How to manage symptoms in pediatric cancer patients enrolled in clinical trials? A review of principal patient-reported outcome screening tools
Keywords:Cancer symptoms, Childhood cancers, Outcome screening tools, Patient-reported outcomes, Pediatrics
Background: In adult oncology, the practice of tracking symptoms and toxicities using patient-reported outcomes (PROs) has increased and correlates with increased survival. In contrast, symptom monitoring using PROs is not common in pediatric oncology. Only in the last couple of years attention has also been paid to the patient’s perception in pediatrics and listening to the voice of children and to making them participate in the treatment.
Methods: A comprehensive literature search was conducted in MEDLINE/PubMed and PsycINFO to identify relevant articles published through December 2022.
Results: From 58 non-duplicate articles, 33 met our eligibility criteria. Of these, 17 were used in clinical trials.
Conclusions: The dissemination and use of these tools will therefore have surprising repercussions on the control of pain and physical symptoms of small patients as well as on physical and psychological aspects. The administration and use of the PROs ensures optimal use of the drugs currently present in clinical trials by researcher and nurse and aims at a safer and more controlled approval of new drugs.
Ward E, DeSantis C, Robbins A, Kohler B, Jemal A. Childhood and adolescent cancer statistics, 2014. CA Cancer J Clin. 2014;64(2):83-103. https://doi.org/10.3322/caac.21219 PMID:24488779 DOI: https://doi.org/10.3322/caac.21219
AIRTUM Working Group. I numeri del cancro in Italia 2013. Intermedia Editore; 2014. https://www.registri-tumori.it/cms/pubblicazioni/i-numeri-del-cancro-italia-2013. Accessed June 2023.
National Cancer Institute. Surveillance, Epidemiology, and End Results Program. https://seer.cancer.gov/archive/csr/1975_2015/. Accessed June 2023.
Ullrich CK, Billett AL, Berde CB. Symptom Management in Children with Cancer. In: Orkin SH, Nathan DG, Ginsburg D, Look AT, Fisher DE, Lux SE, editors. Nathan and Oski's Hematology of Infancy and Childhood. Elsevier Health Sciences; 2009. pp. 2349–96. https://doi.org/10.1016/B978-1-4160-3431-5.00031-5 DOI: https://doi.org/10.1016/B978-1-4160-3431-5.00031-5
Collins JJ, Byrnes ME, Dunkel IJ, et al. The measurement of symptoms in children with cancer. J Pain Symptom Manage. 2000;19(5):363-377. https://doi.org/10.1016/S0885-3924(00)00127-5 PMID:10869877 DOI: https://doi.org/10.1016/S0885-3924(00)00127-5
Leahy AB, Feudtner C, Basch E. Symptom monitoring in pediatric oncology using patient-reported outcomes: why, how, and where next. Patient. 2018;11(2):147-153. https://doi.org/10.1007/s40271-017-0279-z PMID:29071524 DOI: https://doi.org/10.1007/s40271-017-0279-z
Schepers SA, Haverman L, Zadeh S, Grootenhuis MA, Wiener L. Healthcare professionals’ preferences and perceived barriers for routine assessment of patient-reported outcomes in pediatric oncology practice: moving toward international processes of change. Pediatr Blood Cancer. 2016;63(12):2181-2188. https://doi.org/10.1002/pbc.26135 PMID:27511830 DOI: https://doi.org/10.1002/pbc.26135
Basch E, Deal AM, Dueck AC, et al. Overall survival results of a trial assessing patient-reported outcomes for symptom monitoring during routine cancer treatment. JAMA. 2017;318(2):197-198. https://doi.org/10.1001/jama.2017.7156 PMID:28586821 DOI: https://doi.org/10.1001/jama.2017.7156
Leahy AB, Steineck A. Patient-reported outcomes in pediatric oncology: the patient voice as a gold standard. JAMA Pediatr. 2020;174(11):e202868. https://doi.org/10.1001/jamapediatrics.2020.2868 PMID:32832974 DOI: https://doi.org/10.1001/jamapediatrics.2020.2868
Di Maio M. [Outcomes reported by the patient: a tool for respecting the patient’s rights and for involvement in research]. Recenti Prog Med. 2020 Nov;111(11):685-689. [Article in Italian]. https://doi.org/10.1701/3474.34575. Accessed June 2023.
Pinheiro LC, McFatrich M, Lucas N, et al. Child and adolescent self-report symptom measurement in pediatric oncology research: a systematic literature review. Qual Life Res. 2018;27(2):291-319. https://doi.org/10.1007/s11136-017-1692-4 PMID:28879501 DOI: https://doi.org/10.1007/s11136-017-1692-4
OʼSullivan C, Dupuis LL, Sung L. A review of symptom screening tools in pediatric cancer patients. Curr Opin Oncol. 2015;27(4):285-290. https://doi.org/10.1097/CCO.0000000000000197 PMID:26049266 DOI: https://doi.org/10.1097/CCO.0000000000000197
Cheng L, Wang L, He M, Feng S, Zhu Y, Rodgers C. Perspectives of children, family caregivers, and health professionals about pediatric oncology symptoms: a systematic review. Support Care Cancer. 2018;26(9):2957-2971. https://doi.org/10.1007/s00520-018-4257-3 PMID:29774474 DOI: https://doi.org/10.1007/s00520-018-4257-3
The European Society for Paediatric Oncology. SIOP Europe. www.siope.eu. Accessed June 2023.
Laetsch TW, DuBois SG, Bender JG, Macy ME, Moreno L. Opportunities and challenges in drug development for pediatric cancers. Cancer Discov. 2021;11(3):545-559. https://doi.org/10.1158/2159-8290.CD-20-0779 PMID:33277309 DOI: https://doi.org/10.1158/2159-8290.CD-20-0779
Wiener L, Devine KA, Thompson AL. Advances in pediatric psychooncology. Curr Opin Pediatr. 2020;32(1):41-47. https://doi.org/10.1097/MOP.0000000000000851 PMID:31790025 DOI: https://doi.org/10.1097/MOP.0000000000000851
Reeve BB, McFatrich M, Mack JW, et al. Validity and reliability of the pediatric patient-reported outcomes version of the common terminology criteria for adverse events. J Natl Cancer Inst. 2020;112(11):1143-1152. https://doi.org/10.1093/jnci/djaa016 PMID:31999349 DOI: https://doi.org/10.1093/jnci/djaa016
Ilowite MF, Al-Sayegh H, Ma C, et al. The relationship between household income and patient-reported symptom distress and quality of life in children with advanced cancer: a report from the PediQUEST study. Cancer. 2018;124(19):3934-3941. https://doi.org/10.1002/cncr.31668 PMID:30216416 DOI: https://doi.org/10.1002/cncr.31668
Reeve BB, McFatrich M, Mack JW, et al. Expanding construct validity of established and new PROMIS pediatric measures for children and adolescents receiving cancer treatment. Pediatr Blood Cancer. 2020;67(4):e28160. https://doi.org/10.1002/pbc.28160 PMID:31904157 DOI: https://doi.org/10.1002/pbc.28160
Madden K, Magno Charone M, Mills S, et al. Systematic symptom reporting by pediatric palliative care patients with cancer: a preliminary report. J Palliat Med. 2019;22(8):894-901. https://doi.org/10.1089/jpm.2018.0545 PMID:30759031 DOI: https://doi.org/10.1089/jpm.2018.0545
Dussel V, Orellana L, Soto N, et al. Feasibility of conducting a palliative care randomized controlled trial in children with advanced cancer: assessment of the PediQUEST study. J Pain Symptom Manage. 2015;49(6):1059-1069. https://doi.org/10.1016/j.jpainsymman.2014.12.010 PMID:25640275 DOI: https://doi.org/10.1016/j.jpainsymman.2014.12.010
Macpherson CF, Wang J, DeWalt DA, Stern ED, Jacobs S, Hinds PS. Comparison of legacy fatigue measures with the PROMIS pediatric fatigue short form. Oncol Nurs Forum. 2018;45(1):106-114. https://doi.org/10.1188/18.ONF.106-114 PMID:29251294 DOI: https://doi.org/10.1188/18.ONF.106-114
Buckner TW, Wang J, DeWalt DA, Jacobs S, Reeve BB, Hinds PS. Patterns of symptoms and functional impairments in children with cancer. Pediatr Blood Cancer. 2014;61(7):1282-1288. https://doi.org/10.1002/pbc.25029 PMID:24634396 DOI: https://doi.org/10.1002/pbc.25029
Wang J, Jacobs S, Dewalt DA, Stern E, Gross H, Hinds PS. A longitudinal study of PROMIS pediatric symptom clusters in children undergoing chemotherapy. J Pain Symptom Manage. 2018;55(2):359-367. https://doi.org/10.1016/j.jpainsymman.2017.08.021 PMID:28865867 DOI: https://doi.org/10.1016/j.jpainsymman.2017.08.021
Hinds PS, Wang J, Stern ED, et al. Voices of children and adolescents on phase 1 or phase 2 cancer trials: A new trial endpoint? Cancer. 2017;123(19):3799-3806. https://doi.org/10.1002/cncr.30782 PMID:28581685 DOI: https://doi.org/10.1002/cncr.30782
Hinds PS, Wang J, Cheng YI, et al. PROMIS pediatric measures validated in a longitudinal study design in pediatric oncology. Pediatr Blood Cancer. 2019;66(5):e27606. https://doi.org/10.1002/pbc.27606 PMID:30663254 DOI: https://doi.org/10.1002/pbc.27606
Janic A, Bowden S, Levy S, Stinson J, Dimaras H. Patient-reported outcome measures for retinoblastoma: a scoping review. J Patient Rep Outcomes. 2020;4(1):66. https://doi.org/10.1186/s41687-020-00232-7 PMID:32770435 DOI: https://doi.org/10.1186/s41687-020-00232-7
Murugappan MN, King-Kallimanis BL, Reaman GH, et al. Patient-reported outcomes in pediatric cancer registration trials: a US Food and Drug Administration perspective. J Natl Cancer Inst. 2022 Jan 11;114(1):12-19. https://doi.org/10.1093/jnci/djab087 PMID:33930159 DOI: https://doi.org/10.1093/jnci/djab087
How to Cite
Copyright (c) 2023 Giulia Zucchetti , Celeste Cagnazzo, Sabrina Ciappina, Nicoletta Bertorello, Francesca Resente , Manuela Spadea , Valeria Ceolin, Franca Fagioli
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
Authors contributing to AboutOpen agree to publish their articles under the Creative Commons NonCommercial 4.0 International License (CC BY-NC 4.0), which allows third parties to copy and redistribute the material providing appropriate credit and a link to the license but does not allow to use the material for commercial purposes and to use the material if it has been remixed, transformed or built upon.