Patient-reported outcomes are under-utilised in evaluating supportive therapies in paediatric oncology – A systematic review of clinical trial registries
Patient-reported outcomes are under-utilised in evaluating supportive therapies in paediatric oncology – A systematic review of clinical trial registries
Background: Children with cancer suffer from numerous symptoms and side-effects, making supportive interventions indispensable to improve their quality of life. The gold standard for evaluating the latter is patient-reported outcome (PRO) assessment. This systematic review investigates the current practice of clinical outcome assessment (COA) in clinical trials on supportive interventions. Methods: ClinicalTrials.gov and EudraCT were searched for trials including children and adolescents (≤21 years) with cancer receiving supportive care registered 2007–2020. The use of different types of COAs was analysed, focusing on PRO assessment and the domains measured with PRO measures (PROMs). Associations with trial characteristics were investigated using univariate and multivariable analyses. Results: Of 4789 identified trials, 229 were included. Among them, 44.1 % relied on PROMs, the most commonly used COA. The proportion of trials using PROMs did not significantly differ over time. In the multivariable analysis, intervention type (higher PROM use in behavioural vs. medical interventional trials) and cancer type (higher PROM use in mixed and solid tumour samples vs. haematological samples) were significant predictors of PROM use. The majority of trials using PROMs (59.6 %) measured more than one health domain. ‘Physical health’ was the most frequently assessed domain (92.6 %). Conclusion: Less than half of registered clinical trials investigating supportive interventions for children with cancer used PROMs. This result is striking since supportive care explicitly focuses on patients’ quality of life, which is best assessed using PROMs. Our systematic review underlines the need to identify barriers for PROM implementation and to improve PRO research in paediatric oncology.
Childhood cancer, Clinicaltrials.gov, EudraCT, Paediatric oncology, Patient-reported outcomes, Supportive cancer care, Trial registries
Rothmund, Maria
874c229d-a404-4eff-9281-d9ad7eb2c902
Lehmann, Jens
c5d25e2d-62c7-45f5-9d6a-b5eb4ab361d4
Moser, Wiebke
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De Rojas, Teresa
799aa1fb-52f8-4ecf-973a-632cfce7ad2f
Sodergren, Samantha C.
d66fc3fa-2c98-403d-8ae5-410ef95de46e
Darlington, Anne-Sophie
472fcfc9-160b-4344-8113-8dd8760ff962
Riedl, David
dd01d4ed-3140-4f77-9923-883a04a4c136
1 August 2022
Rothmund, Maria
874c229d-a404-4eff-9281-d9ad7eb2c902
Lehmann, Jens
c5d25e2d-62c7-45f5-9d6a-b5eb4ab361d4
Moser, Wiebke
19d31361-8219-4ea9-98f7-9a2c951b2846
De Rojas, Teresa
799aa1fb-52f8-4ecf-973a-632cfce7ad2f
Sodergren, Samantha C.
d66fc3fa-2c98-403d-8ae5-410ef95de46e
Darlington, Anne-Sophie
472fcfc9-160b-4344-8113-8dd8760ff962
Riedl, David
dd01d4ed-3140-4f77-9923-883a04a4c136
Rothmund, Maria, Lehmann, Jens, Moser, Wiebke, De Rojas, Teresa, Sodergren, Samantha C., Darlington, Anne-Sophie and Riedl, David
(2022)
Patient-reported outcomes are under-utilised in evaluating supportive therapies in paediatric oncology – A systematic review of clinical trial registries.
Critical Reviews in Oncology/Hematology, 176, [103755].
(doi:10.1016/j.critrevonc.2022.103755).
Abstract
Background: Children with cancer suffer from numerous symptoms and side-effects, making supportive interventions indispensable to improve their quality of life. The gold standard for evaluating the latter is patient-reported outcome (PRO) assessment. This systematic review investigates the current practice of clinical outcome assessment (COA) in clinical trials on supportive interventions. Methods: ClinicalTrials.gov and EudraCT were searched for trials including children and adolescents (≤21 years) with cancer receiving supportive care registered 2007–2020. The use of different types of COAs was analysed, focusing on PRO assessment and the domains measured with PRO measures (PROMs). Associations with trial characteristics were investigated using univariate and multivariable analyses. Results: Of 4789 identified trials, 229 were included. Among them, 44.1 % relied on PROMs, the most commonly used COA. The proportion of trials using PROMs did not significantly differ over time. In the multivariable analysis, intervention type (higher PROM use in behavioural vs. medical interventional trials) and cancer type (higher PROM use in mixed and solid tumour samples vs. haematological samples) were significant predictors of PROM use. The majority of trials using PROMs (59.6 %) measured more than one health domain. ‘Physical health’ was the most frequently assessed domain (92.6 %). Conclusion: Less than half of registered clinical trials investigating supportive interventions for children with cancer used PROMs. This result is striking since supportive care explicitly focuses on patients’ quality of life, which is best assessed using PROMs. Our systematic review underlines the need to identify barriers for PROM implementation and to improve PRO research in paediatric oncology.
Text
Patient reported outcomes are under-utilised
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Figure_2_revised
- Accepted Manuscript
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Figure_3_PROM_usage_over_time
- Accepted Manuscript
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1-s2.0-S1040842822001792-main
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More information
Accepted/In Press date: 2 July 2022
e-pub ahead of print date: 5 July 2022
Published date: 1 August 2022
Additional Information:
Funding Information:
This study was supported by the European Organisation for Research and Treatment of Cancer (EORTC) Qualify of Life Group. The grant (no. 002-2020) was awarded to Dr. David Riedl and Dr. Samantha Sodergren.
Publisher Copyright:
© 2022 The Authors
Keywords:
Childhood cancer, Clinicaltrials.gov, EudraCT, Paediatric oncology, Patient-reported outcomes, Supportive cancer care, Trial registries
Identifiers
Local EPrints ID: 473250
URI: http://eprints.soton.ac.uk/id/eprint/473250
ISSN: 1040-8428
PURE UUID: ca1c3216-79b4-4111-bbec-36f0acbe0549
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Date deposited: 12 Jan 2023 18:23
Last modified: 17 Mar 2024 07:36
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Contributors
Author:
Maria Rothmund
Author:
Jens Lehmann
Author:
Wiebke Moser
Author:
Teresa De Rojas
Author:
David Riedl
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