Article
Clinical Neurology
Xuexue Bai, Xiaomin Liu, Jun Wen
Summary: By retrospective analysis, we found that bevacizumab could improve progression-free survival and overall survival in patients with high-grade meningiomas, and it was associated with lower preoperative steroid use.
NEUROPSYCHIATRIC DISEASE AND TREATMENT
(2022)
Article
Radiology, Nuclear Medicine & Medical Imaging
Beatrice Detti, Silvia Scoccianti, Maria Ausilia Teriaca, Virginia Maragna, Victoria Lorenzetti, Sara Lucidi, Chiara Bellini, Daniela Greto, Isacco Desideri, Lorenzo Livi
Summary: Overall, the analysis confirms the efficacy of bevacizumab in patients with recurrent high-grade gliomas, with an acceptable toxicity profile. The study found that performance status, age at diagnosis, and the use of corticosteroids during bevacizumab therapy were strongly associated with progression-free survival. Long-term responders accounted for more than half of the cohort.
Article
Oncology
David J. Pinato, Petros Fessas, Alessio Cortellini, Lorenza Rimassa
Summary: Combination immunotherapy is an important treatment approach in hepatocellular carcinoma (HCC) where single-agent immunotherapy has not been successful. The RESCUE trial introduces camrelizumab and apatinib as new options for advanced HCC treatment, sparking debate about the optimal positioning of different combination regimens in liver cancer.
CLINICAL CANCER RESEARCH
(2021)
Article
Clinical Neurology
Katherine Sadler, John Bowes, Charlie F. Rowlands, Cristina Perez-Becerril, C. Mwee van der Meer, Andrew T. King, Scott A. Rutherford, Omar N. Pathmanaban, Charlotte Hammerbeck-Ward, Simon K. W. Lloyd, Simon R. Freeman, Ricky Williams, Cathal John Hannan, Daniel Lewis, Steve Eyre, D. Gareth Evans, Miriam J. Smith
Summary: A genome-wide association study revealed that rs1556516 in the 9p21.3 region is associated with the risk of vestibular schwannoma. The dysregulation of CDKN2B-AS1 and CDKN2A/B genes in this region has been linked to multiple pathologies, and these genes have been shown to influence each other's expression. The recurrent associations of the 9p21.3 region with known oncogenic pathways provide compelling evidence for its involvement in vestibular schwannoma tumorigenesis.
Article
Oncology
Scott R. Plotkin, Jeffrey Allen, Girish Dhall, Jian L. Campian, D. Wade Clapp, Michael J. Fisher, Rakesh K. Jain, James Tonsgard, Nicole J. Ullrich, Coretta Thomas, Lloyd J. Edwards, Bruce Korf, Roger Packer, Matthias A. Karajannis, Jaishri O. Blakeley
Summary: In this prospective multicenter phase II study, the efficacy, safety, and tolerability of bevacizumab for maintenance therapy in persons with NF2-SWN and hearing loss due to VS were evaluated. The results showed that maintenance bevacizumab (5 mg/kg every 3 weeks) was associated with high rates of hearing and tumor stability during 18 months of follow-up. No new unexpected adverse events related to bevacizumab were identified in this population.
Article
Oncology
Justin Z. Wang, Farshad Nassiri, Alexander P. Landry, Vikas Patil, Alexander Rebchuk, Zamir A. Merali, Chloe Gui, Grace Lee, Lauren Rogers, Jessica Sinha, Zeel Patel, Jeffrey A. Zuccato, Mathew R. Voisin, David Munoz, Julian Spears, Michael D. Cusimano, Sunit Das, Serge Makarenko, Stephen Yip, Andrew Gao, Normand Laperriere, Derek S. Tsang, Gelareh Zadeh
Summary: This study assessed the outcomes of surgically resected meningiomas treated with adjuvant and salvage radiotherapy (RT) and identified factors associated with survival rates. Adjuvant RT may be associated with improved survival rates compared to salvage RT.
RADIOTHERAPY AND ONCOLOGY
(2023)
Article
Oncology
Yaru Tian, Hairong Tian, Xiaoyang Zhai, Hui Zhu, Jinming Yu
Summary: This retrospective study found that the addition of bevacizumab does not improve the overall survival of elderly patients with advanced non-squamous NSCLC. Although bevacizumab increased adverse events, the overall efficacy was not significantly improved.
FRONTIERS OF MEDICINE
(2022)
Article
Ophthalmology
Haribalakrishna Balasubramanian, Mythily Sindhur, Ashish Doshi, Lakshmi Srinivasan, Nandkishor S. Kabra, Anupam Malpani, Prachi Agashe
Summary: In infants treated for retinopathy of prematurity, predictors of rescue treatment include lower gestational age, small for gestational age, early packed red blood cell transfusion, and presence of Zone 1 retinopathy. Those who required rescue treatment had a significantly higher incidence of abnormal refractive outcomes.
Article
Oncology
Yosuke Kito, Hisato Kawakami, Seiichiro Mitani, Shinichi Nishina, Toshihiko Matsumoto, Takao Tsuzuki, Yudai Shinohara, Hozumi Shimokawa, Ryosuke Kumanishi, Takashi Ohta, Hiroo Katsuya, Takeshi Kawakami, Tomohiro Nishina, Hiroko Hasegawa, Kohei Akiyoshi, Yasutaka Chiba, Kentaro Yamazaki, Shuichi Hironaka, Kei Muro
Summary: The study evaluated the efficacy and safety of trifluridine/tipiracil plus bevacizumab for patients with previously treated metastatic colorectal cancer who were intolerant of intensive therapy.
Article
Gastroenterology & Hepatology
Salome Ouazana, Emmanuel Coron, Marc Le Rhun, Marianne Le Rhun, Xavier Dray, Lucille Queneherve
Summary: This study aimed to describe the efficacy and safety of percutaneous endoscopic sigmoidopexy (PES) in the treatment of recurrent sigmoid volvulus. The results showed that PES was feasible and effective, and could be considered as an alternative to elective sigmoidectomy in inoperable patients.
Article
Oncology
Jin Suminokura, Morikazu Miyamoto, Tomoyuki Yoshikawa, Hiroko Kouta, Yoshihiro Kikuchi, Taira Hada, Hiroki Ishibashi, Tsubasa Ito, Hideki Iwahashi, Soichiro Kakimoto, Rie Suzuki, Hiroko Matsuura, Naohisa Kishimoto, Masashi Takano
Summary: The study found that weekly low-dose administration of Bevacizumab may improve prognosis and decrease the frequency of adverse effects in patients with ovarian carcinoma. However, prospective studies are needed for confirmation.
Article
Pharmacology & Pharmacy
Ye Peng, Xiaohui Zeng, Liubao Peng, Qiao Liu, Lidan Yi, Xia Luo, Sini Li, Liting Wang, Shuxia Qin, Xiaomin Wan, Chongqing Tan
Summary: This analysis evaluated the cost-effectiveness of sintilimab plus bevacizumab biosimilar as a first-line treatment for unresectable hepatocellular carcinoma. The findings suggested that this treatment option significantly improved survival and quality-adjusted survival compared with sorafenib.
FRONTIERS IN PHARMACOLOGY
(2022)
Article
Oncology
Robert W. Holloway, Alberto A. Mendivil, James E. Kendrick, Lisa N. Abaid, John V. Brown, Jane LeBlanc, Nathalie D. McKenzie, Kristina M. Mori, Sarfraz Ahmad
Summary: The study evaluated the antitumor activity and safety of Olvi-Vec virotherapy combined with platinum-based chemotherapy in patients with advanced ovarian cancer. The results showed promising efficacy and tolerability of this treatment regimen, providing a basis for further clinical investigations.
Article
Pharmacology & Pharmacy
Blaise Pasquiers, Salih Benamara, Mathieu Felices, David Ternant, Xavier Decleves, Alicja Puszkiel
Summary: The objective of this study was to develop a generic physiologically based pharmacokinetic (PBPK) translational approach for monoclonal antibodies (mAb) using open-source software. The translation of bevacizumab was used as a case example to demonstrate the model. The predictions of the model were within an acceptable range for both area under the concentration-time curve (AUC) and maximum concentration (C-max).
Article
Clinical Neurology
Leonel Ampie, M. Harrison Snyder, Jose F. Dominguez, Avery Buchholz, Chun-Po Yen, Mark E. Shaffrey, Hasan R. Syed, Christopher I. Shaffrey, Justin S. Smith
Summary: Primary spinal meningiomas are rare neoplasms located in the intradural-extramedullary compartment, primarily affecting the thoracic spine. Surgical resection is the main treatment for symptomatic tumors, with overall favorable outcomes observed in terms of symptom improvement and limited need for future operations. Ventrally positioned tumors do not necessarily indicate a worse prognosis or limit the extent of resection.
NEUROSURGICAL FOCUS
(2021)
Article
Genetics & Heredity
Pamela L. Wolters, Stephanie Reda, Staci Martin, Nour Al Ghriwati, Melissa Baker, Dale Berg, Gregg Erickson, Barbara Franklin, Vanessa L. Merker, Beverly Oberlander, Stephanie Reeve, Claas Rohl, Tena Rosser, Mary Anne Toledo-Tamula, Ana-Maria Vranceanu
Summary: The survey found that NF patients experienced higher levels of anxiety and stress during the COVID-19 pandemic, especially those with mental health disorders or severe NF symptoms. Although telehealth services were helpful, they were underutilized, indicating a need for increased awareness and utilization of these services.
AMERICAN JOURNAL OF MEDICAL GENETICS PART A
(2022)
Article
Genetics & Heredity
Vanessa L. Merker, Pamela Knight, Heather B. Radtke, Kaleb Yohay, Nicole J. Ullrich, Scott R. Plotkin, Justin T. Jordan
Summary: This study evaluated the level of awareness of and agreement with published neurofibromatosis (NF) clinical guidelines among NF specialists in the United States. The results showed wide variability in both awareness of and agreement with the guidelines among the experts. Future quality improvement efforts should focus on evidence-based, consensus-driven methods to update and disseminate these guidelines to support collaboration among this multi-specialty group of providers.
ORPHANET JOURNAL OF RARE DISEASES
(2022)
Article
Health Care Sciences & Services
Vanessa L. Merker, Justeen K. Hyde, Abigail Herbst, Amanda K. Solch, David C. Mohr, Lauren Gaj, Kelly Dvorin, Eileen M. Dryden
Summary: This study explores the impacts of engaging patients as consultants in research at a Veterans Health Administration research center. It found that patient engagement improved the quality and relevance of research studies, and had personal benefits for both researchers and patients.
JOURNAL OF GENERAL INTERNAL MEDICINE
(2022)
Article
Genetics & Heredity
Vanessa L. Merker, Bronwyn Slobogean, Justin T. Jordan, Shannon Langmead, Mark Meterko, Martin P. Charns, A. Rani Elwy, Jaishri O. Blakeley, Scott R. Plotkin
Summary: This study evaluated the extent and reasons for diagnostic delays and errors in schwannomatosis, a neurogenetic syndrome characterized by nerve sheath tumors and chronic pain. The results showed a long median time to diagnosis and a high misdiagnosis rate in patients with schwannomatosis. The study suggests that interventions in clinician education, genetic testing availability, expert review of pathology findings, and automatic triggers for genetics referrals may improve the diagnosis of schwannomatosis.
AMERICAN JOURNAL OF MEDICAL GENETICS PART A
(2022)
Article
Genetics & Heredity
Scott R. Plotkin, Ludwine Messiaen, Eric Legius, Patrice Pancza, Robert A. Avery, Jaishri O. Blakeley, Dusica Babovic-Vuksanovic, Rosalie Ferner, Michael J. Fisher, Jan M. Friedman, Marco Giovannini, David H. Gutmann, Clemens Oliver Hanemann, Michel Kalamarides, Hildegard Kehrer-Sawatzki, Bruce R. Korf, Victor-Felix Mautner, Mia MacCollin, Laura Papi, Katherine A. Rauen, Vincent Riccardi, Elizabeth Schorry, Miriam J. Smith, Anat Stemmer-Rachamimov, David A. Stevenson, Nicole J. Ullrich, David Viskochil, Katharina Wimmer, Kaleb Yohay, Susan M. Huson, Pierre Wolkenstein, D. Gareth Evans
Summary: The study aims to update the diagnostic criteria for neurofibromatosis type 2 (NF2) and schwannomatosis (SWN) by incorporating recent advances in genetics, ophthalmology, neuropathology, and neuroimaging. The updated criteria include clinical features and genetic testing, emphasizing the phenotypic overlap between the two conditions.
GENETICS IN MEDICINE
(2022)
Article
Clinical Neurology
K. Ina Ly, Vanessa L. Merker, Wenli Cai, Miriam A. Bredella, Alona Muzikansky, Raquel D. Thalheimer, Jennifer Liwei Da, Christina C. Orr, Hamilton P. Herr, Mary E. Morris, Connie Y. Chang, Gordon J. Harris, Scott R. Plotkin, Justin T. Jordan
Summary: The primary objective of this study was to evaluate the long-term growth behavior of internal neurofibromas in adults with NF1. The study found that internal neurofibroma growth behavior in older adults differs fundamentally from that in children and young adults, with most tumors demonstrating spontaneous shrinkage. Patient-specific, tumor-specific, and patient-reported variables were not strong predictors of tumor growth.
Article
Psychology, Educational
Stephanie L. Santoro, Maria J. Cabrera, Kelsey Haugen, Kavita Krell, Vanessa L. Merker
Summary: This study conducted virtual focus groups with 20 parents and 8 individuals with Down syndrome to gather their views on health indicators. The findings showed similarities in the perspectives of individuals with Down syndrome and their parents, with both groups discussing physical, mental, and social health components. However, individuals with Down syndrome provided a more limited range of examples with different nuances compared to parents.
JOURNAL OF APPLIED RESEARCH IN INTELLECTUAL DISABILITIES
(2023)
Article
Genetics & Heredity
Stephanie L. Santoro, Sandra Baker, Jasmine M. Blake, Maria J. Cabrera, Kelli Caughman, Jeanhee Chung, Sarah Cullen, Maureen Gallagher, Kelsey Haugen, Roxanne Hoke-Chandler, Kavita Krell, Julius Maina, Diana McLuckie, Vanessa L. Merker, Carie Michael, Kate O'Neill, Angeles Pena, Albert Pless Jr, Dina Royal, Michelle Slape, Noemi Alice Spinazzi, Amy Torres, Carlos G. Torres, Brian G. Skotko
Summary: This study utilized a mixed methods approach to investigate the impact of COVID-19 on health of individuals with Down syndrome (DS) and their families. Findings from virtual focus groups and a questionnaire revealed that the pandemic had negative effects on the physical, social, and mental health of individuals with DS and their parents. However, there were also unexpected positives, such as improved hygiene and opportunities for online learning and communication.
AMERICAN JOURNAL OF MEDICAL GENETICS PART A
(2023)
Article
Oncology
Scott R. Plotkin, Jeffrey Allen, Girish Dhall, Jian L. Campian, D. Wade Clapp, Michael J. Fisher, Rakesh K. Jain, James Tonsgard, Nicole J. Ullrich, Coretta Thomas, Lloyd J. Edwards, Bruce Korf, Roger Packer, Matthias A. Karajannis, Jaishri O. Blakeley
Summary: In this prospective multicenter phase II study, the efficacy, safety, and tolerability of bevacizumab for maintenance therapy in persons with NF2-SWN and hearing loss due to VS were evaluated. The results showed that maintenance bevacizumab (5 mg/kg every 3 weeks) was associated with high rates of hearing and tumor stability during 18 months of follow-up. No new unexpected adverse events related to bevacizumab were identified in this population.
Editorial Material
Medicine, Research & Experimental
Vanessa L. Merker, Andrea M. Gross, Brigitte C. Widemann, Scott R. Plotkin
Article
Genetics & Heredity
Jineta Banerjee, Jan M. Friedman, Laura J. Klesse, Kaleb H. Yohay, Justin T. Jordan, Scott R. Plotkin, Robert J. Allaway, Jaishri O. Blakeley
Summary: This study investigated whether patients with rare diseases such as neurofibromatosis type 1 (NF1), neurofibromatosis type 2 (NF2), or schwannomatosis (SWN) are more susceptible to SARS-CoV-2 infection and severe COVID-19. The results showed that the proportion of positive cases in NF1, NF2, or SWN patients was not significantly higher than in individuals without these rare diseases. There were no severe outcomes reported in the NF2 or SWN cohorts, and the proportion of severe outcomes in NF1 patients was similar to other rare disease cohorts and the general population.
GENETICS IN MEDICINE
(2023)
Article
Medicine, Research & Experimental
Pamela L. Wolters, Nour Al Ghriwati, Melissa Baker, Staci Martin, Dale Berg, Gregg Erickson, Barbara Franklin, Vanessa L. Merker, Beverly Oberlander, Stephanie Reeve, Claas Rohl, Tena Rosser, Ana-Maria Vranceanu
Summary: This study conducted the largest survey to assess physical symptoms, mental health needs, and preferences for psychosocial trials in adults with neurofibromatosis. The results indicate a high prevalence of disease manifestations, psychosocial difficulties, and untreated mental health problems in adults with neurofibromatosis. The study also found a high degree of willingness among participants to participate in psychosocial clinical trials. Patient preferences should be taken into consideration when designing and implementing psychosocial interventions to develop the most feasible and meaningful studies.
Meeting Abstract
Clinical Neurology
Vanessa L. Merker, Yidan Ma, Lori B. Chibnik, Nicole J. Ullrich, Kaleb H. Yohay, Pamela Knight, Heather B. Radtke, Scott R. Plotkin, Justin T. Jordan
ANNALS OF NEUROLOGY
(2022)
