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490 results on '"Blakeley, Jaishri"'

3. CEST2022: Amide proton transfer-weighted MRI improves the diagnostic performance of multiparametric non-contrast-enhanced MRI techniques in patients with post-treatment high-grade gliomas

Author

Huang, Qianqi, Wu, Jingpu, Le, Nhat, Shen, Yiqing, Guo, Pengfei, Schreck, Karisa C., Kamson, David, Blair, Lindsay, Heo, Hye-Young, Li, Xu, Li, Wenbo, Sair, Haris L., Blakeley, Jaishri O., Laterra, John, Holdhoff, Matthias, Grossman, Stuart A., Mukherjee, Debraj, Bettegowda, Chetan, van Zijl, Peter, Zhou, Jinyuan, and Jiang, Shanshan

Published
2023
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13. Platform trial design for neurofibromatosis type 1, NF2-related schwannomatosis and non-NF2-related schwannomatosis: A potential model for rare diseases.

Author

Dhaenens, Britt A E, Heimann, Günter, Bakker, Annette, Nievo, Marco, Ferner, Rosalie E, Evans, D Gareth, Wolkenstein, Pierre, Leubner, Jonas, Potratz, Cornelia, Carton, Charlotte, Iloeje, Uchenna, Kirk, George, Blakeley, Jaishri O, Plotkin, Scott, Fisher, Michael J, Kim, AeRang, Driever, Pablo Hernáiz, Azizi, Amedeo A, Widemann, Brigitte C, and Gross, Andrea

Subjects
RARE diseases, NEUROFIBROMATOSIS 1, TREATMENT effectiveness, CLINICAL trials, NATURAL history
Abstract

Background Neurofibromatosis type 1, NF2 -related schwannomatosis and non- NF2 -related schwannomatosis (grouped under the abbreviation "NF") are rare hereditary tumor predisposition syndromes. Due to the low prevalence, variability in the range, and severity of manifestations, as well as limited treatment options, these conditions require innovative trial designs to accelerate the development of new treatments. Methods Within European Patient-Centric Clinical Trial Platforms (EU-PEARL), we designed 2 platform-basket trials in NF. The trials were designed by a team of multidisciplinary NF experts and trial methodology experts. Results The trial will consist of an observational and a treatment period. The observational period will serve as a longitudinal natural history study. The platform trial design and randomization to a sequence of available interventions allow for the addition of interventions during the trial. If a drug does not meet the predetermined efficacy endpoint or reveals unacceptable toxicities, participants may stop treatment on that arm and re-enter the observational period, where they can be re-randomized to a different treatment arm if eligible. Intervention-specific eligibility criteria and endpoints are listed in intervention-specific-appendices, allowing the flexibility and adaptability needed for highly variable and rare conditions like NF. Conclusions These innovative platform-basket trials for NF may serve as a model for other rare diseases, as they will enhance the chance of identifying beneficial treatments through optimal learning from a small number of patients. The goal of these trials is to identify beneficial treatments for NF more rapidly and at a lower cost than traditional, single-agent clinical trials. [ABSTRACT FROM AUTHOR]

Published
2024
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16. Cabozantinib for neurofibromatosis type 1–related plexiform neurofibromas: a phase 2 trial

Author

Fisher, Michael J., Shih, Chie-Schin, Rhodes, Steven D., Armstrong, Amy E., Wolters, Pamela L., Dombi, Eva, Zhang, Chi, Angus, Steven P., Johnson, Gary L., Packer, Roger J., Allen, Jeffrey C., Ullrich, Nicole J., Goldman, Stewart, Gutmann, David H., Plotkin, Scott R., Rosser, Tena, Robertson, Kent A., Widemann, Brigitte C., Smith, Abbi E., Bessler, Waylan K., He, Yongzheng, Park, Su-Jung, Mund, Julie A., Jiang, Li, Bijangi-Vishehsaraei, Khadijeh, Robinson, Coretta Thomas, Cutter, Gary R., Korf, Bruce R., Blakeley, Jaishri O., and Clapp, D. Wade

Published
2021
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17. Epigenomic, genomic, and transcriptomic landscape of schwannomatosis

Author

Mansouri, Sheila, Suppiah, Suganth, Mamatjan, Yasin, Paganini, Irene, Liu, Jeffrey C., Karimi, Shirin, Patil, Vikas, Nassiri, Farshad, Singh, Olivia, Sundaravadanam, Yogi, Rath, Prisni, Sestini, Roberta, Gensini, Francesca, Agnihotri, Sameer, Blakeley, Jaishri, Ostrow, Kimberly, Largaespada, David, Plotkin, Scott R., Stemmer-Rachamimov, Anat, Ferrer, Marcela Maria, Pugh, Trevor J., Aldape, Kenneth D., Papi, Laura, and Zadeh, Gelareh

Published
2021
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21. Recommendations for the collection and annotation of biosamples for analysis of biomarkers in neurofibromatosis and schwannomatosis clinical trials.

Author

Sundby, R Taylor, Rhodes, Steven D, Komlodi-Pasztor, Edina, Sarnoff, Herb, Grasso, Vito, Upadhyaya, Meena, Kim, AeRang, Evans, D Gareth, Blakeley, Jaishri O, Hanemann, C Oliver, and Bettegowda, Chetan

Subjects
BIOMARKERS, CLINICAL trials, SCHWANNOMAS, MEDICAL protocols, NEUROFIBROMATOSIS, BODY fluid examination, PREDICTION models
Abstract

Introduction: Neurofibromatosis 1 and schwannomatosis are characterized by potential lifelong morbidity and life-threatening complications. To date, however, diagnostic and predictive biomarkers are an unmet need in this patient population. The inclusion of biomarker discovery correlatives in neurofibromatosis 1/schwannomatosis clinical trials enables study of low-incidence disease. The implementation of a common data model would further enhance biomarker discovery by enabling effective concatenation of data from multiple studies. Methods: The Response Evaluation in Neurofibromatosis and Schwannomatosis biomarker working group reviewed published data on emerging trends in neurofibromatosis 1 and schwannomatosis biomarker research and developed recommendations in a series of consensus meetings. Results: Liquid biopsy has emerged as a promising assay for neurofibromatosis 1/schwannomatosis biomarker discovery and validation. In addition, we review recommendations for a range of biomarkers in clinical trials, neurofibromatosis 1/schwannomatosis–specific data annotations, and common data models for data integration. Conclusion: These Response Evaluation in Neurofibromatosis and Schwannomatosis consensus guidelines are intended to provide best practices for the inclusion of biomarker studies in neurofibromatosis 1/schwannomatosis clinical trials, data, and sample annotation and to lay a framework for data harmonization and concatenation between trials. [ABSTRACT FROM AUTHOR]

Published
2024
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22. Perspectives of adolescents with neurofibromatosis 1 and cutaneous neurofibromas: Implications for clinical trials.

Author

Cannon, Ashley, Sarin, Kavita Y, Petersen, Andrea K, Pichard, Dominique C, Wolters, Pamela L, Erickson, Gregg, Lessing, Andrés J, Li, Peng, Röhl, Claas, Rosser, Tena, Widemann, Brigitte C, Blakeley, Jaishri O, and Plotkin, Scott R

Subjects
CAREGIVER attitudes, SELF-evaluation, DISEASES, NEUROFIBROMA, SKIN tumors, PATIENTS' attitudes, RISK assessment, SURVEYS, COMMUNICATION, RESEARCH funding, NEUROFIBROMATOSIS 1, EMOTIONS, ADOLESCENCE
Abstract

Background/Aims: More than 99% of individuals with neurofibromatosis 1 develop cutaneous neurofibromas, benign nerve sheath tumors that manifest as nodules on the skin. These cutaneous neurofibromas emerge with age, appearing most commonly in adolescence. Nevertheless, few data have been published on how adolescents with neurofibromatosis 1 feel about cutaneous neurofibromas. The purpose of this study was to assess the perspectives of adolescents with neurofibromatosis 1 and their caregivers regarding cutaneous neurofibroma morbidity, treatment options, and acceptable risks-benefits of treatment. Methods: An online survey was distributed through the world's largest NF registry. Eligibility criteria included self-reported neurofibromatosis 1 diagnosis, adolescent child ages 12–17 years, ≥1 cutaneous neurofibroma, and ability to read English. The survey was designed to collect details about the adolescent's cutaneous neurofibromas, views on morbidity related to cutaneous neurofibromas, social and emotional impact of cutaneous neurofibromas, communication regarding cutaneous neurofibromas, and views regarding current and potential future cutaneous neurofibroma treatment. Results: Survey respondents included 28 adolescents and 32 caregivers. Adolescents reported having several negative feelings about cutaneous neurofibromas, particularly feeling worried about the potential progression of their cutaneous neurofibromas (50%). Pruritus (34%), location (34%), appearance (31%), and number (31%) were the most bothersome cutaneous neurofibroma features. Topical medication (77%–96%), followed by oral medication (54%–93%), was the most preferred treatment modality. Adolescents and caregivers most often replied that cutaneous neurofibroma treatment should be initiated when cutaneous neurofibromas become bothersome. The majority of respondents were willing to treat cutaneous neurofibromas for at least 1 year (64%–75%). Adolescent and caregivers were least willing to risk pain (72%–78%) and nausea/vomiting (59%–81%) as a cutaneous neurofibroma treatment side effect. Conclusions: These data indicate that adolescents with neurofibromatosis 1 are negatively impacted by their cutaneous neurofibromas, and that both adolescents and their caregivers would be willing to try longer-term experimental treatments. [ABSTRACT FROM AUTHOR]

Published
2024
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31. Telomere alterations in neurofibromatosis type 1-associated solid tumors

Author

Rodriguez, Fausto J., Graham, Mindy K., Brosnan-Cashman, Jacqueline A., Barber, John R., Davis, Christine, Vizcaino, M. Adelita, Palsgrove, Doreen N., Giannini, Caterina, Pekmezci, Melike, Dahiya, Sonika, Gokden, Murat, Noë, Michael, Wood, Laura D., Pratilas, Christine A., Morris, Carol D., Belzberg, Allan, Blakeley, Jaishri, and Heaphy, Christopher M.

Published
2019
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32. Long-term safety and efficacy of selumetinib in children with neurofibromatosis type 1 on a phase 1/2 trial for inoperable plexiform neurofibromas.

Author

Gross, Andrea M, Dombi, Eva, Wolters, Pamela L, Baldwin, Andrea, Dufek, Anne, Herrera, Kailey, Martin, Staci, Derdak, Joanne, Heisey, Kara S, Whitcomb, Patricia M, Steinberg, Seth M, Venzon, David J, Fisher, Michael J, Kim, AeRang, Bornhorst, Miriam, Weiss, Brian D, Blakeley, Jaishri O, Smith, Malcolm A, and Widemann, Brigitte C

Published
2023
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35. Consensus-Based Best Practice Guidelines for the Management of Spinal Deformity and Associated Tumors in Pediatric Neurofibromatosis Type 1: Screening and Surveillance, Surgical Intervention, and Medical Therapy.

Author

Xu, Amy L., Suresh, Krishna V., Gomez, Jaime A., Emans, John B., Larson, A. Noelle, Cahill, Patrick J., Andras, Lindsay M., White, Klane K., Miller, Daniel J., Murphy, Joshua S., Groves, Mari L., Belzberg, Allan J., Hwang, Steven W., Rosser, Tena L., Staedtke, Verena, Ullrich, Nicole J., Sato, Aimee A., Blakeley, Jaishri O., Schorry, Elizabeth K., and Gross, Andrea M.

Published
2023
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36. Correction to: Epigenomic, genomic, and transcriptomic landscape of schwannomatosis

Author

Mansouri, Sheila, Suppiah, Suganth, Mamatjan, Yasin, Paganini, Irene, Liu, Jeffrey C., Karimi, Shirin, Patil, Vikas, Nassiri, Farshad, Singh, Olivia, Sundaravadanam, Yogi, Rath, Prisni, Sestini, Roberta, Gensini, Francesca, Agnihotri, Sameer, Blakeley, Jaishri, Ostrow, Kimberly, Largaespada, David, Plotkin, Scott R., Stemmer-Rachamimov, Anat, Ferrer, Marcela Maria, Pugh, Trevor J., Aldape, Kenneth D., Papi, Laura, and Zadeh, Gelareh

Published
2021
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39. Cutaneous neurofibromas: Current clinical and pathologic issues

Author

Ortonne, Nicolas, Wolkenstein, Pierre, Blakeley, Jaishri O., Korf, Bruce, Plotkin, Scott R., Riccardi, Vincent M., Miller, Douglas C., Huson, Susan, Peltonen, Juha, Rosenberg, Andrew, Carroll, Steven L., Verma, Sharad K., Mautner, Victor, Upadhyaya, Meena, and Stemmer-Rachamimov, Anat

Published
2018
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48. Pharmacokinetic, pharmacodynamic and biomarker evaluation of transforming growth factor-β receptor I kinase inhibitor, galunisertib, in phase 1 study in patients with advanced cancer

Author

Rodón, Jordi, Carducci, Michael, Sepulveda-Sánchez, Juan M., Azaro, Analía, Calvo, Emiliano, Seoane, Joan, Braña, Irene, Sicart, Elisabet, Gueorguieva, Ivelina, Cleverly, Ann, Pillay, N. Sokalingum, Desaiah, Durisala, Estrem, Shawn T., Paz-Ares, Luis, Holdhoff, Matthias, Blakeley, Jaishri, Lahn, Michael M., and Baselga, Jose

Published
2015
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49. Germline loss-of-function mutations in LZTR1 predispose to an inherited disorder of multiple schwannomas

Author

Piotrowski, Arkadiusz, Xie, Jing, Liu, Ying F, Poplawski, Andrzej B, Gomes, Alicia R, Madanecki, Piotr, Fu, Chuanhua, Crowley, Michael R, Crossman, David K, Armstrong, Linlea, Babovic-Vuksanovic, Dusica, Bergner, Amanda, Blakeley, Jaishri O, Blumenthal, Andrea L, Daniels, Molly S, Feit, Howard, Gardner, Kathy, Hurst, Stephanie, Kobelka, Christine, Lee, Chung, Nagy, Rebecca, Rauen, Katherine A, Slopis, John M, Suwannarat, Pim, Westman, Judith A, Zanko, Andrea, Korf, Bruce R, and Messiaen, Ludwine M

Published
2014
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