Citations 3
Management of neurofibromatosis type 1-associated plexiform neurofibromas
Fisher, Michael J. , Blakeley, Jaishri O. , Dombi, Eva , Akshintala, Srivandana , Belzberg, Allan J. , Bornhorst, Miriam , Bredella, Miriam A. , Ferner, Rosalie E. , Gross, Andrea M. , Harris, Gordon J. , Listernick, Robert , Ly, Ina , Martin, Staci , Mautner, Victor F., Salamon, Johannes, Salerno, Kilian , Spinner, Robert J. , Staedtke, Verena , Ullrich, Nicole J. , Upadhyaya, Meena , Wolters, Pamela L. , Widemann, Brigitte C. , Dombi, Eva , Bornhorst, Miriam , Blakeley, Jaishri O , Gross, Andrea M , Weiss, Brian , Ahlawat, Shivani , Cai, Wenli , Mautner, Victor‐F., Yohay, Kaleb
Neuro-Oncology | June 3, 2022 | Volume: 24 | Issue: 11 | Pages: 1827-1844
Abstract Plexiform Neurofibromas (PN) are a common manifestation of the genetic disorder neurofibromatosis type 1 (NF1). These benign nerve sheath tumors often cause significant morbidity, with treatment options limited historically to surgery. There have been tremendous advances over the past two decades in our understanding of PN, and the recent regulatory approvals of the MEK inhibitor selumetinib are reshaping the landscape for PN management. At present, there is no agreed upon PN definition, diagnostic evaluation, surveillance strategy, or clear indications for when to initiate treatment and selection of treatment modality. In this review, we address these questions via consensus recommendations from a panel of multidisciplinary NF1 experts.
Recommendations for imaging tumor response in neurofibromatosis clinical trials
Ardern‐Holmes, Simone , Babovic‐Vuksanovic, Dusica , Barker, Fred G., Evans, D. Gareth , Fisher, Michael J. , Goutagny, Stéphane , Harris, Gordon J. , Karajannis, Matthias A. , Korf, Bruce R. , Mautner, Victor, Plotkin, Scott R. , Robertson, Kent A. , Shih, Chie‐Schin , Widemann, Brigitte C. , Dombi, Eva , Connor, Steve E. J. , Jaramillo, Diego , Poussaint, Tina Young , Evans, D Gareth , Karajannis, Matthias A
Neurology | November 18, 2013 | Volume: 81 | Issue: 21_supplement_1 | Pages: No page available
Consensus Recommendations to Accelerate Clinical Trials for Neurofibromatosis Type 2
Evans, D. Gareth , Kalamarides, Michel , Hunter‐Schaedle, Kim, Allen, Jeffrey C. , Babovic-Vuskanovic, Dusica, Belzberg, Allan J. , Bollag, Gideon , Chen, Ruihong , DiTomaso, Emmanuelle, Golfinos, John G. , Harris, Gordon J. , Jacob, Abraham, Kalpana, Ganjam V. , Karajannis, Matthias A. , Korf, Bruce R. , Law, Meng , McClatchey, Andrea I. , Packer, Roger J. , Roehm, Pamela C. , Rubenstein, Allan E., Slattery, William H. , Tonsgard, James H. , Widemann, Brigitte C. , Yohay, Kaleb , Giovannini, Marco , Blakeley, Jaishri O. , Kurzrock, Razelle , Welling, D. Bradley , Evans, D Gareth , Karajannis, Matthias A
Clinical Cancer Research | August 12, 2009 | Volume: 15 | Issue: 16 | Pages: 5032-5039
Abstract Purpose: Neurofibromatosis type 2 (NF2) is a rare autosomal dominant disorder associated primarily with bilateral schwannomas seen on the superior vestibular branches of the eighth cranial nerves. Significant morbidity can result from surgical treatment of these tumors. Meningiomas, ependymomas, and other benign central nervous system tumors are also common in NF2. The lack of effective treatments for NF2 marks an unmet medical need. Experimental Design: Here, we provide recommendations from a workshop, cochaired by Drs. D. Gareth Evans and Marco Giovannini, of 36 international researchers, physicians, representatives of the biotechnology industry, and patient advocates on how to accelerate progress toward NF2 clinical trials. Results: Workshop participants reached a consensus that, based on current knowledge, the time is right to plan and implement NF2 clinical trials. Obstacles impeding NF2 clinical trials and how to address them were discussed, as well as the candidate therapeutic pipeline for NF2. Conclusions: Both phase 0 and phase II NF2 trials are near-term options for NF2 clinical trials. The number of NF2 patients in the population remains limited, and successful recruitment will require ongoing collaboration efforts between NF2 clinics. (Clin Cancer Res 2009;15(16):5032–9)