Article
Oncology
Michael J. Fisher, Jaishri O. Blakeley, Brian D. Weiss, Eva Dombi, Shivani Ahlawat, Srivandana Akshintala, Allan J. Belzberg, Miriam Bornhorst, Miriam A. Bredella, Wenli Cai, Rosalie E. Ferner, Andrea M. Gross, Gordon J. Harris, Robert Listernick, Ina Ly, Staci Martin, Victor F. Mautner, Johannes M. Salamon, Kilian E. Salerno, Robert J. Spinner, Verena Staedtke, Nicole J. Ullrich, Meena Upadhyaya, Pamela L. Wolters, Kaleb Yohay, Brigitte C. Widemann
Summary: Plexiform neurofibromas are a common manifestation of neurofibromatosis type 1, with surgery being the main treatment option in the past. However, recent regulatory approvals of the MEK inhibitor selumetinib have provided new options for PN management. Currently, there is no consensus on the definition, diagnostic evaluation, surveillance strategy, and treatment indications for PN. This review provides consensus recommendations from NF1 experts to address these questions.
Article
Oncology
Andrea M. Gross, Eva Dombi, Pamela L. Wolters, Andrea Baldwin, Anne Dufek, Kailey Herrera, Staci Martin, Joanne Derdak, Kara S. Heisey, Patricia M. Whitcomb, Seth M. Steinberg, David J. Venzon, Michael J. Fisher, AeRang Kim, Miriam Bornhorst, Brian D. Weiss, Jaishri O. Blakeley, Malcolm A. Smith, Brigitte C. Widemann
Summary: This study provides 5-year data on the use of Selumetinib in children with NF1-related plexiform neurofibromas. It shows that most children experienced tumor shrinkage, reduced pain, and sustained improvement. No new safety signals were found, but monitoring of known Selumetinib-related adverse events is necessary.
Article
Medicine, General & Internal
Kunihiro Ikuta, Yoshihiro Nishida, Tomohisa Sakai, Hiroshi Koike, Kan Ito, Hiroshi Urakawa, Shiro Imagama
Summary: This study retrospectively reviewed surgical excision cases of deep-seated nodular plexiform neurofibromas in patients with neurofibromatosis type 1 and found that surgical treatment of these tumors is relatively safe with improvement in preoperative symptoms.
JOURNAL OF CLINICAL MEDICINE
(2022)
Article
Medicine, Research & Experimental
Victoria Idowu, Julie Christensen, Andrea M. Gross, Eva Dombi, Jennifer R. Miles, Kelly King, Jennifer Chisholm, Christopher Zalewski, Andrea Baldwin, Patricia Whitcomb, Crystal Burgess, Brigitte C. Widemann, Carmen C. Brewer, Hung Jeffrey Kim
Summary: This study investigated otologic and audiologic manifestations in patients with NF1 and found that the majority of patients had normal hearing sensitivity and middle ear function. However, conductive hearing loss was more common in patients with ear-related plexiform neurofibromas. These findings suggest the importance of addressing hearing concerns and implementing audiological monitoring in NF1 patients.
Review
Oncology
I. Solaresy, D. Vinal, M. Morales-Conejo, N. Rodriguez-Salas, J. Feliu
Summary: From 2016 onwards, targeted therapies for NF1 and PN patients have shown positive results, with MEK inhibitors and other molecular targeted therapies demonstrating success. Selumetinib has shown an overall response rate of 68% in pediatric NF1 patients with symptomatic inoperable PNs, leading to FDA approval in May 2020. Cabozantinib and mirdametinib have also proven efficacy in adults, with ongoing trials and promising preliminary results for other MEK inhibitors.
Review
Clinical Neurology
Ike Iheanacho, Hyun Kyoo Yoo, Xiaoqin Yang, Sophie Dodman, Rachel Hughes, Suvina Amin
Summary: The purpose of the study is to investigate the epidemiology, burden of illness, and outcomes of surgical resection in pediatric patients with NF-1 and associated PNs. The results suggest that NF-1 is rare in children, but has increased morbidity and mortality in those with associated PNs. Surgical outcomes for PNs are often poor, indicating significant unmet needs in these patients.
NEUROLOGICAL SCIENCES
(2022)
Article
Dermatology
Verena Staedtke, Piotr Topilko, Lu Q. Le, Kevin Grimes, David A. Largaespada, Ross L. Cagan, Matthew R. Steensma, Anat Stemmer-Rachamimov, Jaishri O. Blakeley, Steven D. Rhodes, Ina Ly, Carlos G. Romo, Sang Y. Lee, Eduard Serra
Summary: Neurofibromatosis type 1 (NF1) is caused by a nonfunctional NF1 tumor suppressor gene, resulting in the development of cutaneous neurofibromas (cNFs). Incomplete understanding of cNF pathophysiology and limitations in experimental modeling have hindered the development of cNF treatment. Recent advances in preclinical in vitro and in vivo modeling provide unprecedented opportunities for therapeutic discovery and improving our understanding of cNF biology.
JOURNAL OF INVESTIGATIVE DERMATOLOGY
(2023)
Review
Oncology
Amy E. Armstrong, Allan J. Belzberg, John R. Crawford, Angela C. Hirbe, Zhihong J. Wang
Summary: Neurofibromatosis type 1 (NF1) is a common tumor predisposition syndrome caused by NF1 gene variants leading to loss of neurofibromin. Surgery is the traditional treatment for plexiform neurofibromas (PN) in NF1 patients, but some PN are inoperable. MEK1/2 inhibitor selumetinib has shown promising efficacy as a medical treatment option for pediatric patients with symptomatic, inoperable NF1-PN. Other MEK inhibitors and tyrosine kinase inhibitor cabozantinib are also being investigated as medical therapies for NF1-PN.
Article
Clinical Neurology
Eddie Luidy Imada, Diego Strianese, Deepak P. Edward, Rawan AlThaqib, Antionette Price, Antje Arnold, Hailah Al-Hussain, Luigi Marchionni, Fausto J. Rodriguez
Summary: The study identified gene expression differences between orbitofacial NFs and NFs occurring at other locations, suggesting that orbitofacial NFs may have higher local aggressiveness and treatment challenges. Further investigation may be warranted given the disproportionate morbidity associated with orbitofacial NFs.
Article
Oncology
Brian D. Weiss, Pamela L. Wolters, Scott R. Plotkin, Brigitte C. Widemann, James H. Tonsgard, Jaishri Blakeley, Jeffrey C. Allen, Elizabeth Schorry, Bruce Korf, Nathan J. Robison, Stewart Goldman, Alexander A. Vinks, Chie Emoto, Tsuyoshi Fukuda, Coretta T. Robinson, Gary Cutter, Lloyd Edwards, Eva Dombi, Nancy Ratner, Roger Packer, Michael J. Fisher
Summary: This study conducted a phase II trial of mirdametinib in NF1 patients with PNs, revealing a 42% partial response rate and preliminary evidence of reduced pain in some patients. It represents the first characterization of mirdametinib activity and pharmacokinetics in this patient population and is the first published response study for MAPK/ERK kinase inhibitors in adults with NF1 and PNs.
JOURNAL OF CLINICAL ONCOLOGY
(2021)
Article
Biochemistry & Molecular Biology
Michael J. Fisher, Chie-Schin Shih, Steven D. Rhodes, Amy E. Armstrong, Pamela L. Wolters, Eva Dombi, Chi Zhang, Steven P. Angus, Gary L. Johnson, Roger J. Packer, Jeffrey C. Allen, Nicole J. Ullrich, Stewart Goldman, David H. Gutmann, Scott R. Plotkin, Tena Rosser, Kent A. Robertson, Brigitte C. Widemann, Abbi E. Smith, Waylan K. Bessler, Yongzheng He, Su-Jung Park, Julie A. Mund, Li Jiang, Khadijeh Bijangi-Vishehsaraei, Coretta Thomas Robinson, Gary R. Cutter, Bruce R. Korf, Jaishri O. Blakeley, D. Wade Clapp
Summary: Cabozantinib, an inhibitor of multiple receptor tyrosine kinases, has shown efficacy in reducing plexiform neurofibroma volume and improving pain in patients with NF1, meeting the primary outcome of the phase II trial. However, some patients experienced adverse events leading to dose reduction or discontinuation of therapy.
Article
Medicine, Research & Experimental
Liny John, Gurbani Singh, Eva Dombi, Pamela L. Wolters, Staci Martin, Andrea Baldwin, Seth M. Steinberg, Jessica Bernstein, Patricia Whitcomb, Dominique C. Pichard, Anne Dufek, Andy Gillespie, Kara Heisey, Miriam Bornhorst, Michael J. Fisher, Brian D. Weiss, Aerang Kim, Brigitte C. Widemann, Andrea M. Gross
Summary: This study developed an observer disfigurement severity scale for neurofibroma-related plexiform neurofibromas and evaluated its feasibility, reliability, and validity. The scale showed good agreement among different raters and within the same rater across repeated sessions. It also demonstrated correlation with the change in neurofibroma volume with treatment.
Article
Oncology
Hallie Coltin, Sebastien Perreault, Valerie Larouche, Karina Black, Bev Wilson, Magimairajan Issai Vanan, Abha A. Gupta, Daniel A. Morgenstern, Patricia C. Parkin, Eric Bouffet, Vijay Ramaswamy
Summary: This study reports a case series of 19 children treated with selumetinib, showing significant clinical benefit with symptom improvement or stabilization and acceptable toxicity profiles. This real-world experience confirms the previous trials' findings on the efficacy of selumetinib for this patient population.
PEDIATRIC BLOOD & CANCER
(2022)
Article
Clinical Neurology
Helen Toledano, Gad Dotan, Rivka Friedland, Rony Cohen, Iftach Yassur, Hagit Toledano-Alhadef, Shlomi Constantini, Mika Shapira Rootman
Summary: Trametinib can reduce tumor size in some young children with orbital PNF and may prevent progressive disfigurement. The dosages are adjusted for children of different ages, with no changes in visual function observed during treatment, but some children experienced skin and nail toxicities of grades 1-2.
CHILDS NERVOUS SYSTEM
(2021)
Editorial Material
Medicine, General & Internal
Antonella Cacchione, Alessia Carboni, Mariachiara Lodi, Rita De Vito, Andrea Carai, Antonio Marrazzo, Marina Macchiaiolo, Ioan Paul Voicu, Angela Mastronuzzi, Giovanna Stefania Colafati
Summary: Different radiographical imaging modalities were used to diagnose and evaluate the condition of a patient with NF1 and PN, biopsy confirmed the diagnosis. NF1 diagnosis at birth remains challenging, with congenital PN recognized in 20% of cases. Early detection of PN progression allowed for necessary life-saving interventions.
Article
Dentistry, Oral Surgery & Medicine
Kiran Kumar, Yu-Yao Teoh, Laurence J. Walsh
Summary: This laboratory study aimed to evaluate the effectiveness of laser agitation of sodium hypochlorite in removing multispecies biofilms from the mesial root of permanent first molars. The results showed that none of the tested methods were able to completely eradicate biofilms from the most confined regions of the root canal system, with the cleaning of isthmus regions being the most challenging. Additionally, there was a positive correlation between canal cleaning and isthmus cleaning.
AUSTRALIAN ENDODONTIC JOURNAL
(2023)
Article
Dentistry, Oral Surgery & Medicine
Carol Tran, Ambereen Khan, Neil Meredith, Laurence J. Walsh
Summary: This in vitro study evaluated eight debridement protocols on implant surfaces and found that mechanical instruments were the least effective at biofilm removal and caused the greatest surface alterations. Glycine powder in an air polisher and 40% citric acid application had minimal surface alterations and were effective in biofilm removal.
INTERNATIONAL JOURNAL OF DENTAL HYGIENE
(2023)
Article
Public, Environmental & Occupational Health
Clare Mangoyana, Sandra March, Ratilal Lalloo, Laurence J. Walsh
Summary: This study explored the benefits of a partnership between a university dental school and a Community Controlled Health Service in improving the oral health of an underserved rural Indigenous community. Participants expressed positive benefits in general and oral health awareness, improved access to dental care, and viewed the partnership as mutually beneficial. The results suggest that this partnership provides sustainable positive social and health benefits for the targeted Indigenous community and for the wider local population.
AUSTRALIAN JOURNAL OF RURAL HEALTH
(2023)
Article
Dentistry, Oral Surgery & Medicine
Pingping Han, Corey S. Moran, Srinivas Sulugodu Ramachandra, Laurence J. Walsh, Saso Ivanovski
Summary: This prospective cohort study aimed to evaluate antibody responses in gingival crevicular fluid (GCF) and saliva to the SARS-CoV-2 Spike protein following vaccination. The study found that antibody levels peaked at 3 weeks after vaccination, and the concentration of antibodies in GCF was significantly higher than in saliva.
JOURNAL OF PERIODONTOLOGY
(2023)
Article
Oncology
Yashoma Lalla, Sachin Kulkarni, Laurence James Walsh, Roy George
Summary: The aim of this study was to evaluate the influence of luminosity values and smear layer on transmission characteristics of 810 nm near infrared laser energy through dentine. The results showed that both darker tooth shades and the presence of smear layer can attenuate the transmission of laser energy.
PHOTODIAGNOSIS AND PHOTODYNAMIC THERAPY
(2023)
Article
Chemistry, Multidisciplinary
Philip Yuan-Ho Chien, Boyang Wan, Laurence James Walsh, Ove Andreas Peters
Summary: This study evaluated the cyclic fatigue resistance of rotary endodontic nickel-titanium instruments using experimental and finite element methods. The results showed that TRN instruments had the highest cyclic fatigue resistance, while PTU instruments had the lowest. Finite element analysis provided insights into the stress distribution during cyclic fatigue.
APPLIED SCIENCES-BASEL
(2023)
Review
Infectious Diseases
Amjad Almuqrin, Inder Preet Kaur, Laurence J. Walsh, Chaminda Jayampath Seneviratne, Sobia Zafar
Summary: Topical cariostatic agents, such as silver diamine fluoride (SDF), are effective in managing dental caries in young children. However, SDF application often leads to unaesthetic tooth discoloration, which can be addressed by using nano-metallic antimicrobials like selenium nanoparticles (SeNPs). SeNPs have shown antimicrobial, antioxidant, and antiviral activities, making them a potential substitute for silver in SDF.
Article
Dentistry, Oral Surgery & Medicine
M. Masood, L. J. Walsh, S. Zafar
Summary: This laboratory-based study investigated the release of ions from metallic piercings when exposed to organic acids (lactic, citric) and inorganic acids (phosphoric, hydrochloric) that could be encountered in the oral cavity. The study found that inorganic acids caused greater release of material per surface area compared to organic acids. This could lead to corrosion and leaching of ions from metallic oral piercings.
AUSTRALIAN DENTAL JOURNAL
(2023)
Article
Dentistry, Oral Surgery & Medicine
S. March, C. Mangoyana, P. Oakley, R. Lalloo, L. J. Walsh
Summary: This study found that oral health services provided by dental student clinical outplacement positively impacted a rural Indigenous community, and explored the nature of these benefits.
AUSTRALIAN DENTAL JOURNAL
(2023)
Article
Dentistry, Oral Surgery & Medicine
Janice Cheah Ping Chuang, Archana Pradhan, Laurence James Walsh, Claudia Patricia Lopez Silva
Summary: This study assessed dentists' attitudes towards providing dental care for older adults with disabilities in Singapore and identified factors influencing their willingness to provide care. The study found that as the severity of impairments increased, dentists were less willing, confident, and involved in the care of older adults with disabilities. Less than half of the respondents were willing to treat individuals with severe disabilities. The study suggests a need for training in severe impairments and targeted interventions to increase dentists' willingness to provide care.
Letter
Dermatology
Ericson John V. Torralba, Shikshita Singh, Robert F. Short, Christine M. Rapp, Karen M. Henkels, Ravi P. Sahu, Jeffrey B. Travers
SKIN RESEARCH AND TECHNOLOGY
(2023)
Article
Chemistry, Multidisciplinary
Sepanta Hosseinpour, Huan Dai, Laurence J. Walsh, Chun Xu
Summary: Nanoparticles can be used to deliver miRNA-26a to macrophages, leading to the suppression of pro-inflammatory cytokines and the promotion of osteogenic differentiation. The biocompatible MSN-CC nanoparticles showed low toxicity and efficient internalization in macrophages. The conditioned macrophages created a favorable osteoimmune environment for preosteoblasts, enhancing bone production.
Article
Dentistry, Oral Surgery & Medicine
Shenna Yu-En Ho, Laurence James Walsh, Archana Pradhan, Jingrong Yang, Claudia Patricia Lopez Silva
Summary: Oral health therapists play a valuable role in improving dental care for frail elderly in nursing homes, but they face barriers and their skills are under-utilized. This study evaluated the perceptions of oral health therapists regarding barriers to oral care provision in nursing homes. Lack of opportunity, inadequate education and training, limited work scope and registration status, and inadequate financial remuneration and equipment were identified as major barriers. Addressing these barriers can enhance the utilization of oral health therapists' skill-set.
SPECIAL CARE IN DENTISTRY
(2023)
Article
Dentistry, Oral Surgery & Medicine
Fatemeh Arabpour, Maryam Kuzekanani, Laurence James Walsh, Moghaddameh Mirzaei
Summary: This study aimed to evaluate the knowledge, attitudes, and performance of Iranian endodontists regarding patients with chronic kidney disease (CKD). The majority of endodontists had a good understanding of the safe care requirements for dental patients with CKD, but older clinicians may benefit from further education in this area due to limited exposure to formal training.
EUROPEAN ENDODONTIC JOURNAL
(2023)
Article
Oncology
Daniel C. Bowers, Veena Rajaram, Matthias A. Karajannis, Sharon L. Gardner, Jack Meng-Fen Su, Patricia Baxter, Sonia Partap, Laura J. Klesse
Summary: This phase II clinical trial evaluated the efficacy of everolimus, an mTOR pathway inhibitor, for children with recurrent or progressive posterior fossa-A (PF-A) ependymomas. The study showed that everolimus did not lead to objective tumor responses in these patients. Adverse events were generally mild, with only one case of grade 3 pneumonia.
NEURO-ONCOLOGY ADVANCES
(2023)