Literature DB >> 29296950

Targeted inhibition of the MAPK pathway: emerging salvage option for progressive life-threatening multisystem LCH.

Alexandra Kolenová1,2, Raphaela Schwentner3, Gunhild Jug3, Ingrid Simonitsch-Klupp4, Christoph Kornauth4, Lukáš Plank5,6, Júlia Horáková1,2, Ivana Bodová1,2, Tomáš Sýkora1,2, Lucia Geczová1,2, Wolfgang Holter3,7, Milen Minkov3, Caroline Hutter7.   

Abstract

Single-agent vemurafenib leads to a rapid and sustained clinical response in severe multisystem LCH but does not eradicate the disease.Longitudinal assessment of BRAF V600E during treatment shows that clinical remission can occur despite significant amounts of mutated BRAF.

Entities:  

Year:  2017        PMID： 29296950      PMCID： PMC5738993          DOI： 10.1182/bloodadvances.2016003533

Source DB:  PubMed          Journal:  Blood Adv        ISSN： 2473-9529


  24 in total

1.  Response to initial treatment of multisystem Langerhans cell histiocytosis: an important prognostic indicator.

Authors:  Milen Minkov; Nicole Grois; Andreas Heitger; Ulrike Pötschger; T Westermeier; Helmut Gadner
Journal:  Med Pediatr Oncol       Date:  2002-12

2.  Risk organ + LCH gets the one-two punch?

Authors:  Kimo C Stine
Journal:  Blood       Date:  2015-09-17       Impact factor: 22.113

Review 3.  Langerhans cell histiocytosis in children: from the bench to bedside for an updated therapy.

Authors:  Maurizio Aricò
Journal:  Br J Haematol       Date:  2016-02-23       Impact factor: 6.998

Review 4.  Tumor adaptation and resistance to RAF inhibitors.

Authors:  Piro Lito; Neal Rosen; David B Solit
Journal:  Nat Med       Date:  2013-11       Impact factor: 53.440

5.  Personalized medicine: Time for one-person trials.

Authors:  Nicholas J Schork
Journal:  Nature       Date:  2015-04-30       Impact factor: 49.962

6.  The BRAF-V600E mutation in circulating cell-free DNA is a promising biomarker of high-risk adult Langerhans cell histiocytosis.

Authors:  Masayuki Kobayashi; Arinobu Tojo
Journal:  Blood       Date:  2014-10-16       Impact factor: 22.113

7.  Cladribine and cytarabine in refractory multisystem Langerhans cell histiocytosis: results of an international phase 2 study.

Authors:  Jean Donadieu; Frederic Bernard; Max van Noesel; Mohamed Barkaoui; Odile Bardet; Rosella Mura; Maurizio Arico; Christophe Piguet; Virginie Gandemer; Corinne Armari Alla; Niels Clausen; Eric Jeziorski; Anne Lambilliote; Sheila Weitzman; Jan Inge Henter; Cor Van Den Bos
Journal:  Blood       Date:  2015-07-20       Impact factor: 22.113

8.  Recurrent BRAF mutations in Langerhans cell histiocytosis.

Authors:  Gayane Badalian-Very; Jo-Anne Vergilio; Barbara A Degar; Laura E MacConaill; Barbara Brandner; Monica L Calicchio; Frank C Kuo; Azra H Ligon; Kristen E Stevenson; Sarah M Kehoe; Levi A Garraway; William C Hahn; Matthew Meyerson; Mark D Fleming; Barrett J Rollins
Journal:  Blood       Date:  2010-06-02       Impact factor: 22.113

9.  2'-Chlorodeoxyadenosine (2-CdA) as salvage therapy for Langerhans cell histiocytosis (LCH). results of the LCH-S-98 protocol of the Histiocyte Society.

Authors:  Sheila Weitzman; Jorge Braier; Jean Donadieu; R Maarten Egeler; Nicole Grois; Stephan Ladisch; Ulrike Pötschger; David Webb; James Whitlock; Robert J Arceci
Journal:  Pediatr Blood Cancer       Date:  2009-12-15       Impact factor: 3.167

10.  Mutually exclusive recurrent somatic mutations in MAP2K1 and BRAF support a central role for ERK activation in LCH pathogenesis.

Authors:  Rikhia Chakraborty; Oliver A Hampton; Xiaoyun Shen; Stephen J Simko; Albert Shih; Harshal Abhyankar; Karen Phaik Har Lim; Kyle R Covington; Lisa Trevino; Ninad Dewal; Donna M Muzny; Harshavardhan Doddapaneni; Jianhong Hu; Linghua Wang; Philip J Lupo; M John Hicks; Diana L Bonilla; Karen C Dwyer; Marie-Luise Berres; Poulikos I Poulikakos; Miriam Merad; Kenneth L McClain; David A Wheeler; Carl E Allen; D Williams Parsons
Journal:  Blood       Date:  2014-09-08       Impact factor: 22.113
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  9 in total

1.  BRAF-V600E-mutated Rosai-Dorfman-Destombes disease and Langerhans cell histiocytosis with response to BRAF inhibitor.

Authors:  Rosemarie Mastropolo; Allison Close; Steven W Allen; Kenneth L McClain; Scott Maurer; Jennifer Picarsic
Journal:  Blood Adv       Date:  2019-06-25

2.  Clinical responses and persistent BRAF V600E+ blood cells in children with LCH treated with MAPK pathway inhibition.

Authors:  Olive S Eckstein; Johannes Visser; Carlos Rodriguez-Galindo; Carl E Allen
Journal:  Blood       Date:  2019-02-04       Impact factor: 22.113

3.  Response to mitogen-activated protein kinase inhibition of neurodegeneration in Langerhans cell histiocytosis monitored by cerebrospinal fluid neurofilament light as a biomarker: a pilot study.

Authors:  Jan-Inge Henter; Egle Kvedaraite; Daniel Martín Muñoz; Monica Cheng Munthe-Kaas; Bernward Zeller; Tove A Nystad; Caroline Björklund; Isabella Donnér; Magda Lourda; Henrik Zetterberg; Kaj Blennow; Nikolas Herold; Désirée Gavhed; Tatiana von Bahr Greenwood
Journal:  Br J Haematol       Date:  2021-08-25       Impact factor: 8.615

4.  Vemurafenib for Refractory Multisystem Langerhans Cell Histiocytosis in Children: An International Observational Study.

Authors:  Jean Donadieu; Islam Amine Larabi; Mathilde Tardieu; Johannes Visser; Caroline Hutter; Elena Sieni; Nabil Kabbara; Mohamed Barkaoui; Jean Miron; François Chalard; Paul Milne; Julien Haroche; Fleur Cohen; Zofia Hélias-Rodzewicz; Nicolas Simon; Mathilde Jehanne; Alexandra Kolenova; Anne Pagnier; Nathalie Aladjidi; Pascale Schneider; Geneviève Plat; Anne Lutun; Anne Sonntagbauer; Thomas Lehrnbecher; Alina Ferster; Viktoria Efremova; Martina Ahlmann; Laurence Blanc; James Nicholson; Anne Lambilliote; Houda Boudiaf; Andrej Lissat; Karel Svojgr; Fanette Bernard; Sarah Elitzur; Michal Golan; Dmitriy Evseev; Michael Maschan; Ahmed Idbaih; Olga Slater; Milen Minkov; Valerie Taly; Matthew Collin; Jean-Claude Alvarez; Jean-François Emile; Sébastien Héritier
Journal:  J Clin Oncol       Date:  2019-09-12       Impact factor: 44.544

5.  Case Report: Early Association of Vemurafenib to Standard Chemotherapy in Multisystem Langerhans Cell Histiocytosis in a Newborn: Taking a Chance for a Better Outcome?

Authors:  Stefania Gaspari; Valentina Di Ruscio; Francesca Stocchi; Roberto Carta; Marco Becilli; Maria Antonietta De Ioris
Journal:  Front Oncol       Date:  2021-12-13       Impact factor: 6.244

6.  Vemurafenib acts as a molecular on-off switch governing systemic inflammation in Langerhans cell histiocytosis.

Authors:  Sebastian K Eder; Raphaela Schwentner; Philipp Ben Soussia; Giulio Abagnale; Andishe Attarbaschi; Milen Minkov; Florian Halbritter; Caroline Hutter
Journal:  Blood Adv       Date:  2022-02-08

7.  Development of BRAFV600E-positive acute myeloid leukemia in a patient on long-term dabrafenib for multisystem LCH.

Authors:  Marta Salek; Ninad Oak; Melissa Hines; Jamie L Maciaszek; Ruth Tatevossian; Akshay Sharma; Kim E Nichols; Patrick Campbell
Journal:  Blood Adv       Date:  2022-04-26

8.  Vemurafenib in Langerhans cell histiocytosis: report of a pediatric patient and review of the literature.

Authors:  Anne Heisig; Jan Sörensen; Stefanie-Yvonne Zimmermann; Stefan Schöning; Dirk Schwabe; Hans-Michael Kvasnicka; Raphaela Schwentner; Caroline Hutter; Thomas Lehrnbecher
Journal:  Oncotarget       Date:  2018-04-24

9.  High-risk LCH in infants is serially transplantable in a xenograft model but responds durably to targeted therapy.

Authors:  Lynn H Lee; Christa Krupski; Jason Clark; Mark Wunderlich; Robert B Lorsbach; Michael S Grimley; Matthew Burwinkel; Adam Nelson; Ashish R Kumar
Journal:  Blood Adv       Date:  2020-02-25
  9 in total

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