1,200 results on '"Gelderblom, H."'
Search Results
2. Health-related quality of life in patients with gastrointestinal stromal tumor: data from a real-world cohort compared with a normative population
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van de Wal, D., den Hollander, D., Desar, I.M.E., Gelderblom, H., Oosten, A.W., Reyners, A.K.L., Steeghs, N., van der Graaf, W.T.A., and Husson, O.
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- 2024
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3. Results from the UNITED study: a multicenter study validating the prognostic effect of the tumor–stroma ratio in colon cancer
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Petrushevska, Gordana, Bogdanovska, Magdalena, Zdravkoski, Panche, Antovic, Svetozar, Dzambaz, Darko, Karagjozov, Panche, de Cuba, Erienne M.V., Beverdam, Frédérique, Jansen, Jan, Vermaas, Maarten, Gašljević, Gorana, Kjær-Frifeldt, Sanne, Lindebjerg, Jan, Strous, Maud, Vogelaar, Jeroen F., Bulkmans, Nicole W.J., van Baarlen, Joop, Mekenkamp, Leonie, Hoekstra, Ronald, Sie, Mark, Cuatrecasas, Miriam, Simonetti, Sara, Rodrigo, María Teresa, Sanz, Iván Archilla, Guerrero Pineda, Jose, Leeuwis-Fedorovich, Natalja E., Talsma, Koen A., Souza da Silva, Ricella M., Lacle, Miangela M., Koopman, Miriam, Dekker, Jan Willem T., van Tilburg, Arjan, Nuciforo, Paolo, Villalobos Alberú, Xenia, Landolfi, Stefania, Zucchiatti, Adriana, Witteveen, Emma, Bordbar, Arad, Hendriks, Mathijs P., Arensman, René, Natu, Shonali, Maka, Noori, Mesker, Wilma E., Tollenaar, Rob A.E.M., Polack, Meaghan, Smit, Marloes A., van Pelt, Gabi W., Putter, Hein, Meershoek-Kleinenbarg, Elma, Roodvoets, Annet G.H., Crobach, Augustinus S.L.P., Gelderblom, Hans, Fontes e Sousa, Mário, Borralho Nunes, Paula, Cruz, João, Raimundo, Ana, Silva, Nelson, Brito, Maria J., Terpstra, Valeska, Zakhartseva, L.M., Al Dieri, Raed, Fléjou, Jean-François, Feakins, Roger, Dequeker, Els, Vink, Geraldine R., van Krieken, J.Han J.M., Polack, M., Smit, M.A., van Pelt, G.W., Roodvoets, A.G.H., Meershoek-Klein Kranenbarg, E., Putter, H., Gelderblom, H., Crobach, A.S.L.P., Terpstra, V., Petrushevska, G., Gašljević, G., Kjær-Frifeldt, S., de Cuba, E.M.V., Bulkmans, N.W.J., Vink, G.R., Al Dieri, R., Tollenaar, R.A.E.M., van Krieken, J.H.J.M., and Mesker, W.E.
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- 2024
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4. Efficacy of immune checkpoint inhibitors in alveolar soft-part sarcoma: results from a retrospective worldwide registry
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Hindi, N., Razak, A., Rosenbaum, E., Jonczak, E., Hamacher, R., Rutkowski, P., Bhadri, V.A., Skryd, A., Brahmi, M., Alshibany, A., Jagodzinska-Mucha, P., Bauer, S., Connolly, E., Gelderblom, H., Boye, K., Henon, C., Bae, S., Bogefors, K., Vincenzi, B., Martinez-Trufero, J., Lopez-Martin, J.A., Redondo, A., Valverde, C., Blay, J.-Y., Moura, D.S., Gutierrez, A., Tap, W., and Martin-Broto, J.
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- 2023
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5. Synovial sarcoma: characteristics, challenges, and evolving therapeutic strategies
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Blay, J.-Y., von Mehren, M., Jones, R.L., Martin-Broto, J., Stacchiotti, S., Bauer, S., Gelderblom, H., Orbach, D., Hindi, N., Dei Tos, A., and Nathenson, M.
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- 2023
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6. Sex differences in patients with gastrointestinal stromal tumours: do they exist and does it affect survival?
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IJzerman, N.S., van Werkhoven, E., Mohammadi, M., Hollander, D. den, Bleckman, R.F., Reyners, A.K.L., Desar, I.M.E., Gelderblom, H., Grünhagen, D.J., Mathijssen, R.H.J., Steeghs, N., and van der Graaf, W.T.A.
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- 2022
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7. Systemic treatment of advanced clear cell sarcoma: results from a retrospective international series from the World Sarcoma Network
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Smrke, A., Frezza, A.M., Giani, C., Somaiah, N., Brahmi, M., Czarnecka, A.M., Rutkowski, P., Van der Graaf, W., Baldi, G.G., Connolly, E., Duffaud, F., Huang, P.H., Gelderblom, H., Bhadri, V., Grimison, P., Mahar, A., Stacchiotti, S., and Jones, R.L.
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- 2022
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8. Results from the UNITED study: a multicenter study validating the prognostic effect of the tumor–stroma ratio in colon cancer
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Polack, M., primary, Smit, M.A., additional, van Pelt, G.W., additional, Roodvoets, A.G.H., additional, Meershoek-Klein Kranenbarg, E., additional, Putter, H., additional, Gelderblom, H., additional, Crobach, A.S.L.P., additional, Terpstra, V., additional, Petrushevska, G., additional, Gašljević, G., additional, Kjær-Frifeldt, S., additional, de Cuba, E.M.V., additional, Bulkmans, N.W.J., additional, Vink, G.R., additional, Al Dieri, R., additional, Tollenaar, R.A.E.M., additional, van Krieken, J.H.J.M., additional, Mesker, W.E., additional, Petrushevska, Gordana, additional, Bogdanovska, Magdalena, additional, Zdravkoski, Panche, additional, Antovic, Svetozar, additional, Dzambaz, Darko, additional, Karagjozov, Panche, additional, de Cuba, Erienne M.V., additional, Beverdam, Frédérique, additional, Jansen, Jan, additional, Vermaas, Maarten, additional, Gašljević, Gorana, additional, Kjær-Frifeldt, Sanne, additional, Lindebjerg, Jan, additional, Strous, Maud, additional, Vogelaar, Jeroen F., additional, Bulkmans, Nicole W.J., additional, van Baarlen, Joop, additional, Mekenkamp, Leonie, additional, Hoekstra, Ronald, additional, Sie, Mark, additional, Cuatrecasas, Miriam, additional, Simonetti, Sara, additional, Rodrigo, María Teresa, additional, Sanz, Iván Archilla, additional, Guerrero Pineda, Jose, additional, Leeuwis-Fedorovich, Natalja E., additional, Talsma, Koen A., additional, Souza da Silva, Ricella M., additional, Lacle, Miangela M., additional, Koopman, Miriam, additional, Dekker, Jan Willem T., additional, van Tilburg, Arjan, additional, Nuciforo, Paolo, additional, Villalobos Alberú, Xenia, additional, Landolfi, Stefania, additional, Zucchiatti, Adriana, additional, Witteveen, Emma, additional, Bordbar, Arad, additional, Hendriks, Mathijs P., additional, Arensman, René, additional, Natu, Shonali, additional, Maka, Noori, additional, Mesker, Wilma E., additional, Tollenaar, Rob A.E.M., additional, Polack, Meaghan, additional, Smit, Marloes A., additional, van Pelt, Gabi W., additional, Putter, Hein, additional, Meershoek-Kleinenbarg, Elma, additional, Roodvoets, Annet G.H., additional, Crobach, Augustinus S.L.P., additional, Gelderblom, Hans, additional, Fontes e Sousa, Mário, additional, Borralho Nunes, Paula, additional, Cruz, João, additional, Raimundo, Ana, additional, Silva, Nelson, additional, Brito, Maria J., additional, Terpstra, Valeska, additional, Zakhartseva, L.M., additional, Al Dieri, Raed, additional, Fléjou, Jean-François, additional, Feakins, Roger, additional, Dequeker, Els, additional, Vink, Geraldine R., additional, and van Krieken, J.Han J.M., additional
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- 2024
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9. Priorities and preferences of advanced soft tissue sarcoma patients starting palliative chemotherapy: baseline results from the HOLISTIC study
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Younger, E., Jones, R.L., den Hollander, D., Soomers, V.L.M.N., Desar, I.M.E., Benson, C., Young, R.J., Oosten, A.W., de Haan, J.J., Miah, A., Zaidi, S., Gelderblom, H., Steeghs, N., Husson, O., and van der Graaf, W.T.A.
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- 2021
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10. Epithelioid hemangioendothelioma, an ultra-rare cancer: a consensus paper from the community of experts
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Stacchiotti, S., Miah, A.B., Frezza, A.M., Messiou, C., Morosi, C., Caraceni, A., Antonescu, C.R., Bajpai, J., Baldini, E., Bauer, S., Biagini, R., Bielack, S., Blay, J.Y., Bonvalot, S., Boukovinas, I., Bovee, J.V.M.G., Boye, K., Brodowicz, T., Callegaro, D., De Alava, E., Deoras-Sutliff, M., Dufresne, A., Eriksson, M., Errani, C., Fedenko, A., Ferraresi, V., Ferrari, A., Fletcher, C.D.M., Garcia del Muro, X., Gelderblom, H., Gladdy, R.A., Gouin, F., Grignani, G., Gutkovich, J., Haas, R., Hindi, N., Hohenberger, P., Huang, P., Joensuu, H., Jones, R.L., Jungels, C., Kasper, B., Kawai, A., Le Cesne, A., Le Grange, F., Leithner, A., Leonard, H., Lopez Pousa, A., Martin Broto, J., Merimsky, O., Merriam, P., Miceli, R., Mir, O., Molinari, M., Montemurro, M., Oldani, G., Palmerini, E., Pantaleo, M.A., Patel, S., Piperno-Neumann, S., Raut, C.P., Ravi, V., Razak, A.R.A., Reichardt, P., Rubin, B.P., Rutkowski, P., Safwat, A.A., Sangalli, C., Sapisochin, G., Sbaraglia, M., Scheipl, S., Schöffski, P., Strauss, D., Strauss, S.J., Sundby Hall, K., Tap, W.D., Trama, A., Tweddle, A., van der Graaf, W.T.A., Van De Sande, M.A.J., Van Houdt, W., van Oortmerssen, G., Wagner, A.J., Wartenberg, M., Wood, J., Zaffaroni, N., Zimmermann, C., Casali, P.G., Dei Tos, A.P., and Gronchi, A.
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- 2021
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11. Final analysis of the randomized trial on imatinib as an adjuvant in localized gastrointestinal stromal tumors (GIST) from the EORTC Soft Tissue and Bone Sarcoma Group (STBSG), the Australasian Gastro-Intestinal Trials Group (AGITG), UNICANCER, French Sarcoma Group (FSG), Italian Sarcoma Group (ISG), and Spanish Group for Research on Sarcomas (GEIS)☆
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Casali, P.G., Le Cesne, A., Velasco, A.P., Kotasek, D., Rutkowski, P., Hohenberger, P., Fumagalli, E., Judson, I.R., Italiano, A., Gelderblom, H., Penel, N., Hartmann, J.T., Duffaud, F., Goldstein, D., Martin-Broto, J., Gronchi, A., Wardelmann, E., Marréaud, S., Zalcberg, J.R., Litière, S., and Blay, J.-Y.
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- 2021
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12. Gastrointestinal stromal tumours: ESMO–EURACAN–GENTURIS Clinical Practice Guidelines for diagnosis, treatment and follow-up
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Casali, P.G., Blay, J.Y., Abecassis, N., Bajpai, J., Bauer, S., Biagini, R., Bielack, S., Bonvalot, S., Boukovinas, I., Bovee, J.V.M.G., Boye, K., Brodowicz, T., Buonadonna, A., De Álava, E., Dei Tos, A.P., Del Muro, X.G., Dufresne, A., Eriksson, M., Fedenko, A., Ferraresi, V., Ferrari, A., Frezza, A.M., Gasperoni, S., Gelderblom, H., Gouin, F., Grignani, G., Haas, R., Hassan, A.B., Hindi, N., Hohenberger, P., Joensuu, H., Jones, R.L., Jungels, C., Jutte, P., Kasper, B., Kawai, A., Kopeckova, K., Krákorová, D.A., Le Cesne, A., Le Grange, F., Legius, E., Leithner, A., Lopez-Pousa, A., Martin-Broto, J., Merimsky, O., Messiou, C., Miah, A.B., Mir, O., Montemurro, M., Morosi, C., Palmerini, E., Pantaleo, M.A., Piana, R., Piperno-Neumann, S., Reichardt, P., Rutkowski, P., Safwat, A.A., Sangalli, C., Sbaraglia, M., Scheipl, S., Schöffski, P., Sleijfer, S., Strauss, D., Strauss, S.J., Hall, K Sundby, Trama, A., Unk, M., van de Sande, M.A.J., van der Graaf, W.T.A., van Houdt, W.J., Frebourg, T., Gronchi, A., and Stacchiotti, S.
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- 2021
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13. 58MO Evaluation of three pexidartinib doses in a global phase IV clinical study of patients with tenosynovial giant cell tumor (TGCT) who chose to continue treatment
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Stacchiotti, S, Wagner, AJ, Garcia, ICC, Cesari, M, Gordon, M, Lin, C-C, Papai, Z, Ryan, CW, Tap, WD, Trent, JC, Gelderblom, H, Grimison, P, Pousa, AL, Van Tine, BA, Rubinacci, M, Tecson, K, Wooddell, M, Desai, J, Stacchiotti, S, Wagner, AJ, Garcia, ICC, Cesari, M, Gordon, M, Lin, C-C, Papai, Z, Ryan, CW, Tap, WD, Trent, JC, Gelderblom, H, Grimison, P, Pousa, AL, Van Tine, BA, Rubinacci, M, Tecson, K, Wooddell, M, and Desai, J
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- 2024
14. Referral patterns of GIST patients: data from a nationwide study
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Roets, E., Ijzerman, N.S., Ho, V.K., Desar, I.M.E., Reyners, A.K.L., Gelderblom, H., Grünhagen, D.J., Etten, B. van, Houdt, W.J. van, Graaf, W.T.A. van der, Steeghs, N., Roets, E., Ijzerman, N.S., Ho, V.K., Desar, I.M.E., Reyners, A.K.L., Gelderblom, H., Grünhagen, D.J., Etten, B. van, Houdt, W.J. van, Graaf, W.T.A. van der, and Steeghs, N.
- Abstract
Contains fulltext : 305391.pdf (Publisher’s version ) (Open Access), BACKGROUND: This study compares the characteristics, referral and treatment patterns and overall survival (OS) of gastrointestinal stromal tumor (GIST) patients treated in reference and non-reference centers in the Netherlands. PATIENTS AND METHODS: This retrospective cohort study on patients diagnosed between 2016 and 2019, utilises data from the Netherlands Cancer Registry and the Dutch Nationwide Pathology Database. Patients were categorized into two groups: patients diagnosed in or referred to reference centers and patients diagnosed in non-reference centers without referral. RESULTS: This study included 1,550 GIST patients with a median age of 67.0 in reference and 68.0 years in non-reference centers. Eighty-seven per cent of patients were diagnosed in non-reference centers, of which 36.5% (493/1,352) were referred to a reference center. Referral rates were higher for high-risk (62.2% [74/119]) and metastatic patients (67.2% [90/134]). Mutation analysis was performed in 96.9% and 87.6% of these cases in reference and in non-reference centers (p < 0.01), respectively. Systemic therapy was given in reference centers versus non-reference in 89.5% versus 82.0% (p < 0.01) of high-risk and in 94.1% versus 65.9% (p < 0.01) of metastatic patients, respectively. The proportion of positive resection margins and tumor rupture did not differ between reference and non-reference centers. Median OS was not reached. CONCLUSION: A substantial amount of metastatic GIST patients in non-reference centers did not receive systemic treatment. This might be due to valid reasons. However, optimisation of the referral strategy of GIST patients in the Netherlands could benefit patients. Further research is needed to explore reasons for not starting systemic treatment in metastatic GIST patients.
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- 2024
15. Diagnosis and management of tropomyosin receptor kinase (TRK) fusion sarcomas: expert recommendations from the World Sarcoma Network
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Demetri, G.D., Antonescu, C.R., Bjerkehagen, B., Bovée, J.V.M.G., Boye, K., Chacón, M., Dei Tos, A.P., Desai, J., Fletcher, J.A., Gelderblom, H., George, S., Gronchi, A., Haas, R.L., Hindi, N., Hohenberger, P., Joensuu, H., Jones, R.L., Judson, I., Kang, Y.-K., Kawai, A., Lazar, A.J., Le Cesne, A., Maestro, R., Maki, R.G., Martín, J., Patel, S., Penault-Llorca, F., Premanand Raut, C., Rutkowski, P., Safwat, A., Sbaraglia, M., Schaefer, I.-M., Shen, L., Serrano, C., Schöffski, P., Stacchiotti, S., Sundby Hall, K., Tap, W.D., Thomas, D.M., Trent, J., Valverde, C., van der Graaf, W.T.A., von Mehren, M., Wagner, A., Wardelmann, E., Naito, Y., Zalcberg, J., and Blay, J.-Y.
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- 2020
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16. 62P Updated overall survival and safety with ripretinib vs sunitinib in patients with advanced gastrointestinal stromal tumor previously treated with imatinib and harboring KIT exon 11 + 17/18 mutations: ctDNA analysis from INTRIGUE
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Blay, J-Y., primary, Jones, R.L., additional, Gelderblom, H., additional, George, S., additional, Schöffski, P., additional, von Mehren, M., additional, Zalcberg, J.R., additional, Kang, Y-K., additional, Razak, A.R. Abdul, additional, Trent, J.C., additional, Attia, S., additional, Le Cesne, A., additional, Davis, E., additional, Sprott, K., additional, Cox, P., additional, Sherman, M.L., additional, Ruiz-Soto, R., additional, Heinrich, M., additional, and Bauer, S., additional
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- 2024
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17. 141TiP Cemiplimab treatment in patients with locally advanced and metastatic secondary angiosarcomas (CEMangio): A phase II clinical trial in progress
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van Ravensteijn, S., primary, de Haan, J.J., additional, Gelderblom, H., additional, Versleijen-Jonkers, Y., additional, Speetjens, F., additional, Kaal, S., additional, Smits, M., additional, van Herpen, C., additional, and Desar, I., additional
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- 2024
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18. EURAMOS-1, an international randomised study for osteosarcoma: results from pre-randomisation treatment †
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Whelan, JS, Bielack, SS, Marina, N, Smeland, S, Jovic, G, Hook, JM, Krailo, M, Anninga, J, Butterfass-Bahloul, T, Böhling, T, Calaminus, G, Capra, M, Deffenbaugh, C, Dhooge, C, Eriksson, M, Flanagan, AM, Gelderblom, H, Goorin, A, Gorlick, R, Gosheger, G, Grimer, RJ, Hall, KS, Helmke, K, Hogendoorn, PCW, Jundt, G, Kager, L, Kuehne, T, Lau, CC, Letson, GD, Meyer, J, Meyers, PA, Morris, C, Mottl, H, Nadel, H, Nagarajan, R, Randall, RL, Schomberg, P, Schwarz, R, Teot, LA, Sydes, MR, Bernstein, M, Pickering, James, Joffe, Nicola, Kevric, Matthias, Sorg, Benjamin, Villaluna, Doojduen, Wang, Caroline, Perisoglou, Martha, Trani, Leonardo, Potratz, Jenny, Carrle, Dorothe, Wilhelm, Miriam, Zils, Katja, and Teske, Carmen
- Subjects
Pediatric ,Cancer ,Clinical Trials and Supportive Activities ,Clinical Research ,Pediatric Cancer ,Evaluation of treatments and therapeutic interventions ,6.1 Pharmaceuticals ,Adolescent ,Antineoplastic Combined Chemotherapy Protocols ,Bone Neoplasms ,Child ,Cisplatin ,Combined Modality Therapy ,Doxorubicin ,Etoposide ,Female ,Humans ,Ifosfamide ,Interferon-alpha ,Male ,Methotrexate ,Neoadjuvant Therapy ,Osteosarcoma ,Polyethylene Glycols ,Quality of Life ,Research Design ,Young Adult ,EURAMOS collaborators ,international collaboration ,osteosarcoma ,randomised controlled trial ,trial conduct ,Oncology and Carcinogenesis ,Oncology & Carcinogenesis - Abstract
BackgroundFour international study groups undertook a large study in resectable osteosarcoma, which included two randomised controlled trials, to determine the effect on survival of changing post-operative chemotherapy based on histological response.Patients and methodsPatients with resectable osteosarcoma aged ≤40 years were treated with the MAP regimen, comprising pre-operatively of two 5-week cycles of cisplatin 120 mg/m(2), doxorubicin 75 mg/m(2), methotrexate 12 g/m(2) × 2 (MAP) and post-operatively two further cycles of MAP and two cycles of just MA. Patients were randomised after surgery. Those with ≥10% viable tumour in the resected specimen received MAP or MAP with ifosfamide and etoposide. Those with
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- 2015
19. Effect of CYP2C19 genotypes on tamoxifen metabolism and early-breast cancer relapse
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Sanchez-Spitman, A. B., Swen, J. J., Dezentjé, V. O., Moes, D. J. A. R., Gelderblom, H., and Guchelaar, H. J.
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- 2021
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20. Analytical and experimental characterization of a miniature calorimetric sensor in pulsatile flow
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Gelderblom, H., van der Horst, A., Haartsen, J. R., Rutten, M. C. M., van de Ven, A. A. F., and van de Vosse, F. N.
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Physics - Fluid Dynamics - Abstract
The behaviour of a miniature calorimetric sensor, which is under consideration for catheter-based coronary artery flow assessment, is investigated in both steady and pulsatile tube flow. The sensor is composed of a heating element operated at constant power, and two thermopiles that measure flow-induced temperature differences over the sensor surface. An analytical sensor model is developed, which includes axial heat conduction in the fluid and a simple representation of the solid wall, assuming a quasi-steady sensor response to the pulsatile flow. To reduce the mathematical problem, described by a two-dimensional advection-diffusion equation, a spectral method is applied. A Fourier transform is then used to solve the resulting set of ordinary differential equations and an analytical expression for the fluid temperature is found. To validate the analytical model, experiments with the sensor mounted in a tube have been performed in steady and pulsatile water flow with various amplitudes and Strouhal numbers. Experimental results are generally in good agreement with theory and show a quasi-steady sensor response in the coronary flow regime. The model can therefore be used to optimize the sensor design for coronary flow assessment.
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- 2011
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21. Bone sarcomas: ESMO–PaedCan–EURACAN Clinical Practice Guidelines for diagnosis, treatment and follow-up
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Casali, P.G., Bielack, S., Abecassis, N., Aro, H.T., Bauer, S., Biagini, R., Bonvalot, S., Boukovinas, I., Bovee, J V M G, Brennan, B., Brodowicz, T., Broto, J.M., Brugières, L., Buonadonna, A., De Álava, E., Dei Tos, A.P., Del Muro, X.G., Dileo, P., Dhooge, C., Eriksson, M., Fagioli, F., Fedenko, A., Ferraresi, V., Ferrari, A., Ferrari, S., Frezza, A.M., Gaspar, N., Gasperoni, S., Gelderblom, H., Gil, T., Grignani, G., Gronchi, A., Haas, R.L., Hassan, B., Hecker-Nolting, S., Hohenberger, P., Issels, R., Joensuu, H., Jones, R.L., Judson, I., Jutte, P., Kaal, S., Kager, L., Kasper, B., Kopeckova, K., Krákorová, D.A., Ladenstein, R., Le Cesne, A., Lugowska, I., Merimsky, O., Montemurro, M., Morland, B., Pantaleo, M.A., Piana, R., Picci, P., Piperno-Neumann, S., Pousa, A.L., Reichardt, P., Robinson, M.H., Rutkowski, P., Safwat, A.A., Schöffski, P., Sleijfer, S., Stacchiotti, S., Strauss, S.J., Sundby Hall, K., Unk, M., Van Coevorden, F., van der Graaf, W.T.A., Whelan, J., Wardelmann, E., Zaikova, O., and Blay, J.Y.
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- 2018
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22. Soft tissue and visceral sarcomas: ESMO–EURACAN Clinical Practice Guidelines for diagnosis, treatment and follow-up
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Casali, P.G., Abecassis, N., Bauer, S., Biagini, R., Bielack, S., Bonvalot, S., Boukovinas, I., Bovee, J V M G, Brodowicz, T., Broto, J.M., Buonadonna, A., De Álava, E., Dei Tos, A.P., Del Muro, X.G., Dileo, P., Eriksson, M., Fedenko, A., Ferraresi, V., Ferrari, A., Ferrari, S., Frezza, A.M., Gasperoni, S., Gelderblom, H., Gil, T., Grignani, G., Gronchi, A., Haas, R.L., Hannu, A., Hassan, B., Hohenberger, P., Issels, R., Joensuu, H., Jones, R.L., Judson, I., Jutte, P., Kaal, S., Kasper, B., Kopeckova, K., Krákorová, D.A., Le Cesne, A., Lugowska, I., Merimsky, O., Montemurro, M., Pantaleo, M.A., Piana, R., Picci, P., Piperno-Neumann, S., Pousa, A.L., Reichardt, P., Robinson, M.H., Rutkowski, P., Safwat, A.A., Schöffski, P., Sleijfer, S., Stacchiotti, S., Sundby Hall, K., Unk, M., Van Coevorden, F., Van der Graaf, W., Whelan, J., Wardelmann, E., Zaikova, O., and Blay, J.Y.
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- 2018
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23. Gastrointestinal stromal tumours: ESMO–EURACAN Clinical Practice Guidelines for diagnosis, treatment and follow-up
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Casali, P.G., Abecassis, N., Bauer, S., Biagini, R., Bielack, S., Bonvalot, S., Boukovinas, I., Bovee, J.V.M.G., Brodowicz, T., Broto, J.M., Buonadonna, A., De Álava, E., Dei Tos, A.P., Del Muro, X.G., Dileo, P., Eriksson, M., Fedenko, A., Ferraresi, V., Ferrari, A., Ferrari, S., Frezza, A.M., Gasperoni, S., Gelderblom, H., Gil, T., Grignani, G., Gronchi, A., Haas, R.L., Hannu, A., Hassan, B., Hohenberger, P., Issels, R., Joensuu, H., Jones, R.L., Judson, I., Jutte, P., Kaal, S., Kasper, B., Kopeckova, K., Krákorová, D.A., Le Cesne, A., Lugowska, I., Merimsky, O., Montemurro, M., Pantaleo, M.A., Piana, R., Picci, P., Piperno-Neumann, S., Pousa, A.L., Reichardt, P., Robinson, M.H., Rutkowski, P., Safwat, A.A., Schöffski, P., Sleijfer, S., Stacchiotti, S., Sundby Hall, K., Unk, M., Van Coevorden, F., Van der Graaf, W., Whelan, J., Wardelmann, E., Zaikova, O., and Blay, J.Y.
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- 2018
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24. Activity and safety of crizotinib in patients with alveolar soft part sarcoma with rearrangement of TFE3: European Organization for Research and Treatment of Cancer (EORTC) phase II trial 90101 ‘CREATE’
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Schöffski, P., Wozniak, A., Kasper, B., Aamdal, S., Leahy, M.G., Rutkowski, P., Bauer, S., Gelderblom, H., Italiano, A., Lindner, L.H., Hennig, I., Strauss, S., Zakotnik, B., Anthoney, A., Albiges, L., Blay, J.-Y., Reichardt, P., Sufliarsky, J., van der Graaf, W.T.A., Debiec-Rychter, M., Sciot, R., Van Cann, T., Marréaud, S., Raveloarivahy, T., Collette, S., and Stacchiotti, S.
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- 2018
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25. The Drug Rediscovery protocol facilitates the expanded use of existing anticancer drugs
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van der Velden, D. L., Hoes, L. R., van der Wijngaart, H., van Berge Henegouwen, J. M., van Werkhoven, E., Roepman, P., Schilsky, R. L., de Leng, W. W. J., Huitema, A. D. R., Nuijen, B., Nederlof, P. M., van Herpen, C. M. L., de Groot, D. J. A., Devriese, L. A., Hoeben, A., de Jonge, M. J. A., Chalabi, M., Smit, E. F., de Langen, A. J., Mehra, N., Labots, M., Kapiteijn, E., Sleijfer, S., Cuppen, E., Verheul, H. M. W., Gelderblom, H., and Voest, E. E.
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- 2019
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26. Best practices for the management of local-regional recurrent chordoma: a position paper by the Chordoma Global Consensus Group
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Stacchiotti, S., Gronchi, A., Fossati, P., Akiyama, T., Alapetite, C., Baumann, M., Blay, J.Y., Bolle, S., Boriani, S., Bruzzi, P., Capanna, R., Caraceni, A., Casadei, R., Colia, V., Debus, J., Delaney, T., Desai, A., Dileo, P., Dijkstra, S., Doglietto, F., Flanagan, A., Froelich, S., Gardner, P.A., Gelderblom, H., Gokaslan, Z.L., Haas, R., Heery, C., Hindi, N., Hohenberger, P., Hornicek, F., Imai, R., Jeys, L., Jones, R.L., Kasper, B., Kawai, A., Krengli, M., Leithner, A., Logowska, I., Martin Broto, J., Mazzatenta, D., Morosi, C., Nicolai, P., Norum, O.J., Patel, S., Penel, N., Picci, P., Pilotti, S., Radaelli, S., Ricchini, F., Rutkowski, P., Scheipl, S., Sen, C., Tamborini, E., Thornton, K.A., Timmermann, B., Torri, V., Tunn, P.U., Uhl, M., Yamada, Y., Weber, D.C., Vanel, D., Varga, P.P., Vleggeert-Lankamp, C.L.A., Casali, P.G., and Sommer, J.
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- 2017
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27. A phase II study of a human anti-PDGFRα monoclonal antibody (olaratumab, IMC-3G3) in previously treated patients with metastatic gastrointestinal stromal tumors
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Wagner, A.J., Kindler, H., Gelderblom, H., Schöffski, P., Bauer, S., Hohenberger, P., Kopp, H.-G., Lopez-Martin, J.A., Peeters, M., Reichardt, P., Qin, A., Nippgen, J., Ilaria, R.L., and Rutkowski, P.
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- 2017
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28. Gastrointestinal stromal tumours
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Casali, P.G., Blay, J.Y., Abecassis, N., Bajpai, J., Bauer, S., Biagini, R., Bielack, S., Bonvalot, S., Boukovinas, I., Bovee, J.V.M.G., Boye, K., Brodowicz, T., Buonadonna, A., Alava, E. de, Tos, A.P. dei, Muro, X.G. del, Dufresne, A., Eriksson, M., Fedenko, A., Ferraresi, V., Ferrari, A., Frezza, A.M., Gasperoni, S., Gelderblom, H., Gouin, F., Grignani, G., Haas, R., Hassan, A.B., Hindi, N., Hohenberger, P., Joensuu, H., Jones, R.L., Jungels, C., Jutte, P., Kasper, B., Kawai, A., Kopeckova, K., Krakorova, D.A., Cesne, A. le, Grange, F. le, Legius, E., Leithner, A., Lopez-Pousa, A., Martin-Broto, J., Merimsky, O., Messiou, C., Miah, A.B., Mir, O., Montemurro, M., Morosi, C., Palmerini, E., Pantaleo, M.A., Piana, R., Piperno-Neumann, S., Reichardt, P., Rutkowski, P., Safwat, A.A., Sangalli, C., Sbaraglia, M., Scheipl, S., Schoffski, P., Sleijfer, S., Strauss, D., Strauss, S.J., Hall, K.S., Trama, A., Unk, M., Sande, M.A.J. van de, Graaf, W.T.A. van der, Houdt, W.J. van, Frebourg, T., Gronchi, A., Stacchiotti, S., ESMO Guidelines Comm, EURACAN, GENTURIS, Casali P.G., Blay J.Y., Abecassis N., Bajpai J., Bauer S., Biagini R., Bielack S., Bonvalot S., Boukovinas I., Bovee J.V.M.G., Boye K., Brodowicz T., Buonadonna A., De Alava E., Dei Tos A.P., Del Muro X.G., Dufresne A., Eriksson M., Fedenko A., Ferraresi V., Ferrari A., Frezza A.M., Gasperoni S., Gelderblom H., Gouin F., Grignani G., Haas R., Hassan A.B., Hindi N., Hohenberger P., Joensuu H., Jones R.L., Jungels C., Jutte P., Kasper B., Kawai A., Kopeckova K., Krakorova D.A., Le Cesne A., Le Grange F., Legius E., Leithner A., Lopez-Pousa A., Martin-Broto J., Merimsky O., Messiou C., Miah A.B., Mir O., Montemurro M., Morosi C., Palmerini E., Pantaleo M.A., Piana R., Piperno-Neumann S., Reichardt P., Rutkowski P., Safwat A.A., Sangalli C., Sbaraglia M., Scheipl S., Schoffski P., Sleijfer S., Strauss D., Strauss S.J., Hall K.S., Trama A., Unk M., van de Sande M.A.J., van der Graaf W.T.A., van Houdt W.J., Frebourg T., Gronchi A., Stacchiotti S., Medical Oncology, Basic and Translational Research and Imaging Methodology Development in Groningen (BRIDGE), Public Health Research (PHR), and Man, Biomaterials and Microbes (MBM)
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GIST ,clinical practice guidelines ,gastrointestinal stromal tumour ,surgery ,tyrosine kinase inhibitor ,Oncology ,Medizin ,Hematology ,clinical practice guideline - Abstract
Gastrointestinal stromal tumours (GISTs) are malignant mesenchymal tumours with a variable clinical behaviour, marked by differentiation towards the interstitial cells of Cajal.1 GISTs belong to the family of soft tissue sarcomas (STSs) but are treated separately due to their peculiar histogenesis, clinical behaviour and specific therapy. This European Society for Medical Oncology (ESMO)–European Reference Network for Rare Adult Solid Cancers (EURACAN)–European Reference Network for Genetic Tumour Risk Syndromes (GENTURIS) Clinical Practice Guideline (CPG) will cover GISTs while other STSs are covered in the ESMO–EURACAN–European Reference Network for Paediatric Oncology (ERN PaedCan)–GENTURIS STS CPG.
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- 2022
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29. Efficacy of eye movement desensitization and reprocessing therapy for fear of cancer recurrence among cancer survivors: a randomized single-case experimental design
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Bruin, J., primary, van Rood, Y. R., additional, Peeters, K.C.M.J., additional, de Roos, C., additional, Tanious, R., additional, Portielje, J.E.A., additional, Gelderblom, H., additional, and Hinnen, S.C.H., additional
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- 2023
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30. Genome-wide analyses of platinum-induced ototoxicity in childhood cancer patients: Results of GO-CAT and United Kingdom MAGIC consortia
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Hurkmans, E.G.E., Klumpers, M.J., Dello Russo, Cinzia, Witte, Ward De, Guchelaar, H.J., Gelderblom, H., Vermeulen, S.H., Kaal, S.E., Graaf, W.T.A. van der, Flucke, U.E., Coenen, M.J.H., Loo, D.M.W.M. te, Hurkmans, E.G.E., Klumpers, M.J., Dello Russo, Cinzia, Witte, Ward De, Guchelaar, H.J., Gelderblom, H., Vermeulen, S.H., Kaal, S.E., Graaf, W.T.A. van der, Flucke, U.E., Coenen, M.J.H., and Loo, D.M.W.M. te
- Abstract
Contains fulltext : 290144.pdf (Publisher’s version ) (Open Access)
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- 2023
31. Local recurrence in primary localised resected gastrointestinal stromal tumours: A registry observational national cohort study including 912 patients.
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Bleckman, R.F., Roets, E., Ijzerman, N.S., Mohammadi, M., Bonenkamp, H., Gelderblom, H., Mathijssen, R.H., Steeghs, N., Reyners, A.K., Etten, B. van, Bleckman, R.F., Roets, E., Ijzerman, N.S., Mohammadi, M., Bonenkamp, H., Gelderblom, H., Mathijssen, R.H., Steeghs, N., Reyners, A.K., and Etten, B. van
- Abstract
01 juni 2023, Item does not contain fulltext, BACKGROUND AND OBJECTIVES: Previous literature showed a high risk of recurrence following surgical treatment in patients with gastrointestinal stromal tumours (GISTs). However, little is known about the patient- and treatment characteristics of local recurrences (LRs) in GIST patients. Therefore, this study aimed to better understand patterns of LR in surgically treated localised GIST and to describe treatment options based on our Dutch GIST Registry (DGR). METHODS: Data of primary surgically treated localised GIST between January 2009 until July 2021 were retrospectively retrieved from the DGR. RESULTS: Of 1452 patients registered in the DGR, 912 patients were included in this study. Only 3.8% (35/912) of patients developed LR, including 20 patients with LR only and 15 patients with simultaneous LR and distant metastases (DM). Median time to LR was 30 (interquartile range 8-53) months from date of surgery. Eleven percent (100/912) of patients developed only DM. A total of 2.3% (6/259) of patients treated with adjuvant treatment developed an LR during adjuvant therapy. Seventy percent of patients with LR only (14/20) were treated with surgery (85.7% R0), which was mostly combined with systemic treatment. CONCLUSIONS: Patients with primary surgically treated localised GIST have a limited risk of developing recurrence. Fifteen percent developed recurrence, of which one quarter developed an LR. Therefore, less intensified follow-up schedules could be considered, especially during treatment with adjuvant imatinib. In patients with LR only, potentially curative treatment strategies, including surgical (re-)resection, are often possible as treatment for LR.
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- 2023
32. Improved Efficacy of First-Line Imatinib in Advanced Gastrointestinal Stromal Tumors (GIST): The Dutch GIST Registry Data.
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Mohammadi, M., Ijzerman, N.S., Hollander, D. den, Bleckman, R.F., Oosten, A.W., Desar, I.M.E., Reyners, A.K., Steeghs, N., Gelderblom, H., Mohammadi, M., Ijzerman, N.S., Hollander, D. den, Bleckman, R.F., Oosten, A.W., Desar, I.M.E., Reyners, A.K., Steeghs, N., and Gelderblom, H.
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Item does not contain fulltext, BACKGROUND: Patients with unresectable and metastasized gastrointestinal stromal tumor (GIST) experienced a remarkable improvement of progression-free survival (PFS) and overall survival (OS) after the introduction of imatinib. Our hypothesis is that the outcomes of treatment with imatinib are even better nowadays compared with the registration trials that were performed two decades ago. To study this, we used real-life data from a contemporary registry. METHODS: A multicenter, retrospective study was performed by exploring clinical data from a prospective real-life clinical database, the Dutch GIST Registry (DGR). Patients with advanced GIST treated with first-line imatinib were included and PFS (primary outcome) and OS (secondary outcome) were analyzed. Results of our study were compared with published results of the European Organisation for Research and Treatment of Cancer (EORTC) 62005 trial, which marked the first era of imatinib in the treatment of GIST. RESULTS: Overall, 420 of the 435 patients treated with imatinib in the DGR had recorded response evaluation and were included in the analysis. During a median follow-up of 35.0 months (range 2.0-136.0), progression of GIST was eventually observed in 217 patients (51.2%). The DGR cohort showed a longer median PFS (33.0 months, 95% confidence interval [CI] 28.4-37.6) compared with the EORTC 62005 trial (an estimated PFS of 19.5 months). Additionally, the median OS of 68.0 months (95% CI 56.1-80.0) was longer than the exposed median OS (46.8 months) published in the long-term follow-up results of the EORTC 62005 trial (median follow-up duration 10.9 years). CONCLUSION: This study provides an update on outcomes of imatinib in the treatment of advanced GIST patients and demonstrates improved clinical outcomes since the first randomized studies of imatinib 2 decades ago. Furthermore, these results represent outcomes in real-world clinical practice and can serve as a reference when evaluating effectiveness of imatin
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- 2023
33. Local treatment in metastatic GIST patients: A multicentre analysis from the Dutch GIST Registry.
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Brink, P., Kalisvaart, G.M., Schrage, Y.M., Mohammadi, M., Ijzerman, N.S., Bleckman, R.F., Wal, T., Geus-Oei, L.F. de, Hartgrink, H.H., Grunhagen, D.J., Verhoef, C., Sleijfer, S., Oosten, A.W., Been, L.B., Ginkel, R.J. van, Reyners, A.K.L., Bonenkamp, H., Desar, I.M.E., Gelderblom, H., Houdt, W.J. van, Steeghs, N., Fiocco, M., Hage, J.A. van der, Brink, P., Kalisvaart, G.M., Schrage, Y.M., Mohammadi, M., Ijzerman, N.S., Bleckman, R.F., Wal, T., Geus-Oei, L.F. de, Hartgrink, H.H., Grunhagen, D.J., Verhoef, C., Sleijfer, S., Oosten, A.W., Been, L.B., Ginkel, R.J. van, Reyners, A.K.L., Bonenkamp, H., Desar, I.M.E., Gelderblom, H., Houdt, W.J. van, Steeghs, N., Fiocco, M., and Hage, J.A. van der
- Abstract
01 september 2023, Contains fulltext : 296539.pdf (Publisher’s version ) (Open Access), BACKGROUND: The added value of local treatment in selected metastatic GIST patients is unclear. This study aims to provide insight into the usefulness of local treatment in metastatic GIST by use of a survey study and retrospective analyses in a clinical database. METHODS: A survey study was conducted among clinical specialists to select most relevant characteristics of metastatic GIST patients considered for local treatment, defined as elective surgery or ablation. Patients were selected from the Dutch GIST Registry. A multivariate Cox-regression model for overall survival since time of diagnosis of metastatic disease was estimated with local treatment as a time-dependent variable. An additional model was estimated to assess prognostic factors since local treatment. RESULTS: The survey's response rate was 14/16. Performance status, response to TKIs, location of active disease, number of lesions, mutation status, and time between primary diagnosis and metastases, were regarded the 6 most important characteristics. Of 457 included patients, 123 underwent local treatment, which was associated with better survival after diagnosis of metastases (HR = 0.558, 95%CI = 0.336-0.928). Progressive disease during systemic treatment (HR = 3.885, 95%CI = 1.195-12.627) and disease confined to the liver (HR = 0.269, 95%CI = 0.082-0.880) were associated with worse and better survival after local treatment, respectively. CONCLUSION: Local treatment is associated with better survival in selected patients with metastatic GIST. Locally treated patients with response to TKIs and disease confined to the liver have good clinical outcome. These results might be considered for tailoring treatment, but should be interpreted with care because only specific patients are provided with local treatment in this retrospective study.
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- 2023
34. Limited clinical activity of palbociclib and ribociclib monotherapy in advanced cancers with cyclin D-CDK4/6 pathway alterations in the Dutch DRUP and Australian MoST trials.
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Zeverijn, L.J., Looze, E.J., Thavaneswaran, S., Berge Henegouwen, J.M. van, Simes, R.J., Hoes, L.R., Sjoquist, K.M., Wijngaart, H. van der, Sebastian, L., Geurts, B.S., Lee, C.K., Wit, G.F. de, Espinoza, D., Roepman, P., Lin, F.P., Jansen, A.M.L, Leng, W.W.J. de, Noort, V. van der, Leek, L.V.M., Vos, F.Y.F.L. de, Herpen, C.M.L. van, Gelderblom, H., Verheul, H.M.W., Thomas, D.M., Voest, E.E., Zeverijn, L.J., Looze, E.J., Thavaneswaran, S., Berge Henegouwen, J.M. van, Simes, R.J., Hoes, L.R., Sjoquist, K.M., Wijngaart, H. van der, Sebastian, L., Geurts, B.S., Lee, C.K., Wit, G.F. de, Espinoza, D., Roepman, P., Lin, F.P., Jansen, A.M.L, Leng, W.W.J. de, Noort, V. van der, Leek, L.V.M., Vos, F.Y.F.L. de, Herpen, C.M.L. van, Gelderblom, H., Verheul, H.M.W., Thomas, D.M., and Voest, E.E.
- Abstract
Contains fulltext : 295941.pdf (Publisher’s version ) (Closed access), The Dutch Drug Rediscovery Protocol (DRUP) and the Australian Cancer Molecular Screening and Therapeutic (MoST) Program are similar nonrandomized, multidrug, pan-cancer trial platforms that aim to identify signals of clinical activity of molecularly matched targeted therapies or immunotherapies outside their approved indications. Here, we report results for advanced or metastatic cancer patients with tumors harboring cyclin D-CDK4/6 pathway alterations treated with CDK4/6 inhibitors palbociclib or ribociclib. We included adult patients that had therapy-refractory solid malignancies with the following alterations: amplifications of CDK4, CDK6, CCND1, CCND2 or CCND3, or complete loss of CDKN2A or SMARCA4. Within MoST, all patients were treated with palbociclib, whereas in DRUP, palbociclib and ribociclib were assigned to different cohorts (defined by tumor type and alteration). The primary endpoint for this combined analysis was clinical benefit, defined as confirmed objective response or stable disease ≥16 weeks. We treated 139 patients with a broad variety of tumor types; 116 with palbociclib and 23 with ribociclib. In 112 evaluable patients, the objective response rate was 0% and clinical benefit rate at 16 weeks was 15%. Median progression-free survival was 4 months (95% CI: 3-5 months), and median overall survival 5 months (95% CI: 4-6 months). In conclusion, only limited clinical activity of palbociclib and ribociclib monotherapy in patients with pretreated cancers harboring cyclin D-CDK4/6 pathway alterations was observed. Our findings indicate that monotherapy use of palbociclib or ribociclib is not recommended and that merging data of two similar precision oncology trials is feasible.
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- 2023
35. Atrial fibrillation in cancer: thromboembolism and bleeding in daily practice
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Chu, G., Seelig, J., Cannegieter, S.C., Gelderblom, H., Hovens, M.M.C., Huisman, M.V., Hulle, T. van der, Trines, S.A., Vlot, A.J., Versteeg, H.H., Hemels, M.E.W., Klok, F.A., Chu, G., Seelig, J., Cannegieter, S.C., Gelderblom, H., Hovens, M.M.C., Huisman, M.V., Hulle, T. van der, Trines, S.A., Vlot, A.J., Versteeg, H.H., Hemels, M.E.W., and Klok, F.A.
- Abstract
Contains fulltext : 294501.pdf (Publisher’s version ) (Open Access), BACKGROUND: Cancer is suggested to confer thromboembolic and bleeding risk in patients with atrial fibrillation (AF). OBJECTIVES: We aimed to describe current anticoagulant practice in patients with AF and active cancer, present incidences of thromboembolic and bleeding complications, and evaluate the association between cancer type or anticoagulant management strategy with AF-related complications. METHODS: This retrospective study identified patients with AF and active cancer in 2 hospitals between January 1, 2012, and December 31, 2017. Follow-up lasted for 2 years. Data on cancer and anticoagulant treatment were collected. The outcomes of interest included ischemic stroke or transient ischemic attack (TIA) and clinically relevant nonmajor bleeding (CRNMB/MB). Incidence rates (IRs) per 100 patient-years and subdistribution hazard ratios (SHRs) with corresponding 95% Cis were estimated. RESULTS: We identified 878 patients with AF who developed cancer (cohort 1) and 335 patients with cancer who developed AF (cohort 2). IRs for ischemic stroke/TIA and MB/CRNMB were 3.9 (2.8-5.3) and 15.7 (13.3-18.5) for cohort 1 and 4.0 (2.2-6.7) and 16.7 (12.6-21.7) for cohort 2. 14.2% (cohort 1) and 19.1% (cohort 2) of patients with a CHA(2)DS(2)-VASc score of ≥2 did not receive anticoagulant treatment. Withholding anticoagulants was associated with thromboembolic complications (SHR: 5.1 [3.20-8.0]). In nonanticoagulated patients with a CHA(2)DS(2)-VASc score of <2, IRs for stroke/TIA were 4.5 (0.75-15.0; cohort 1) and 16.0 (5.1-38.7; cohort 2). CONCLUSION: Patients with AF and active cancer experience high rates of thromboembolic and bleeding complications, underlying the complexity of anticoagulant management in these patients. Our data suggest that the presence of cancer is an important factor in determining the indication for anticoagulants in patients with a low CHA(2)DS(2)-VASc score.
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- 2023
36. Thromboembolic and bleeding complications during interruptions and after discontinuation of anticoagulant treatment in patients with atrial fibrillation and active cancer: A daily practice evaluation.
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Chu, G., Seelig, J., Cannegieter, S.C., Gelderblom, H., Hovens, M.M.C., Huisman, M.V., Hulle, T. van der, Trines, S.A., Vlot, A.J., Versteeg, H.H., Hemels, M.E.W., Klok, F.A., Chu, G., Seelig, J., Cannegieter, S.C., Gelderblom, H., Hovens, M.M.C., Huisman, M.V., Hulle, T. van der, Trines, S.A., Vlot, A.J., Versteeg, H.H., Hemels, M.E.W., and Klok, F.A.
- Abstract
Item does not contain fulltext, BACKGROUND AND AIMS: Cancer provides challenges to the continuity of anticoagulant treatment in patients with atrial fibrillation (AF), e.g. through cancer-related surgery or complications. We aimed to provide data on the incidence and reasons for interrupting and discontinuing anticoagulant treatment in AF patients with cancer and to assess its contribution to the risk of thromboembolism (TE) and major bleeding (MB). METHODS: This retrospective study identified AF patients with cancer in two hospitals between 2012 and 2017. Data on anticoagulant treatment, TE and MB were collected during two-year follow-up. Incidence rates (IR) per 100 patient-years and adjusted hazard ratios (aHR) were obtained for TE and MB occurring during on- and off-anticoagulant treatment, during interruption and after resumption, and after permanent discontinuation. RESULTS: 1213 AF patients with cancer were identified, of which 140 patients permanently discontinued anticoagulants and 426 patients experienced one or more interruptions. Anticoagulation was most often interrupted or discontinued due to cancer-related treatment (n = 441, 62 %), bleeding (n = 129, 18 %) or end of life (n = 36, 5 %). The risk of TE was highest off-anticoagulation and during interruptions, with IRs of 19 (14-25)) and 105 (64-13), and aHRs of 3.1 (1.9-5.0) and 4.6 (2.4-9.0), respectively. Major bleeding risk were not only increased during an interruption, but also in the first 30 days after resumption, with IRs of 33 (12-72) and 30 (17-48), and aHRs of 3.3 (1.1-9.8) and 2.4 (1.2-4.6), respectively. CONCLUSIONS: Interruption of anticoagulation therapy harbors high TE and MB risk in AF patients with cancer. The high incidence rates call for better (periprocedural) anticoagulant management strategies tailored to the cancer setting.
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- 2023
37. The association of having a monitoring or blunting coping style with psychological distress, health-related quality of life and satisfaction with healthcare in gastrointestinal stromal tumour (GIST) patients.
- Author
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Wal, D. van de, Doorn, B. van, Hollander, D. den, Desar, I.M.E., Gelderblom, H., Oosten, A.W., Reyners, A.K.L., Steeghs, N., Graaf, W.T.A. van der, Husson, O., Wal, D. van de, Doorn, B. van, Hollander, D. den, Desar, I.M.E., Gelderblom, H., Oosten, A.W., Reyners, A.K.L., Steeghs, N., Graaf, W.T.A. van der, and Husson, O.
- Abstract
Contains fulltext : 300113.pdf (Publisher’s version ) (Open Access), BACKGROUND: There are two main coping styles regarding information seeking under medical threat; monitoring (information-seeking) and blunting (information-avoiding). The aim of this study is to (1) determine factors associated with a monitoring or blunting coping style in gastro-intestinal stromal tumour (GIST) patients and (2) investigate its association with psychological distress, cancer-related concerns, health-related quality of life and satisfaction with healthcare. METHODS: In a cross-sectional study, Dutch GIST patients completed the shortened version of the Threatening Medical Situations Inventory to determine their coping style, the Hospital Anxiety and Depression Scale, Cancer Worry Scale, EORTC QLQ-C30 and part of the EORTC QLQ-INFO25. RESULTS: A total of 307 patients were classified as blunters (n = 175, 57%) or monitors (n = 132, 43%). Coping style was not associated with tumour or treatment variables, but being a female (OR 2.5; 95%CI 1.5-4.1; p= <.001) and higher educated (OR 5.5; 95%CI 2.5-11.9, p= <.001) were associated with higher odds of being a monitor. Monitors scored significantly lower on emotional functioning (mean = 86.8 vs mean = 90.9, p=.044), which is considered a trivial difference, more often experienced severe fear of cancer recurrence or progression (53.0% vs 37.7%, p=.007), and had more concerns about dying from GIST in the future (60.6% vs 47.4%, p=.025). Compared to blunters, monitors were less satisfied with the received healthcare and information, and would have liked to receive more information. CONCLUSION: GIST patients with a monitoring coping style experience a higher emotional burden. Additionally, monitors exhibit a greater need for information. Although this need for information could potentially result in fears and concerns, recognising it may also create an opening for tailored communication and information., 01 december 2023
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- 2023
38. Genome-wide analyses of platinum-induced ototoxicity in childhood cancer patients: Results of GO-CAT and United Kingdom MAGIC consortia
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Hurkmans, E. G. E., Klumpers, M. J., Dello Russo, Cinzia, De Witte, W., Guchelaar, H. -J., Gelderblom, H., Cleton-Jansen, A. -M., Vermeulen, S. H., Kaal, S., van der Graaf, W. T. A., Flucke, U., Gidding, C. E. M., Schreuder, H. W. B., de Bont, E. S. J. M., Caron, H. N., Gattuso, G., Schiavello, E., Terenziani, M., Massimino, M., Mccowage, G., Nagabushan, S., Limaye, A., Rose, V., Catchpoole, D., Jorgensen, A. L., Barton, C., Delaney, L., Hawcutt, D. B., Pirmohamed, M., Pizer, B., Coenen, M. J. H., te Loo, D. M. W. M., Dello Russo C. (ORCID:0000-0002-2538-3832), Hurkmans, E. G. E., Klumpers, M. J., Dello Russo, Cinzia, De Witte, W., Guchelaar, H. -J., Gelderblom, H., Cleton-Jansen, A. -M., Vermeulen, S. H., Kaal, S., van der Graaf, W. T. A., Flucke, U., Gidding, C. E. M., Schreuder, H. W. B., de Bont, E. S. J. M., Caron, H. N., Gattuso, G., Schiavello, E., Terenziani, M., Massimino, M., Mccowage, G., Nagabushan, S., Limaye, A., Rose, V., Catchpoole, D., Jorgensen, A. L., Barton, C., Delaney, L., Hawcutt, D. B., Pirmohamed, M., Pizer, B., Coenen, M. J. H., te Loo, D. M. W. M., and Dello Russo C. (ORCID:0000-0002-2538-3832)
- Abstract
Hearing loss (ototoxicity) is a major adverse effect of cisplatin and carboplatin chemotherapy. The aim of this study is to identify novel genetic variants that play a role in platinum-induced ototoxicity. Therefore, a genome-wide association study was performed in the Genetics of Childhood Cancer Treatment (GO-CAT) cohort (n = 261) and the United Kingdom Molecular Genetics of Adverse Drug Reactions in Children Study (United Kingdom MAGIC) cohort (n = 248). Results of both cohorts were combined in a meta-analysis. In primary analysis, patients with SIOP Boston Ototoxicity Scale grade ≥1 were considered cases, and patients with grade 0 were controls. Variants with a p-value <10-5 were replicated in previously published data by the PanCareLIFE cohort (n = 390). No genome-wide significant associations were found, but variants in TSPAN5, RBBP4P5, AC010090.1 and RNU6-38P were suggestively associated with platinum-induced ototoxicity. The lowest p-value was found for rs7671702 in TSPAN5 (odds ratio 2.0 (95% confidence interval 1.5-2.7), p-value 5.0 × 10-7). None of the associations were significant in the replication cohort, although the effect directions were consistent among all cohorts. Validation and functional understanding of these genetic variants could lead to more insights in the development of platinum-induced ototoxicity.
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- 2023
39. Bone sarcomas: ESMO–EURACAN–GENTURIS–ERN PaedCan Clinical Practice Guideline for diagnosis, treatment and follow-up
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Strauss, S.J., Frezza, A.M., Abecassis, N., Bajpai, J., Bauer, S., Biagini, R., Bielack, S., Blay, J.Y., Bolle, S., Bonvalot, S., Boukovinas, I., Bovee, J.V.M.G., Boye, K., Brennan, B., Brodowicz, T., Buonadonna, A., Alava, E. de, Tos, A.P. dei, Muro, X.G. del, Dufresne, A., Eriksson, M., Fagioli, F., Fedenko, A., Ferraresi, V., Ferrari, A., Gaspar, N., Gasperoni, S., Gelderblom, H., Gouin, F., Grignani, G., Gronchi, A., Haas, R., Hassan, A.B., Hecker-Nolting, S., Hindi, N., Hohenberger, P., Joensuu, H., Jones, R.L., Jungels, C., Jutte, P., Kager, L., Kasper, B., Kawai, A., Kopeckova, K., Krakorova, D.A., Cesne, A. le, Grange, F. le, Legius, E., Leithner, A., Pousa, A.L., Martin-Broto, J., Merimsky, O., Messiou, C., Miah, A.B., Mir, O., Montemurro, M., Morland, B., Morosi, C., Palmerini, E., Pantaleo, M.A., Piana, R., Piperno-Neumann, S., Reichardt, P., Rutkowski, P., Safwat, A.A., Sangalli, C., Sbaraglia, M., Scheipl, S., Schoffski, P., Sleijfer, S., Strauss, D., Hall, K.S., Trama, A., Unk, M., Sande, M.A.J. van de, Graaf, W.T.A. van der, Houdt, W.J. van, Frebourg, T., Ladenstein, R., Casali, P.G., Stacchiotti, S., ESMO Guidelines Comm, EURACAN, GENTURIS, ERN PaedCan, European Society for Medical Oncology, Strauss S.J., Frezza A.M., Abecassis N., Bajpai J., Bauer S., Biagini R., Bielack S., Blay J.Y., Bolle S., Bonvalot S., Boukovinas I., Bovee J.V.M.G., Boye K., Brennan B., Brodowicz T., Buonadonna A., de Alava E., Dei Tos A.P., Garcia del Muro X., Dufresne A., Eriksson M., Fagioli F., Fedenko A., Ferraresi V., Ferrari A., Gaspar N., Gasperoni S., Gelderblom H., Gouin F., Grignani G., Gronchi A., Haas R., Hassan A.B., Hecker-Nolting S., Hindi N., Hohenberger P., Joensuu H., Jones R.L., Jungels C., Jutte P., Kager L., Kasper B., Kawai A., Kopeckova K., Krakorova D.A., Le Cesne A., Le Grange F., Legius E., Leithner A., Lopez Pousa A., Martin-Broto J., Merimsky O., Messiou C., Miah A.B., Mir O., Montemurro M., Morland B., Morosi C., Palmerini E., Pantaleo M.A., Piana R., Piperno-Neumann S., Reichardt P., Rutkowski P., Safwat A.A., Sangalli C., Sbaraglia M., Scheipl S., Schoffski P., Sleijfer S., Strauss D., Sundby Hall K., Trama A., Unk M., van de Sande M.A.J., van der Graaf W.T.A., van Houdt W.J., Frebourg T., Ladenstein R., Casali P.G., Stacchiotti S., Basic and Translational Research and Imaging Methodology Development in Groningen (BRIDGE), Public Health Research (PHR), and Man, Biomaterials and Microbes (MBM)
- Subjects
medicine.medical_specialty ,diagnosis ,Medizin ,bone sarcoma ,Bone Neoplasm ,Bone Sarcoma ,Follow-Up Studie ,03 medical and health sciences ,0302 clinical medicine ,follow-up ,medicine ,030304 developmental biology ,Osteosarcoma ,0303 health sciences ,treatment ,business.industry ,Sarcoma ,Hematology ,Guideline ,clinical practice guideline ,management ,3. Good health ,Clinical Practice ,diagnosi ,Oncology ,Diagnosis treatment ,030220 oncology & carcinogenesis ,Radiology ,business ,Human - Abstract
A. Kawai43, K. Kopeckova44, D. A. Krakorova45, A. Le Cesne46, F. Le Grange1, E. Legius47, A. Leithner48, A. Lopez Pousa49, J. Martin-Broto36, O. Merimsky50, C. Messiou51, A. B. Miah52, O. Mir53, M. Montemurro54, B. Morland55, C. Morosi56, E. Palmerini57, M. A. Pantaleo58, R. Piana59, S. Piperno-Neumann60, P. Reichardt61, P. Rutkowski62, A. A. Safwat63, C. Sangalli64, M. Sbaraglia19, S. Scheipl48, P. Schoffski65, S. Sleijfer66, D. Strauss67, K. Sundby Hall13, A. Trama68, M. Unk69, M. A. J. van de Sande70, W. T. A. van der Graaf66,71, W. J. van Houdt72, T. Frebourg73x, R. Ladenstein41z, P. G. Casali2,74z &
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- 2021
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40. Long-term efficacy of imatinib mesylate in patients with advanced Tenosynovial Giant Cell Tumor
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Verspoor, F. G. M., Mastboom, M. J. L., Hannink, G., Maki, R. G., Wagner, A., Bompas, E., Desai, J., Italiano, A., Seddon, B. M., van der Graaf, W. T. A., Blay, J.-Y., Brahmi, M., Eberst, L., Stacchiotti, S., Mir, O., van de Sande, M. A. J., Gelderblom, H., and Cassier, P. A.
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- 2019
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41. Easy-to-use clinical tool for survival estimation in Ewing sarcoma at diagnosis and after surgery
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Bosma, S. E., Lancia, C., Rueten-Budde, A. J., Ranft, A., Gelderblom, H., Fiocco, M., van de Sande, M. A. J., Dijkstra, P. D. S., and Dirksen, U.
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- 2019
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42. Nationwide evaluation of mutation-tailored anti-EGFR therapy selection in patients with colorectal cancer in daily clinical practice
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Steeghs, E.M.P., Vink, G.R., Elferink, M.A.G., Voorham, Q.J.M., Gelderblom, H., Nagtegaal, I.D., Grunberg, K., Ligtenberg, M.J.L., PATH Consortium Members, and Pathology
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Oncology ,medicine.medical_specialty ,business.industry ,Colorectal cancer ,Stage iv disease ,General Medicine ,colorectal neoplasms ,medicine.disease ,Pathology and Forensic Medicine ,Cancer registry ,Clinical Practice ,diagnostic techniques and procedures ,SDG 3 - Good Health and Well-being ,Internal medicine ,Mutation (genetic algorithm) ,Mutation testing ,Medicine ,pathology ,In patient ,molecular ,business - Abstract
For a nationwide real-word data study on the application of predictive mutation testing of patients with colorectal cancer (CRC) for anti-epidermal growth factor receptor (EGFR) therapy stratification, pathology data were collected from the Dutch Pathology Registry from October 2017 until June 2019 (N=4060) and linked with the Netherlands Cancer Registry. Mutation testing rates increased from 24% at diagnosis of stage IV disease to 60% after 20–23 months of follow-up (pKRAS/NRAS wild-type patients was mainly observed from the third treatment line onwards (65% vs 17% in first/second treatment line (pKRAS/NRAS/BRAF mutation rate was 63.9%, being similar for next-generation sequencing (NGS)-based approaches and single gene tests (64.4% vs 61.2%, p=ns). NGS-based approaches detected more additional potential biomarkers, for example, ERBB2 amplifications (p
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- 2022
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43. Gamma delta T cells are effectors of immunotherapy in cancers with HLA class I defects
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Vries, N.L. de, Haar, J. van de, Veninga, V., Chalabi, M., Ijsselsteijn, M.E., Ploeg, M. van der, Bulk, J. van den, Ruano, D., Berg, J.G. van den, Haanen, J.B., Zeverijn, L.J., Geurts, B.S., Wit, G.F. de, Battaglia, T.W., Gelderblom, H., Verheul, H.M.W., Schumacher, T.N., Wessels, L.F.A., Koning, F., Miranda, N.F.C.C. de, and Voest, E.E.
- Abstract
DNA mismatch repair-deficient (MMR-d) cancers present an abundance of neoantigens that is thought to explain their exceptional responsiveness to immune checkpoint blockade (ICB)(1,2). Here, in contrast to other cancer types(3-5), we observed that 20 out of 21 (95%) MMR-d cancers with genomic inactivation of beta 2-microglobulin (encoded by B2M) retained responsiveness to ICB, suggesting the involvement of immune effector cells other than CD8(+) T cells in this context. We next identified a strong association between B2M inactivation and increased infiltration by gamma delta T cells in MMR-d cancers. These gamma delta T cells mainly comprised the V delta 1 and V delta 3 subsets, and expressed high levels of PD-1, other activation markers, including cytotoxic molecules, and a broad repertoire of killer-cell immunoglobulin-like receptors. In vitro, PD-1(+) gamma delta T cells that were isolated from MMR-d colon cancers exhibited enhanced reactivity to human leukocyte antigen (HLA)-class-I-negative MMR-d colon cancer cell lines and B2M-knockout patient-derived tumour organoids compared with antigen-presentation-proficient cells. By comparing paired tumour samples from patients with MMR-d colon cancer that were obtained before and after dual PD-1 and CTLA-4 blockade, we found that immune checkpoint blockade substantially increased the frequency of gamma delta T cells in B2M-deficient cancers. Taken together, these data indicate that gamma delta T cells contribute to the response to immune checkpoint blockade in patients with HLA-class-I-negative MMR-d colon cancers, and underline the potential of gamma delta T cells in cancer immunotherapy.
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- 2023
44. Risk of venous thromboembolism and major bleeding in the clinical course of osteosarcoma and Ewing sarcoma
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Kaptein, F.H.J., Stals, M.A.M., Evenhuis, R.E., Gelderblom, H., Huisman, M.V., Karis, D.S.A., Noten, R.W.D., Cannegieter, S.C., Speetjens, F.M., Verschoor, A.J., Versteeg, H.H., Sande, M.A.J. van de, and Klok, F.A.
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Osteosarcoma ,Anticoagulants ,Hemorrhage ,Hematology ,Age groups ,Prognosis ,Venous thromboembolism - Abstract
Background: Patients with osteosarcoma (OS) and Ewing sarcoma (ES) are considered to have a high venous thromboembolism (VTE) risk, although the exact incidence and prognostic impact are under-researched in general as well as in relevant age groups. Aims: To study the impact of VTE and major bleeding (MB) in OS and ES patients, subdivided in children, Ad-olescents Young Adults (AYAs; aged 18-39) and older adults. Methods: Retrospective single-center chart review in 519 OS and 165 ES patients treated between 1980 and 2018. Patients were followed from sarcoma diagnosis until an outcome of interest (VTE, MB) or death occurred. Cu-mulative incidences were estimated with death as competing risk. Cox models were used to determine prognostic impact. Results: Five-year cumulative incidences of VTE were 12 % (95%CI 9.1-15) for OS and 6.7 % (95%CI 3.5-11) for ES patients, mostly happening in patients >= 18 years; the most frequent VTE presentation was catheter-related upper-extremity thrombosis (OS: 18/65, ES: 7/11). Five-year cumulative incidences for MB were 5.8 % (95% CI 4.0-8.1) in OS and 5.4 % (95%CI 2.5-9.8) in ES patients. 192 OS and 77 ES AYAs were included, who faced similar VTE and MB incidences as older adults. In OS, VTE and MB were both associated with mortality (adjusted HRs 2.0 [95%CI 1.4-2.9] and 2.4 [95%CI 1.4-4.0], respectively), whereas in ES this association was only present for MB (aHR 3.4 [95%CI 1.2-9.6]). Conclusions: VTE is a frequent complication in adult OS and to a lesser extent in ES patients, while the rate of MB was comparably high in both sarcoma types.
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- 2023
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45. Sunitinib-induced hypertension in CYP3A4 rs4646437 A-allele carriers with metastatic renal cell carcinoma
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Diekstra, M H, Belaustegui, A, Swen, J J, Boven, E, Castellano, D, Gelderblom, H, Mathijssen, R H, García-Donas, J, Rodríguez-Antona, C, Rini, B I, and Guchelaar, H-J
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- 2017
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46. Intradermal vaccination of HPV-16 E6 synthetic peptides conjugated to an optimized Toll-like receptor 2 ligand shows safety and potent T cell immunogenicity in patients with HPV-16 positive (pre-)malignant lesions
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Speetjens, F.M., Welters, M.J.P., Slingerland, M., Poelgeest, M.I.E. van, Steenwijk, P.J.D. van, Roozen, I., Boekestijn, S., Loof, N.M., Zom, G.G., Valentijn, A.R.P.M., Krebber, W.J., Meeuwenoord, N.J., Janssen, C.A.H., Melief, C.J.M., Marel, G.A. van der, Filippov, D.V., Burg, S.H. van der, Gelderblom, H., Ossendorp, F., Obstetrie & Gynaecologie, MUMC+: MA Medische Staf Obstetrie Gynaecologie (9), and RS: GROW - R2 - Basic and Translational Cancer Biology
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Inflammation ,Pharmacology ,Human papillomavirus 16 ,Cancer Research ,Immunodominant Epitopes ,T-Lymphocytes ,Papillomavirus Infections ,Vaccination ,Immunology ,Uterine Cervical Neoplasms ,Ligands ,Cancer Vaccines ,Toll-Like Receptor 2 ,Oncology ,Inflammation/drug therapy ,Humans ,Molecular Medicine ,Immunology and Allergy ,Female ,Papillomavirus Vaccines ,Peptides - Abstract
BackgroundAmplivant is a molecularly optimized Toll-like receptor 2 ligand that can be covalently conjugated to tumor peptide antigens. In preclinical models, amplivant-adjuvanted synthetic long peptides (SLPs) strongly enhanced antigen presentation by dendritic cells, T cell priming and induction of effective antitumor responses. The current study is a first-in-human trial to investigate safety and immunogenicity of amplivant conjugated to human papillomavirus (HPV) 16-SLP.MethodsA dose escalation phase I vaccination trial was performed in 25 patients treated for HPV16 positive (pre-)malignant lesions. Amplivant was conjugated to two SLPs derived from the two most immunodominant regions of the HPV16 E6 oncoprotein. The vaccine, containing a mix of these two conjugates in watery solution without any other formulation, was injected intradermally three times with a 3-week interval in four dose groups (1, 5, 20 or 50 µg per conjugated peptide). Safety data were collected during the study. Peptide-specific T cell immune responses were determined in blood samples taken before, during and after vaccination using complementary immunological assays.ResultsToxicity after three amplivant-conjugated HPV16-SLP vaccinations was limited to grade 1 or 2, observed as predominantly mild skin inflammation at the vaccination site and sometimes mild flu-like symptoms. Adverse events varied from none in the lowest dose group to mild/moderate vaccine-related inflammation in all patients and flu-like symptoms in three out of seven patients in the highest dose group, after at least one injection. In the lowest dose group, vaccine-induced T cell responses were observed in the blood of three out of six vaccinated persons. In the highest dose group, all patients displayed a strong HPV16-specific T cell response after vaccination. These HPV16-specific T cell responses lasted until the end of the trial.ConclusionsAmplivant-conjugated SLPs can safely be used as an intradermal therapeutic vaccine to induce robust HPV16-specific T cell immunity in patients previously treated for HPV16 positive (pre-) malignancies. Increased vaccine dose was associated with a higher number of mild adverse events and with stronger systemic T cell immunity.Trial registration numbersNCT02821494and 2014-000658-12.
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- 2022
47. P63 Patient-Reported Outcomes Following Treatment with Vimseltinib for Tenosynovial Giant Cell Tumour in a Phase 2 Expansion Study
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Blay, JY, primary, Gelderblom, H, additional, Rutkowski, P, additional, Wagner, AJ, additional, van de Sande, M, additional, Stacchiotti, S, additional, Le Cesne, A, additional, Palmerini, E, additional, Vallee, M, additional, Harrow, B, additional, Becker, C, additional, Jarecha, R, additional, Sharma, M, additional, and Tap, WD, additional
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- 2022
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48. P61 Health State Utility Values and Quality of Life in Patients Receiving Ripretinib in the Phase 3 Invictus Trial and a Real-World Evidence Study in China
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Jones, RL, primary, Blay, JY, additional, Chi, P, additional, Bauer, S, additional, Gelderblom, H, additional, Shen, L, additional, He, YL, additional, Heinrich, MC, additional, Schöffski, P, additional, Zalcberg, JR, additional, Harrow, B, additional, Sherman, ML, additional, Ruiz-Soto, R, additional, Becker, C, additional, and von Mehren, M, additional
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- 2022
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49. Therapeutic drug monitoring-based precision dosing of oral targeted therapies in oncology: a prospective multicenter study
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Groenland, S.L., primary, van Eerden, R.A.G., additional, Westerdijk, K., additional, Meertens, M., additional, Koolen, S.L.W., additional, Moes, D.J.A.R., additional, de Vries, N., additional, Rosing, H., additional, Otten, H., additional, Vulink, A.J.E., additional, Desar, I.M.E., additional, Imholz, A.L.T., additional, Gelderblom, H., additional, van Erp, N.P., additional, Beijnen, J.H., additional, Mathijssen, R.H.J., additional, Huitema, A.D.R., additional, and Steeghs, N., additional
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- 2022
- Full Text
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50. Long-term follow-up of nilotinib in patients with advanced tenosynovial giant cell tumours long-term follow-up of nilotinib in TGCT
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Spierenburg, G., Grimison, P., Chevreau, C., Stacchiotti, S., Piperno-Neumann, S., Cesne, A. le, Ferraresi, V., Italiano, A., Duffaud, F., Penel, N., Metzger, S., Chabaud, S., Heijden, L. van der, Perol, D., Sande, M.A.J. van de, Blay, J.Y., and Gelderblom, H.
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PVNS ,TGCT ,CSF1 ,Tyrosine kinase inhibitor ,Nilotinib ,CSF1R inhibitor - Abstract
Background: Diffuse-type tenosynovial giant cell tumour (D-TGCT) is a nonmalignant but locally aggressive tumour driven by overexpression of colony-stimulating factor-1 (CSF1). CSF1R inhibitors are potential therapeutic strategies for patients not amenable to surgery. We report here the long-term outcome of nilotinib in patients with advanced D-TGCT treated within a phase II prospective international study (ClinicalTrials.gov: NCT01261429). Methods: Patients were enrolled between December 2010-September 2012 at 11 cancer centres. Eligible patients had histologically confirmed D-TGCT, not amenable to surgery. Patients received nilotinib until evidence of progression, toxicity or a maximum of one year. Long-term data were retrospectively collected after the completion of the phase II trial. Patients with nilotinib treatment >= 12 weeks and follow-up >= 12 months were included for long-term analysis. Results: Forty-eight of 56 enrolled patients were included. Median treatment duration was 11 months; 31 (65%) patients completed the treatment protocol. After 102 months of follow-up (median; range 12-129), 25 patients (52%) had progression. The median progression-free survival (PFS) was 77 months. The five-year PFS rate was 53%. Fifteen patients (n=15/46; 33%) experienced clinical worsening after 11 months (median). Twenty-seven patients (58%) received additional treatment, after which eleven patients (n = 11/27; 41%) had a second relapse. Nine patients required a subsequent treatment, primarily other CSF1R inhibitors (n = 6/9; 67%). No unfavourable long-term effects were observed. Conclusion: This long-term analysis of nilotinib for advanced D-TGCT showed that about half of the patients had progression and underwent additional treatment after 8.5 years follow-up. Contrarily, several patients had ongoing disease control after limited treatment duration, demonstrating the mixed effect of nilotinib.
- Published
- 2022
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