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2. Treatment at Relapse for Synovial Sarcoma of Children, Adolescents and Young Adults: From the State of Art to Future Clinical Perspectives

Author

Ferrari A, Berlanga P, Gatz SA, Schoot RA, van Noesel MM, Hovsepyan S, Chiaravalli S, Bergamaschi L, Minard-Colin V, Corradini N, Alaggio R, Gasparini P, Brennan B, Casanova M, Pasquali S, and Orbach D

Subjects
synovial sarcoma, relapse, second line therapy, surgery, new agents, tcr-t cell therapy, children, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Abstract

Andrea Ferrari,1 Pablo Berlanga,2 Susanne Andrea Gatz,3 Reineke A Schoot,4 Max M van Noesel,4,5 Shushan Hovsepyan,6 Stefano Chiaravalli,1 Luca Bergamaschi,1 Veronique Minard-Colin,2 Nadege Corradini,7 Rita Alaggio,8 Patrizia Gasparini,9 Bernadette Brennan,10 Michela Casanova,1 Sandro Pasquali,11,12 Daniel Orbach13 1Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; 2Department of Pediatric and Adolescent Oncology, Gustave Roussy Cancer Campus, Université Paris-Saclay, Villejuif, France; 3Cancer Research UK Clinical Trials Unit, Institute of Cancer and Genomic Sciences, University of Birmingham, Birmingham, UK; 4Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands; 5Division Imaging & Cancer, University Medical Center Utrecht, Utrecht, the Netherlands; 6Pediatric Cancer and Blood Disorders Center of Armenia, Yerevan, Armenia; 7Department of Pediatric Hematology and Oncology-IHOPe, Léon Bérard Center, Lyon, France; 8Pathology Department, Ospedale Pediatrico Bambino Gesù IRCCS, Roma, Italy; 9Tumor Genomics Unit, Department of Research, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; 10Pediatric Oncology, Royal Manchester Children’s Hospital, Manchester, UK; 11Molecular Pharmacology Unit, Department of Applied Research and Technological Development, Molecular Pharmacology Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; 12Sarcoma Service, Department of Surgery, Department of Surgery, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; 13SIREDO Oncology Center(Care, Innovation and Research for Children, Adolescents and Young Adults with Cancer), Institut Curie, PSL University, Paris, FranceCorrespondence: Andrea Ferrari, Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Via G. Venezian, Milano, MI, 1 20133, Italy, Tel +39 02 23902588, Fax +39 02 23902648, Email andrea.ferrari@istitutotumori.mi.itAbstract: While the overall prognosis is generally quite satisfactory in children, adolescents and young adults with localised synovial sarcoma at first diagnosis, the outcome remains poor for patients after relapse. Conversely to the front-line standardised treatment options, patients with relapse generally have an individualised approach and to date, there is still a lack of consensus regarding standard treatment approaches. Studies on relapsed synovial sarcoma were able to identify some prognostic variables that influence post-relapse survival, in order to plan risk-adapted salvage protocols. Treatment proposals must consider previous first-line treatments, potential toxicities, and the possibility of achieving an adequate local treatment by new surgery and/or re-irradiation. Effective second-line drug therapies are urgently needed. Notably, experimental treatments such as adoptive engineered TCR-T cell immunotherapy seem promising in adults and are currently under validation also in paediatric patients.Keywords: synovial sarcoma, relapse, second line therapy, surgery, new agents, TCR-T cell therapy, children

Published
2023

4. Pediatric Non-Rhabdomyosarcoma Soft Tissue Sarcomas: Standard of Care and Treatment Recommendations from the European Paediatric Soft Tissue Sarcoma Study Group (EpSSG)

Author

Ferrari A, Brennan B, Casanova M, Corradini N, Berlanga P, Schoot RA, Ramirez-Villar GL, Safwat A, Guillen Burrieza G, Dall'Igna P, Alaggio R, Lyngsie Hjalgrim L, Gatz SA, Orbach D, and van Noesel MM

Subjects
non-rhabdomyosarcoma soft tissue sarcomas, nrsts, epssg, treatment, recommendations, pediatric, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Abstract

Andrea Ferrari,1 Bernadette Brennan,2 Michela Casanova,1 Nadege Corradini,3 Pablo Berlanga,4 Reineke A Schoot,5 Gema L Ramirez-Villar,6 Akmal Safwat,7 Gabriela Guillen Burrieza,8 Patrizia Dall’Igna,9 Rita Alaggio,10 Lisa Lyngsie Hjalgrim,11 Susanne Andrea Gatz,12 Daniel Orbach,13 Max M van Noesel5 1Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy; 2Pediatric Oncology, Royal Manchester Children’s Hospital, Manchester, UK; 3Department of Pediatric Oncology, Institut d’Hematologie et d’Oncologie Pédiatrique/Centre, Léon Bérard, Lyon, France; 4Department of Pediatric and Adolescent Oncology, Gustave-Roussy, Cancer Campus, Université Paris-Saclay, Villejuif, France; 5Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands; 6Pediatric Oncology Unit, Hospital Universitario Virgen del Rocío, Seville, Spain; 7Oncology Department and Danish Center for Particle Therapy, Aarhus University Hospital, Aarhus, Denmark; 8Surgical Oncology and Neonatal Surgery, Pediatric Surgery Department, Hospital Infantil Universitari Vall d’Hebron, Barcelona, Spain; 9Department of Emergencies and Organ Transplantation, Pediatric Surgery, University of Bari, Bari, Italy; 10Pathology Department, Ospedale Pediatrico Bambino Gesù IRCCS, Rome, Italy; 11Department of Pediatrics and Adolescent Medicine, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark; 12Cancer Research UK Clinical Trials Unit, Institute of Cancer and Genomic Sciences, University of Birmingham, Birmingham, UK; 13SIREDO Oncology Center, Institut Curie, PSL University, Paris, FranceCorrespondence: Andrea Ferrari, Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Via G. Venezian, 1, Milan, MI, 20133, Italy, Tel +39 02 23902588, Fax +39 02 23902648, Email andrea.ferrari@istitutotumori.mi.itAbstract: This paper describes the standard of care for patients with non-rhabdomyosarcoma soft tissue sarcomas (NRSTS) and the therapeutic recommendations developed by the European paediatric Soft tissue sarcoma Study Group (EpSSG). NRSTS form a very mixed group of mesenchymal extraskeletal malignancies. Their rarity, heterogeneity, and aggressiveness make the management of children and adolescents with these tumors complex and challenging. The overall cure rate for patients with NRSTS is around 70%, but survival depends on several prognostic variables, such as histotype and tumor grade, extent of disease and stage, tumor size, and tumor site. While surgery remains the mainstay of treatment for most of these tumors, a multimodal therapeutic approach including radiotherapy and chemotherapy is required in many cases. The EpSSG NRSTS 2005 study was the first prospective protocol tailored specifically to NRSTS. Together with the ARST0332 study developed by the North-American Soft Tissue Sarcoma Committee of the Children’s Oncology Group (COG), the EpSSG NRSTS 2005 study currently represents the benchmark for these tumors, establishing risk-adapted standards of care. The EpSSG has developed common treatment recommendations for the large group of adult-type NRSTS (including synovial sarcoma), and specific treatment recommendations for other particular adult-type histologies (ie, alveolar soft-part sarcoma, clear cell sarcoma and dermatofibrosarcoma protuberans); other highly malignant tumors with a biology and clinical behavior differing from those of adult-type NRSTS (ie, rhabdoid tumors and desmoplastic small round cell tumor); and soft tissue tumors of intermediate malignancy (ie desmoid-type fibromatosis, inflammatory myofibroblastic tumors, and infantile fibrosarcoma). New effective drugs are needed for patients whose NRSTS carries the worst prognosis, ie, those with unresectable tumors, metastases at diagnosis, or relapsing disease. Progress in this area relies on our ability to develop international integrated prospective collaborations, both within existing pediatric oncology networks and, importantly, between the communities of specialists treating pediatric and adult sarcoma.Keywords: non-rhabdomyosarcoma soft tissue sarcomas, NRSTS, EpSSG, treatment, recommendations, pediatric

Published
2022

15. Desmoid type fibromatosis in pediatric and young adults from French databases: Clinical characteristics and initial outcome according to age

Author

Bouttefroy, S., primary, Penel, N., additional, Orbach, D., additional, Minard-Colin, V., additional, Le Cesne, A., additional, Blay, JY, additional, Marec-Berard, P., additional, Verité, C., additional, Laurence, V., additional, Piperno-Neumann, S., additional, Defachelles, AS., additional, Bompas, E., additional, Chevreau, C., additional, Duffaud, F., additional, Salas, S., additional, Morelle, M., additional, Jean-Denis, M., additional, Italiano, A., additional, Bonvalot, S., additional, and Corradini, N., additional

Published
2023
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17. Quantitative diffusion-weighted MRI response assessment in rhabdomyosarcoma: an international retrospective study on behalf of the European paediatric Soft tissue sarcoma Study Group Imaging Committee.

Author

Ewijk, R. van, Chatziantoniou, C., Adams, M., Bertolini, P., Bisogno, G., Bouhamama, A., Caro-Dominguez, P., Charon, V., Coma, A., Dandis, R., Devalck, C., Donno, G. De, Ferrari, Andrea, Fiocco, M., Gallego, S., Giraudo, C., Glosli, H., Horst, S.A.J. Ter, Jenney, M., Klein, W.M., Leemans, A., Leseur, J., Mandeville, H.C., McHugh, K., Merks, J.H.M., Minard-Colin, V., Moalla, S., Morosi, C., Orbach, D., Ording Muller, L.S., Pace, E., Paolo, P.L. Di, Perruccio, K., Quaglietta, L., Renard, M., Rijn, R.R. van, Ruggiero, A., Sirvent, S.I., Luca, A. De, Schoot, R.A., Ewijk, R. van, Chatziantoniou, C., Adams, M., Bertolini, P., Bisogno, G., Bouhamama, A., Caro-Dominguez, P., Charon, V., Coma, A., Dandis, R., Devalck, C., Donno, G. De, Ferrari, Andrea, Fiocco, M., Gallego, S., Giraudo, C., Glosli, H., Horst, S.A.J. Ter, Jenney, M., Klein, W.M., Leemans, A., Leseur, J., Mandeville, H.C., McHugh, K., Merks, J.H.M., Minard-Colin, V., Moalla, S., Morosi, C., Orbach, D., Ording Muller, L.S., Pace, E., Paolo, P.L. Di, Perruccio, K., Quaglietta, L., Renard, M., Rijn, R.R. van, Ruggiero, A., Sirvent, S.I., Luca, A. De, and Schoot, R.A.

Abstract

Contains fulltext : 300161.pdf (Publisher’s version ) (Open Access), OBJECTIVE: To investigate the feasibility of diffusion-weighted magnetic resonance imaging (DW-MRI) as a predictive imaging marker after neoadjuvant chemotherapy in patients with rhabdomyosarcoma. MATERIAL AND METHODS: We performed a multicenter retrospective study including pediatric, adolescent and young adult patients with rhabdomyosarcoma, Intergroup Rhabdomyosarcoma Study group III/IV, treated according to the European paediatric Soft tissue sarcoma Study Group (EpSSG) RMS2005 or MTS2008 studies. DW-MRI was performed according to institutional protocols. We performed two-dimensional single-slice tumor delineation. Areas of necrosis or hemorrhage were delineated to be excluded in the primary analysis. Mean, median and 5th and 95th apparent diffusion coefficient (ADC) were extracted. RESULTS: Of 134 included patients, 82 had measurable tumor at diagnosis and response and DW-MRI scans of adequate quality and were included in the analysis. Technical heterogeneity in scan acquisition protocols and scanners was observed. Mean ADC at diagnosis was 1.1 (95% confidence interval [CI]: 1.1-1.2) (all ADC expressed in * 10(-3) mm(2)/s), versus 1.6 (1.5-1.6) at response assessment. The 5th percentile ADC was 0.8 (0.7-0.9) at diagnosis and 1.1 (1.0-1.2) at response. Absolute change in mean ADC after neoadjuvant chemotherapy was 0.4 (0.3-0.5). Exploratory analyses for association between ADC and clinical parameters showed a significant difference in mean ADC at diagnosis for alveolar versus embryonal histology. Landmark analysis at nine weeks after the date of diagnosis showed no significant association (hazard ratio 1.3 [0.6-3.2]) between the mean ADC change and event-free survival. CONCLUSION: A significant change in the 5th percentile and the mean ADC after chemotherapy was observed. Strong heterogeneity was identified in DW-MRI acquisition protocols between centers and in individual patients., 01 november 2023

Published
2023

18. Genetic Tools to Investigate the Consequences of Sex

Author

Würsig, Bernd, Orbach, Dara N, Würsig, B ( Bernd ), Orbach, D N ( Dara N ), Gerber, Livia; https://orcid.org/0000-0002-1247-2262, Krützen, Michael; https://orcid.org/0000-0003-1055-5299, Würsig, Bernd, Orbach, Dara N, Würsig, B ( Bernd ), Orbach, D N ( Dara N ), Gerber, Livia; https://orcid.org/0000-0002-1247-2262, and Krützen, Michael; https://orcid.org/0000-0003-1055-5299

Abstract

The primary purpose of sex is reproduction. However, because not all mating events result in fertilization and only a small number of species provide biparental care to their young, successfully reproducing individuals can rarely be identified from behavioral observations alone. Genetic tools permit reliable identification of an individual’s parents and thus of successfully reproducing individuals, because each parent passes on half of their genetic material to their offspring. In cetaceans, genetic tools are required to identify a female’s already weaned offspring and to detect successfully reproducing males due to the absence of paternal care. To date, relatively few studies have investigated variables linked to reproductive success in this taxon, owed to the difficulty of sampling entire cetacean populations. We summarize currently known factors that are linked to successful reproduction in whales, porpoises, and dolphins, as well as in terrestrial mammals with comparable life histories that give birth to single young.

Published
2023

24. 1512P Pain in patients with desmoid fibromatosis (DF)

Author

Penel, N., primary, Bonvalot, S., additional, Bimbai, A-M., additional, Italiano, A., additional, Orbach, D., additional, Verret, B., additional, Toulmonde, M., additional, Dufresne, A., additional, Bay, J-O., additional, Chaigneau, L., additional, Kurtz, J.E., additional, Bompas, E., additional, Salas, S., additional, Bertucci, F., additional, Guillemet, C., additional, Ryckewaert, T., additional, Thery, J., additional, Le Deley, M-C., additional, Blay, J-Y., additional, and Le Cesne, A., additional

Published
2022
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27. Training guidelines for endovascular stroke intervention: an international multi-society consensus document

Author

Lavine, S. D., Cockroft, K., Hoh, B., Bambakidis, N., Khalessi, A. A., Woo, H., Riina, H., Siddiqui, A., Hirsch, J. A., Chong, W., Rice, H., Wenderoth, J., Mitchell, P., Coulthard, A., Signh, T. J., Phatorous, C., Khangure, M., Klurfan, P., ter Brugge, K., Iancu, D, Gunnarsson, T., Jansen, O., Muto, M., Szikora, I., Pierot, L., Brouwer, P., Gralla, J., Renowden, S., Andersson, T., Fiehler, J., Turjman, F., White, P., Januel, A. C., Spelle, L., Kulcsar, Z., Chapot, R., Biondi, A., Dima, S., Taschner, C., Szajner, M., Krajina, A., Sakai, N., Matsumaru, Y., Yoshimura, S., Ezura, M., Fujinaka, T., Iihara, K., Ishii, A., Higashi, T., Hirohata, M., Hyodo, A., Ito, Y., Kawanishi, M., Kiyosue, H., Kobayashi, E., Kobayashi, S., Kuwayama, N., Matsumoto, Y., Miyachi, S., Murayama, Y., Nagata, I., Nakahara, I., Nemoto, S., Niimi, Y., Oishi, H., Satomi, J., Satow, T., Sugiu, K., Tanaka, M., Terada, T., Yamagami, H., Diaz, O., Lylyk, P., Jayaraman, M. V., Patsalides, A., Gandhi, C. D., Lee, S. K., Abruzzo, T., Albani, B., Ansari, S. A., Arthur, A. S., Baxter, B. W., Bulsara, K. R., Chen, M., Almandoz, J. E. Delgado, Fraser, J. F., Heck, D. V., Hetts, S. W., Hussain, M. S., Klucznik, R. P., Leslie-Mawzi, T. M., Mack, W. J., McTaggart, R. A., Meyers, P. M., Mocco, J., Prestigiacomo, C. J., Pride, G. L., Rasmussen, P. A., Starke, R. M., Sunenshine, P. J., Tarr, R. W., Frei, D. F., Ribo, M., Nogueira, R. G., Zaidat, O. O., Jovin, T., Linfante, I., Yavagal, D., Liebeskind, D., Novakovic, R., Pongpech, S., Rodesch, G., Soderman, M., Taylor, A., Krings, T., Orbach, D., Picard, L., Suh, D. C., and Zhang, H. Q.

Published
2016
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30. Clinical characteristics and clinical evolution of a large cohort of pediatric patients with primary central nervous system (cns) tumors and tropomyosin receptor kinase (trk) fusion.

Author

Lamoureux A.-A., Fisher M., Lemelle L., Pfaff E., Kramm C., De Wilde B., Kazanowska B., Hutter C., Pfister S.M., Sturm D., Jones D., Orbach D., Pierron G., Raskin S., Drilon A., Diamond E., Harada G., Zapotocky M., Ellezam B., Weil A.G., Venne D., Barritault M., Leblond P., Coltin H., Hammad R., Tabori U., Hawkins C., Hansford J.R., Meyran D., Erker C., McFadden K., Sato M., Gottardo N.G., Dholaria H., Noroxe D.S., Goto H., Ziegler D.S., Lin F.Y., Parsons D.W., Lindsay H., Wong T.-T., Liu Y.-L., Wu K.-S., Franson A.F., Hwang E., Aguilar-Bonilla A., Cheng S., Cacciotti C., Massimino M., Schiavello E., Wood P., Hoffman L.M., Cappellano A., Lassaletta A., Van Damme A., Llort A., Gerber N.U., Ceruso M.S., Bendel A.E., Skrypek M., Hamideh D., Mushtaq N., Walter A., Jabado N., Alsahlawi A., Farmer J.-P., Abadi C.C., Mueller S., Mazewski C., Aguilera D., Robison N., O'Halloran K., Abbou S., Berlanga P., Geoerger B., Ora I., Moertel C.L., Razis E.D., Vernadou A., Doz F., Laetsch T.W., Perreault S., Lamoureux A.-A., Fisher M., Lemelle L., Pfaff E., Kramm C., De Wilde B., Kazanowska B., Hutter C., Pfister S.M., Sturm D., Jones D., Orbach D., Pierron G., Raskin S., Drilon A., Diamond E., Harada G., Zapotocky M., Ellezam B., Weil A.G., Venne D., Barritault M., Leblond P., Coltin H., Hammad R., Tabori U., Hawkins C., Hansford J.R., Meyran D., Erker C., McFadden K., Sato M., Gottardo N.G., Dholaria H., Noroxe D.S., Goto H., Ziegler D.S., Lin F.Y., Parsons D.W., Lindsay H., Wong T.-T., Liu Y.-L., Wu K.-S., Franson A.F., Hwang E., Aguilar-Bonilla A., Cheng S., Cacciotti C., Massimino M., Schiavello E., Wood P., Hoffman L.M., Cappellano A., Lassaletta A., Van Damme A., Llort A., Gerber N.U., Ceruso M.S., Bendel A.E., Skrypek M., Hamideh D., Mushtaq N., Walter A., Jabado N., Alsahlawi A., Farmer J.-P., Abadi C.C., Mueller S., Mazewski C., Aguilera D., Robison N., O'Halloran K., Abbou S., Berlanga P., Geoerger B., Ora I., Moertel C.L., Razis E.D., Vernadou A., Doz F., Laetsch T.W., and Perreault S.

Abstract

BACKGROUND: TRK fusions are detected in less than 3% of CNS tumors. Given their rarity, there are limited data on the clinical course of these patients. METHOD(S): We contacted 166 oncology centers worldwide to retrieve data on patients with TRK fusion-driven CNS tumors. Data extracted included demographics, histopathology, NTRK gene fusion, treatment modalities and outcomes. Patients less than 18 years of age at diagnosis were included in this analysis. RESULT(S): Seventy-three pediatric patients with TRK fusion-driven primary CNS tumors were identified. Median age at diagnosis was 2.4 years (range 0.0-17.8) and 60.2 % were male. NTRK2 gene fusions were found in 37 patients (50.7%), NTRK1 and NTRK3 aberrations were detected in 19 (26.0%) and 17 (23.3%), respectively. Tumor types included 38 high-grade gliomas (HGG; 52.1%), 20 low-grade gliomas (LGG; 27.4%), 4 embryonal tumors (5.5%) and 11 others (15.1%). Median follow-up was 46.5 months (range 3-226). During the course of their disease, a total of 62 (84.9%) patients underwent surgery with a treatment intent, 50 (68.5%) patients received chemotherapy, 35 (47.9%) patients received radiation therapy, while 34 (46.6%) patients received NTRK inhibitors (3 as first line treatment). Twenty-four (32.9%) had no progression including 9 LGG (45%) and 9 HGG (23.6%). At last follow-up, only one (5.6%-18 evaluable) patient with LGG died compared to 11 with HGG (35.5%-31 evaluable). For LGG the median progression-free survival (PFS) after the first line of treatment was 17 months (95% CI: 0.0-35.5) and median overall survival (OS) was not reached. For patients with HGG the median PFS was 30 months (95% CI: 11.9-48.1) and median OS was 182 months (95% CI 20.2-343.8). CONCLUSION(S): We report the largest cohort of pediatric patients with TRK fusion-driven primary CNS tumors. These results will help us to better understand clinical evolution and compare outcomes with ongoing clinical trials.

Published
2022

31. HGG-11. Clinical characteristics and clinical evolution of a large cohort of pediatric patients with primary central nervous system (CNS) tumors and tropomyosin receptor kinase (TRK) fusion.

Author

Lamoureux, A-A, Fisher, M, Lemelle, L, Pfaff, E, Kramm, C, De Wilde, B, Kazanowska, B, Hutter, C, Pfister, SM, Sturm, D, Jones, D, Orbach, D, Pierron, G, Raskin, S, Drilon, A, Diamond, E, Harada, G, Zapotocky, M, Ellezam, B, Weil, AG, Venne, D, Barritault, M, Leblond, P, Coltin, H, Hammad, R, Tabori, U, Hawkins, C, Hansford, JR, Meyran, D, Erker, C, McFadden, K, Sato, M, Gottardo, NG, Dholaria, H, Nørøxe, DS, Goto, H, Ziegler, DS, Lin, FY, Parsons, DW, Lindsay, H, Wong, T-T, Liu, Y-L, Wu, K-S, Franson, AF, Hwang, E, Aguilar-Bonilla, A, Cheng, S, Cacciotti, C, Massimino, M, Schiavello, E, Wood, P, Hoffman, LM, Cappellano, A, Lassaletta, A, Van Damme, A, Llort, A, Gerber, NU, Ceruso, MS, Bendel, AE, Skrypek, M, Hamideh, D, Mushtaq, N, Walter, A, Jabado, N, Alsahlawi, A, Farmer, J-P, Abadi, CC, Mueller, S, Mazewski, C, Aguilera, D, Robison, N, O’Halloran, K, Abbou, S, Berlanga, P, Geoerger, B, Øra, I, Moertel, CL, Razis, ED, Vernadou, A, Doz, F, Laetsch, TW, Perreault, S, Lamoureux, A-A, Fisher, M, Lemelle, L, Pfaff, E, Kramm, C, De Wilde, B, Kazanowska, B, Hutter, C, Pfister, SM, Sturm, D, Jones, D, Orbach, D, Pierron, G, Raskin, S, Drilon, A, Diamond, E, Harada, G, Zapotocky, M, Ellezam, B, Weil, AG, Venne, D, Barritault, M, Leblond, P, Coltin, H, Hammad, R, Tabori, U, Hawkins, C, Hansford, JR, Meyran, D, Erker, C, McFadden, K, Sato, M, Gottardo, NG, Dholaria, H, Nørøxe, DS, Goto, H, Ziegler, DS, Lin, FY, Parsons, DW, Lindsay, H, Wong, T-T, Liu, Y-L, Wu, K-S, Franson, AF, Hwang, E, Aguilar-Bonilla, A, Cheng, S, Cacciotti, C, Massimino, M, Schiavello, E, Wood, P, Hoffman, LM, Cappellano, A, Lassaletta, A, Van Damme, A, Llort, A, Gerber, NU, Ceruso, MS, Bendel, AE, Skrypek, M, Hamideh, D, Mushtaq, N, Walter, A, Jabado, N, Alsahlawi, A, Farmer, J-P, Abadi, CC, Mueller, S, Mazewski, C, Aguilera, D, Robison, N, O’Halloran, K, Abbou, S, Berlanga, P, Geoerger, B, Øra, I, Moertel, CL, Razis, ED, Vernadou, A, Doz, F, Laetsch, TW, and Perreault, S

Abstract

BACKGROUND: TRK fusions are detected in less than 3% of CNS tumors. Given their rarity, there are limited data on the clinical course of these patients. METHODS: We contacted 166 oncology centers worldwide to retrieve data on patients with TRK fusion-driven CNS tumors. Data extracted included demographics, histopathology, NTRK gene fusion, treatment modalities and outcomes. Patients less than 18 years of age at diagnosis were included in this analysis. RESULTS: Seventy-three pediatric patients with TRK fusion-driven primary CNS tumors were identified. Median age at diagnosis was 2.4 years (range 0.0–17.8) and 60.2 % were male. NTRK2 gene fusions were found in 37 patients (50.7%), NTRK1 and NTRK3 aberrations were detected in 19 (26.0%) and 17 (23.3%), respectively. Tumor types included 38 high-grade gliomas (HGG; 52.1%), 20 low-grade gliomas (LGG; 27.4%), 4 embryonal tumors (5.5%) and 11 others (15.1%). Median follow-up was 46.5 months (range 3-226). During the course of their disease, a total of 62 (84.9%) patients underwent surgery with a treatment intent, 50 (68.5%) patients received chemotherapy, 35 (47.9%) patients received radiation therapy, while 34 (46.6%) patients received NTRK inhibitors (3 as first line treatment). Twenty-four (32.9%) had no progression including 9 LGG (45%) and 9 HGG (23.6%). At last follow-up, only one (5.6%-18 evaluable) patient with LGG died compared to 11 with HGG (35.5%-31 evaluable). For LGG the median progression-free survival (PFS) after the first line of treatment was 17 months (95% CI: 0.0-35.5) and median overall survival (OS) was not reached. For patients with HGG the median PFS was 30 months (95% CI: 11.9-48.1) and median OS was 182 months (95% CI 20.2-343.8). CONCLUSIONS: We report the largest cohort of pediatric patients with TRK fusion-driven primary CNS tumors. These results will help us to better understand clinical evolution and compare outcomes with ongoing clinical trials.

Published
2022

32. Essential medicines for childhood cancer in Europe: a pan-European, systematic analysis by SIOPE

Author

Maria, O, Eva, B, Pamela R, K, Olga, K, Marko, O, Reineke A, S, Gilles, V, Achini, F, Balduzzi, A, Beck Popovic, M, Beishuizen, A, Bergamaschi, L, Biondi, A, Bourdeaut, F, Braicu, E, Brok, J, Brugières, L, Burke, A, Calaminus, G, Casanova, M, Choucair, M, Cleirec, M, Corbaciouglu, S, Genoveva Correa Llano, M, De Rojas, T, Domínguez Pinilla, N, Elmaraghi, C, Ferrari, A, Fossa, A, Gaspar, N, Herold, N, Karapiperi, K, Karu, M, Kjærsgaar, M, Knörr, F, Koenig, C, Kranjcec, I, Krawczyk, M, Lehmberg, K, Lehrnbecher, T, Lunesink, M, Massano, D, Matijasic, N, Merks, H, Metzler, M, Michalski, A, Minkov, M, Morland, B, Niktoreh, N, Oltenau, E, Orbach, D, Owens, C, Papachristidou, S, Pasqualini, C, Pavlovic, M, Perez Albert, P, Poyer, F, Radulovic, I, Reinhardt, D, Rebelo, J, Roser, E, Russo, I, Scheinemann, K, Schindera, C, Schrappe, M, Sehested, A, Sehouli, J, Spreafico, F, J Strauss, S, Stutterheim, J, Svojgr, K, Tzotzola, V, Van Ewijk, R, Verschuur, A, Vora, A, Woessmann, W, Zajac-Spychala, O, Zwaan, M, Otth, Maria, Brack, Eva, Kearns, Pamela R, Kozhaeva, Olga, Ocokoljic, Marko, Schoot, Reineke A, Vassal, Gilles, Federica Achini, Adriana Balduzzi, Maja Beck Popovic, Auke Beishuizen, Luca Bergamaschi, Andrea Biondi, Franck Bourdeaut, Elena Braicu, Jesper Brok, Laurence Brugières, Amos Burke, Gabriele Calaminus, Michela Casanova, Marie-Louise Choucair, Morgane Cleirec, Selim Corbaciouglu, Maria Genoveva Correa Llano, Teresa De Rojas, Nerea Domínguez Pinilla, Caroline Elmaraghi, Andrea Ferrari, Alexander Fossa, Nathalie Gaspar, Nikolas Herold, Kyriaki Karapiperi, Maarja Karu, Mimi Kjærsgaar, Fabian Knörr, Christa Koenig, Izabela Kranjcec, Malgorzata Krawczyk, Kai Lehmberg, Thomas Lehrnbecher, Maaike Lunesink, Davide Massano, Nuša Matijasic, Hans Merks, Markus Metzler, Anthony Michalski, Milen Minkov, Bruce Morland, Naghmeh Niktoreh, Elena Oltenau, Daniel Orbach, Cormac Owens, Smaragda Papachristidou, Claudia Pasqualini, Maja Pavlovic, Paula Perez Albert, Fiona Poyer, Ivana Radulovic, Dirk Reinhardt, Joana Rebelo, Eva Roser, Ida Russo, Katrin Scheinemann, Christina Schindera, Martin Schrappe, Astrid Sehested, Jalid Sehouli, Filippo Spreafico, Sandra J Strauss, Janine Stutterheim, Karel Svojgr, Vasiliki Tzotzola, Roelof Van Ewijk, Arnauld Verschuur, Ajay Vora, Willi Woessmann, Olga Zajac-Spychala, Michel Zwaan, Maria, O, Eva, B, Pamela R, K, Olga, K, Marko, O, Reineke A, S, Gilles, V, Achini, F, Balduzzi, A, Beck Popovic, M, Beishuizen, A, Bergamaschi, L, Biondi, A, Bourdeaut, F, Braicu, E, Brok, J, Brugières, L, Burke, A, Calaminus, G, Casanova, M, Choucair, M, Cleirec, M, Corbaciouglu, S, Genoveva Correa Llano, M, De Rojas, T, Domínguez Pinilla, N, Elmaraghi, C, Ferrari, A, Fossa, A, Gaspar, N, Herold, N, Karapiperi, K, Karu, M, Kjærsgaar, M, Knörr, F, Koenig, C, Kranjcec, I, Krawczyk, M, Lehmberg, K, Lehrnbecher, T, Lunesink, M, Massano, D, Matijasic, N, Merks, H, Metzler, M, Michalski, A, Minkov, M, Morland, B, Niktoreh, N, Oltenau, E, Orbach, D, Owens, C, Papachristidou, S, Pasqualini, C, Pavlovic, M, Perez Albert, P, Poyer, F, Radulovic, I, Reinhardt, D, Rebelo, J, Roser, E, Russo, I, Scheinemann, K, Schindera, C, Schrappe, M, Sehested, A, Sehouli, J, Spreafico, F, J Strauss, S, Stutterheim, J, Svojgr, K, Tzotzola, V, Van Ewijk, R, Verschuur, A, Vora, A, Woessmann, W, Zajac-Spychala, O, Zwaan, M, Otth, Maria, Brack, Eva, Kearns, Pamela R, Kozhaeva, Olga, Ocokoljic, Marko, Schoot, Reineke A, Vassal, Gilles, Federica Achini, Adriana Balduzzi, Maja Beck Popovic, Auke Beishuizen, Luca Bergamaschi, Andrea Biondi, Franck Bourdeaut, Elena Braicu, Jesper Brok, Laurence Brugières, Amos Burke, Gabriele Calaminus, Michela Casanova, Marie-Louise Choucair, Morgane Cleirec, Selim Corbaciouglu, Maria Genoveva Correa Llano, Teresa De Rojas, Nerea Domínguez Pinilla, Caroline Elmaraghi, Andrea Ferrari, Alexander Fossa, Nathalie Gaspar, Nikolas Herold, Kyriaki Karapiperi, Maarja Karu, Mimi Kjærsgaar, Fabian Knörr, Christa Koenig, Izabela Kranjcec, Malgorzata Krawczyk, Kai Lehmberg, Thomas Lehrnbecher, Maaike Lunesink, Davide Massano, Nuša Matijasic, Hans Merks, Markus Metzler, Anthony Michalski, Milen Minkov, Bruce Morland, Naghmeh Niktoreh, Elena Oltenau, Daniel Orbach, Cormac Owens, Smaragda Papachristidou, Claudia Pasqualini, Maja Pavlovic, Paula Perez Albert, Fiona Poyer, Ivana Radulovic, Dirk Reinhardt, Joana Rebelo, Eva Roser, Ida Russo, Katrin Scheinemann, Christina Schindera, Martin Schrappe, Astrid Sehested, Jalid Sehouli, Filippo Spreafico, Sandra J Strauss, Janine Stutterheim, Karel Svojgr, Vasiliki Tzotzola, Roelof Van Ewijk, Arnauld Verschuur, Ajay Vora, Willi Woessmann, Olga Zajac-Spychala, and Michel Zwaan

Abstract

Background: Shortages and unequal access to anticancer medicines for children and adolescents are a reality in Europe. The aim of the European Society for Paediatric Oncology (SIOPE) Essential Anticancer Medicines Project was to provide a list of anticancer medicines that are considered essential in the treatment of paediatric cancers to help ensure their continuous access to all children and adolescents with cancer across Europe. Methods: This pan-European project, done between Jan 20, 2020, and Feb 18, 2022, was designed to be a systematic collection and review of treatment protocols and strategies that are used to treat childhood cancer in Europe. We formed 16 working groups on the basis of paediatric cancer types, and which were based on the existing SIOPE Clinical Trial Groups. Workings groups consisted of representatives from the SIOPE Clinical Trial Groups, Young SIOPE members, and senior paediatric oncology experts. Each group collected existing treatment protocols that are used to treat the respective cancer types in Europe. Medicines from the standard group of each protocol were extracted. For medicines not on the WHO Essential Medicines List for children (EMLc) 2017, working groups did a literature search to determine whether the medicines should be defined as essential, promising, or neither essential nor promising. Each group provided an individual summary, and all medicines that were considered essential by at least one group were combined in a joint list. Findings: The working groups identified 73 treatment protocols used in Europe and defined 66 medicines as essential. For several newer medicines, such as kinase inhibitors or tisagenlecleucel, the supporting evidence was insufficient to consider them essential, so these medicines were defined as promising. 25 medicines were considered promising by at least one working group. 22 (33%) of the 66 essential and none of the promising medicines were included in the WHO EMLc 2017. The WHO EMLc 2021 included

Published
2022

35. 1522MO Hormonal contraception and pregnancy and risk of progression or relapse in desmoid-type fibromatosis (DF)

Author

Debaudringhien, M., primary, Blay, J-Y., additional, Bimbai, A.M., additional, Bonvalot, S., additional, Italiano, A., additional, Rousset-Jablonski, C., additional, Corradini, N., additional, Piperno-Neumann, S., additional, Chevreau, C.M., additional, Kurtz, J.E., additional, Guillemet, C., additional, Bompas, E., additional, Collard, O., additional, Salas, S., additional, Le Cesne, A., additional, Orbach, D., additional, Thery, J., additional, Deley, M-C. Le, additional, Mir, O., additional, and Penel, N., additional

Published
2021
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36. PD-0923 Increasing access to highly specialized radiation treatments: the example of pediatric brachytherapy

Author

Chargari, C., primary, Haie-Meder, C., additional, Espenel, S., additional, Ben-Arush, M., additional, Bolle, S., additional, Borjesson, A., additional, Cesen, M., additional, Costa Lago, A., additional, Desfachelles, A., additional, De Moerloose, B., additional, Valck, D., additional, Dos Reis Farinha, N., additional, Francotte, N., additional, Glosli, H., additional, Guillen Burrieza, G., additional, Helfre, S., additional, Irtan, S., additional, Kattamis, A., additional, Lacerda, A., additional, Levy, A., additional, Lyngsie, L., additional, Mansuy, L., additional, Mascard, E., additional, Mlynarski, W., additional, Orbach, D., additional, Owens, C., additional, Philippe-Chomette, P., additional, Peek, A., additional, Pizer, B., additional, Pluchart, C., additional, Rognlien, A.G., additional, Rome, A., additional, Sarnacki, S., additional, Safwat, A., additional, Schiavetti, A., additional, Serre, J., additional, Verite, C., additional, Von der Weid, N., additional, Wendtland, P., additional, Wysocki, M., additional, Valteau-Couanet, D., additional, Deutsch, E., additional, Minard-Colin, V., additional, Martelli, H., additional, and Guérin, F., additional

Published
2021
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40. Malformations, Genetic Abnormalities, and Wilms Tumor

Author

Dumoucel, S., Gauthier-Villars, M., Stoppa-Lyonnet, D., Parisot, P., Brisse, H., Philippe-Chomette, P., Sarnacki, S., Boccon-Gibod, L., Rossignol, S., Baumann, C., Aerts, I., Bourdeaut, F., Doz, F., Orbach, D., Pacquement, H., Michon, J., and Schleiermacher, G.

Published
2014
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43. Surgical Management of Ovarian Mature Teratoma in Children: Lessons From the French Pediatric Surgery Oncology Group (FREPSO)

Author

Delehaye, F., Orbach, D., Cheikhelard, A., Rouger, J., J Parienti, J., Faure-Conter, C., Hameury, F., Dijoud, F., Aubry, E., Wacrenier, A., Habonimana, E., Joseph, S., Alliot, H., Scalabre, A., Duchesne, C., Chaussy, Y., Poidevin, G., C Rousselet-Chapeau, M., Elodie Haraux, Pommepuy, I., Ballouhey, Q., Lavrand, F., Matthieu Peycelon, Laquinto, M., Irtan, S., Dariel, A., Borrione, C., Galmiche, L., Sarnacki, S., Rod, J., and DESSAIVRE, Louise

Subjects
[SDV] Life Sciences [q-bio]
Published
2020

46. Fusion status in patients with lymph node‐positive (N1) alveolar rhabdomyosarcoma is a powerful predictor of prognosis: Experience of the European Paediatric Soft Tissue Sarcoma Study Group (EpSSG)

Author

Gallego, S, Zanetti, I, Orbach, D, Ranchère, D, Shipley, J, Zin, A, Bergeron, C, DE SALVO, GIAN LUCA, Chisholm, J, Ferrari, A, Jenney, M, Mandeville, Hc, Rogers, T, Merks, Jhm, Mudry, P, Glosli, H, Milano, Gm, Ferman, S, Bisogno, G, European Paediatric Soft Tissue Sarcoma Study Group (EpSSG), Paediatric Oncology, and CCA - Imaging and biomarkers

Subjects
Adult, Male, 0301 basic medicine, Oncology, Cancer Research, medicine.medical_specialty, alveolar rhabdomyosarcoma, Adolescent, Pediatrics, Disease-Free Survival, lymph node involvement, Young Adult, 03 medical and health sciences, 0302 clinical medicine, Maintenance therapy, Risk Factors, Internal medicine, Antineoplastic Combined Chemotherapy Protocols, medicine, Humans, Child, Rhabdomyosarcoma, Lymph node, Survival rate, Rhabdomyosarcoma, Alveolar, Neoplasm Staging, Forkhead Box Protein O1, business.industry, Soft tissue sarcoma, prognostic factors, Infant, Induction chemotherapy, Cancer, Prognosis, medicine.disease, 030104 developmental biology, medicine.anatomical_structure, Child, Preschool, Lymphatic Metastasis, 030220 oncology & carcinogenesis, Alveolar rhabdomyosarcoma, Female, paired box (PAX)-forkhead box protein O1 (FOXO1) fusion, rhabdomyosarcoma, Lymph Nodes, Neoplasm Recurrence, Local, business
Abstract

Background Alveolar rhabdomyosarcoma (aRMS) with lymph node involvement (N1 classification) accounts for up to 10% of all cases of RMS. The prognosis is poor, and is comparable to that of distant metastatic disease. In the European Paediatric Soft Tissue Sarcoma Study Group (EpSSG) RMS2005 protocol, patients with a histologic diagnosis of aRMS/N1 received intensified chemotherapy with systematic locoregional treatment. Methods Patients with aRMS/N1 were enrolled prospectively after primary surgery/biopsy and fusion status was assessed in tumor samples. All patients received 9 cycles of induction chemotherapy and 6 months of maintenance therapy. Local treatment included radiotherapy to the primary site and lymph nodes with or without secondary surgical resection. Results A total of 103 patients were enrolled. The clinical characteristics of the patients were predominantly unfavorable: 90% had macroscopic residual disease after initial surgery/biopsy, 63% had locally invasive tumors, 77% had a tumor measuring >5 cm, and 81% had disease at unfavorable sites. Fusion genes involving forkhead box protein O1 (FOXO1) were detected in 56 of 84 patients. Events occurred in 52 patients: 43 developed disease recurrence, 7 had disease that was refractory to treatment, and 2 patients developed second neoplasms. On univariate analysis, unfavorable disease site, tumor invasiveness, Intergroup Rhabdomyosarcoma Study group III, and fusion-positive status correlated with worse prognosis. The 5-year event-free survival rate of patients with fusion-positive tumors was 43% compared with 74% in patients with fusion-negative tumors (P = .01). On multivariate analysis, fusion positivity and tumor invasiveness proved to be unfavorable prognostic markers. Conclusions Fusion status and tumor invasiveness appear to have a strong impact on prognosis in patients with aRMS/N1. Fusion status will be used to stratify these patients in the next EpSSG RMS study, and treatment will be intensified in patients with fusion-positive tumors. Cancer 2018. © 2018 American Cancer Society.

Published
2018

47. Erratum to: Training guidelines for endovascular stroke intervention: an international multi-society consensus document

Author

Lavine, S. D., Cockroft, K., Hoh, B., Bambakidis, N., Khalessi, A. A., Woo, H., Riina, H., Siddiqui, A., Hirsch, J. A., Chong, W., Rice, H., Wenderoth, J., Mitchell, P., Coulthard, A., Signh, T. J., Phatouros, C., Khangure, M., Klurfan, P., ter Brugge, K., Iancu, D, Gunnarsson, T., Jansen, O., Muto, M., Szikora, I., Pierot, L., Brouwer, P., Gralla, J., Renowden, S., Andersson, T., Fiehler, J., Turjman, F., White, P., Januel, A. C., Spelle, L., Kulcsar, Z., Chapot, R., Biondi, A., Dima, S., Taschner, C., Szajner, M., Krajina, A., Sakai, N., Matsumaru, Y., Yoshimura, S., Ezura, M., Fujinaka, T., Iihara, K., Ishii, A., Higashi, T., Hirohata, M., Hyodo, A., Ito, Y., Kawanishi, M., Kiyosue, H., Kobayashi, E., Kobayashi, S., Kuwayama, N., Matsumoto, Y., Miyachi, S., Murayama, Y., Nagata, I., Nakahara, I., Nemoto, S., Niimi, Y., Oishi, H., Satomi, J., Satow, T., Sugiu, K., Tanaka, M., Terada, T., Yamagami, H., Diaz, O., Lylyk, P., Jayaraman, M. V., Patsalides, A., Gandhi, C. D., Lee, S. K., Abruzzo, T., Albani, B., Ansari, S. A., Arthur, A. S., Baxter, B. W., Bulsara, K. R., Chen, M., Delgado Almandoz, J. E., Fraser, J. F., Heck, D. V., Hetts, S.W., Hussain, M. S., Klucznik, R. P., Leslie-Mawzi, T. M., Mack, W. J., McTaggart, R. A., Meyers, P. M., Mocco, J., Prestigiacomo, C. J., Pride, G. L., Rasmussen, P. A., Starke, R. M., Sunenshine, P. J., Tarr, R. W., Frei, D. F., Ribo, M., Nogueira, R. G., Zaidat, O. O., Jovin, T., Linfante, I., Yavagal, D., Liebeskind, D., Novakovic, R., Pongpech, S., Rodesch, G., Soderman, M., Taylor, A., Krings, T., Orbach, D., Picard, L., Suh, D. C., and Zhang, H. Q.

Published
2017
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50. 1650P Desmoid type fibromatosis in patients

Author

Bouttefroy, S., primary, Penel, N., additional, Minard-Colin, V., additional, Orbach, D., additional, Le Cesne, A., additional, Blay, J-Y., additional, Marec Berard, P., additional, Verité, C., additional, Laurence, V., additional, Piperno-Neumann, S., additional, Defachelles, A-S., additional, Bompas, E., additional, Chevreau, C.M., additional, Duffaud, F., additional, Salas, S., additional, Morelle, M., additional, Jean Denis, M., additional, Italiano, A., additional, Bonvalot, S., additional, and Corradini, N., additional

Published
2020
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