Skip to main content
Erschienen in: Cancer and Metastasis Reviews 1/2020

15.01.2020

Clinical research tools in pediatric oncology: challenges and opportunities

verfasst von: Teresa de Rojas, Anouk Neven, Alexander J. Towbin, Fernando Carceller, Francisco Bautista, David Riedl, Samantha Sodergren, Anne-Sophie Darlington, Ana Fernandez-Teijeiro, Lucas Moreno

Erschienen in: Cancer and Metastasis Reviews | Ausgabe 1/2020

Einloggen, um Zugang zu erhalten

Abstract

Survival for childhood cancers has improved significantly over the last decades. However, patient outcomes have plateaued over the last decade for difficult-to-treat diseases. With high cure rates, decreasing long-term toxicities and sequelae remains crucial. Since many advances in childhood cancer research come from the adult oncology world, one of the key areas is improving the adaptation of tools that are essential for clinical trial conduct that were developed for adults into pediatrics. These include tools to evaluate toxicity, quality of life, radiological response, statistical methodology, or indicators of cancer care quality. In this review, we present ongoing international efforts to validate and adapt these tools for children and adolescents and discuss remaining challenges. These efforts will hopefully accelerate and improve the quality of pediatric oncology research in the upcoming years.
Literatur
2.
Zurück zum Zitat Trama, A., Botta, L., Foschi, R., Ferrari, A., Stiller, C., Desandes, E., Maule, M. M., Merletti, F., Gatta, G., & EUROCARE-5 Working Group. (2016). Survival of European adolescents and young adults diagnosed with cancer in 2000-07: Population-based data from EUROCARE-5. The Lancet. Oncology, 17(7), 896–906. https://doi.org/10.1016/S1470-2045(16)00162-5.CrossRefPubMed Trama, A., Botta, L., Foschi, R., Ferrari, A., Stiller, C., Desandes, E., Maule, M. M., Merletti, F., Gatta, G., & EUROCARE-5 Working Group. (2016). Survival of European adolescents and young adults diagnosed with cancer in 2000-07: Population-based data from EUROCARE-5. The Lancet. Oncology, 17(7), 896–906. https://​doi.​org/​10.​1016/​S1470-2045(16)00162-5.CrossRefPubMed
3.
Zurück zum Zitat Noone AM, Howlader N, Krapcho M, Miller D, Brest A, Yu M, Ruhl J, Tatalovich Z, Mariotto A, Lewis DR, Chen HS, Feuer EJ, C. K. (eds). (2018). SEER Cancer Statistics Review, 1975–2015, National Cancer Institute. Retrieved September 3, 2018, from https://seer.cancer.gov/csr/1975_2015/ Noone AM, Howlader N, Krapcho M, Miller D, Brest A, Yu M, Ruhl J, Tatalovich Z, Mariotto A, Lewis DR, Chen HS, Feuer EJ, C. K. (eds). (2018). SEER Cancer Statistics Review, 1975–2015, National Cancer Institute. Retrieved September 3, 2018, from https://​seer.​cancer.​gov/​csr/​1975_​2015/​
7.
Zurück zum Zitat Vassal, G., Rousseau, R., Blanc, P., Moreno, L., Bode, G., Schwoch, S., et al. (2015). Creating a unique, multi-stakeholder Paediatric Oncology Platform to improve drug development for children and adolescents with cancer. European journal of cancer (Oxford, England : 1990), 51(2), 218–224. https://doi.org/10.1016/j.ejca.2014.10.029.CrossRef Vassal, G., Rousseau, R., Blanc, P., Moreno, L., Bode, G., Schwoch, S., et al. (2015). Creating a unique, multi-stakeholder Paediatric Oncology Platform to improve drug development for children and adolescents with cancer. European journal of cancer (Oxford, England : 1990), 51(2), 218–224. https://​doi.​org/​10.​1016/​j.​ejca.​2014.​10.​029.CrossRef
10.
Zurück zum Zitat Vassal, G., Fitzgerald, E., Schrappe, M., Arnold, F., Kowalczyk, J., Walker, D., et al. (2014). Challenges for children and adolescents with cancer in Europe: The SIOP-Europe agenda. Pediatric Blood & Cancer. https://doi.org/10.1002/pbc.25044. Vassal, G., Fitzgerald, E., Schrappe, M., Arnold, F., Kowalczyk, J., Walker, D., et al. (2014). Challenges for children and adolescents with cancer in Europe: The SIOP-Europe agenda. Pediatric Blood & Cancer. https://​doi.​org/​10.​1002/​pbc.​25044.
14.
Zurück zum Zitat Aaronson, N. K., Ahmedzai, S., Bergman, B., Bullinger, M., Cull, A., Duez, N. J., et al. (1993). The European Organisation for Research and Treatment of Cancer QLQ-C30: A quality-of-life instrument for use in international clinical trials in oncology. Journal of the National Cancer Institute, 85, 365–376. https://doi.org/10.1093/jnci/85.5.365.CrossRefPubMed Aaronson, N. K., Ahmedzai, S., Bergman, B., Bullinger, M., Cull, A., Duez, N. J., et al. (1993). The European Organisation for Research and Treatment of Cancer QLQ-C30: A quality-of-life instrument for use in international clinical trials in oncology. Journal of the National Cancer Institute, 85, 365–376. https://​doi.​org/​10.​1093/​jnci/​85.​5.​365.CrossRefPubMed
15.
Zurück zum Zitat Therasse, P., Arbuck, S. G., Eisenhauer, E. A., Wanders, J., Kaplan, R. S., Rubinstein, L., et al. (2000). New guidelines to evaluate the response to treatment in solid tumors. European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada. Journal of the National Cancer Institute, 92(3), 205–216 Retrieved from http://www.ncbi.nlm.nih.gov/pubmed/10655437.CrossRef Therasse, P., Arbuck, S. G., Eisenhauer, E. A., Wanders, J., Kaplan, R. S., Rubinstein, L., et al. (2000). New guidelines to evaluate the response to treatment in solid tumors. European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada. Journal of the National Cancer Institute, 92(3), 205–216 Retrieved from http://​www.​ncbi.​nlm.​nih.​gov/​pubmed/​10655437.CrossRef
16.
Zurück zum Zitat Eisenhauer, E. A., Therasse, P., Bogaerts, J., Schwartz, L. H., Sargent, D., Ford, R., Dancey, J., Arbuck, S., Gwyther, S., Mooney, M., Rubinstein, L., Shankar, L., Dodd, L., Kaplan, R., Lacombe, D., & Verweij, J. (2009). New response evaluation criteria in solid tumours: Revised RECIST guideline (version 1.1). European Journal of Cancer, 45(2), 228–247. https://doi.org/10.1016/j.ejca.2008.10.026.CrossRefPubMed Eisenhauer, E. A., Therasse, P., Bogaerts, J., Schwartz, L. H., Sargent, D., Ford, R., Dancey, J., Arbuck, S., Gwyther, S., Mooney, M., Rubinstein, L., Shankar, L., Dodd, L., Kaplan, R., Lacombe, D., & Verweij, J. (2009). New response evaluation criteria in solid tumours: Revised RECIST guideline (version 1.1). European Journal of Cancer, 45(2), 228–247. https://​doi.​org/​10.​1016/​j.​ejca.​2008.​10.​026.CrossRefPubMed
17.
Zurück zum Zitat de Rojas, T., Bautista, F. J., Madero, L., & Moreno, L. (2016). The first step to integrating adapted common terminology criteria for adverse events for children. Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology, 34(18), 2196–2197. https://doi.org/10.1200/JCO.2016.67.7104.CrossRef de Rojas, T., Bautista, F. J., Madero, L., & Moreno, L. (2016). The first step to integrating adapted common terminology criteria for adverse events for children. Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology, 34(18), 2196–2197. https://​doi.​org/​10.​1200/​JCO.​2016.​67.​7104.CrossRef
18.
Zurück zum Zitat Schmiegelow, K., Attarbaschi, A., Barzilai, S., Escherich, G., Frandsen, T. L., Halsey, C., et al. (2016). Consensus definitions of 14 severe acute toxic effects for childhood lymphoblastic leukaemia treatment: A Delphi consensus. The Lancet Oncology. https://doi.org/10.1016/S1470-2045(16)30035-3. Schmiegelow, K., Attarbaschi, A., Barzilai, S., Escherich, G., Frandsen, T. L., Halsey, C., et al. (2016). Consensus definitions of 14 severe acute toxic effects for childhood lymphoblastic leukaemia treatment: A Delphi consensus. The Lancet Oncology. https://​doi.​org/​10.​1016/​S1470-2045(16)30035-3.
26.
Zurück zum Zitat Carceller, F., Bautista, F. J., Fowkes, L. A., Marshall, L. V., Sirvent, S. I., Chisholm, J. C., Pearson, A. D., Koh, D. M., & Moreno, L. (2016). Response assessment in paediatric phase I trials according to RECIST guidelines: Survival outcomes, patterns of progression and relevance of changes in tumour measurements. Pediatric Blood & Cancer, 63(8), 1400–1406. https://doi.org/10.1002/pbc.26039.CrossRef Carceller, F., Bautista, F. J., Fowkes, L. A., Marshall, L. V., Sirvent, S. I., Chisholm, J. C., Pearson, A. D., Koh, D. M., & Moreno, L. (2016). Response assessment in paediatric phase I trials according to RECIST guidelines: Survival outcomes, patterns of progression and relevance of changes in tumour measurements. Pediatric Blood & Cancer, 63(8), 1400–1406. https://​doi.​org/​10.​1002/​pbc.​26039.CrossRef
30.
Zurück zum Zitat Weeda, V. B., Aronson, D. C., Verheij, J., & Lamers, W. H. (2019). Is hepatocellular carcinoma the same disease in children and adults? Comparison of histology, molecular background, and treatment in pediatric and adult patients. Pediatric Blood & Cancer, 66(2), e27475. https://doi.org/10.1002/pbc.27475.CrossRef Weeda, V. B., Aronson, D. C., Verheij, J., & Lamers, W. H. (2019). Is hepatocellular carcinoma the same disease in children and adults? Comparison of histology, molecular background, and treatment in pediatric and adult patients. Pediatric Blood & Cancer, 66(2), e27475. https://​doi.​org/​10.​1002/​pbc.​27475.CrossRef
31.
Zurück zum Zitat Ferrari, A., Miceli, R., Meazza, C., Zaffignani, E., Gronchi, A., Piva, L., Collini, P., Podda, M., Massimino, M., Luksch, R., Cefalo, G., Terenziani, M., Spreafico, F., Polastri, D., Fossati-Bellani, F., Casanova, M., & Mariani, L. (2009). Soft tissue sarcomas of childhood and adolescence: The prognostic role of tumor size in relation to patient body size. Journal of Clinical Oncology, 27(3), 371–376. https://doi.org/10.1200/JCO.2007.15.4542.CrossRefPubMed Ferrari, A., Miceli, R., Meazza, C., Zaffignani, E., Gronchi, A., Piva, L., Collini, P., Podda, M., Massimino, M., Luksch, R., Cefalo, G., Terenziani, M., Spreafico, F., Polastri, D., Fossati-Bellani, F., Casanova, M., & Mariani, L. (2009). Soft tissue sarcomas of childhood and adolescence: The prognostic role of tumor size in relation to patient body size. Journal of Clinical Oncology, 27(3), 371–376. https://​doi.​org/​10.​1200/​JCO.​2007.​15.​4542.CrossRefPubMed
32.
Zurück zum Zitat Nguyen, R., McCarville, M. B., Sykes, A., Mao, S., Wu, J., Langham, M. R., & Furman, W. L. (2018). Rapid decrease of serum alpha-fetoprotein and tumor volume predicts outcome in children with hepatoblastoma treated with neoadjuvant chemotherapy. International Journal of Clinical Oncology, 23, 900–907. https://doi.org/10.1007/s10147-018-1285-4.CrossRefPubMed Nguyen, R., McCarville, M. B., Sykes, A., Mao, S., Wu, J., Langham, M. R., & Furman, W. L. (2018). Rapid decrease of serum alpha-fetoprotein and tumor volume predicts outcome in children with hepatoblastoma treated with neoadjuvant chemotherapy. International Journal of Clinical Oncology, 23, 900–907. https://​doi.​org/​10.​1007/​s10147-018-1285-4.CrossRefPubMed
33.
Zurück zum Zitat O’Neill, A. F., Towbin, A. J., Krailo, M. D., Xia, C., Gao, Y., McCarville, M. B., et al. (2017). Characterization of pulmonary metastases in children with hepatoblastoma treated on Children’s Oncology Group Protocol AHEP0731 (the treatment of children with all stages of hepatoblastoma): A report from the Children’s Oncology Group. Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology, 35(30), 3465–3473. https://doi.org/10.1200/JCO.2017.73.5654.CrossRef O’Neill, A. F., Towbin, A. J., Krailo, M. D., Xia, C., Gao, Y., McCarville, M. B., et al. (2017). Characterization of pulmonary metastases in children with hepatoblastoma treated on Children’s Oncology Group Protocol AHEP0731 (the treatment of children with all stages of hepatoblastoma): A report from the Children’s Oncology Group. Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology, 35(30), 3465–3473. https://​doi.​org/​10.​1200/​JCO.​2017.​73.​5654.CrossRef
34.
Zurück zum Zitat Schoot, R. A., McHugh, K., van Rijn, R. R., Kremer, L. C. M., Chisholm, J. C., Caron, H. N., & Merks, J. H. M. (2013). Response assessment in pediatric rhabdomyosarcoma: Can Response Evaluation Criteria in Solid Tumors replace three-dimensional volume assessments? Radiology, 269(3), 870–878. https://doi.org/10.1148/radiol.13122607.CrossRefPubMed Schoot, R. A., McHugh, K., van Rijn, R. R., Kremer, L. C. M., Chisholm, J. C., Caron, H. N., & Merks, J. H. M. (2013). Response assessment in pediatric rhabdomyosarcoma: Can Response Evaluation Criteria in Solid Tumors replace three-dimensional volume assessments? Radiology, 269(3), 870–878. https://​doi.​org/​10.​1148/​radiol.​13122607.CrossRefPubMed
36.
Zurück zum Zitat Guenther, L. M., Rowe, R. G., Acharya, P. T., Swenson, D. W., Meyer, S. C., Clinton, C. M., et al. (2017). Response Evaluation Criteria in Solid Tumors (RECIST) following neoadjuvant chemotherapy in osteosarcoma. Pediatric Blood & Cancer, e26896. https://doi.org/10.1002/pbc.26896. Guenther, L. M., Rowe, R. G., Acharya, P. T., Swenson, D. W., Meyer, S. C., Clinton, C. M., et al. (2017). Response Evaluation Criteria in Solid Tumors (RECIST) following neoadjuvant chemotherapy in osteosarcoma. Pediatric Blood & Cancer, e26896. https://​doi.​org/​10.​1002/​pbc.​26896.
37.
Zurück zum Zitat Park, J. R., Bagatell, R., Cohn, S. L., Pearson, A. D., Villablanca, J. G., Berthold, F., Burchill, S., Boubaker, A., McHugh, K., Nuchtern, J. G., London, W. B., Seibel, N. L., Lindwasser, O. W., Maris, J. M., Brock, P., Schleiermacher, G., Ladenstein, R., Matthay, K. K., & Valteau-Couanet, D. (2017). Revisions to the international neuroblastoma response criteria: A consensus statement from the National Cancer Institute Clinical Trials Planning Meeting. Journal of Clinical Oncology, 35(22), 2580–2587. https://doi.org/10.1200/JCO.2016.72.0177.CrossRefPubMedPubMedCentral Park, J. R., Bagatell, R., Cohn, S. L., Pearson, A. D., Villablanca, J. G., Berthold, F., Burchill, S., Boubaker, A., McHugh, K., Nuchtern, J. G., London, W. B., Seibel, N. L., Lindwasser, O. W., Maris, J. M., Brock, P., Schleiermacher, G., Ladenstein, R., Matthay, K. K., & Valteau-Couanet, D. (2017). Revisions to the international neuroblastoma response criteria: A consensus statement from the National Cancer Institute Clinical Trials Planning Meeting. Journal of Clinical Oncology, 35(22), 2580–2587. https://​doi.​org/​10.​1200/​JCO.​2016.​72.​0177.CrossRefPubMedPubMedCentral
42.
Zurück zum Zitat Reeve, B. B., McFatrich, M., Pinheiro, L. C., Weaver, M. S., Sung, L., Withycombe, J. S., Baker, J. N., Mack, J. W., Waldron, M. K., Gibson, D., Tomlinson, D., Freyer, D. R., Mowbray, C., Jacobs, S., Palma, D., Martens, C. E., Gold, S. H., Jackson, K. D., & Hinds, P. S. (2017). Eliciting the child’s voice in adverse event reporting in oncology trials: Cognitive interview findings from the Pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events initiative. Pediatric Blood & Cancer, 64(3), e26261. https://doi.org/10.1002/pbc.26261.CrossRef Reeve, B. B., McFatrich, M., Pinheiro, L. C., Weaver, M. S., Sung, L., Withycombe, J. S., Baker, J. N., Mack, J. W., Waldron, M. K., Gibson, D., Tomlinson, D., Freyer, D. R., Mowbray, C., Jacobs, S., Palma, D., Martens, C. E., Gold, S. H., Jackson, K. D., & Hinds, P. S. (2017). Eliciting the child’s voice in adverse event reporting in oncology trials: Cognitive interview findings from the Pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events initiative. Pediatric Blood & Cancer, 64(3), e26261. https://​doi.​org/​10.​1002/​pbc.​26261.CrossRef
43.
Zurück zum Zitat Gipson, D. S., Kirkendall, E. S., Gumbs-Petty, B., Quinn, T., Steen, A., Hicks, A., McMahon, A., Nicholas, S., Zhao-Wong, A., Taylor-Zapata, P., Turner, M., Herreshoff, E., Jones, C., Davis, J. M., Haber, M., & Hirschfeld, S. (2017). Development of a pediatric adverse events terminology. Pediatrics, 139(1). https://doi.org/10.1542/peds.2016-0985. Gipson, D. S., Kirkendall, E. S., Gumbs-Petty, B., Quinn, T., Steen, A., Hicks, A., McMahon, A., Nicholas, S., Zhao-Wong, A., Taylor-Zapata, P., Turner, M., Herreshoff, E., Jones, C., Davis, J. M., Haber, M., & Hirschfeld, S. (2017). Development of a pediatric adverse events terminology. Pediatrics, 139(1). https://​doi.​org/​10.​1542/​peds.​2016-0985.
47.
Zurück zum Zitat Heath, J. A., Clarke, N. E., Donath, S. M., McCarthy, M., Anderson, V. A., & Wolfe, J. (2010). Symptoms and suffering at the end of life in children with cancer: An Australian perspective. The Medical Journal of Australia, 192(2), 71–75 Retrieved from http://www.ncbi.nlm.nih.gov/pubmed/20078405 .CrossRef Heath, J. A., Clarke, N. E., Donath, S. M., McCarthy, M., Anderson, V. A., & Wolfe, J. (2010). Symptoms and suffering at the end of life in children with cancer: An Australian perspective. The Medical Journal of Australia, 192(2), 71–75 Retrieved from http://www.ncbi.nlm.nih.gov/pubmed/20078405 .CrossRef
49.
Zurück zum Zitat Matza, L. S., Patrick, D. L., Riley, A. W., Alexander, J. J., Rajmil, L., Pleil, A. M., & Bullinger, M. (2013). Pediatric patient-reported outcome instruments for research to support medical product labeling: Report of the ISPOR PRO good research practices for the assessment of children and adolescents task force. Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research, 16(4), 461–479. https://doi.org/10.1016/j.jval.2013.04.004.CrossRef Matza, L. S., Patrick, D. L., Riley, A. W., Alexander, J. J., Rajmil, L., Pleil, A. M., & Bullinger, M. (2013). Pediatric patient-reported outcome instruments for research to support medical product labeling: Report of the ISPOR PRO good research practices for the assessment of children and adolescents task force. Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research, 16(4), 461–479. https://​doi.​org/​10.​1016/​j.​jval.​2013.​04.​004.CrossRef
50.
Zurück zum Zitat Pinheiro, L. C., McFatrich, M., Lucas, N., Walker, J. S., Withycombe, J. S., Hinds, P. S., Sung, L., Tomlinson, D., Freyer, D. R., Mack, J. W., Baker, J. N., & Reeve, B. B. (2018). Child and adolescent self-report symptom measurement in pediatric oncology research: A systematic literature review. Quality of Life Research, 27(2), 291–319. https://doi.org/10.1007/s11136-017-1692-4.CrossRefPubMed Pinheiro, L. C., McFatrich, M., Lucas, N., Walker, J. S., Withycombe, J. S., Hinds, P. S., Sung, L., Tomlinson, D., Freyer, D. R., Mack, J. W., Baker, J. N., & Reeve, B. B. (2018). Child and adolescent self-report symptom measurement in pediatric oncology research: A systematic literature review. Quality of Life Research, 27(2), 291–319. https://​doi.​org/​10.​1007/​s11136-017-1692-4.CrossRefPubMed
51.
Zurück zum Zitat Anthony, S. J., Selkirk, E., Sung, L., Klaassen, R. J., Dix, D., Scheinemann, K., & Klassen, A. F. (2014). Considering quality of life for children with cancer: A systematic review of patient-reported outcome measures and the development of a conceptual model. Quality of Life Research, 23(3), 771–789. https://doi.org/10.1007/s11136-013-0482-x.CrossRefPubMed Anthony, S. J., Selkirk, E., Sung, L., Klaassen, R. J., Dix, D., Scheinemann, K., & Klassen, A. F. (2014). Considering quality of life for children with cancer: A systematic review of patient-reported outcome measures and the development of a conceptual model. Quality of Life Research, 23(3), 771–789. https://​doi.​org/​10.​1007/​s11136-013-0482-x.CrossRefPubMed
60.
Zurück zum Zitat Varni, J. W., Limbers, C. A., & Burwinkle, T. M. (2007). How young can children reliably and validly self-report their health-related quality of life? An analysis of 8,591 children across age subgroups with the PedsQL™ 4.0 Generic Core Scales. Health and Quality of Life Outcomes, 5. https://doi.org/10.1186/1477-7525-5-1. Varni, J. W., Limbers, C. A., & Burwinkle, T. M. (2007). How young can children reliably and validly self-report their health-related quality of life? An analysis of 8,591 children across age subgroups with the PedsQL™ 4.0 Generic Core Scales. Health and Quality of Life Outcomes, 5. https://​doi.​org/​10.​1186/​1477-7525-5-1.
61.
Zurück zum Zitat Mokkink, L. B., Terwee, C. B., Knol, D. L., Stratford, P. W., Alonso, J., Patrick, D. L., et al. (2010). The COSMIN checklist for evaluating the methodological quality of studies on measurement properties: A clarification of its content. BMC Medical Research Methodology, 10. https://doi.org/10.1186/1471-2288-10-22. Mokkink, L. B., Terwee, C. B., Knol, D. L., Stratford, P. W., Alonso, J., Patrick, D. L., et al. (2010). The COSMIN checklist for evaluating the methodological quality of studies on measurement properties: A clarification of its content. BMC Medical Research Methodology, 10. https://​doi.​org/​10.​1186/​1471-2288-10-22.
72.
Zurück zum Zitat Sodergren, S. C., Husson, O., Rohde, G. E., Tomasewska, I. M., Griffiths, H., Pessing, A., Yarom, N., Hooker, L., Din, A., Darlington, A. S., EORTC Quality of Life Group, & EORTC Quality of Life Group. (2018). Does age matter? A comparison of health-related quality of life issues of adolescents and young adults with cancer. European Journal of Cancer Care, 27(6), e12980. https://doi.org/10.1111/ecc.12980.CrossRefPubMed Sodergren, S. C., Husson, O., Rohde, G. E., Tomasewska, I. M., Griffiths, H., Pessing, A., Yarom, N., Hooker, L., Din, A., Darlington, A. S., EORTC Quality of Life Group, & EORTC Quality of Life Group. (2018). Does age matter? A comparison of health-related quality of life issues of adolescents and young adults with cancer. European Journal of Cancer Care, 27(6), e12980. https://​doi.​org/​10.​1111/​ecc.​12980.CrossRefPubMed
74.
Zurück zum Zitat Sodergren, S. C., Husson, O., Rohde, G. E., Tomasewska, I. M., Vivat, B., Yarom, N., Griffiths, H., & Darlington, A. S. (2018). A life put on pause: An exploration of the health-related quality of life issues relevant to adolescents and young adults with cancer. Journal of Adolescent and Young Adult Oncology, 7(4), 453–464. https://doi.org/10.1089/jayao.2017.0110.CrossRefPubMed Sodergren, S. C., Husson, O., Rohde, G. E., Tomasewska, I. M., Vivat, B., Yarom, N., Griffiths, H., & Darlington, A. S. (2018). A life put on pause: An exploration of the health-related quality of life issues relevant to adolescents and young adults with cancer. Journal of Adolescent and Young Adult Oncology, 7(4), 453–464. https://​doi.​org/​10.​1089/​jayao.​2017.​0110.CrossRefPubMed
78.
Zurück zum Zitat Hilgers, R.-D., König, F., Molenberghs, G., & Senn, S. (2016). Design and analysis of clinical trials for small rare disease populations. Dis Res Treat, 1 Retrieved from www.rarediseasesjournal.com. Hilgers, R.-D., König, F., Molenberghs, G., & Senn, S. (2016). Design and analysis of clinical trials for small rare disease populations. Dis Res Treat, 1 Retrieved from www.​rarediseasesjour​nal.​com.
81.
Zurück zum Zitat Casali, P. G., Bruzzi, P., Bogaerts, J., Blay, J.-Y., & Rare Cancers Europe (RCE) Consensus Panel. (2015). Rare Cancers Europe (RCE) methodological recommendations for clinical studies in rare cancers: A European consensus position paper. Annals of oncology : official journal of the European Society for Medical Oncology, 26(2), 300–306. https://doi.org/10.1093/annonc/mdu459.CrossRef Casali, P. G., Bruzzi, P., Bogaerts, J., Blay, J.-Y., & Rare Cancers Europe (RCE) Consensus Panel. (2015). Rare Cancers Europe (RCE) methodological recommendations for clinical studies in rare cancers: A European consensus position paper. Annals of oncology : official journal of the European Society for Medical Oncology, 26(2), 300–306. https://​doi.​org/​10.​1093/​annonc/​mdu459.CrossRef
89.
Zurück zum Zitat Kelly, L. E., Dyson, M. P., Butcher, N. J., Balshaw, R., London, A. J., Neilson, C. J., Junker, A., Mahmud, S. M., Driedger, S. M., & Wang, X. (2018). Considerations for adaptive design in pediatric clinical trials: Study protocol for a systematic review, mixed-methods study, and integrated knowledge translation plan. Trials, 19(1), 572. https://doi.org/10.1186/s13063-018-2934-7.CrossRefPubMedPubMedCentral Kelly, L. E., Dyson, M. P., Butcher, N. J., Balshaw, R., London, A. J., Neilson, C. J., Junker, A., Mahmud, S. M., Driedger, S. M., & Wang, X. (2018). Considerations for adaptive design in pediatric clinical trials: Study protocol for a systematic review, mixed-methods study, and integrated knowledge translation plan. Trials, 19(1), 572. https://​doi.​org/​10.​1186/​s13063-018-2934-7.CrossRefPubMedPubMedCentral
91.
Zurück zum Zitat European Medicines Agency. (2017). ICH E11(R1) guideline on clinical investigation of medicinal products in the pediatric population. European Medicines Agency. (2017). ICH E11(R1) guideline on clinical investigation of medicinal products in the pediatric population.
96.
Zurück zum Zitat Molenberghs, G., & Orman, C. (n.d.). Surrogate endpoints: Application in pediatric clinical trials. Molenberghs, G., & Orman, C. (n.d.). Surrogate endpoints: Application in pediatric clinical trials.
98.
Zurück zum Zitat de Rojas, T., Puertas, M., Bautista, F., de Prada, I., López-Pino, M. Á., Rivero, B., Gonzalez-San Segundo, C., Gonzalez-Vicent, M., Lassaletta, A., Madero, L., & Moreno, L. (2019). Improving the quality of care in the molecular era for children and adolescents with medulloblastoma. Clinical & Translational Oncology, 21, 1687–1698. https://doi.org/10.1007/s12094-019-02101-2.CrossRef de Rojas, T., Puertas, M., Bautista, F., de Prada, I., López-Pino, M. Á., Rivero, B., Gonzalez-San Segundo, C., Gonzalez-Vicent, M., Lassaletta, A., Madero, L., & Moreno, L. (2019). Improving the quality of care in the molecular era for children and adolescents with medulloblastoma. Clinical & Translational Oncology, 21, 1687–1698. https://​doi.​org/​10.​1007/​s12094-019-02101-2.CrossRef
104.
Metadaten
Titel
Clinical research tools in pediatric oncology: challenges and opportunities
verfasst von
Teresa de Rojas
Anouk Neven
Alexander J. Towbin
Fernando Carceller
Francisco Bautista
David Riedl
Samantha Sodergren
Anne-Sophie Darlington
Ana Fernandez-Teijeiro
Lucas Moreno
Publikationsdatum
15.01.2020
Verlag
Springer US
Erschienen in
Cancer and Metastasis Reviews / Ausgabe 1/2020
Print ISSN: 0167-7659
Elektronische ISSN: 1573-7233
DOI
https://doi.org/10.1007/s10555-020-09856-z

Weitere Artikel der Ausgabe 1/2020

Cancer and Metastasis Reviews 1/2020 Zur Ausgabe

EditorialNotes

Preface

Update Onkologie

Bestellen Sie unseren Fach-Newsletter und bleiben Sie gut informiert.