Article
Oncology
Mark Agulnik, Brian Schulte, Steven Robinson, Angela C. Hirbe, Kevin Kozak, Sant P. Chawla, Steven Attia, Alfred Rademaker, Hui Zhang, Susan Abbinanti, Rasima Cehic, Varun Monga, Mohammed Milhem, Scott Okuno, Brian A. Van Tine
Summary: Regorafenib is a safe and effective treatment for refractory metastatic and unresectable angiosarcoma, showing good efficacy with acceptable rates of adverse events.
EUROPEAN JOURNAL OF CANCER
(2021)
Article
Oncology
Diana M. Carvalho, Peter J. Richardson, Nagore Olaciregui, Reda Stankunaite, Cinzia Lavarino, Valeria Molinari, Elizabeth A. Corley, Daniel P. Smith, Ruth Ruddle, Adam Donovan, Akos Pal, Florence Raynaud, Sara Temelso, Alan Mackay, John P. Overington, Anne Phelan, David Sheppard, Andrew Mackinnon, Bassel Zebian, Safa Al-Sarraj, Ashirwad Merve, Jeremy Pryce, Jacques Grill, Michael Hubank, Ofelia Cruz, Andres Morales La Madrid, Sabine Mueller, Angel M. Carcaboso, Fernando Carceller, Chris Jones
Summary: Using artificial intelligence, researchers have identified and validated the combination of vandetanib and everolimus as a potential therapeutic approach for ACVR1-mutant DIPG. This combination showed synergistic effects in vitro and in vivo, significantly improving survival and reducing tumor burden in patients.
Article
Oncology
Siren Morken, Seppo W. Langer, Anna Sundlov, Lene Weber Vestermark, Morten Ladekarl, Geir Olav Hjortland, Johanna B. Svensson, Elizaveta Mitkina Tabaksblat, Torjan Magne Haslerud, Jorg Assmus, Sonke Detlefsen, Anne Couvelard, Aurel Perren, Halfdan Sorbye
Summary: This study evaluated the efficacy and safety of everolimus and temozolomide as first-line treatment for metastatic high-grade gastroenteropancreatic neuroendocrine neoplasms. The results showed that this treatment option can effectively control the disease and prolong progression-free survival and overall survival. The treatment response was better in NET G3 patients, but there were some toxicities reported.
BRITISH JOURNAL OF CANCER
(2023)
Article
Multidisciplinary Sciences
Min Qiu, Yan Tang, Jinjin Chen, Rachel Muriph, Zhongfeng Ye, Changfeng Huang, Jason Evans, Elizabeth P. Henske, Qiaobing Xu
Summary: The challenge of delivering mRNA to specific organs and cells in vivo is being addressed, with research showing that N-series LNPs can selectively deliver mRNA to the lungs while O-series LNPs tend to deliver mRNA to the liver. By analyzing the protein corona on targeted LNPs, unique plasma proteins were identified that contribute to their targetability. This research demonstrates the potential of LNPs as a promising therapeutic intervention for lung diseases like lymphangioleiomyomatosis.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
(2022)
Article
Oncology
Karen D. Wright, Xiaopan Yao, Wendy B. London, Pei-Chi Kao, Lia Gore, Stephen Hunger, Russ Geyer, Kenneth J. Cohen, Jeffrey C. Allen, Howard M. Katzenstein, Amy Smith, Jessica Boklan, Kellie Nazemi, Tanya Trippett, Matthias Karajannis, Cynthia Herzog, Joseph Destefano, Jennifer Direnzo, Jay Pietrantonio, Lianne Greenspan, Danielle Cassidy, Debra Schissel, John Perentesis, Mitali Basu, Tomoyuki Mizuno, Alexander A. Vinks, Sanjay P. Prabhu, Susan N. Chi, Mark W. Kieran
Summary: Everolimus shows efficacy as an alternative treatment for pediatric patients with radiographically progressive low-grade glioma, with a well-tolerated profile. Some patients experienced toxicities, but overall survival rates were relatively high.
PEDIATRIC BLOOD & CANCER
(2021)
Article
Genetics & Heredity
Chongsheng Cheng, Wenshuai Xu, Yani Wang, Tengyue Zhang, Luning Yang, Wangji Zhou, Danjing Hu, Yanli Yang, Xinlun Tian, Kai-Feng Xu
Summary: This study found that high CT grade and age at presentation ≤ 35 years were associated with an increased risk of pneumothorax in patients with LAM, while postmenopausal status correlated with a lower risk of pneumothorax. Additionally, sirolimus was found to reduce the risk of pneumothorax recurrence in LAM patients.
ORPHANET JOURNAL OF RARE DISEASES
(2022)
Article
Oncology
Philip Zeuschner, Sebastian Hoelters, Michael Stoeckle, Barbara Seliger, Anja Mueller, Hagen S. Bachmann, Viktor Gruenwald, Daniel C. Christoph, Arnulf Stenzl, Marc-Oliver Grimm, Fabian Bruening, Peter J. Goebell, Marinela Augustin, Frederik Roos, Johanna Harde, Iris Benz-Rued, Michael Staehler, Kerstin Junker
Summary: In patients with metastatic renal cell carcinoma treated with the mTOR inhibitor everolimus, lower levels of TSP-2 and higher levels of LDH were found to be predictive blood biomarkers associated with therapy response. These biomarkers had higher predictive value than baseline patient parameters or risk classifications, highlighting the need for predictive biomarkers in mRCC therapy. Polymorphisms in the mTOR gene may be associated with therapy response, but further research is needed to confirm this finding.
Article
Oncology
S. Phadnis, X. Wang, N. C. Daw, I. M. Subbiah, W. Zaky, M. A. Gouda, A. C. Morani, B. Amini, D. J. Harrison, S. A. Piha-Paul, F. Meric-Bernstam, R. Gorlick, C. L. Schwartz, V. Subbiah
Summary: The combination of vandetanib and everolimus showed early activity and tolerable toxicity profile in pediatric patients with advanced cancers.
Article
Oncology
Hiroya Takeuchi, Yoshinori Ito, Ryunosuke Machida, Ken Kato, Masakatsu Onozawa, Keiko Minashi, Tomonori Yano, Kenichi Nakamura, Takahiro Tsushima, Hiroki Hara, Tatsuya Okuno, Shuichi Hironaka, Isao Nozaki, Takashi Ura, Keisho Chin, Takashi Kojima, Shiko Seki, Katsuyuki Sakanaka, Haruhiko Fukuda, Yuko Kitagawa
Summary: This study aimed to confirm the efficacy of CRT modifications in reducing CRT-related toxicities and facilitating salvage treatment for improved survival in patients with locally advanced EC who refuse surgery.
INTERNATIONAL JOURNAL OF RADIATION ONCOLOGY BIOLOGY PHYSICS
(2022)
Article
Multidisciplinary Sciences
Eva Revilla-Lopez, Cristina Berastegui, Alejandra Mendez, Berta Saez-Gimenez, Victoria Ruiz de Miguel, Manuel Lopez-Meseguer, Victor Monforte, Carlos Bravo, Miguel Angel Pujana, Maria Antonia Ramon, Susana Gomez-Olles, Antonio Roman
Summary: This study found that sirolimus treatment had a positive long-term effect on most LAM patients, with 59% showing a positive response, 30% showing a partial response, and 11% showing a negative response after 5 years of treatment.
SCIENTIFIC REPORTS
(2021)
Article
Gastroenterology & Hepatology
Walter Reinisch, William J. Sandborn, Silvio Danese, Xavier Hebuterne, Maria Klopocka, Dino Tarabar, Tomas Vanasek, Milos Gregus, Paul A. Hellstern, Joo Sung Kim, Miles P. Sparrow, Kenneth J. Gorelick, Michael Hoy, Martina Goetsch, Caleb Bliss, Charu Gupta, Fabio Cataldi, Severine Vermeire
Summary: Ontamalimab showed efficacy and safety in patients with moderate-to-severe ulcerative colitis compared to placebo in a controlled study, with a 72-week follow-up demonstrating good tolerability of the drug and significant reduction in the occurrence of certain serious adverse events.
JOURNAL OF CROHNS & COLITIS
(2021)
Article
Medicine, General & Internal
Ji Zhang, Dong Liu, Bingqing Yue, Le Ban, Min Zhou, Hongmei Wang, Jian Lv, Bo Wu, Zhenguo Zhai, Kai-Feng Xu, Wenhui Chen, Jingyu Chen
Summary: This study summarized the clinical data of 25 patients with pulmonary LAM who underwent lung transplantation at two hospitals in China between 2010 and 2018. The study highlighted the importance of lung transplantation for LAM patients in the Asian population and emphasized the need for further research on sirolimus use and LAM-related complications.
FRONTIERS IN MEDICINE
(2021)
Article
Pharmacology & Pharmacy
Roberto Previtali, Giorgia Prontera, Enrico Alfei, Luisa Nespoli, Silvia Masnada, Pierangelo Veggiotti, Savina Mannarino
Summary: Tuberous sclerosis complex (TSC) is a genetic disease characterized by abnormal cell proliferation and differentiation, leading to the growth of hamartomas. The disease is caused by the activation of mTOR protein. Symptomatic therapies have been attempted in the past, but they are not fully effective in modifying long-term outcomes. Everolimus, an allosteric inhibitor of mTORC1, has shown promising results in reducing symptoms and correcting the molecular defect associated with TSC.
PHARMACOLOGICAL RESEARCH
(2023)
Article
Medicine, General & Internal
Maxi Pia Bretschneider, Lena Roth, Peter E. H. Schwarz
Summary: This study aimed to provide preliminary evidence for the effectiveness of the mebix app-based treatment program in patients with type II diabetes mellitus. The results showed that after 3 months of using mebix, participants significantly reduced their HbA1c levels and experienced improvements in weight, well-being, and self-management. The app-generated data supported the finding that higher baseline HbA1c values led to greater reductions.
JOURNAL OF CLINICAL MEDICINE
(2023)
Article
Oncology
Toni Choueiri, Camillo Porta, Cristina Suarez, John Hainsworth, Eric Voog, Ignacio Duran, James Reeves, Piotr Czaykowski, Daniel Castellano, Jingjing Chen, Farhad Sedarati, Thomas Powles
Summary: This study evaluated the efficacy and safety of single-agent sapanisertib and sapanisertib plus the PI3K alpha inhibitor TAK-117 versus everolimus in patients with advanced clear cell renal cell carcinoma that had progressed on or after VEGF-targeted therapy. The results showed that sapanisertib, with or without TAK-117, was not more effective than everolimus in improving outcomes in patients with relapsed or refractory advanced ccRCC, and had lower tolerability.
Article
Respiratory System
Asa Lina M. Jonsson, Elisabeth Bendstrup, Susie Mogensen, Elizabeth J. Kopras, Francis X. McCormack, Ilaria Campo, Francesca Mariani, Amparo Escribano-Montaner, Are M. Holm, Maria del Mar Martinez-Colls, Guillem Pintos-Morell, Camille Taille, Bruno Crestani, Ole Hilberg, Jane Hvarregaard Christensen, Ulf Simonsen
EUROPEAN RESPIRATORY JOURNAL
(2020)
Article
Cardiac & Cardiovascular Systems
Vera P. Krymskaya, Andrew M. Courtwright, Victoria Fleck, Daniel Dorgan, Robert Kotloff, Francis X. McCormack, Maryl Kreider
RESPIRATORY MEDICINE
(2020)
Editorial Material
Critical Care Medicine
Nishant Gupta, Jack H. Bleesing, Francis X. McCormack
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
(2020)
Editorial Material
Medicine, Research & Experimental
Michael T. Dellinger, Francis X. McCormack
EMBO MOLECULAR MEDICINE
(2020)
Article
Respiratory System
Saisakul Chernbumroong, Janice Johnson, Nishant Gupta, Suzanne Miller, Francis X. McCormack, Jonathan M. Garibaldi, Simon R. Johnson
Summary: Machine learning was used to identify clinically relevant clusters associated with complications and outcomes in Lymphangioleiomyomatosis (LAM) patients, dividing them into two to three groups. Assigning individuals to these clusters could improve decision-making and prognostic information for patients.
EUROPEAN RESPIRATORY JOURNAL
(2021)
Article
Respiratory System
Patrick Kosciuk, Cristopher Meyer, Kathryn A. Wikenheiser-Brokamp, Francis X. McCormack
EUROPEAN RESPIRATORY REVIEW
(2020)
Article
Medicine, General & Internal
Martina Bonifazi, Federico Mei, Edlira Skrami, Lara Letizia Latini, Donatella Amico, Elisabetta Balestro, Francesco Bini, Floriano Bonifazi, Antonella Caminati, Piero Candoli, Saverio Cinti, Susanna Contucci, Alessandro Di Marco Berardino, Sergio Harari, Guido Levi, Sara Lococo, Vincenzo Menditto, Giampietro Marchetti, Sara Piciucchi, Venerino Poletti, Claudia Ravaglia, Marina Saetta, Gianluca Svegliati-Baroni, Sara Tomassetti, Mario Tamburrini, Alessandro Zanforlin, Umberto Zuccon, Lina Zuccatosta, Stefano Gasparini, Flavia Carle
Summary: Obesity and older age are significant independent predictors for the need of mechanical ventilation in young and middle-aged adults, while comorbidities such as hypertension, diabetes, asthma, and increased D-dimer levels are significantly associated with higher mortality risk. Factors like age of onset, body mass index, and mechanical ventilation do not change the impact of these comorbidities on survival.
JOURNAL OF CLINICAL MEDICINE
(2021)
Article
Cardiac & Cardiovascular Systems
Krishnan Warrior, Lorriana E. Leard, Arun R. Nair, Cynthia J. Gries, Andrew J. Fisher, Simon R. Johnson, Francis X. McCormack, Daniel F. Dilling
Summary: There is significant variability in the use of mTOR inhibitors in LAM patients on the lung transplant waiting list worldwide. Our survey suggests favorable outcomes for those patients who continue mTOR inhibitor use up to the time of transplant.
RESPIRATORY MEDICINE
(2022)
Article
Medicine, General & Internal
Alexander Poellinger, Sabina Berezowska, Jeffrey Leon Myers, Adrian Huber, Manuela Funke-Chambour, Sabina Guler, Thomas Geiser, Sergio Harari, Antonella Caminati, Maurizio Zompatori, Nicola Sverzellati
Summary: The study aimed to determine the frequency and distribution of the octopus sign on high-resolution CT images in patients with PLCH. The radiological presence of the octopus sign seems to be dependent on the stage of PLCH, particularly in cases of nodular and cystic lung disease.
Meeting Abstract
Critical Care Medicine
N. Gupta, A. Palipana, H. Lee, S. Johnson, F. X. McCormack, R. Szczesniak
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
(2020)
Meeting Abstract
Critical Care Medicine
Y. Hasegawa, Y. Tanaka, C. M. Shaver, K. K. Brown, C. Perkins, L. B. Pistick, C. G. McKnight, H. Wu, E. J. Kopras, F. X. McCormack
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
(2020)
Meeting Abstract
Critical Care Medicine
T. Hwalek, A. Osterburg, M. T. Borchers, F. X. McCormack
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
(2020)
Meeting Abstract
Critical Care Medicine
A. Kelly, T. D. Le Cras, E. J. Kopras, M. O'Callaghan, T. J. McDonnell, J. Song, C. E. Girod, M. Baqir, T. Moua, J. H. Ryu, F. X. McCormack, C. McCarthy
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
(2020)
Meeting Abstract
Critical Care Medicine
Y. Lu, E. Zhang, M. Guo, X. Zhao, M. Adam, N. Gupta, E. J. Kopras, A. T. Perl, K. A. Wikenheiser-Brokamp, M. Soleimani, K. Setchell, S. Sherman, S. Potter, J. A. Whitsett, F. X. McCormack, Y. Xu, J. J. Yu
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
(2020)
Article
Medicine, Research & Experimental
Huan Liu, Andrew R. Osterburg, Jennifer Flury, Zulma Swank, Dennis W. McGraw, Nishant Gupta, Kathryn A. Wikenheiser-Brokamp, Ashish Kumar, Abdellatif Tazi, Yoshikazu Inoue, Masaki Hirose, Francis X. McCormack, Michael T. Borchers