude Eric, Souchet, Laëtitia, Bernardi, Massimo, Chiusolo, Patrizia, Borin, Lorenza, Follo, Matilde Yung, Consagra, Angela , Passamonti, Francesco, Diral, Elisa , Vucinic, Vladan, Bergonzi, Gregorio Maria , Hou, Hsin-An, Russo, Antonio, Tentori, Cristina Astrid , Buizza, Alessandro , Ferrari, Ivan, Matteo, Maria Di, Crespi, Alessandra, Figini, Giulia , Delponte, Camilla, Zampini, Matteo, Riva, Elena, Delleani, Mattia , Iascone, Eleonora, Ficara, Francesca , Ventura, Denise, Pinocchio, Nicole, Santoro, Armando, Kiwan, Ahmad , Ali, Najla H. Al, Zeidan, Amer M. , Robin, Marie , Dall’Olio, Daniele , Riva, Elena , Gagelmann, Nico , Robin, Marie , Sala, Claudia , Gurnari, Carmelo , Wang, Yu-Hung , Shih, Lee‐Yung , Alsugair, Ali , Fathima, Saubia , Gregorio, Caterina , Rollo, Cesare , Platzbecker, Anne Sophie , Asti, Gianluca , Itzykson, Raphael , Sallman, David A , Fariselli, Piero , Kern, Wolfgang , Garcia-Manero, Guillermo , Platzbecker, Uwe , Solé, Francesc , Diez-Campelo, Maria , Maciejewski, Jaroslaw P , Bejar, Rafael , Thol, Felicitas , Savona, Michael R , Sanz, Guillermo F , Kordasti, Shahram , Santini, Valeria , Zeidan, Amer M , Komrokji, Rami S , Castellani, Gastone , D'Amico, Saverio , Patnaik, Mrinal , Ieva, Francesca , Solary, Eric , Padron, Eric , Della Porta, Matteo Giovanni , Buizza, Alessandro , Ficara, Francesca , Lanino, Luca , Gagelmann, Nico , Gurnari, Carmelo , Xicoy, Blanca , Montalban‐Bravo, Guillermo , Fathima, Saubia , Gregorio, Caterina , Palomo, Laura , Itzykson, Raphaël , Sallman, David A. , Kern, Wolfgang , Garcia‐Manero, Guillermo , Platzbecker, Uwe , Solé, Françesc , Díez‐Campelo, María , Thol, Felicitas , Kröger, Nicolaus , Savona, Michael R. , Fenaux, Pierre , Sanz, Guillermo , Santini, Valeria , Fontenay, Michaëla , Komrokji, Rami S. , Haferlach, Torsten , Germing, Ulrich , Solary, Éric , Tefferi, Ayalew , Haque, Tamanna , Bataller, Alex , Sauta, Elisabetta , Sanavia, Tiziana , Dall'Olio, Lorenzo, Díaz‐Beyá, Marina , Roldan, Veronica, Fiallo, Dolly Viviana , Such, Esperanza , Castilla‐Llorente, Cristina , Bulabois, Claude‐Eric , Awada, Hussein , Chiusolo, Patrizia , Curti, Antonio , Giaccone, Luisa , Finelli, Carlo , Casetti, Ilaria Carola , Onida, Francesco , Passamonti, Francesco , Vučinić, Vladan , Voso, Maria Teresa , Hou, Hsin-An, Chou, Wen‐Chien , Yao, Chi‐Yuan , Lin, Chien‐Chin , Tien, Hwei‐Fang , Campagna, Alessia , Ubezio, Marta , Todisco, Gabriele , Maggioni, Giulia , Matteo, Maria Di, Pesce, Francesco , Zampini, Matteo , Crisafulli, Laura , Ventura, Denise , Pinocchio, Nicole , Brindisi, Matteo , VanOudenhove, Jennifer , Ali, Najla H. Al, Graubert, Timothy Aaron , Thota, Swapna , Griffiths, Elizabeth A.

Journal of Clinical Oncology | March 27, 2026 | Volume: 44 | Issue: 17 | Pages: 1608-1623

10.1200/jco-25-02116 | PMID 41894646 Get It

PURPOSE Chronic myelomonocytic leukemia (CMML) is a rare myeloid neoplasm characterized by clinical heterogeneity and is associated with poor outcomes. To date, limited molecular information has been incorporated into disease classification and risk stratification. We aimed to integrate genomic features into the clinical decision-making process for CMML. PATIENTS AND METHODS We analyzed a retrospective cohort of 3013 patients with CMML (training set) and a prospective population of 516 patients (validation set). Using an innovative framework for multimodal data analysis, we developed molecular-based disease taxonomy and prognostication. RESULTS Unsupervised clustering identified nine entities with distinct genomic features and outcomes ( P < .001), including splicing machinery, transcription factors, signal transduction and tyrosine kinase pathways aberrations, and high-risk molecular signatures. Notably, 15% of patients showed molecular/clinical overlap with other myeloid neoplasms. We integrated molecular and clinical information to build the international CMML Prognostic Scoring System (iCPSS), incorporating mutations in nine genes together with hematologic parameters and cytogenetic abnormalities. The iCPSS identified five groups with distinct probability of overall and leukemia-free survival in both training and validation cohorts ( P < .001), outperforming existing prognostic models. Importantly, 55% of patients were reassigned to higher or lower risk groups by the iCPSS. Decision analysis demonstrated that iCPSS could refine the optimal timing of allogeneic transplantation at the individual level; compared with conventional prognostic tools, iCPSS-based decision modeling changed transplantation strategy in 31% of cases, resulting in a significant gain-in-life expectancy for eligible patient population ( P < .001). A federated learning platform was implemented to enable continuous, privacy-preserving model update across multiple centers. CONCLUSION Molecular information improves CMML classification and prognostication, supports more effective clinical decision making, and potentially refines the design of clinical trials.
The landscape of dementia research, diagnosis, treatment, and care in Latin America
Suemoto, Claudia K., Custodio, Nilton, Avila‐Funes, José A., Baez, Sandra, Bagnati, Pablo , Barnes, Lisa Laverne, D., Brucki Sonia M., Calandri, Ismael L., Cornejo‐Olivas, Mario, Derio, Carolina Delgado, García, Adolfo M., Grinberg, Lea T., Ibanez, Agustin M., Isasi, Rosario, Josephy‐Hernandez, Sylvia , Porras, Maria Lazo, Llibre‐Guerra, Jorge J., Llibre‐Rodriguez, Juan J., Meza, B. Marcela Mar, Migeot, Joaquin, Miranda, J. Jaime, Miranda‐Castillo, Claudia, Mummery, Catherine J., Parodi, José F., Castro, Natalia Pozo, Contreras, Rosa María Salinas, Slachevsky, Andrea, Souza‐Talarico, Juliana Nery, Takada, Leonel T., Tsoy, Elena, Pilalumbo, Martha Unaucho, Zimmer, Eduardo R., Fontana, Igor C. , Snyder, Heather M. , Carrillo, Marı́a C., Carrillo, María C. , Takada, Leonel Tadao , Custodio, Nilton , Ibáñez, Agustín , Suemoto, Cláudia Kimie , Delgado, Carolina , Mahinrad, Simin , Ibañez, Agustín , Suemoto, Claudia Kimie , Aguilar, Diego, Ávila‐Funes, José Alberto , Báez, Sandra , Barnes, Lisa L. , Brucki, Sônia Maria Dozzi , Caramelli, Paulo , Cornejo-Olivas, Mario , Ferreira, Sérgio T. , García, A.R. , Grinberg, Lea T. , Llibre‐Guerra, Jorge J. , Rodríguez, Juan J. Llibre , Lourenco, Mychael V. , Migeot, Joaquín , Miranda, J. Jaime , Miranda‐Castillo, Claudia , Mummery, Catherine J. , Parodi, Jose Francisco , Salinas‐Contreras, Rosa Maria, Santamaría‐García, Hernando A , Slachevsky, Andrea , Souza‐Talarico, Juliana Nery , Surace, Ezequiel , Tsoy, Elena , Unaucho‐Pilalumbo, Martha , Zimmer, Eduardo Rigon , Mahinrad, Simin

Alzheimer's & Dementia | March 27, 2026 | Volume: 22 | Issue: 3 | Pages: No page available

10.1002/alz.71309 | PMID 41894159 Get It

Abstract Latin America is undergoing rapid population aging alongside a rising burden of dementia. While the region holds substantial potential for dementia risk reduction, challenges remain, such as delayed diagnoses, limited access to specialized care and biomarker testing, persistent stigma, and deep‐rooted structural inequities. To address these gaps and foster regionally informed solutions, the Alzheimer's Association convened the 2025 Alzheimer's Association International Conference (AAIC) Satellite Symposium in Lima, Peru, on May 14–15, in collaboration with the Global Brain Health Institute (GBHI) and the Atlantic Fellows for Equity in Brain Health. The meeting aimed to bring core elements of the global AAIC meeting to regional Latin American settings, recognizing that national and cultural contexts demand tailored approaches to dementia prevention, risk reduction, treatment and care all aimed at promoting brain health in the region. This manuscript synthesizes the symposium's key discussions, scientific advances, and opportunities for collaboration across the region.
BASECAMP-1 screening study: a model for efficient enrolment in precision oncology clinical trials
Hecht, J Randolph , Molina, Julian R , Liechty, Kirstin B., Welling, Theodore H., Grierson, Patrick , Patel, Sandip Pravin , Kirtane, Kedar , Morelli, M Pia, Locke, Frederick L, Maloney, David G. , Nikiforow, Sarah, Lin, Yi, Ulrickson, Matthew, Specht, J. M., Lozac’hmeur, Ariane, Garde, Ryan J., Rangel, Gena A, Ng, Eric W, Welch, John S. , Tebbets, Jessica , Go, William Y., Specht, Jennifer M. , Tebbets, Jessica C , Specht, Jennifer , Molina, Julian R. , Morelli, Maria Pia , Locke, Frederick L. , Punekar, Salman R. , Nikiforow, Sarah , Ulrickson, Matthew L. , Osterman, Chelsea Kendall , Ng, Eric W., Simeone, Diane M.

BMJ Oncology | March 27, 2026 | Volume: 5 | Issue: 1 | Pages: e001033

10.1136/bmjonc-2025-001033 | PMID 41918932 Get It

Objective Identifying eligible patients for precision oncology clinical trials is challenging, particularly for rare molecular subpopulations. To address this challenge, A2 Biotherapeutics developed BASECAMP-1 ( NCT04981119 ), a non-interventional master screening study to identify patients eligible for interventional studies of logic-gated Tmod chimeric antigen receptor T-cell therapies. Eligible patients for these interventional trials have an advanced solid malignancy and are germline human leucocyte antigen (HLA)-A*02 heterozygous, with tumour-associated HLA-A loss of heterozygosity (LOH). HLA-A LOH occurs in ~16% of advanced solid malignancies; therefore, an efficient screening strategy is required. This report describes BASECAMP-1; compares the efficiency of two screening methods; and discusses the broader advantages of BASECAMP-1 beyond efficient enrolment. Methods and analysis Patients are identified for BASECAMP-1 using two approaches. In the traditional approach, common for clinical trials, investigators consent and screen all patients who might be good candidates for cell therapy trials, with no prior knowledge of patient HLA-A type or LOH status. To further optimise our approach, we co-developed with Tempus AI (Tempus) the bioinformatic programme Aware, which identifies potentially eligible patients with tumour-associated HLA-A*02 LOH within a clinico-genomic database that includes linked genomic and transcriptomic sequencing and clinical data collected during routine care. Results Over 42 months of using a traditional approach to identify eligible patients, 1918 patients at 13 study sites were consented and screened for BASECAMP-1; of these, 30 patients with tumour-associated HLA-A*02 LOH were enrolled (~0.7 participants per month). Over the last 30 months of that same period, Tempus Aware screening was implemented and 55 patients with tumour-associated HLA-A*02 LOH were enrolled (~1.8 participants per month). The bioinformatic approach identified more patients than the traditional approach and used sequencing results produced as part of the standard clinical tumour sequencing workflow, reducing resource use and study staff burden. Additional advantages of using a screening study, such as BASECAMP-1, include manufacturing efficiencies and collection of a large dataset of molecular and clinical parameters that can be used to supplement trial analyses. Conclusions The BASECAMP-1 study demonstrates a clinico-genomic screening approach can more efficiently identify patients for precision oncology trials. Furthermore, precision oncology can be enhanced through collaborative data-sharing. Trial registration number NCT04981119 .
Naturalistic Driving Outcomes and Sensorimotor Function in Cognitively Normal Older Adults
Bekena, Semere , Singh, Ramkrishna K. , Zhu, Yiqi, Harrison, Kimystian L. , Williams, Jonathan P., Laurido‐Soto, Osvaldo J., Dickerson, Anne, Carr, David B. , Babulal, Ganesh M., Singh, Ramkrishna K. , Laurido-Soto, Osvaldo , Al‐Hammadi, Noor , Dickerson, Anne D., Babulal, Ganesh M.

Journal of the American Geriatrics Society | March 26, 2026 | Volume: 74 | Issue: 5 | Pages: 1358-1368

10.1111/jgs.70400 | PMID 41884999 Get It

ABSTRACT Background Driving supports independence and quality of life in later life but is vulnerable to age‐related and/or disease‐related decline. While cognitive impairment is a well‐recognized risk for unsafe mobility, the contribution of sensorimotor function is less understood. Methods We studied 374 cognitively normal older adults (Clinical Dementia Rating = 0 at baseline) enrolled in the DRIVES Project. A SensoryMotor Impairment Index (SMI) was constructed from six domains (grip strength, gait speed, reaction time, hearing, vision, olfaction) and categorized as 0, 1, 2, or ≥ 3 impairments. Naturalistic driving was continuously monitored using in‐vehicle GPS dataloggers between 2019 and 2025. Linear mixed‐effects models tested whether longitudinal driving trajectories differed by baseline SMI, adjusting for age, sex, race, education, body mass index, and cognition (Preclinical Alzheimer's Cognitive Composite). Results At baseline, participants with higher SMI burden were older and had lower cognitive scores (both p < 0.01). Over time, greater sensorymotor impairment was associated with faster declines in driving exposure and spatial range. Compared with SMI = 0, participants with SMI ≥ 3 showed steeper reductions in nighttime trips (−0.050 vs. −0.037 trips/month), long‐distance trips (> 20 miles; p < 0.001), and maximum trip distance ( p = 0.009), and greater contraction of driving space (entropy and radius of gyration; p < 0.001). Self‐reported driving behaviors showed that participants with higher SMI drove fewer days per‐week and were more likely to avoid night driving ( p < 0.01). Rates of self‐reported adverse driving events (e.g., crashes or citations) did not differ significantly across SMI groups, consistent with compensatory self‐regulatory behaviors. Conclusion Greater sensorymotor impairment predicts accelerated decline in naturalistic driving among cognitively normal older adults, independent of cognition. A composite SMI may provide a feasible, low‐cost approach to identify older drivers at risk for declining mobility and support timely interventions to prolong safe driving.
Minimal Clinically Important Difference of the Sinonasal Outcome Test in Sinonasal Malignancy
Maoz, Sabrina L. , Lee, Michelle , Fischer, Jakob L. , Choby, Garret M., Hwang, Peter H. , Wang, En, Adappa, Nithin D., Getz, Anne E., Humphreys, Ian M. , Pinheiro-Neto, Carlos D., Chan, Erik, Roozdar, Pooya, Chang, Eugene H., Jafari, Aria, Kingdom, Todd T. , Kohanski, Michael A. , Lee, Jivianne T. , Lazor, Jillian W. , Nabavizadeh, Ali, Palmer, James N., Patel, Zara M. , Smith, Tim L., Snyderman, Carl H., John, Maie A. St, Storm, Phillip B., Wang, Marilene B., Sim, Myung‐Shin , Beswick, Daniel M., Fischer, Jakob Louis , Humphreys, Ian M , Chang, Eugene H , Snyderman, Carl H , Patel, Zara , Hwang, Peter H , Maoz, Sabrina L , Choby, Garret , Kuan, Edward C , Adappa, Nithin D. , Geltzeiler, Mathew , Le, Christopher H. , Pinheiro‐Neto, Carlos , Abuzeid, Waleed M. , Chang, Eugene H. , Jafari, Aria , Nayak, Jayakar V. , Adappa, Nithin D. , Resnick, Adam , John, Maie A. St., Storm, Phillip B. , Suh, Jeffrey D. , Beswick, Daniel M.

Annals of Otology, Rhinology & Laryngology | March 26, 2026 | Volume: 135 | Issue: 7 | Pages: 543-549

10.1177/00034894261428393 | PMID 41881811 Get It

Background: Sinonasal malignancies (SNM) are rare cancers associated with poor prognosis and impaired quality of life (QOL). The 22-question Sino-Nasal Outcome Test (SNOT-22) has been used to assess QOL in patients with SNM. Objective: To determine the minimal clinically important difference (MCID) for the SNOT-22 in patients with SNM. Methods: About 264 patients with SNM were drawn from a prospective, longitudinal, multi-institutional study. SNOT-22 scores were collected at pre-treatment baseline and follow-up through 5 years post-treatment. Cronbach’s alpha was used to assess the internal consistency of the SNOT-22. Distribution-based approaches were used to estimate the MCID. Multivariable regression identified factors associated with SNOT-22 improvement exceeding the determined MCID. Results: Cronbach’s alpha was >.85. The estimated MCIDs were: 1.44 (Cohen’s effect size), 1.32 (standard error of the mean), 10.45 (1/2 of baseline standard deviation), and 3.67 (minimum detectable change). The mean MCID across methods was 4.22. Multivariable regression demonstrated higher odds of clinically meaningful improvement from baseline in patients with early-stage disease (odds ratio (OR) 3.510, P = .035), no neck irradiation (OR 11.050, P = .014), and neoadjuvant therapy (OR 16.667, P = .047). Conclusions: The SNOT-22 MCID was estimated as 4.22 in a large multi-center cohort of patients with SNM, suggesting that relatively small QOL changes are clinically relevant. When applying the MCID to the current cohort, advanced stage, treatment of neck disease, and not requiring neoadjuvant therapy were predictive of worse QOL.
NT-I7, a Long-Acting Interleukin 7, Increases Lymphocyte Counts and Induces CD8+ T-cell Clonotype Expansion in Patients with Newly Diagnosed High-Grade Gliomas
Butt, Omar H. , Singhal, Kartik , Luo, Jingqin , Rettig, Michael P. , Foltz, Jennifer A. , Huang, Jiayi , Zhou, Alice Y. , Tao, Yu , Griffith, Obi L., Griffith, Malachi, Johanns, Tanner M. , Ansstas, George , Zhang, Chaocai , Tang, Justin , Christ, Stephanie , Ferrando-Martinez, Sara , Lee, Byung Ha , Chheda, Milan G., Fehniger, Todd A. , Campian, Jian L., Griffith, Malachi , Griffith, Obi L. , Huang, Jiayi , Griffith, Obi L , Griffith, Malachi , DiPersio, John F , Chheda, Milan G , Fehniger, Todd A , Campian, Jian L , Luo, Jingqin , Ansstas, George , DiPersio, John F. , Chheda, Milan G. , Campian, Jian

Clinical Cancer Research | March 25, 2026 | Volume: 32 | Issue: 13 | Pages: 2581-2592

10.1158/1078-0432.ccr-25-2953 | PMID 41880597 Get It

Abstract Purpose: Standard care for high-grade gliomas (HGG) involves maximal surgical resection followed by radiation and temozolomide. Postoperative adjuvant therapy frequently causes lymphopenia, which is associated with poor prognosis. Interleukin 7 (IL7) is essential for lymphocyte development, homeostasis, and survival. NT-I7 (efineptakin alfa), a long-acting recombinant IL7, reverses lymphopenia and improves survival in murine glioma models. However, the safety, maximum tolerated dose (MTD), and impact of NT-I7 on immune cells in patients with HGG remain unknown. Patients and Methods: We conducted a phase I trial (NCT03687957) examining the MTD and effect of NT-I7 on lymphocytes in patients with newly diagnosed HGG. The primary endpoint was dose-limiting toxicity; secondary endpoints included absolute lymphocyte count (ALC) changes over time, overall response, progression-free survival, and overall survival. Exploratory endpoints included immune profiling at different time points using single-cell RNA sequencing (scRNA-seq) in a subset of patients. Results: NT-I7 was well tolerated with a MTD of 720 µg/kg. Moreover, NT-I7 significantly increased ALCs for more than 12 weeks in duration. Early elevations in CD4+, CD8+ T cells and NK cells further coincided with increased TNF and CXCL9 cytokine levels. Comprehensive immune profiling of peripheral blood T cells revealed selective clonotype expansion within CD8+, but not CD4+, T cells following NT-I7 administration. Finally, a subset of our patients with MGMT promoter–unmethylated glioblastoma, which are typically associated with a poorer prognosis, demonstrated promising clinical responses. Conclusions: NT-I7 has the potential to maintain and increase lymphocyte counts in patients with HGG and warrants further investigation, particularly in combination with immune-based therapies.