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753 results on '"Vassal, G."'

51. 061 - INTER-B NHL-RITUX-2010 TRIAL FOR CHILDREN/ADOLESCENTS WITH HIGH-RISK MATURE B-NHL: SAFETY AND EFFICACY IN PATIENTS TREATED WITH RITUXIMAB AND LMB CHEMOTHERAPY

Author

Minard-Colin, V., Aupérin, A., Burke, A., Alexander, S., Moreno, M., Buffardi, S., Uyttebroeck, A., Bollard, C., Zsiros, J., Csoka, M., Kazanowska, B., Chiang, A., Verschuur, A., Miles, R., Wotherspoon, A., Barkauskas, D., Wheatley, K., Vassal, G., Adamson, P., Gross, T., Patte, C., and Pillon, M.

Published
2022
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53. Defining and listing very rare cancers of paediatric age: consensus of the Joint Action on Rare Cancers in cooperation with the European Cooperative Study Group for Pediatric Rare Tumors

Author

Ferrari, A, Brecht, I, Gatta, G, Schneider, D, Orbach, D, Cecchetto, G, Godzinski, J, Reguerre, Y, Bien, E, Stachowicz-Stencel, T, Ost, M, Magni, C, Kearns, P, Vassal, G, Massimino, M, Biondi, A, Bisogno, G, Trama, A, Brecht, IB, Schneider, DT, Stachowicz-Stencel, Te, Ferrari, A, Brecht, I, Gatta, G, Schneider, D, Orbach, D, Cecchetto, G, Godzinski, J, Reguerre, Y, Bien, E, Stachowicz-Stencel, T, Ost, M, Magni, C, Kearns, P, Vassal, G, Massimino, M, Biondi, A, Bisogno, G, Trama, A, Brecht, IB, Schneider, DT, and Stachowicz-Stencel, Te

Abstract

Although all tumours are rare in childhood, there are some particularly rare paediatric cancers which have not benefited from advances made by the international paediatric oncology network. To establish a shared definition and produce a list of these entities, the European Union Joint Action on Rare Cancers (JARC) promoted a consensus effort. The definition was based on the incidence rates estimated using the information network on rare cancers (RARECAREnet) database, pooling data from 94 population-based cancer registries and 27 countries. The RARECAREnet list of cancers was used to estimate the incidence rates. This list groups cancers by combining the International Classification of Diseases for Oncology, third edition, morphology and topography codes. According to the consensus, very rare paediatric cancers were identified as those with an annual incidence <2/1000000 and corresponded to 11% of all cancers in patients aged 0–14 years. Two subgroups were identified: tumour types typical of childhood (i.e. hepatoblastoma, pleuropulmonary blastoma, pancreatoblastoma) and those typical of adult age (i.e. carcinomas, melanoma). The threshold of 2/1000000 could also be adopted in populations aged 0–19 years: in this case, three tumour types had an incidence rate which was >2/1000000 (i.e. thyroid and testicular cancers and skin melanoma), but the consensus experts considered them as ‘very rare’ according to their clinical needs (e.g. shortage of knowledge and clinical expertise as the other rare paediatric cancers). The JARC consensus produced a definition and a list of very rare paediatric cancers which may represent a starting point for prioritising research on these tumours, based on data and patients’ clinical needs.

Published
2019

63. The SIOPE strategic plan: A European cancer plan for children and adolescents

Author

Vassal, G, Schrappe, M, Pritchard-Jones, K, Arnold, F, Basset, L, Biondi, A, Bode, G, Eggert, A, Hjorth, L, Kameric, L, Kameric, N, Karner, S, Kearns, P, Kienesberger, A, Kowalczyk, J, Lack, P, Perilongo, G, Sullivan, R, Tsirou, A, Essiaf, S, Ladenstein, R, Vassal G., Schrappe M., Pritchard-Jones K., Arnold F., Basset L., Biondi A., Bode G., Eggert A., Hjorth L., Kameric L., Kameric N., Karner S., Kearns P., Kienesberger A., Kowalczyk J., Lack P., Perilongo G., Sullivan R., Tsirou A., Essiaf S., Ladenstein R., Vassal, G, Schrappe, M, Pritchard-Jones, K, Arnold, F, Basset, L, Biondi, A, Bode, G, Eggert, A, Hjorth, L, Kameric, L, Kameric, N, Karner, S, Kearns, P, Kienesberger, A, Kowalczyk, J, Lack, P, Perilongo, G, Sullivan, R, Tsirou, A, Essiaf, S, Ladenstein, R, Vassal G., Schrappe M., Pritchard-Jones K., Arnold F., Basset L., Biondi A., Bode G., Eggert A., Hjorth L., Kameric L., Kameric N., Karner S., Kearns P., Kienesberger A., Kowalczyk J., Lack P., Perilongo G., Sullivan R., Tsirou A., Essiaf S., and Ladenstein R.

Published
2016

64. Multiple synchronous mechanisms may contribute to osimertinib resistance in non-small cell lung cancer (NSCLC) patients: Insights of the MATCH-R study

Author

Enrico, D.H., primary, Lacroix, L., additional, Rouleau, E., additional, Scoazec, J.-Y., additional, Loriot, Y., additional, Tselikas, L., additional, Jovelet, C., additional, Planchard, D., additional, Gazzah, A., additional, Mezquita, L., additional, Ngo, M., additional, Michiels, S., additional, Massard, C., additional, Facchinetti, F., additional, Chen, J., additional, Soria, J.-C., additional, André, F., additional, Vassal, G., additional, Friboulet, L., additional, and Besse, B., additional

Published
2019
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65. Evaluation of the Implementation of the Response Assessment in Neuro-Oncology Criteria in the HERBY Trial of Pediatric Patients with Newly Diagnosed High-Grade Gliomas

Author

Rodriguez, D., primary, Chambers, T., additional, Warmuth-Metz, M., additional, Aliaga, E. Sanchez, additional, Warren, D., additional, Calmon, R., additional, Hargrave, D., additional, Garcia, J., additional, Vassal, G., additional, Grill, J., additional, Zahlmann, G., additional, Morgan, P.S., additional, and Jaspan, T., additional

Published
2019
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70. OA12.03 Activity of Crizotinib in MET or ROS1 Positive (+) NSCLC: Results of the AcSé Trial.

Author

Moro-Sibilot, D., primary, Cozic, N., additional, Pérol, M., additional, Otto, J., additional, Mazieres, J., additional, Souquet, P., additional, Bahleda, R., additional, Wislez, M., additional, De Guibert, S., additional, Mennecier, B., additional, Chouaid, C., additional, Sabatier, R., additional, Bota, S., additional, Gervais, R., additional, Verriele, V., additional, Haddad, V., additional, Ferretti, G., additional, Nowak, F., additional, Mahier - Ait Oukhatar, C., additional, and Vassal, G., additional

Published
2018
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71. Biomarker-driven access to crizotinib in ALK, MET or ROS1 positive (+) malignancies in adults and children: The French national AcSé program

Author

Vassal, G., primary, Cozic, N., additional, Houot, R., additional, Brugières, L., additional, Aparicio, T., additional, Blay, J.-Y., additional, Perol, M., additional, Brice, P., additional, Meriaux, E., additional, Geoerger, B., additional, El Bejjani, M.R., additional, Moalla, S., additional, Bièche, I., additional, Lantuejoul, S., additional, Mahier Ait Oukhatar, C., additional, Hoog Labouret, N., additional, and Moro-Sibilot, D., additional

Published
2018
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72. Tumors in Adolescents and Young Adults

Author

Stark, D.P, Vassal, G., Stark, D.P, and Vassal, G.

Subjects
Neoplasms, Adolescent, Young Adult
Abstract

The field of adolescents and young adult (AYA) oncology is experiencing a very challenging time. This book is a guide to the key issues for any clinician and health professional managing AYA with cancer in Europe. Emphasis is on collaboration between adult and pediatric specialists. Authors present their perception of the current state of the most prominent primary issues in AYA oncology. Chapters cover cross-cutting issues such as disease epidemiology, systems of care, access to innovative therapy and late effects of treatment and survivorship for AYA-onset cancers. There are discussions of the latest developments and the most important cancer types for AYA, including the shared perspectives of adult and pediatric specialists. Throughout the book recurrent challenges to the AYA community are exposed and solutions proposed. Tumors in Adolescents and Young Adults is highly recommended to any oncologist or haematologist treating patients aged 15 to 39 diagnosed with cancer. It will also be of interest to other members of the multidisciplinary teams involved with this patient group.

Published
2016

73. Roles of Clinical Research Networks in Pediatric Drug Development

Author

Turner, M.A., Attar, S., Wildt, S.N. de, Vassal, G., Mangiarini, L., Giaquinto, C., Turner, M.A., Attar, S., Wildt, S.N. de, Vassal, G., Mangiarini, L., and Giaquinto, C.

Abstract

Item does not contain fulltext, The evaluation of drugs that are used in children has been neglected historically but is now well established as an essential part of clinical drug development. The increase in pediatric activity among industry, and other sectors, has highlighted the importance of joint working. All participants in pediatric drug development need to be aware of the "big picture." An increasingly important part of this big picture in pediatrics, as in other populations, is the design and conduct of clinical trials in networks. This narrative review provides an overview of the roles of clinical research networks in pediatric drug development. Networks take many forms as specialty networks and geographic networks but work toward common principles, including sharing resources between trials, and using experience with trial conduct to improve trial design. Networks develop standardized processes for trial conduct (including performance management) that increase the speed and predictability of trial conduct while reducing burdens on sites, sponsors, and intermediaries. Networks can provide validated, real-world information about natural history, participant distribution, and standards of care to inform planning of development programs, including extrapolation and clinical trial simulation. Networks can work across geographic and jurisdictional barriers to promote global interoperability of drug development. Networks support participant centrality. Networks offer an opportunity to develop relationships with investigators, sites, and methodological experts that span pre-competitive foundations for drug development and specific products. Sustainable networks benefit all stakeholders by providing a multifunctional platform that promotes the quality and timeliness of clinical drug development.

Published
2017

74. Polo-like Kinase Inhibitor Volasertib Exhibits Antitumor Activity and Synergy with Vincristine in Pediatric Malignancies

Author

Abbou, S., Lanvers-Kaminsky, C., Daudigeos-Dubus, E., Le Dret, L., Laplace-Builhe, C., Molenaar, J., Vassal, G., birgit geoerger, Paediatric Oncology, and Oncogenomics

Abstract

Polo-like kinase 1 (PLK1) controls the main cell-cycle checkpoints, suggesting utility of its inhibition for cancer treatment, including of highly proliferative pediatric cancer. This preclinical study explored the selective PLK1 inhibitor volasertib (BI 6727) alone and combined with chemotherapy in pediatric malignancies. Inhibition of proliferation was explored in vitro using dimethylthiazol carboxymethoxyphenyl sulfophenyl tetrazolium (MTS) assay. Mice bearing human xenografts were treated with weekly intravenous injections of volasertib. Volasertib inhibited proliferation in all 40 cell lines tested, with a mean half-maximal growth inhibitory concentration of 313 nmol/l (range: 4-5000 nmol/l). Volasertib was highly active against RMS-1 alveolar rhabdomyosarcoma xenografts, resulting in 100% tumor regression. Activity was associated with complete and prolonged G2/M arrest and subsequent apoptotic cell death. Volasertib showed synergistic activity with vincristine but antagonistic effects with etoposide. These findings support the further exploration of volasertib for pediatric malignancies, particularly alveolar rhabdomyosarcoma, and its combination with mitotic spindle poison

Published
2016

77. Rare Cancers Europe (RCE) methodological recommendations for clinical studies in rare cancers: A European consensus position paper

Author

Casali, Paolo G., Bruzzi, P., Bogaerts, Jan, Blay, Jean-Yves, Aapro, M. S., Adamou, A., Berruti, A., Bressington, J., Bruzzi, B., Capocaccia, R., Cardoso, Fatima, Celis, J. E., Cervantes, A., Ciardiello, F., Claussen, C., Coleman, M., Comis, S., Craine, S., Boltz, D. De, Lorenzo, F. De, P, Angelo Dei Tos, Gatta, G., Geissler, J., Giuliani, R., Grande, E., Gronchi, A., Jezdic, S., Jonsson, B., Jost, Lorenz M., Keulen, H., Lacombe, D., Lamory, G., Cam, Y. Le, Priolo, S. Leto di, Licitra, Lisa, Macchia, F., Margulies, A., Marreaud, S., McVie, G., Narbutas, S., Oliver, K., Pavlidis, Nicholas, Pelouchova, J., Pentheroudakis, George, Piccart, M., Pierotti, M. A., Pravettoni, G., Redmond, K., Riegman, P., Ruffilli, M. P., Ryner, D., Sandrucci, S., Seymour, M., Torri, V., Trama, A., Belle, S. Van, Vassal, G., Wartenberg, M., Watts, C., Wilson, A., Yared, W., Pavlidis, Nicholas [0000-0002-2195-9961], and Pentheroudakis, George [0000-0002-6632-2462]

Subjects
Research design, Pathology, Data base, Research methodology, Electronic medical record, Disease, Review, Procedures, Treatment response, Clinical trials, Rare cancers, Clinical Studies as Topic, Humans, Neoplasms, Rare Diseases, Research Design, Hematology, Oncology, Reimbursement, Priority journal, education.field_of_study, Clinical studies as topic, Rare diseases, Europe, Clinical decision making, Human, medicine.medical_specialty, Practice guideline, Case finding, Population, Health care quality, Reviews, Cancer research, Clinical study, SDG 3 - Good Health and Well-being, Conceptual framework, medicine, Tumor marker, Intensive care medicine, education, Antineoplastic activity, Flexibility (engineering), Surrogate endpoint, business.industry, Methodology, Rare cancer, Study design, Cancer survival, Clinical trial, Patient information, Clinical effectiveness, Position paper, Neoplasm, business, Rare disease
Abstract

While they account for one-fifth of new cancer cases, rare cancers are difficult to study. A higher than average degree of uncertainty should be accommodated for clinical as well as for population-based decision making. Rules of rational decision making in conditions of uncertainty should be rigorously followed and would need widely informative clinical trials. In principle, any piece of new evidence would need to be exploited in rare cancers. Methodologies to explicitly weigh and combine all the available evidence should be refined, and the Bayesian logic can be instrumental to this end. Likewise, Bayesian-design trials may help optimize the low number of patients liable to be enrolled in clinical studies on rare cancers, as well as adaptive trials in general, with their inherent potential of flexibility when properly applied. While clinical studies are the mainstay to test hypotheses, the potential of electronic patient records should be exploited to generate new hypotheses, to create external controls for future studies (when internal controls are unpractical), to study effectiveness of new treatments in real conditions. Framework study protocols in specific rare cancers to stepwisely test sets of new agents, as from the early post-phase I development stage, should be encouraged. Also the compassionate and the off-label settings should be exploited to generate new evidence, and flexible regulatory innovations such as adaptive licensing could convey new agents early to rare cancer patients, while generating evidence. Though validation of surrogate end points is problematic in rare cancers, the use of an updated notion of tumor response may be of great value in the single patient to optimize the use of therapies, all the more the new ones. Disease-based communities, involving clinicians and patients, should be regularly consulted by regulatory bodies when setting their policies on drug approval and reimbursement in specific rare cancers ., While they account for one-fifth of new cancer cases, rare cancers are difficult to study. A higher than average degree of uncertainty should be accommodated for clinical as well as for population-based decision making. Rules of rational decision making in conditions of uncertainty should be rigorously followed and would need widely informative clinical trials. In principle, any piece of new evidence would need to be exploited in rare cancers. Methodologies to explicitly weigh and combine all the available evidence should be refined, and the Bayesian logic can be instrumental to this end. Likewise, Bayesian-design trials may help optimize the low number of patients liable to be enrolled in clinical studies on rare cancers, as well as adaptive trials in general, with their inherent potential of flexibility when properly applied. While clinical studies are the mainstay to test hypotheses, the potential of electronic patient records should be exploited to generate new hypotheses, to create external controls for future studies (when internal controls are unpractical), to study effectiveness of new treatments in real conditions. Framework study protocols in specific rare cancers to sequentially test sets of new agents, as from the early post-phase I development stage, should be encouraged. Also the compassionate and the off-label settings should be exploited to generate new evidence, and flexible regulatory innovations such as adaptive licensing could convey new agents early to rare cancer patients, while generating evidence. Though validation of surrogate end points is problematic in rare cancers, the use of an updated notion of tumor response may be of great value in the single patient to optimize the use of therapies, all the more the new ones. Disease-based communities, involving clinicians and patients, should be regularly consulted by regulatory bodies when setting their policies on drug approval and reimbursement in specific rare cancers.

Published
2015

78. The SIOPE strategic plan: A European cancer plan for children and adolescents

Author

Universitat Politècnica de València. Departamento de Mecánica de los Medios Continuos y Teoría de Estructuras - Departament de Mecànica dels Medis Continus i Teoria d'Estructures, Vassal, G., Schrappe, M., Pritchard-Jones, K., Arnold, F., Basset-Salom, Luisa, Biondi, A., Bode, G., Eggert, A., Hjorth, L., Kameric, L., Kameric, N., Karner, S., Kearns, P., Kienesberger, A., Kowalczyk, J., Lack, P., Perilongo, G., Sullivan, R., Tsirou, A., Essiaf, S., Landenstein, R., Universitat Politècnica de València. Departamento de Mecánica de los Medios Continuos y Teoría de Estructuras - Departament de Mecànica dels Medis Continus i Teoria d'Estructures, Vassal, G., Schrappe, M., Pritchard-Jones, K., Arnold, F., Basset-Salom, Luisa, Biondi, A., Bode, G., Eggert, A., Hjorth, L., Kameric, L., Kameric, N., Karner, S., Kearns, P., Kienesberger, A., Kowalczyk, J., Lack, P., Perilongo, G., Sullivan, R., Tsirou, A., Essiaf, S., and Landenstein, R.

Abstract

[EN] Within the European Network for Cancer research in Children and Adolescents (ENCCA), SIOPE and the European paediatric haematology-oncology community have established a long-term sustainable Strategic Plan to increase the cure rate and the quality of survivorship for children and young people with cancer over the next ten years. The ultimate goal is to increase the diseaseand late-effect- free survival after 10 years from the diagnosis, and beyond. As a result of several initiatives to involve all stakeholders and ensure that all their points of view would be taken into account in the document, this long-term sustainable Strategic Plan has achieved a broad consensus, and will serve as the "European Cancer Plan for Children and Adolescents".

Published
2016

79. The ENCCA-WP7/EuroSarc/EEC/PROVABES/EURAMOS 3rd European Bone Sarcoma Networking Meeting/Joint Workshop of EU Bone Sarcoma Translational Research Networks; Vienna, Austria, September 24-25, 2015. Workshop Report

Author

Kager, L., Whelan, J., Dirksen, U., Hassan, B., Anninga, J., Bennister, L., Bovee, J.V., Brennan, B., Broto, J.M., Brugieres, L., Cleton-Jansen, A.M., Copland, C., Dutour, A., Fagioli, F., Ferrari, S., Fiocco, M., Fleuren, E.D., Gaspar, N., Gelderblom, H., Gerrand, C., Gerss, J., Gonzato, O., Graaf, W.T.A. van der, Hecker-Nolting, S., Herrero-Martin, D., Klco-Brosius, S., Kovar, H., Ladenstein, R., Lancia, C., Ledeley, M.C., McCabe, M.G., Metzler, M., Myklebost, O., Nathrath, M., Picci, P., Potratz, J., Redini, F., Richter, G.H., Reinke, D., Rutkowski, P., Scotlandi, K., Strauss, S., Thomas, D, Tirado, O.M., Tirode, F., Vassal, G., Bielack, S.S., Kager, L., Whelan, J., Dirksen, U., Hassan, B., Anninga, J., Bennister, L., Bovee, J.V., Brennan, B., Broto, J.M., Brugieres, L., Cleton-Jansen, A.M., Copland, C., Dutour, A., Fagioli, F., Ferrari, S., Fiocco, M., Fleuren, E.D., Gaspar, N., Gelderblom, H., Gerrand, C., Gerss, J., Gonzato, O., Graaf, W.T.A. van der, Hecker-Nolting, S., Herrero-Martin, D., Klco-Brosius, S., Kovar, H., Ladenstein, R., Lancia, C., Ledeley, M.C., McCabe, M.G., Metzler, M., Myklebost, O., Nathrath, M., Picci, P., Potratz, J., Redini, F., Richter, G.H., Reinke, D., Rutkowski, P., Scotlandi, K., Strauss, S., Thomas, D, Tirado, O.M., Tirode, F., Vassal, G., and Bielack, S.S.

Abstract

Contains fulltext : 167540.pdf (publisher's version ) (Open Access), This report summarizes the results of the 3rd Joint ENCCA-WP7, EuroSarc, EEC, PROVABES, and EURAMOS European Bone Sarcoma Network Meeting, which was held at the Children's Cancer Research Institute in Vienna, Austria on September 24-25, 2015. The joint bone sarcoma network meetings bring together European bone sarcoma researchers to present and discuss current knowledge on bone sarcoma biology, genetics, immunology, as well as results from preclinical investigations and clinical trials, to generate novel hypotheses for collaborative biological and clinical investigations. The ultimate goal is to further improve therapy and outcome in patients with bone sarcomas.

Published
2016

80. ECCO Essential Requirements for Quality Cancer Care: Soft Tissue Sarcoma in Adults and Bone Sarcoma. A critical review

Author

Andritsch, E, Beishon, M, Bielack, S, Bonvalot, S, Casali, P, Crul, M, Delgado-Bolton, R, Donati, D, Douis, H, Haas, R, Hogendoorn, P, Kozhaeva, O, Lavender, V, Lovely, J, Negrouk, A, Pereira, P, Roca, P, Rochette de Lempdes, G, Saarto, T, van Berck, B, Vassal, G, Wartenberg, M, Yared, W, Costa, A, Naredi, P, Andritsch, E, Beishon, M, Bielack, S, Bonvalot, S, Casali, P, Crul, M, Delgado-Bolton, R, Donati, D, Douis, H, Haas, R, Hogendoorn, P, Kozhaeva, O, Lavender, V, Lovely, J, Negrouk, A, Pereira, P, Roca, P, Rochette de Lempdes, G, Saarto, T, van Berck, B, Vassal, G, Wartenberg, M, Yared, W, Costa, A, and Naredi, P

Abstract

ECCO essential requirements for quality cancer care (ERQCC) are checklists and explanations of organisation and actions that are necessary to give high-quality care to patients who have a specific tumour type. They are written by European experts representing all disciplines involved in cancer care. ERQCC papers give oncology teams, patients, policymakers and managers an overview of the elements needed in any healthcare system to provide high quality of care throughout the patient journey. References are made to clinical guidelines and other resources where appropriate, and the focus is on care in Europe. Sarcoma: essential requirements for quality care • Sarcomas – which can be classified into soft tissue and bone sarcomas – are rare, but all rare cancers make up more than 20% of cancers in Europe, and there are substantial inequalities in access to high-quality care. Sarcomas, of which there are many subtypes, comprise a particularly complex and demanding challenge for healthcare systems and providers. This paper presents essential requirements for quality cancer care of soft tissue sarcomas in adults and bone sarcomas. • High-quality care must only be carried out in specialised sarcoma centres (including paediatric cancer centres) which have both a core multidisciplinary team and an extended team of allied professionals, and which are subject to quality and audit procedures. Access to such units is far from universal in all European countries. • It is essential that, to meet European aspirations for high-quality comprehensive cancer control, healthcare organisations implement the requirements in this paper, paying particular attention to multidisciplinarity and patient-centred pathways from diagnosis and follow-up, to treatment, to improve survival and quality of life for patients. Conclusion Taken together, the information presented in this paper provides a comprehensive description of the essential requirements for establishing a high-quality service for soft t

Published
2016

83. Trajectoires scolaires après un cancer pédiatrique : une contribution à l’hypothèse de la sélection par la santé

Author

Dumas, A., primary, Berger, C., additional, Auquier, P., additional, Michel, G., additional, Vassal, G., additional, Valteau-Couanet, D., additional, Fresneau, B., additional, Thouvenin-Doulet, S., additional, Casagranda, L., additional, Pacquement, H., additional, El-Fayech, C., additional, Oberlin, O., additional, Guibout, C., additional, and De Vathaire, F., additional

Published
2016
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84. Rare Cancers Europe (RCE) methodological recommendations for clinical studies in rare cancers: A European consensus position paper

Author

Casali, P.G. (Paolo), Bruzzi, P. (P.), Bogaerts, J. (Jan), Blay, J.Y. (Jean Yves), Aapro, M. (Matti), Adamous, A., Berruti, A. (Alfredo), Bressington, J., Bruzzi, B., Capocaccia, R. (Riccardo), Cardoso, F. (Fatima), Celis, J.E., Cervantes, A. (Andres), Ciardiello, F., Claussen, C., Coleman, M., Comis, S., Craine, S., De Boltz, D., De Lorenzo, F., Dei Tos, A.P. (Angelo), Gatta, G. (Gemma), Geissler, J. (Jan), Giuliani, R., Grande, E. (Enrico), Gronchi, A. (Alessandro), Jezdic, S., Jonsson, B., Jost, L., Keulen, H., Lacombe, D. (Denis), Lamory, G., Le Cam, Y., Leto di Priolo, S., Licitra, L., Macchia, F., Margulies, A., Marreaud, S. (Sandrine), McVie, G., Narbutas, S., Oliver, K., Pavlidis, N., Pelouchova, J., Pentheroudakis, G., Piccart, M.J. (Martine), Pierotti, M. (Marco Alessandro), Pravettoni, G., Redmond, K., Riegman, P.H.J. (Peter), Ruffilli, M.P., Ryner, D., Sandrucci, S., Seymour, M., Torri, V. (Valter), Trama, A., Belle, S. (S.) van, Vassal, G., Wartenberg, M., Watts, C., Wilson, A., Yared, W., Casali, P.G. (Paolo), Bruzzi, P. (P.), Bogaerts, J. (Jan), Blay, J.Y. (Jean Yves), Aapro, M. (Matti), Adamous, A., Berruti, A. (Alfredo), Bressington, J., Bruzzi, B., Capocaccia, R. (Riccardo), Cardoso, F. (Fatima), Celis, J.E., Cervantes, A. (Andres), Ciardiello, F., Claussen, C., Coleman, M., Comis, S., Craine, S., De Boltz, D., De Lorenzo, F., Dei Tos, A.P. (Angelo), Gatta, G. (Gemma), Geissler, J. (Jan), Giuliani, R., Grande, E. (Enrico), Gronchi, A. (Alessandro), Jezdic, S., Jonsson, B., Jost, L., Keulen, H., Lacombe, D. (Denis), Lamory, G., Le Cam, Y., Leto di Priolo, S., Licitra, L., Macchia, F., Margulies, A., Marreaud, S. (Sandrine), McVie, G., Narbutas, S., Oliver, K., Pavlidis, N., Pelouchova, J., Pentheroudakis, G., Piccart, M.J. (Martine), Pierotti, M. (Marco Alessandro), Pravettoni, G., Redmond, K., Riegman, P.H.J. (Peter), Ruffilli, M.P., Ryner, D., Sandrucci, S., Seymour, M., Torri, V. (Valter), Trama, A., Belle, S. (S.) van, Vassal, G., Wartenberg, M., Watts, C., Wilson, A., and Yared, W.

Abstract

While they account for one-fifth of new cancer cases, rare cancers are difficult to study. A higher than average degree of uncertainty should be accommodated for clinical as well as for population-based decision making. Rules of rational decision making in conditions of uncertainty should be rigorously followed and would need widely informative clinical trials. In principle, any piece of new evidence would need to be exploited in rare cancers. Methodologies to explicitly weigh and combine all the available evidence should be refined, and the Bayesian logic can be instrumental to this end. Likewise, Bayesian-design trials may help optimize the low number of patients liable to be enrolled in clinical studies on rare cancers, as well as adaptive trials in general, with their inherent potential of flexibility when properly applied. While clinical studies are the mainstay to test hypotheses, the potential of electronic patient records should be exploited to generate new hypotheses, to create external controls for future studies (when internal controls are unpractical), to study effectiveness of new treatments in real conditions. Framework study protocols in specific rare cancers to stepwisely test sets of new agents, as from the early post-phase I dev

Published
2015
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87. 12LBA Biomarker-driven access to crizotinib in ALK, MET or ROS1 positive (+) malignancies in adults and children: The French national AcSe Program

Author

Vassal, G., primary, Moro-sibilot, D., additional, Le Deley, M.C., additional, Hoog-labouret, N., additional, Nowak, F., additional, Jimenez, M., additional, Tournigand, C., additional, Houot, R., additional, Malka, D., additional, Aparicio, T., additional, Escudier, B., additional, Ray-coquard, I., additional, Godbert, Y., additional, Taillandier, L., additional, Bieche, I., additional, Lantuejoul, S., additional, Ferretti, G., additional, Menu, Y., additional, Blay, J.Y., additional, and Buzyn, A., additional

Published
2015
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88. 1407 The European Strategic Plan for children and adolescents with cancer

Author

Vassal, G., primary, Schrappe, M., additional, Pritchard-Jones, K., additional, Arnold, F., additional, Basset, L., additional, Biondi, A., additional, Bode, G., additional, Eggert, A., additional, Hjorth, L., additional, Kamerić, L., additional, Karner, S., additional, Kearns, P., additional, Kienesberger, A., additional, Kowalczyk, J., additional, Lack, P., additional, Perilongo, G., additional, Sullivan, R., additional, Tsirou, A., additional, Kamerić, N., additional, Essiaf, S., additional, and Ladenstein, R., additional

Published
2015
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89. Challenges for children and adolescents with cancer in Europe: the SIOP-Europe agenda

Author

Vassal, G, Fitzgerald, E, Schrappe, M, Arnold, F, Kowalczyk, J, Walker, D, Hjorth, L, Riccardi, R, Kienesberger, A, Jones, K, Valsecchi, M, Janic, D, Hasle, H, Kearns, P, Petrarulo, G, Florindi, F, Essiaf, S, Ladenstein, R, Ladenstein, R., VALSECCHI, MARIA GRAZIA, Vassal, G, Fitzgerald, E, Schrappe, M, Arnold, F, Kowalczyk, J, Walker, D, Hjorth, L, Riccardi, R, Kienesberger, A, Jones, K, Valsecchi, M, Janic, D, Hasle, H, Kearns, P, Petrarulo, G, Florindi, F, Essiaf, S, Ladenstein, R, Ladenstein, R., and VALSECCHI, MARIA GRAZIA

Abstract

In Europe, 6,000 young people die of cancer yearly, the commonest disease causing death beyond the age of 1 year. In addition, 300,000-500,000 European citizens are survivors of a childhood cancer and up to 30% of them have severe long-term sequelae of their treatment. Increasing both cure and quality of cure are the two goals of the European paediatric haematology/oncology community. SIOPE coordinates and facilitates research, care and training which are implemented by the 18 European study groups and 23 national paediatric haematology/oncology societies. SIOPE is the European branch of the International Society of Paediatric Oncology and one of the six founding members of the European Cancer Organisation. SIOPE is preparing its strategic agenda to assure long-term sustainability of clinical and translational research in paediatric malignancies over the next 15 years. SIOPE tackles the issues of equal access to standard care and research across Europe and improvement of long term follow up. SIOPE defined a comprehensive syllabus for training European specialists. A strong partnership with parent, patient and survivor organisations is being developed to successfully achieve the goals of this patient-centred agenda. SIOPE is advocating in the field of EU policies, such as the Clinical Trials Regulation and the Paediatric Medicine Regulation, to warrant that the voice of young people is heard and their needs adequately addressed. SIOPE and the European community are entirely committed to the global agenda against childhood cancers to overcome the challenges to increasing both cure and quality of cure of young people with cancer.

Published
2014

90. In vivo echographic evidence of tumoral vascularization and microenvironment interactions in metastatic orthotopic human neuroblastoma xenografts

Author

Joseph, Jm, Lassau, N., Rouffiac, V., Opolon, Paule, Laudani, L., Auderset, K., Geay, Jf, Muhlethaler-Mottet, A., Vassal, G., Vectorologie et transfert de gènes (VTG / UMR8121), and Université Paris-Sud - Paris 11 (UP11)-Institut Gustave Roussy (IGR)-Centre National de la Recherche Scientifique (CNRS)

Subjects
[SDV.BC]Life Sciences [q-bio]/Cellular Biology
Abstract

Human neuroblastoma (NB) is the second most frequent solid tumor of childhood and represents a highly heterogeneous disease at clinical and biologic levels. Little progress has been made to improve the poor prognosis of patients with high-stage NB. Tumor progression and metastatic dissemination still represent major obstacles to the successful treatment of advanced stage disease. In order to develop and evaluate new, targeted, therapeutic strategies, fully defined and biologically relevant in vivo models of NB are strongly needed. We have developed an orthotopic model of metastatic human NB in the nude mouse, using 2 well-characterized NB cell lines. Tumor growth, vascular properties and metastatic patterns were investigated using a sensitive and newly developed in vivo echographic technology in addition to immunohistochemistry and PCR analyses. Results show that implantation of low numbers of NB cells directly into the adrenal gland of nude mice resulted in rapid and homogeneous tumor growth without tumor morbidity. Nude mice were shown to rapidly develop highly vascularized adrenal tumors that selectively metastasized to the liver and bone marrow. In addition, the newly formed mouse vessels in orthotopic but not in heterotopic tumors, were found to express the highly angiogenic alphavbeta3 integrin marker, indicating the development of a truly malignant neovasculature in orthotopic conditions only. This observation confirms the impact of the regional microenvironment on tumor biology and suggests the existence of cross-talk with the tumor cells. In conclusion, such model faithfully reproduces the growth, vascular and metastatic patterns as observed in patients. It therefore represents a powerful and biologically relevant tool to improve our understanding of the biology of NB and to develop and assess new antiangiogenic and metastasis-targeted therapies. (c) 2004 Wiley-Liss, Inc.

Published
2005

91. L'aléa incendie et le massif des Maures, aménagement de la RD25 et du COSMA, exposé no 2 sur les feux de forêt et les infrastuctures routières

Author

Gerbeaud Maulin, F., Gasquy, P., Guglielmetti, A., López, S., Faure Vassal, G., Jappiot, M., Lampin, C., Morge, D., and Irstea Publications, Migration

Subjects
[SDE] Environmental Sciences
Abstract

To take into account forest fire hazard in road development: improvement,rehabilitation ( RD25) and new road conception( COSMA)., Prise en compte de l'aléa incendie de forêt dans l'aménagement routier: amélioration (RD25) et choix de nouveau tracé (COSMA).

Published
2004

93. The Herby Study: a Phase 2 Open-Label, Randomized, Multicenter Study of Bevacizumab-Based Therapy in Pediatric Patients with Newly Diagnosed High-Grade Glioma

Author

Grill, J., primary, Hargrave, D., additional, Varlet, P., additional, Jaspan, T., additional, Jones, C., additional, Massimino, M., additional, Cañete, A., additional, Azizi, A.A., additional, Le Deley, M., additional, Saran, F., additional, Morgan, P., additional, Zahlmann, G., additional, Zheng, M., additional, Fuerst-Recktenwald, S., additional, Berger, C., additional, Bouffet, E., additional, and Vassal, G., additional

Published
2014
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94. HIGH GRADE GLIOMAS AND DIPG

Author

Classen, C. F., primary, William, D., additional, Linnebacher, M., additional, Farhod, A., additional, Kedr, W., additional, Elsabe, B., additional, Fadel, S., additional, Van Gool, S., additional, De Vleeschouwer, S., additional, Koks, C., additional, Garg, A., additional, Ehrhardt, M., additional, Riva, M., additional, Agostinis, P., additional, Graf, N., additional, Yao, T.-W., additional, Yoshida, Y., additional, Zhang, J., additional, Ozawa, T., additional, James, D., additional, Nicolaides, T., additional, Kebudi, R., additional, Cakir, F. B., additional, Gorgun, O., additional, Agaoglu, F. Y., additional, Darendeliler, E., additional, Al-Kofide, A., additional, Al-Shail, E., additional, Khafaga, Y., additional, Al-Hindi, H., additional, Dababo, M., additional, Haq, A. U., additional, Anas, M., additional, Barria, M. G., additional, Siddiqui, K., additional, Hassounah, M., additional, Ayas, M., additional, van Zanten, S. V., additional, Jansen, M., additional, van Vuurden, D., additional, Huisman, M., additional, Vugts, D., additional, Hoekstra, O., additional, van Dongen, G., additional, Kaspers, G., additional, Cockle, J., additional, Ilett, E., additional, Scott, K., additional, Bruning-Richardson, A., additional, Picton, S., additional, Short, S., additional, Melcher, A., additional, Benesch, M., additional, Warmuth-Metz, M., additional, von Bueren, A. O., additional, Hoffmann, M., additional, Pietsch, T., additional, Kortmann, R.-D., additional, Eyrich, M., additional, Rutkowski, S., additional, Fruhwald, M. C., additional, Faber, J., additional, Kramm, C., additional, Porkholm, M., additional, Valanne, L., additional, Lonnqvist, T., additional, Holm, S., additional, Lannering, B., additional, Riikonen, P., additional, Wojcik, D., additional, Sehested, A., additional, Clausen, N., additional, Harila-Saari, A., additional, Schomerus, E., additional, Thorarinsdottir, H. K., additional, Lahteenmaki, P., additional, Arola, M., additional, Thomassen, H., additional, Saarinen-Pihkala, U. M., additional, Kivivuori, S.-M., additional, Buczkowicz, P., additional, Hoeman, C., additional, Rakopoulos, P., additional, Pajovic, S., additional, Morrison, A., additional, Bouffet, E., additional, Bartels, U., additional, Becher, O., additional, Hawkins, C., additional, Gould, T. W. A., additional, Rahman, C. V., additional, Smith, S. J., additional, Barrett, D. A., additional, Shakesheff, K. M., additional, Grundy, R. G., additional, Rahman, R., additional, Barua, N., additional, Cronin, D., additional, Gill, S., additional, Lowisl, S., additional, Hochart, A., additional, Maurage, C.-A., additional, Rocourt, N., additional, Vinchon, M., additional, Kerdraon, O., additional, Escande, F., additional, Grill, J., additional, Pick, V. K., additional, Leblond, P., additional, Burzynski, G., additional, Janicki, T., additional, Burzynski, S., additional, Marszalek, A., additional, Ramani, N., additional, Zaky, W., additional, Kannan, G., additional, Morani, A., additional, Sandberg, D., additional, Ketonen, L., additional, Maher, O., additional, Corrales-Medina, F., additional, Meador, H., additional, Khatua, S., additional, Brassesco, M., additional, Delsin, L., additional, Roberto, G., additional, Silva, C., additional, Ana, L., additional, Rego, E., additional, Scrideli, C., additional, Umezawa, K., additional, Tone, L., additional, Kim, S. J., additional, Kim, C.-Y., additional, Kim, I.-A., additional, Han, J. H., additional, Choi, B.-S., additional, Ahn, H. S., additional, Choi, H. S., additional, Haque, F., additional, Layfield, R., additional, Grundy, R., additional, Gandola, L., additional, Pecori, E., additional, Biassoni, V., additional, Schiavello, E., additional, Chiruzzi, C., additional, Spreafico, F., additional, Modena, P., additional, Bach, F., additional, Pignoli, E., additional, Massimino, M., additional, Drogosiewicz, M., additional, Dembowska-Baginska, B., additional, Jurkiewicz, E., additional, Filipek, I., additional, Perek-Polnik, M., additional, Swieszkowska, E., additional, Perek, D., additional, Bender, S., additional, Jones, D. T., additional, Warnatz, H.-J., additional, Hutter, B., additional, Zichner, T., additional, Gronych, J., additional, Korshunov, A., additional, Eils, R., additional, Korbel, J. O., additional, Yaspo, M.-L., additional, Lichter, P., additional, Pfister, S. M., additional, Yadavilli, S., additional, Becher, O. J., additional, Kambhampati, M., additional, Packer, R. J., additional, Nazarian, J., additional, Lechon, F. C., additional, Fowkes, L., additional, Khabra, K., additional, Martin-Retortillo, L. M., additional, Marshall, L. V., additional, Vaidya, S., additional, Koh, D.-M., additional, Leach, M. O., additional, Pearson, A. D., additional, Zacharoulis, S., additional, Schrey, D., additional, Barone, G., additional, Panditharatna, E., additional, Stampar, M., additional, Siu, A., additional, Gordish-Dressman, H., additional, Devaney, J., additional, Hwang, E. I., additional, Chung, A. H., additional, Mittapalli, R. K., additional, Elmquist, W. F., additional, Castel, D., additional, Debily, M.-A., additional, Philippe, C., additional, Truffaux, N., additional, Taylor, K., additional, Calmon, R., additional, Boddaert, N., additional, Le Dret, L., additional, Saulnier, P., additional, Lacroix, L., additional, Mackay, A., additional, Jones, C., additional, Puget, S., additional, Sainte-Rose, C., additional, Blauwblomme, T., additional, Varlet, P., additional, Entz-Werle, N., additional, Maugard, C., additional, Bougeard, G., additional, Nguyen, A., additional, Chenard, M. P., additional, Schneider, A., additional, Gaub, M. P., additional, Tsoli, M., additional, Vanniasinghe, A., additional, Luk, P., additional, Dilda, P., additional, Haber, M., additional, Hogg, P., additional, Ziegler, D., additional, Simon, S., additional, Monje, M., additional, Gurova, K., additional, Gudkov, A., additional, Zapotocky, M., additional, Churackova, M., additional, Malinova, B., additional, Zamecnik, J., additional, Kyncl, M., additional, Tichy, M., additional, Puchmajerova, A., additional, Stary, J., additional, Sumerauer, D., additional, Boult, J., additional, Vinci, M., additional, Perryman, L., additional, Box, G., additional, Jury, A., additional, Popov, S., additional, Ingram, W., additional, Eccles, S., additional, Robinson, S., additional, Emir, S., additional, Demir, H. A., additional, Bayram, C., additional, Cetindag, F., additional, Kabacam, G. B., additional, Fettah, A., additional, Li, J., additional, Jamin, Y., additional, Cummings, C., additional, Bamber, J., additional, Sinkus, R., additional, Nandhabalan, M., additional, Bjerke, L., additional, Burford, A., additional, von Bueren, A., additional, Baudis, M., additional, Clarke, P., additional, Collins, I., additional, Workman, P., additional, Olaciregui, N., additional, Mora, J., additional, Carcaboso, A., additional, Bullock, A., additional, Alonso, M., additional, de Torres, C., additional, Cruz, O., additional, Pencreach, E., additional, Moussalieh, F. M., additional, Guenot, D., additional, Namer, I., additional, Pollack, I., additional, Jakacki, R., additional, Butterfield, L., additional, Hamilton, R., additional, Panigrahy, A., additional, Potter, D., additional, Connelly, A., additional, Dibridge, S., additional, Whiteside, T., additional, Okada, H., additional, Ahsan, S., additional, Raabe, E., additional, Haffner, M., additional, Warren, K., additional, Quezado, M., additional, Ballester, L., additional, Eberhart, C., additional, Rodriguez, F., additional, Ramachandran, C., additional, Nair, S., additional, Quirrin, K.-W., additional, Khatib, Z., additional, Escalon, E., additional, Melnick, S., additional, Classen, C. F., additional, Hofmann, M., additional, Schmid, I., additional, Simon, T., additional, Maass, E., additional, Russo, A., additional, Fleischhack, G., additional, Becker, M., additional, Hauch, H., additional, Sander, A., additional, Grasso, C., additional, Berlow, N., additional, Liu, L., additional, Davis, L., additional, Huang, E., additional, Woo, P., additional, Tang, Y., additional, Ponnuswami, A., additional, Chen, S., additional, Huang, Y., additional, Hutt-Cabezas, M., additional, Dret, L., additional, Meltzer, P., additional, Mao, H., additional, Abraham, J., additional, Fouladi, M., additional, Svalina, M. N., additional, Wang, N., additional, Hulleman, E., additional, Li, X.-N., additional, Keller, C., additional, Spellman, P. T., additional, Pal, R., additional, Jansen, M. H. A., additional, Sewing, A. C. P., additional, Lagerweij, T., additional, Vuchts, D. J., additional, van Vuurden, D. G., additional, Caretti, V., additional, Wesseling, P., additional, Kaspers, G. J. L., additional, Cohen, K., additional, Pearl, M., additional, Kogiso, M., additional, Zhang, L., additional, Qi, L., additional, Lindsay, H., additional, Lin, F., additional, Berg, S., additional, Muscal, J., additional, Amayiri, N., additional, Tabori, U., additional, Campbel, B., additional, Bakry, D., additional, Aronson, M., additional, Durno, C., additional, Gallinger, S., additional, Malkin, D., additional, Qaddumi, I., additional, Musharbash, A., additional, Swaidan, M., additional, Al-Hussaini, M., additional, Shandilya, S., additional, McCully, C., additional, Murphy, R., additional, Akshintala, S., additional, Cole, D., additional, Macallister, R. P., additional, Cruz, R., additional, Widemann, B., additional, Salloum, R., additional, Smith, A., additional, Glaunert, M., additional, Ramkissoon, A., additional, Peterson, S., additional, Baker, S., additional, Chow, L., additional, Sandgren, J., additional, Pfeifer, S., additional, Popova, S., additional, Alafuzoff, I., additional, de Stahl, T. D., additional, Pietschmann, S., additional, Kerber, M. J., additional, Zwiener, I., additional, Henke, G., additional, Muller, K., additional, Sieow, N. Y.-F., additional, Hoe, R. H. M., additional, Tan, A. M., additional, Chan, M. Y., additional, Soh, S. Y., additional, Burrell, K., additional, Chornenkyy, Y., additional, Remke, M., additional, Golbourn, B., additional, Barzczyk, M., additional, Taylor, M., additional, Rutka, J., additional, Dirks, P., additional, Zadeh, G., additional, Agnihotri, S., additional, Hashizume, R., additional, Ihara, Y., additional, Andor, N., additional, Chen, X., additional, Lerner, R., additional, Huang, X., additional, Tom, M., additional, Solomon, D., additional, Mueller, S., additional, Petritsch, C., additional, Zhang, Z., additional, Gupta, N., additional, Waldman, T., additional, Dujua, A., additional, Co, J., additional, Hernandez, F., additional, Doromal, D., additional, Hegde, M., additional, Wakefield, A., additional, Brawley, V., additional, Grada, Z., additional, Byrd, T., additional, Chow, K., additional, Krebs, S., additional, Heslop, H., additional, Gottschalk, S., additional, Yvon, E., additional, Ahmed, N., additional, Cornilleau, G., additional, Paulsson, J., additional, Andreiuolo, F., additional, Guerrini-Rousseau, L., additional, Geoerger, B., additional, Vassal, G., additional, Ostman, A., additional, Parsons, D. W., additional, Trevino, L. R., additional, Gao, F., additional, Shen, X., additional, Hampton, O., additional, Kosigo, M., additional, Baxter, P. A., additional, Su, J. M., additional, Chintagumpala, M., additional, Dauser, R., additional, Adesina, A., additional, Plon, S. E., additional, Wheeler, D. A., additional, Lau, C. C., additional, Gielen, G., additional, Muehlen, A. z., additional, Kwiecien, R., additional, Wolff, J., additional, Lulla, R. R., additional, Laskowski, J., additional, Goldman, S., additional, Gopalakrishnan, V., additional, Fangusaro, J., additional, Kieran, M., additional, Fontebasso, A., additional, Papillon-Cavanagh, S., additional, Schwartzentruber, J., additional, Nikbakht, H., additional, Gerges, N., additional, Fiset, P.-O., additional, Bechet, D., additional, Faury, D., additional, De Jay, N., additional, Ramkissoon, L., additional, Corcoran, A., additional, Jones, D., additional, Sturm, D., additional, Johann, P., additional, Tomita, T., additional, Nagib, M., additional, Bendel, A., additional, Goumnerova, L., additional, Bowers, D. C., additional, Leonard, J. R., additional, Rubin, J. B., additional, Alden, T., additional, DiPatri, A., additional, Browd, S., additional, Leary, S., additional, Jallo, G., additional, Prados, M. D., additional, Banerjee, A., additional, Carret, A.-S., additional, Ellezam, B., additional, Crevier, L., additional, Klekner, A., additional, Bognar, L., additional, Hauser, P., additional, Garami, M., additional, Myseros, J., additional, Dong, Z., additional, Siegel, P. M., additional, Gump, W., additional, Ayyanar, K., additional, Ragheb, J., additional, Krieger, M., additional, Kiehna, E., additional, Robison, N., additional, Harter, D., additional, Gardner, S., additional, Handler, M., additional, Foreman, N., additional, Brahma, B., additional, MacDonald, T., additional, Malkin, H., additional, Chi, S., additional, Manley, P., additional, Bandopadhayay, P., additional, Greenspan, L., additional, Ligon, A., additional, Albrecht, S., additional, Ligon, K. L., additional, Majewski, J., additional, Jabado, N., additional, Cordero, F., additional, Halvorson, K., additional, Taylor, I., additional, Hutt, M., additional, Weingart, M., additional, Price, A., additional, Kantar, M., additional, Onen, S., additional, Kamer, S., additional, Turhan, T., additional, Kitis, O., additional, Ertan, Y., additional, Cetingul, N., additional, Anacak, Y., additional, Akalin, T., additional, Ersahin, Y., additional, Mason, G., additional, Ho, C., additional, Crozier, F., additional, Vezina, G., additional, Packer, R., additional, Hwang, E., additional, Gilheeney, S., additional, Millard, N., additional, DeBraganca, K., additional, Khakoo, Y., additional, Kramer, K., additional, Wolden, S., additional, Donzelli, M., additional, Fischer, C., additional, Petriccione, M., additional, Dunkel, I., additional, Afzal, S., additional, Fleming, A., additional, Larouche, V., additional, Zelcer, S., additional, Johnston, D. L., additional, Kostova, M., additional, Mpofu, C., additional, Decarie, J.-C., additional, Strother, D., additional, Lafay-Cousin, L., additional, Eisenstat, D., additional, Fryer, C., additional, Hukin, J., additional, Hsu, M., additional, Lasky, J., additional, Moore, T., additional, Liau, L., additional, Davidson, T., additional, Prins, R., additional, Hassal, T., additional, Baugh, J., additional, Kirkendall, J., additional, Doughman, R., additional, Leach, J., additional, Jones, B., additional, Miles, L., additional, Hargrave, D., additional, Jacques, T., additional, Savage, S., additional, Saunders, D., additional, Wallace, R., additional, Flutter, B., additional, Morgenestern, D., additional, Blanco, E., additional, Howe, K., additional, Lowdell, M., additional, Samuel, E., additional, Michalski, A., additional, Anderson, J., additional, Arakawa, Y., additional, Umeda, K., additional, Watanabe, K.-i., additional, Mizowaki, T., additional, Hiraoka, M., additional, Hiramatsu, H., additional, Adachi, S., additional, Kunieda, T., additional, Takagi, Y., additional, Miyamoto, S., additional, Venneti, S., additional, Santi, M., additional, Felicella, M. M., additional, Sullivan, L. M., additional, Dolgalev, I., additional, Martinez, D., additional, Perry, A., additional, Lewis, P. W., additional, Allis, D. C., additional, Thompson, C. B., additional, and Judkins, A. R., additional

Published
2014
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95. New policies to address the global burden of childhood cancers

Author

Sullivan, R, Kowalczyk, J, Agarwal, B, Ladenstein, R, Fitzgerald, E, Barr, R, Steliarova Foucher, E, Magrath, I, Howard, S, Kruger, M, Valsecchi, M, Biondi, A, Grundy, P, Smith, M, Adamson, P, Vassal, G, Pritchard Jones, K, Kowalczyk, JR, Howard, SC, Smith, MA, Pritchard Jones, K., VALSECCHI, MARIA GRAZIA, BIONDI, ANDREA, Sullivan, R, Kowalczyk, J, Agarwal, B, Ladenstein, R, Fitzgerald, E, Barr, R, Steliarova Foucher, E, Magrath, I, Howard, S, Kruger, M, Valsecchi, M, Biondi, A, Grundy, P, Smith, M, Adamson, P, Vassal, G, Pritchard Jones, K, Kowalczyk, JR, Howard, SC, Smith, MA, Pritchard Jones, K., VALSECCHI, MARIA GRAZIA, and BIONDI, ANDREA

Abstract

Childhood cancer is a major global health issue. Every year, almost 100 000 children die from cancer before the age of 15 years, more than 90% of them in resource-limited countries. Here, we review the key policy issues for the delivery of better care, research, and education of professionals and patients. We present a key list of time-limited proposals focusing on change to health systems and research and development. These include sector and system reforms to make care affordable to all, policies to promote growth of civil society around both cancer and Millennium Development Goals, major improvements to public health services (particularly the introduction of national cancer plans), improved career development, and increased remuneration of specialist health-care workers and government support for childhood cancer registries. Research and development proposals focus on sustainable funding, the establishment of more research networks, and clinical research specifically targeted at the needs of low-income and middle-income countries. Finally, we present proposals to address the need for clinical trial innovation, the complex dichotomy of regulations, and the threats to the availability of data for childhood cancers. © 2013 Elsevier Ltd.

Published
2013

96. PEDIATRICS CLINICAL RESEARCH

Author

Antony, R., primary, Zagardo, M., additional, Gujrati, M., additional, Lin, J., additional, Antony, R., additional, Al-Rahawan, M., additional, Broniscer, A., additional, Bhardwaj, R., additional, Hampton, C., additional, Ozols, V., additional, Chakravadhanula, M., additional, Bouffet, E., additional, Hawkins, C., additional, Scheinemann, K., additional, Zelcer, S., additional, Johnston, D., additional, Lafay-Cousin, L., additional, Larouche, V., additional, Jabado, N., additional, Carret, A. S., additional, Hukin, J., additional, Eisenstat, D., additional, Pond, G., additional, Poskitt, K., additional, Wilson, B., additional, Bartels, U., additional, Tabori, U., additional, Dhall, G., additional, Haley, K., additional, Finlay, J., additional, Rushing, T., additional, Sposto, R., additional, Seeger, R., additional, Garvin, J., additional, Rupani, K., additional, Stark, E., additional, Anderson, R., additional, Feldstein, N., additional, Grill, J., additional, Hargrave, D., additional, Massimino, M., additional, Jaspan, T., additional, Varlet, P., additional, Jones, C., additional, Morgan, P., additional, Le Deley, M. C., additional, Azizi, A., additional, Canete, A., additional, Saran, F., additional, Bachir, J., additional, Bubuteishvili-Pacaud, L., additional, Rousseau, R., additional, Vassal, G., additional, Gupta, S., additional, Robinson, N., additional, Dhir, N., additional, Wong, K., additional, Zhou, S., additional, Kumabe, T., additional, Kawaguchi, T., additional, Saito, R., additional, Kanamori, M., additional, Yamashita, Y., additional, Sonoda, Y., additional, Tominaga, T., additional, Miyagawa, T., additional, Nwachukwu, C., additional, Youland, R., additional, Laack, N., additional, Filipek, I., additional, Drogosiewicz, M., additional, Polnik, M. P.-, additional, Swieszkowska, E., additional, Dembowska-Baginska, B., additional, Jurkiewicz, E., additional, Perek, D., additional, Grajkowska, W., additional, Roszkowski, M., additional, Sobol, G., additional, Musiol, K., additional, Wachowiak, J., additional, Kazmierczak, B., additional, Pogorzelski, J. 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Published
2013
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97. The state of research into children with cancer across Europe : new policies for a new decade

Author

Pritchard-Jones, K, Lewison, G, Camporesi, S, Vassal, G, Ladenstein, R, Benoit, Y, Predojevic, Js, Sterba, J, Stary, J, Eckschlager, T, Schroeder, H, Doz, F, Creutzig, U, Klingebiel, T, Kosmidis, Hv, Garami, M, Pieters, R, O'Meara, A, Dini, G, Riccardi, R, Rascon, J, Rageliene, L, Calvagna, V, Czauderna, P, Kowalczyk, Jr, Gil-da-Costa, Mj, Norton, L, Pereira, F, Janic, D, Puskacova, J, Jazbec, J, Canete, A, Hjorth, L, Ljungman, Gustaf, Kutluk, T, Morland, B, Stevens, M, Walker, D, Sullivan, R, Pritchard-Jones, K, Lewison, G, Camporesi, S, Vassal, G, Ladenstein, R, Benoit, Y, Predojevic, Js, Sterba, J, Stary, J, Eckschlager, T, Schroeder, H, Doz, F, Creutzig, U, Klingebiel, T, Kosmidis, Hv, Garami, M, Pieters, R, O'Meara, A, Dini, G, Riccardi, R, Rascon, J, Rageliene, L, Calvagna, V, Czauderna, P, Kowalczyk, Jr, Gil-da-Costa, Mj, Norton, L, Pereira, F, Janic, D, Puskacova, J, Jazbec, J, Canete, A, Hjorth, L, Ljungman, Gustaf, Kutluk, T, Morland, B, Stevens, M, Walker, D, and Sullivan, R

Abstract

Overcoming childhood cancers is critically dependent on the state of research. Understanding how, with whom and what the research community is doing with childhood cancers is essential for ensuring the evidence-based policies at national and European level to support children, their families and researchers. As part of the European Union funded EUROCANCERCOMS project to study and integrate cancer communications across Europe, we have carried out new research into the state of research in childhood cancers. We are very grateful for all the support we have received from colleagues in the European paediatric oncology community, and in particular from Edel Fitzgerald and Samira Essiaf from the SIOP Europe office. This report and the evidence-based policies that arise from it come at a important junction for Europe and its Member States. They provide a timely reminder that research into childhood cancers is critical and needs sustainable long-term support.

Published
2011
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98. Phase II study of gemcitabine combined with oxaliplatin in relapsed or refractory paediatric solid malignancies: An innovative therapy for children with Cancer European Consortium Study.

Author

Geoerger, B, Chisholm, J, Le Deley, Mc, Gentet, Jc, Zwaan, Cm, Dias, N, Jaspan, T, Hugh, K, Couanet, D, Hain, S, Devos, A, Riccardi, Riccardo, Cesare, C, Boos, J, Frappaz, D, Leblond, P, Aerts, I, Vassal, G., Riccardi, Riccardo (ORCID:0000-0001-7515-6622), Geoerger, B, Chisholm, J, Le Deley, Mc, Gentet, Jc, Zwaan, Cm, Dias, N, Jaspan, T, Hugh, K, Couanet, D, Hain, S, Devos, A, Riccardi, Riccardo, Cesare, C, Boos, J, Frappaz, D, Leblond, P, Aerts, I, Vassal, G., and Riccardi, Riccardo (ORCID:0000-0001-7515-6622)

Abstract

AIM: To assess objective response rates after 4 cycles of gemcitabine in combination with oxaliplatin in children and adolescents with relapsed or refractory solid tumours. METHODS: This multicentre, non-randomised Phase II study included five strata: neuroblastoma, osteosarcoma, medulloblastoma and other CNS tumours strata with two-stage Simon designs and a miscellaneous, extra-cranial solid tumour stratum with descriptive design. Eligibility criteria included: age 6 months to 21 years; measurable, relapsed or refractory solid malignancy; no more than one previous salvage therapy. Gemcitabine was administered intravenously at 1000 mg/m(2) over 100 min followed by oxaliplatin at 100mg/m(2) over 120 min on Day 1 of a 14-d cycle. Tumour response was assessed every 4 cycles according to WHO criteria. RESULTS: Ninety-three out of 95 patients enrolled in 25 centres received treatment: 12 neuroblastoma; 12 osteosarcoma; 14 medulloblastoma; 13 other CNS tumours and 42 miscellaneous non-CNS solid tumours. Median age was 11.7 years (range, 1.3-20.8 years). Tumour control (CR+PR+SD) at 4 cycles was obtained in 30/93 evaluable patients (32.3%; 95% confidence interval (CI), 22.9-42.7%), including four PR: 1/12 patients with osteosarcoma, 1/12 with medulloblastoma, 1/12 with rhabdomyosarcoma and 1/4 with other sarcoma. Five out of 12 eligible patients with neuroblastoma experienced stable disease. During a total of 481 treatment cycles (median 4, range 1-24 per patient), the most common treatment-related toxicities were haematologic (leukopenia, neutropenia, thrombocytopenia) and neurological (dysesthesia, paresthesia). CONCLUDING STATEMENT: The gemcitabine-oxaliplatin combination administered in a bi-weekly schedule has acceptable safety profile with limited activity in children with relapsed or refractory solid tumours.

Published
2011

99. Innovative Therapies for Children with Cancer pediatric phase I study of erlotinib in brainstem glioma and relapsing/refractory brain tumors.

Author

Geoerger, B, Hargrave, D, Thomas, F, Ndiaye, A, Frappaz, D, Andreiuolo, F, Varlet, P, Aerts, I, Riccardi, Riccardo, Jaspan, T, Chatelut, E, Le Deley, Mc, Paoletti, X, Saint Rose, C, Leblond, P, Morland, B, Gentet, Jc, Meresse, V, Vassal, G., Riccardi, Riccardo (ORCID:0000-0001-7515-6622), Geoerger, B, Hargrave, D, Thomas, F, Ndiaye, A, Frappaz, D, Andreiuolo, F, Varlet, P, Aerts, I, Riccardi, Riccardo, Jaspan, T, Chatelut, E, Le Deley, Mc, Paoletti, X, Saint Rose, C, Leblond, P, Morland, B, Gentet, Jc, Meresse, V, Vassal, G., and Riccardi, Riccardo (ORCID:0000-0001-7515-6622)

Abstract

This multicenter phase I study aimed to establish the recommended dose (RD) of the epidermal growth factor receptor (EGFR) inhibitor erlotinib, given as monotherapy or with radiotherapy to children with malignant brain tumors. Group 1 included patients with refractory or relapsing brain tumors receiving erlotinib alone, and group 2 included newly diagnosed patients with brainstem gliomas receiving radiotherapy and erlotinib. A conventional 3 + 3 dose escalation and a continual reassessment method, respectively, were utilized in 4 dose levels: 75, 100, 125, and 150 mg/m2 per day. Fifty-one children were enrolled (30 and 21, respectively); 50 received treatment. The RD of erlotinib was 125 mg/m2 per day as monotherapy or in combination with radiotherapy. Overall, 230 adverse events in 44 patients were possibly treatment related (216, grades 1 and 2; 9, grade 3; 1, grade 4; 4, grade 5). Dermatologic and neurologic symptoms were common; intratumoral hemorrhage was confirmed in 3 patients. In group 1, 8 of 29 patients (28%) had stable disease with tumor regression approaching 50% in a malignant glioma and an anaplastic oligoastrocytoma. In group 2, overall survival was 12.0 months. EGFR overexpression by immunohistochemistry was found in 17 of 38 (45%) tumor samples analyzed, with a partial gain of 7p11.2 in 1 glioblastoma; phosphate and tensin homolog loss was frequent in brainstem glioma (15 of 19). Mean (95% CI) apparent clearance and volume of distribution for erlotinib were 4.0 L/h (3.4-4.5 L/h) and 98.6 L (69.8-127.0 L), respectively, and were independent of the dose level; mean half-life was 16.6 hours. Thus, erlotinib 125 mg/m2 per day has an acceptable tolerability profile in pediatric patients with brain tumors and can be combined with radiotherapy

Published
2011

100. The role of the Innovative Therapies for Children with Cancer' (ITCC) European consortium

Author

Zwaan, Cm, Kearns, P, Caron, H, Verschuur, A, Riccardi, Riccardo, Boos, J, Doz, F, Geoerger, B, Morland, B, Vassal, G., Riccardi, Riccardo (ORCID:0000-0001-7515-6622), Zwaan, Cm, Kearns, P, Caron, H, Verschuur, A, Riccardi, Riccardo, Boos, J, Doz, F, Geoerger, B, Morland, B, Vassal, G., and Riccardi, Riccardo (ORCID:0000-0001-7515-6622)

Abstract

Overall survival from childhood malignancies has dramatically improved, with survival rates now reaching over 70%. Nevertheless, some types of childhood cancer remain a difficult challenge, and for those who survive the burden of treatment can be considerable. The current paradigm for new cancer therapies is to increase our knowledge of the molecular basis of carcinogenesis, followed by the development of cancer-cell specific therapies. Historically, drug development was focused on adult cancers, and the potential efficacy in childhood malignancies was not considered. Recently, a European academic consortium was established, namely 'innovative therapies for children with cancer' (ITCC), to address this unmet need. This initiative is focused on the evaluation of novel agents in pediatric cancer pre-clinical models, and early clinical development of promising new drugs. The number of pediatric patients eligible to participate in such trials is limited, and accurate pre-clinical evaluation may provide evidence-based prioritization for clinical development. Until recently, clinical development of new drugs in childhood cancer was restricted by the limited accessibility of such agents. Recent changes in EU legislation oblige pharmaceutical companies to provide pediatric clinical data for all new drugs relevant to children, including anti-cancer drugs. Pediatric consortiums like ITCC have established networks of expertise with the specific aim of evaluating new drugs for the treatment of childhood cancers. Through proper evaluation in collaborative clinical trials we will learn how best to use these new therapeutic approaches and improve the survival rates and reduce toxicity for children with cancer.

Published
2010

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