Literature DB >> 27398937

Profound clinical and radiological response to BRAF inhibition in a 2-month-old diencephalic child with hypothalamic/chiasmatic glioma.

Alvaro Lassaletta1,2, Ana Guerreiro Stucklin1,2,3, Vijay Ramaswamy1,2,3, Michal Zapotocky1,2,3, Tara McKeown1, Cynthia Hawkins2,3,4, Eric Bouffet1,2,3, Uri Tabori5,6,7.   

Abstract

Infants with low-grade glioma (LGG) have a poor survival. BRAFV600E mutation has been identified in pediatric LGG; however, the use of BRAF inhibitors in infants has never been reported. A 2-month-old with V600E mutant hypothalamic/chiasmatic glioma progressed on chemotherapy resulting in profound visual loss, massive ascites, and diencephalic syndrome. Initiation of dabrafenib resulted in rapid and sustained disappearance of clinical symptoms and a profound sustained cytoreduction. BRAF inhibition was safely tolerated with dramatic clinicoradiological response, suggesting early targeted therapy is a viable option in infants with LGG. A re-evaluation of current management paradigms in this population is warranted to leverage the potential benefit of upfront-targeted therapies.
© 2016 Wiley Periodicals, Inc.

Entities:  

Keywords:  dabrafenib; glioma; hypothalamic-chiasmatic; infant; low grade

Mesh：

Substances：

Year:  2016        PMID： 27398937     DOI： 10.1002/pbc.26086

Source DB:  PubMed          Journal:  Pediatr Blood Cancer        ISSN： 1545-5009            Impact factor:   3.167


  15 in total

1.  Therapeutic and Prognostic Implications of BRAF V600E in Pediatric Low-Grade Gliomas.

Authors:  Alvaro Lassaletta; Michal Zapotocky; Matthew Mistry; Vijay Ramaswamy; Marion Honnorat; Rahul Krishnatry; Ana Guerreiro Stucklin; Nataliya Zhukova; Anthony Arnoldo; Scott Ryall; Catriona Ling; Tara McKeown; Jim Loukides; Ofelia Cruz; Carmen de Torres; Cheng-Ying Ho; Roger J Packer; Ruth Tatevossian; Ibrahim Qaddoumi; Julie H Harreld; James D Dalton; Jean Mulcahy-Levy; Nicholas Foreman; Matthias A Karajannis; Shiyang Wang; Matija Snuderl; Amulya Nageswara Rao; Caterina Giannini; Mark Kieran; Keith L Ligon; Maria Luisa Garre; Paolo Nozza; Samantha Mascelli; Alessandro Raso; Sabine Mueller; Theodore Nicolaides; Karen Silva; Romain Perbet; Alexandre Vasiljevic; Cécile Faure Conter; Didier Frappaz; Sarah Leary; Courtney Crane; Aden Chan; Ho-Keung Ng; Zhi-Feng Shi; Ying Mao; Elizabeth Finch; David Eisenstat; Bev Wilson; Anne Sophie Carret; Peter Hauser; David Sumerauer; Lenka Krskova; Valerie Larouche; Adam Fleming; Shayna Zelcer; Nada Jabado; James T Rutka; Peter Dirks; Michael D Taylor; Shiyi Chen; Ute Bartels; Annie Huang; David W Ellison; Eric Bouffet; Cynthia Hawkins; Uri Tabori
Journal:  J Clin Oncol       Date:  2017-07-20       Impact factor: 44.544

2.  Marked functional recovery and imaging response of refractory optic pathway glioma to BRAFV600E inhibitor therapy: a report of two cases.

Authors:  Santhosh A Upadhyaya; Giles W Robinson; Julie H Harreld; Paul D Klimo; Mary Ellen Hoehn; Brent A Orr; Ibrahim A Qaddoumi
Journal:  Childs Nerv Syst       Date:  2018-02-01       Impact factor: 1.475

3.  Outcomes of BRAF V600E Pediatric Gliomas Treated With Targeted BRAF Inhibition.

Authors:  Liana Nobre; Michal Zapotocky; Vijay Ramaswamy; Scott Ryall; Julie Bennett; Daniel Alderete; Julia Balaguer Guill; Lorena Baroni; Ute Bartels; Abhishek Bavle; Miriam Bornhorst; Daniel R Boue; Adela Canete; Murali Chintagumpala; Scott L Coven; Ofelia Cruz; Sonika Dahiya; Peter Dirks; Ira J Dunkel; David Eisenstat; Cecile Faure Conter; Elizabeth Finch; Jonathan L Finlay; Didier Frappaz; Maria Luisa Garre; Karen Gauvain; Anne Grete Bechensteen; Jordan R Hansford; Inga Harting; Peter Hauser; Lili-Naz Hazrati; Annie Huang; Sarah G Injac; Valentina Iurilli; Matthias Karajannis; Gurcharanjeet Kaur; Martin Kyncl; Lenka Krskova; Normand Laperriere; Valerie Larouche; Alvaro Lassaletta; Sarah Leary; Frank Lin; Samantha Mascelli; Tara McKeown; Till Milde; Andres Morales La Madrid; Giovanni Morana; Helena Morse; Naureen Mushtaq; Diana S Osorio; Roger Packer; Zdenek Pavelka; Eduardo Quiroga-Cantero; James Rutka; Magnus Sabel; Duarte Salgado; Palma Solano; Jaroslav Sterba; Jack Su; David Sumerauer; Michael D Taylor; Helen Toledano; Derek S Tsang; Mariana Valente Fernandes; Frank van Landeghem; Cornelis M van Tilburg; Bev Wilson; Olaf Witt; Josef Zamecnik; Eric Bouffet; Cynthia Hawkins; Uri Tabori
Journal:  JCO Precis Oncol       Date:  2020-05-20

4.  Efficacy of Dabrafenib for three children with brainstem BRAFV600E positive ganglioglioma.

Authors:  Laflamme Philippe; Kondyli Maria; Aljared Tariq; Miconiatis Sofia; Saint-Martin Christine; Farmer Jean-Pierre; Roy W Dudley; Perreault Sébastien; Jabado Nada; Larouche Valérie
Journal:  J Neurooncol       Date:  2019-09-09       Impact factor: 4.130

Review 5.  Pediatric low-grade gliomas: next biologically driven steps.

Authors:  David T W Jones; Mark W Kieran; Eric Bouffet; Sanda Alexandrescu; Pratiti Bandopadhayay; Miriam Bornhorst; David Ellison; Jason Fangusaro; Michael J Fisher; Nicholas Foreman; Maryam Fouladi; Darren Hargrave; Cynthia Hawkins; Nada Jabado; Maura Massimino; Sabine Mueller; Giorgio Perilongo; Antoinette Y N Schouten van Meeteren; Uri Tabori; Katherine Warren; Angela J Waanders; David Walker; William Weiss; Olaf Witt; Karen Wright; Yuan Zhu; Daniel C Bowers; Stefan M Pfister; Roger J Packer
Journal:  Neuro Oncol       Date:  2018-01-22       Impact factor: 12.300

6.  Dabrafenib in patients with recurrent, BRAF V600E mutated malignant glioma and leptomeningeal disease.

Authors:  Michael C Burger; Michael W Ronellenfitsch; Nadja I Lorenz; Marlies Wagner; Martin Voss; David Capper; Theophilos Tzaridis; Ulrich Herrlinger; Joachim P Steinbach; Gabriele Stoffels; Karl-Josef Langen; Christian Brandts; Christian Senft; Patrick N Harter; Oliver Bähr
Journal:  Oncol Rep       Date:  2017-10-02       Impact factor: 3.906

7.  Papillary craniopharyngioma in a 4-year-old girl with BRAF V600E mutation: a case report and review of the literature.

Authors:  R Borrill; E Cheesman; S Stivaros; I D Kamaly-Asl; K Gnanalingham; John-Paul Kilday
Journal:  Childs Nerv Syst       Date:  2018-08-01       Impact factor: 1.475

8.  Alterations in ALK/ROS1/NTRK/MET drive a group of infantile hemispheric gliomas.

Authors:  Ana S Guerreiro Stucklin; Scott Ryall; Kohei Fukuoka; Michal Zapotocky; Alvaro Lassaletta; Christopher Li; Taylor Bridge; Byungjin Kim; Anthony Arnoldo; Paul E Kowalski; Yvonne Zhong; Monique Johnson; Claire Li; Arun K Ramani; Robert Siddaway; Liana Figueiredo Nobre; Pasqualino de Antonellis; Christopher Dunham; Sylvia Cheng; Daniel R Boué; Jonathan L Finlay; Scott L Coven; Inmaculada de Prada; Marta Perez-Somarriba; Claudia C Faria; Michael A Grotzer; Elisabeth Rushing; David Sumerauer; Josef Zamecnik; Lenka Krskova; Miguel Garcia Ariza; Ofelia Cruz; Andres Morales La Madrid; Palma Solano; Keita Terashima; Yoshiko Nakano; Koichi Ichimura; Motoo Nagane; Hiroaki Sakamoto; Maria Joao Gil-da-Costa; Roberto Silva; Donna L Johnston; Jean Michaud; Bev Wilson; Frank K H van Landeghem; Angelica Oviedo; P Daniel McNeely; Bruce Crooks; Iris Fried; Nataliya Zhukova; Jordan R Hansford; Amulya Nageswararao; Livia Garzia; Mary Shago; Michael Brudno; Meredith S Irwin; Ute Bartels; Vijay Ramaswamy; Eric Bouffet; Michael D Taylor; Uri Tabori; Cynthia Hawkins
Journal:  Nat Commun       Date:  2019-09-25       Impact factor: 14.919

9.  Targetable BRAF and RAF1 Alterations in Advanced Pediatric Cancers.

Authors:  Andrew Rankin; Adrienne Johnson; Alison Roos; Geoffrey Kannan; Jeffrey Knipstein; Nicholas Britt; Mark Rosenzweig; James Haberberger; Dean Pavlick; Eric Severson; Jo-Anne Vergilio; Rachel Squillace; Rachel Erlich; Pratheesh Sathyan; Stuart Cramer; David Kram; Jeffrey Ross; Vince Miller; Prasanth Reddy; Brian Alexander; Siraj M Ali; Shakti Ramkissoon
Journal:  Oncologist       Date:  2020-09-25

10.  A phase 2 study of trametinib for patients with pediatric glioma or plexiform neurofibroma with refractory tumor and activation of the MAPK/ERK pathway: TRAM-01.

Authors:  Sébastien Perreault; Valérie Larouche; Uri Tabori; Cynthia Hawkin; Sarah Lippé; Benjamin Ellezam; Jean-Claude Décarie; Yves Théoret; Marie-Élaine Métras; Serge Sultan; Édith Cantin; Marie-Ève Routhier; Maxime Caru; Geneviève Legault; Éric Bouffet; Lucie Lafay-Cousin; Juliette Hukin; Craig Erker; Nada Jabado
Journal:  BMC Cancer       Date:  2019-12-27       Impact factor: 4.430
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