Biology-guided precision medicine in rare cancers: Lessons from sarcomas and neuroendocrine tumours
Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparati...
Ausführliche Beschreibung
Gespeichert in:
| Autor*in: |
van der Graaf, W.T.A. [verfasserIn] |
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| Format: |
E-Artikel |
|---|---|
| Sprache: |
Englisch |
| Erschienen: |
2022transfer abstract |
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| Umfang: |
14 |
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| Übergeordnetes Werk: |
Enthalten in: Effect of co-doping of sodium on the thermoluminescence dosimetry properties of copper-doped zinc lithium borate glass system - Saidu, A. ELSEVIER, 2016transfer abstract, London |
|---|---|
| Übergeordnetes Werk: |
volume:84 ; year:2022 ; pages:228-241 ; extent:14 |
| Links: |
|---|
| DOI / URN: |
10.1016/j.semcancer.2022.05.011 |
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| 520 | |a Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. | ||
| 520 | |a Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. | ||
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10.1016/j.semcancer.2022.05.011 doi /cbs_pica/cbs_olc/import_discovery/elsevier/einzuspielen/GBV00000000001815.pica (DE-627)ELV058255869 (ELSEVIER)S1044-579X(22)00122-5 DE-627 ger DE-627 rakwb eng 530 VZ 610 VZ 540 VZ 660 VZ 38.51 bkl 57.36 bkl van der Graaf, W.T.A. verfasserin aut Biology-guided precision medicine in rare cancers: Lessons from sarcomas and neuroendocrine tumours 2022transfer abstract 14 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. Tesselaar, M.E.T. oth McVeigh, T.P. oth Oyen, W.J.G. oth Fröhling, S. oth Enthalten in Academic Press Saidu, A. ELSEVIER Effect of co-doping of sodium on the thermoluminescence dosimetry properties of copper-doped zinc lithium borate glass system 2016transfer abstract London (DE-627)ELV019642644 volume:84 year:2022 pages:228-241 extent:14 https://doi.org/10.1016/j.semcancer.2022.05.011 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U SSG-OLC-PHA SSG-OPC-GGO GBV_ILN_70 GBV_ILN_252 38.51 Geologie fossiler Brennstoffe VZ 57.36 Erdölgewinnung Erdgasgewinnung VZ AR 84 2022 228-241 14 |
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10.1016/j.semcancer.2022.05.011 doi /cbs_pica/cbs_olc/import_discovery/elsevier/einzuspielen/GBV00000000001815.pica (DE-627)ELV058255869 (ELSEVIER)S1044-579X(22)00122-5 DE-627 ger DE-627 rakwb eng 530 VZ 610 VZ 540 VZ 660 VZ 38.51 bkl 57.36 bkl van der Graaf, W.T.A. verfasserin aut Biology-guided precision medicine in rare cancers: Lessons from sarcomas and neuroendocrine tumours 2022transfer abstract 14 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. Tesselaar, M.E.T. oth McVeigh, T.P. oth Oyen, W.J.G. oth Fröhling, S. oth Enthalten in Academic Press Saidu, A. ELSEVIER Effect of co-doping of sodium on the thermoluminescence dosimetry properties of copper-doped zinc lithium borate glass system 2016transfer abstract London (DE-627)ELV019642644 volume:84 year:2022 pages:228-241 extent:14 https://doi.org/10.1016/j.semcancer.2022.05.011 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U SSG-OLC-PHA SSG-OPC-GGO GBV_ILN_70 GBV_ILN_252 38.51 Geologie fossiler Brennstoffe VZ 57.36 Erdölgewinnung Erdgasgewinnung VZ AR 84 2022 228-241 14 |
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10.1016/j.semcancer.2022.05.011 doi /cbs_pica/cbs_olc/import_discovery/elsevier/einzuspielen/GBV00000000001815.pica (DE-627)ELV058255869 (ELSEVIER)S1044-579X(22)00122-5 DE-627 ger DE-627 rakwb eng 530 VZ 610 VZ 540 VZ 660 VZ 38.51 bkl 57.36 bkl van der Graaf, W.T.A. verfasserin aut Biology-guided precision medicine in rare cancers: Lessons from sarcomas and neuroendocrine tumours 2022transfer abstract 14 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. Tesselaar, M.E.T. oth McVeigh, T.P. oth Oyen, W.J.G. oth Fröhling, S. oth Enthalten in Academic Press Saidu, A. ELSEVIER Effect of co-doping of sodium on the thermoluminescence dosimetry properties of copper-doped zinc lithium borate glass system 2016transfer abstract London (DE-627)ELV019642644 volume:84 year:2022 pages:228-241 extent:14 https://doi.org/10.1016/j.semcancer.2022.05.011 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U SSG-OLC-PHA SSG-OPC-GGO GBV_ILN_70 GBV_ILN_252 38.51 Geologie fossiler Brennstoffe VZ 57.36 Erdölgewinnung Erdgasgewinnung VZ AR 84 2022 228-241 14 |
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10.1016/j.semcancer.2022.05.011 doi /cbs_pica/cbs_olc/import_discovery/elsevier/einzuspielen/GBV00000000001815.pica (DE-627)ELV058255869 (ELSEVIER)S1044-579X(22)00122-5 DE-627 ger DE-627 rakwb eng 530 VZ 610 VZ 540 VZ 660 VZ 38.51 bkl 57.36 bkl van der Graaf, W.T.A. verfasserin aut Biology-guided precision medicine in rare cancers: Lessons from sarcomas and neuroendocrine tumours 2022transfer abstract 14 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. Tesselaar, M.E.T. oth McVeigh, T.P. oth Oyen, W.J.G. oth Fröhling, S. oth Enthalten in Academic Press Saidu, A. ELSEVIER Effect of co-doping of sodium on the thermoluminescence dosimetry properties of copper-doped zinc lithium borate glass system 2016transfer abstract London (DE-627)ELV019642644 volume:84 year:2022 pages:228-241 extent:14 https://doi.org/10.1016/j.semcancer.2022.05.011 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U SSG-OLC-PHA SSG-OPC-GGO GBV_ILN_70 GBV_ILN_252 38.51 Geologie fossiler Brennstoffe VZ 57.36 Erdölgewinnung Erdgasgewinnung VZ AR 84 2022 228-241 14 |
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10.1016/j.semcancer.2022.05.011 doi /cbs_pica/cbs_olc/import_discovery/elsevier/einzuspielen/GBV00000000001815.pica (DE-627)ELV058255869 (ELSEVIER)S1044-579X(22)00122-5 DE-627 ger DE-627 rakwb eng 530 VZ 610 VZ 540 VZ 660 VZ 38.51 bkl 57.36 bkl van der Graaf, W.T.A. verfasserin aut Biology-guided precision medicine in rare cancers: Lessons from sarcomas and neuroendocrine tumours 2022transfer abstract 14 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. Tesselaar, M.E.T. oth McVeigh, T.P. oth Oyen, W.J.G. oth Fröhling, S. oth Enthalten in Academic Press Saidu, A. ELSEVIER Effect of co-doping of sodium on the thermoluminescence dosimetry properties of copper-doped zinc lithium borate glass system 2016transfer abstract London (DE-627)ELV019642644 volume:84 year:2022 pages:228-241 extent:14 https://doi.org/10.1016/j.semcancer.2022.05.011 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U SSG-OLC-PHA SSG-OPC-GGO GBV_ILN_70 GBV_ILN_252 38.51 Geologie fossiler Brennstoffe VZ 57.36 Erdölgewinnung Erdgasgewinnung VZ AR 84 2022 228-241 14 |
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Biology-guided precision medicine in rare cancers: Lessons from sarcomas and neuroendocrine tumours |
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Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. |
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Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. |
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Rare cancers, which collectively account for almost 25 % of all malignancies, are poorly understood in terms of their aetiology and pathogenesis and are infrequently the focus of translational and clinical research to improve their diagnosis and treatment. Consequently, those affected have comparatively few treatment options, and their prognosis is worse than that of patients with more common entities. Here we review two relevant groups of rare cancers, bone and soft-tissue sarcomas and neuroendocrine tumours (NET), to illustrate recent efforts towards individualised, biology-guided clinical management to improve long-term outcomes. Specifically, we address how comprehensive, multi-layered molecular analyses, including the assessment of predisposing hereditary factors, and innovative imaging approaches can improve the diagnosis of these diseases, allow for better prognostic assessment, and provide new targets for pharmacologic and, in the case of NET, nuclear medicine interventions, whose clinical value must be determined in controlled trials optimally tailored to the particular patient population most likely to benefit. Furthermore, we describe the importance of multidisciplinary collaboration in dedicated reference centres for rare cancers and the increasingly acknowledged potential of networking across institutions at a national and international level. Finally, we illustrate the value of a learning health system based on the systematic collection and sharing of the biological and clinical profiles of patients with rare cancers to achieve continuous cross-fertilisation of scientific and clinical efforts, making the vision of stratified precision medicine in these long-overlooked diseases a reality. |
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