ACCELERATE and European Medicine Agency Paediatric Strategy
Forum for medicinal product development for mature B-cell
malignancies in children

J 2019

ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children

PEARSON, Andrew D. J., Nicole SCOBIE, Koenraad NORGA, Franca LIGAS, Davy CHIODIN et. al.

Základní údaje

Originální název

ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children

Autoři

PEARSON, Andrew D. J. (840 Spojené státy, garant), Nicole SCOBIE (756 Švýcarsko), Koenraad NORGA (56 Belgie), Franca LIGAS (826 Velká Británie a Severní Irsko), Davy CHIODIN (840 Spojené státy), Amos BURKE (826 Velká Británie a Severní Irsko), Veronique MINARD-COLIN (840 Spojené státy), Peter ADAMSON (826 Velká Británie a Severní Irsko), Lynley V. MARSHALL (840 Spojené státy), Arun BALAKUMARAN (840 Spojené státy), Bouchra BENETTAIB (840 Spojené státy), Pankaj BHARGAVA (826 Velká Británie a Severní Irsko), Catherine M. BOLLARD (840 Spojené státy), Ellen BOLOTIN (840 Spojené státy), Simon BOMKEN (826 Velká Británie a Severní Irsko), Jochen BUECHNER (578 Norsko), Birgit BURKHARDT (276 Německo), Hubert CARON (756 Švýcarsko), Christopher COPLAND (756 Švýcarsko), Pierre DEMOLIS (250 Francie), Anton EGOROV (250 Francie), Mahdi FARHAN (756 Švýcarsko), Gerhard ZUGMAIER (276 Německo), Thomas GROSS (840 Spojené státy), Danielle HORTON-TAYLOR (826 Velká Británie a Severní Irsko), Wolfram KLAPPER (276 Německo), Giovanni LESA (826 Velká Británie a Severní Irsko), Robert MARCUS (826 Velká Británie a Severní Irsko), Rodney R. MILES (840 Spojené státy), Kerri NOTTAGE (840 Spojené státy), Lida PACAUD (840 Spojené státy), Rosanna RICAFORT (840 Spojené státy), Martin SCHRAPPE (276 Německo), Jaroslav ŠTĚRBA (203 Česká republika, domácí), Remus VEZAN (840 Spojené státy), Susan WEINER (840 Spojené státy), Su Young KIM (840 Spojené státy), Gregory REAMAN (840 Spojené státy) a Gilles VASSAL (250 Francie)

Vydání

European Journal of Cancer, Oxford, Elsevier Science Inc. 2019, 0959-8049

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30204 Oncology

Stát vydavatele

Velká Británie a Severní Irsko

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 7.275

Kód RIV

RIV/00216224:14110/19:00112994

Organizační jednotka

Lékařská fakulta

DOI

http://dx.doi.org/10.1016/j.ejca.2019.01.013

UT WoS

000460123000012

Klíčová slova anglicky

Paediatric oncology; Mature B-cell malignancies; Medicinal product development

Štítky

14110321, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 27. 1. 2021 12:45, Mgr. Tereza Miškechová

Anotace

V originále

Paediatric Strategy Forums have been created by the multistakeholder organisation, ACCELERATE, and the European Medicines Agency to facilitate dialogue between all relevant stakeholders and suggest strategies in critical areas of paediatric oncology drug development. As there are many medicines being developed for B-cell malignancies in adults but comparatively few in children with these malignancies, a Paediatric Strategy Forum was held to discuss the best approach to develop these products for children. It was concluded that as current frontline therapy is highly successful, despite associated acute toxicity, de-escalation of this or substitution of presently used drugs with new medicines can only be undertaken when there is an effective salvage regimen, which is currently not available. Therefore priority should be given to developing treatment for patients with relapsed and refractory mature B-cell lymphomas. The consensus of the clinicians attending the meeting was that CAR T-cells, T-cell engagers and antibody drug conjugates (excluding those with a vinca alkaloidelike drug) presently have the greatest probability of providing benefit in relapse in view of their mechanism of action. However, as producing autologous CAR T-cells currently takes at least 4 weeks, they are not products which could be quickly employed initially at relapse in rapidly progressing mature B-cell malignancies but only for the consolidation phase of the treatment. Global, industry-supported, academic-sponsored studies testing compounds from different pharmaceutical companies simultaneously should be considered in rare populations, and it was proposed that an international working group be formed to develop an overarching clinical trials strategy for these disease groups. Future Forums are planned for other relevant paediatric oncologic diseases with a high unmet medical need and relevant molecular targets. (C) 2019 Elsevier Ltd. All rights reserved.

Návaznosti

LM2015090, projekt VaV

Název: Český národní uzel Evropské sítě infrastruktur klinického výzkumu (Akronym: CZECRIN)

Investor: Ministerstvo školství, mládeže a tělovýchovy ČR, CZECRIN - Český národní uzel Evropské sítě infrastruktur klinického výzkumu

MUNI/A/1586/2018, interní kód MU

Název: Personalizovaná léčba v dětské onkologii: na cestě k "liquid dynamic medecine" a "N-of-1 clinical trials" (Akronym: Personalizovaná léčba v dětské onkologii)

Investor: Masarykova univerzita, Personalizovaná léčba v dětské onkologii: na cestě k "liquid dynamic medecine" a "N-of-1 clinical trials", DO R. 2020_Kategorie A - Specifický výzkum - Studentské výzkumné projekty

Citovat

PEARSON, Andrew D. J., Nicole SCOBIE, Koenraad NORGA, Franca LIGAS, Davy CHIODIN, Amos BURKE, Veronique MINARD-COLIN, Peter ADAMSON, Lynley V. MARSHALL, Arun BALAKUMARAN, Bouchra BENETTAIB, Pankaj BHARGAVA, Catherine M. BOLLARD, Ellen BOLOTIN, Simon BOMKEN, Jochen BUECHNER, Birgit BURKHARDT, Hubert CARON, Christopher COPLAND, Pierre DEMOLIS, Anton EGOROV, Mahdi FARHAN, Gerhard ZUGMAIER, Thomas GROSS, Danielle HORTON-TAYLOR, Wolfram KLAPPER, Giovanni LESA, Robert MARCUS, Rodney R. MILES, Kerri NOTTAGE, Lida PACAUD, Rosanna RICAFORT, Martin SCHRAPPE, Jaroslav ŠTĚRBA, Remus VEZAN, Susan WEINER, Su Young KIM, Gregory REAMAN a Gilles VASSAL. ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children. European Journal of Cancer. Oxford: Elsevier Science Inc., 2019, roč. 110, 1879-0852, s. 74-85. ISSN 0959-8049. Dostupné z: https://dx.doi.org/10.1016/j.ejca.2019.01.013.

@article{1624117,
   author = {Pearson, Andrew D. J. and Scobie, Nicole and Norga, Koenraad and Ligas, Franca and Chiodin, Davy and Burke, Amos and MinardandColin, Veronique and Adamson, Peter and Marshall, Lynley V. and Balakumaran, Arun and Benettaib, Bouchra and Bhargava, Pankaj and Bollard, Catherine M. and Bolotin, Ellen and Bomken, Simon and Buechner, Jochen and Burkhardt, Birgit and Caron, Hubert and Copland, Christopher and Demolis, Pierre and Egorov, Anton and Farhan, Mahdi and Zugmaier, Gerhard and Gross, Thomas and HortonandTaylor, Danielle and Klapper, Wolfram and Lesa, Giovanni and Marcus, Robert and Miles, Rodney R. and Nottage, Kerri and Pacaud, Lida and Ricafort, Rosanna and Schrappe, Martin and Štěrba, Jaroslav and Vezan, Remus and Weiner, Susan and Kim, Su Young and Reaman, Gregory and Vassal, Gilles},
   article_location = {Oxford},
   article_number = {1879-0852},
   doi = {http://dx.doi.org/10.1016/j.ejca.2019.01.013},
   keywords = {Paediatric oncology; Mature B-cell malignancies; Medicinal product development},
   language = {eng},
   issn = {0959-8049},
   journal = {European Journal of Cancer},
   title = {ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children},
   url = {http://dx.doi.org/10.1016/j.ejca.2019.01.013},
   volume = {110},
   year = {2019}
}

TY  - JOUR
ID  - 1624117
AU  - Pearson, Andrew D. J. - Scobie, Nicole - Norga, Koenraad - Ligas, Franca - Chiodin, Davy - Burke, Amos - Minard-Colin, Veronique - Adamson, Peter - Marshall, Lynley V. - Balakumaran, Arun - Benettaib, Bouchra - Bhargava, Pankaj - Bollard, Catherine M. - Bolotin, Ellen - Bomken, Simon - Buechner, Jochen - Burkhardt, Birgit - Caron, Hubert - Copland, Christopher - Demolis, Pierre - Egorov, Anton - Farhan, Mahdi - Zugmaier, Gerhard - Gross, Thomas - Horton-Taylor, Danielle - Klapper, Wolfram - Lesa, Giovanni - Marcus, Robert - Miles, Rodney R. - Nottage, Kerri - Pacaud, Lida - Ricafort, Rosanna - Schrappe, Martin - Štěrba, Jaroslav - Vezan, Remus - Weiner, Susan - Kim, Su Young - Reaman, Gregory - Vassal, Gilles
PY  - 2019
TI  - ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children
JF  - European Journal of Cancer
VL  - 110
IS  - 1879-0852
SP  - 74-85
EP  - 74-85
PB  - Elsevier Science Inc.
SN  - 09598049
KW  - Paediatric oncology
KW  - Mature B-cell malignancies
KW  - Medicinal product development
UR  - http://dx.doi.org/10.1016/j.ejca.2019.01.013
L2  - http://dx.doi.org/10.1016/j.ejca.2019.01.013
N2  - Paediatric Strategy Forums have been created by the multistakeholder organisation, ACCELERATE, and the European Medicines Agency to facilitate dialogue between all relevant stakeholders and suggest strategies in critical areas of paediatric oncology drug development. As there are many medicines being developed for B-cell malignancies in adults but comparatively few in children with these malignancies, a Paediatric Strategy Forum was held to discuss the best approach to develop these products for children. It was concluded that as current frontline therapy is highly successful, despite associated acute toxicity, de-escalation of this or substitution of presently used drugs with new medicines can only be undertaken when there is an effective salvage regimen, which is currently not available. Therefore priority should be given to developing treatment for patients with relapsed and refractory mature B-cell lymphomas. The consensus of the clinicians attending the meeting was that CAR T-cells, T-cell engagers and antibody drug conjugates (excluding those with a vinca alkaloidelike drug) presently have the greatest probability of providing benefit in relapse in view of their mechanism of action. However, as producing autologous CAR T-cells currently takes at least 4 weeks, they are not products which could be quickly employed initially at relapse in rapidly progressing mature B-cell malignancies but only for the consolidation phase of the treatment. Global, industry-supported, academic-sponsored studies testing compounds from different pharmaceutical companies simultaneously should be considered in rare populations, and it was proposed that an international working group be formed to develop an overarching clinical trials strategy for these disease groups. Future Forums are planned for other relevant paediatric oncologic diseases with a high unmet medical need and relevant molecular targets. (C) 2019 Elsevier Ltd. All rights reserved.
ER  -

PEARSON, Andrew D. J., Nicole SCOBIE, Koenraad NORGA, Franca LIGAS, Davy CHIODIN, Amos BURKE, Veronique MINARD-COLIN, Peter ADAMSON, Lynley V. MARSHALL, Arun BALAKUMARAN, Bouchra BENETTAIB, Pankaj BHARGAVA, Catherine M. BOLLARD, Ellen BOLOTIN, Simon BOMKEN, Jochen BUECHNER, Birgit BURKHARDT, Hubert CARON, Christopher COPLAND, Pierre DEMOLIS, Anton EGOROV, Mahdi FARHAN, Gerhard ZUGMAIER, Thomas GROSS, Danielle HORTON-TAYLOR, Wolfram KLAPPER, Giovanni LESA, Robert MARCUS, Rodney R. MILES, Kerri NOTTAGE, Lida PACAUD, Rosanna RICAFORT, Martin SCHRAPPE, Jaroslav ŠTĚRBA, Remus VEZAN, Susan WEINER, Su Young KIM, Gregory REAMAN a Gilles VASSAL. ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children. \textit{European Journal of Cancer}. Oxford: Elsevier Science Inc., 2019, roč.~110, 1879-0852, s.~74-85. ISSN~0959-8049. Dostupné z: https://dx.doi.org/10.1016/j.ejca.2019.01.013.

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