Safety and efficacy of low-dose PI3K inhibitor taselisib in adult patients with CLOVES and Klippel–Trenaunay syndrome (KTS): the TOTEM trial, a phase 1/2 multicenter, open-label, single-arm study
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J. Clayton-Smith | R. Loffroy | C. Chiaverini | L. Faivre | Pierre Vabres | M. Bardou | R. Semple | T. Mirault | M. Jacquemont | F. Petit | D. Bessis | H. Devilliers | V. Parker | A. Maruani | M. Willems | L. Martin | M. Luu | F. Morice-Picard | A. Phan | A. Maurer | M. Carpentier | C. Fleck | M. Yousfi
[1] A. Delucchi,et al. CLOVES syndrome: Treatment with oral Rapamycin. Report of two cases. , 2019, Revista chilena de pediatria.
[2] H. Rugo,et al. Alpelisib for PIK3CA‐Mutated, Hormone Receptor–Positive Advanced Breast Cancer , 2019, The New England journal of medicine.
[3] M. Genuardi,et al. Old treatments for new genetic conditions: Sirolimus therapy in a child affected by mosaic overgrowth with fibroadipose hyperplasia , 2019, Clinical genetics.
[4] C. Arteaga,et al. Challenges for the Clinical Development of PI3K Inhibitors: Strategies to Improve Their Impact in Solid Tumors. , 2019, Cancer discovery.
[5] P. Fasching,et al. A Phase II Randomized Study of Neoadjuvant Letrozole Plus Alpelisib for Hormone Receptor-Positive, Human Epidermal Growth Factor Receptor 2-Negative Breast Cancer (NEO-ORB) , 2019, Clinical Cancer Research.
[6] J. Baselga,et al. Alpelisib Plus Fulvestrant in PIK3CA-Altered and PIK3CA-Wild-Type Estrogen Receptor–Positive Advanced Breast Cancer: A Phase 1b Clinical Trial , 2019, JAMA oncology.
[7] Julie C. Sapp,et al. Safety and efficacy of low-dose sirolimus in the PIK3CA-related overgrowth spectrum , 2018, Genetics in Medicine.
[8] N. Boddaert,et al. Targeted therapy in patients with PIK3CA-related overgrowth syndrome , 2018, Nature.
[9] L. Pusztai,et al. Phase II Study of Taselisib (GDC-0032) in Combination with Fulvestrant in Patients with HER2-Negative, Hormone Receptor–Positive Advanced Breast Cancer , 2018, Clinical Cancer Research.
[10] C. Simone,et al. In vitro efficacy of ARQ 092, an allosteric AKT inhibitor, on primary fibroblast cells derived from patients with PIK3CA-related overgrowth spectrum (PROS) , 2018, neurogenetics.
[11] A. Halbert,et al. Fibroadipose vascular anomaly treated with sirolimus: Successful outcome in two patients , 2017, Pediatric dermatology.
[12] J. Martinez-Agosto,et al. Somatic overgrowth disorders of the PI3K/AKT/mTOR pathway & therapeutic strategies , 2016, American journal of medical genetics. Part C, Seminars in medical genetics.
[13] X. Paoletti,et al. Innovations for phase I dose-finding designs in pediatric oncology clinical trials. , 2016, Contemporary clinical trials.
[14] S. Mccarroll,et al. Lymphatic and other vascular malformative/overgrowth disorders are caused by somatic mutations in PIK3CA. , 2015, The Journal of pediatrics.
[15] Julie C. Sapp,et al. PIK3CA‐related overgrowth spectrum (PROS): Diagnostic and testing eligibility criteria, differential diagnosis, and evaluation , 2015, American journal of medical genetics. Part A.
[16] H. Soares,et al. Suppression of Feedback Loops Mediated by PI3K/mTOR Induces Multiple Overactivation of Compensatory Pathways: An Unintended Consequence Leading to Drug Resistance , 2014, Molecular Cancer Therapeutics.
[17] Steve Price,et al. Discovery of 2-{3-[2-(1-isopropyl-3-methyl-1H-1,2-4-triazol-5-yl)-5,6-dihydrobenzo[f]imidazo[1,2-d][1,4]oxazepin-9-yl]-1H-pyrazol-1-yl}-2-methylpropanamide (GDC-0032): a β-sparing phosphoinositide 3-kinase inhibitor with high unbound exposure and robust in vivo antitumor activity. , 2013, Journal of medicinal chemistry.
[18] Jeffrey S Barrett,et al. Shortening the timeline of pediatric phase I trials: the rolling six design. , 2008, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.
[19] T. Perneger,et al. The French SF-36 Health Survey: translation, cultural adaptation and preliminary psychometric evaluation. , 1998, Journal of clinical epidemiology.
[20] C. McHorney,et al. The MOS 36‐Item Short‐Form Health Survey (SF‐36): II. Psychometric and Clinical Tests of Validity in Measuring Physical and Mental Health Constructs , 1993, Medical care.
[21] C. Sherbourne,et al. The MOS 36-Item Short-Form Health Survey (SF-36) , 1992 .
[22] Ware J.E.Jr.,et al. THE MOS 36- ITEM SHORT FORM HEALTH SURVEY (SF- 36) CONCEPTUAL FRAMEWORK AND ITEM SELECTION , 1992 .