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Rare use of Patient Reported Outcomes (PROs) in childhood cancer clinical trials – a systematic review of clinical trial registries

Rare use of Patient Reported Outcomes (PROs) in childhood cancer clinical trials – a systematic review of clinical trial registries
Rare use of Patient Reported Outcomes (PROs) in childhood cancer clinical trials – a systematic review of clinical trial registries
Background: patient-reported outcomes (PROs) are the gold standard to assess the patients’ subjective health-status. While both the FDA and EMA recommend the use of PROs as endpoints in paediatric clinical trials to support claims for medical product labelling, it is not known how often PROs are actually used. The aim of this study was to assess the usage of PRO instruments in childhood cancer clinical trials investigating anti-cancer medication. Methods : in June 2020 ClinicalTrials and EudraCT were systematically searched for all trials including children and adolescents (21 years) with cancer registered between 2007-2020. The use of PRO measures and trials characteristics were analysed. To investigate which trial characteristics are associated with the use of PROs, a binary logistic regression was calculated. Results: of 4789 identified trials, 711 were included. The most frequent reason for exclusion was age limitation (age >21 years). Of all included trials, only 8.2% used PROs as endpoints; 0.6% as the primary endpoint. The most commonly used questionnaire was the PedsQLTM (32.8%), followed by the PROMIS scales (12.1%). No association was observed between the use of PROs and trial region, number of centres, trial phase, time period or intervention type (all p > .05). The use of PROs did not substantially increase over time. Only 20.3% of the closed studies had published their results. Conclusion: despite recommendations of regulatory agencies, PRO assessment is extremely rare in paediatric oncology clinical trials. More efforts should be undertaken to facilitate implementation of PRO in paediatric trials to guarantee patient-centred research and treatments.
0959-8049
Riedl, David
dd01d4ed-3140-4f77-9923-883a04a4c136
Rothmund, Maria
874c229d-a404-4eff-9281-d9ad7eb2c902
Darlington, Anne-Sophie
472fcfc9-160b-4344-8113-8dd8760ff962
Sodergren, Samantha
d66fc3fa-2c98-403d-8ae5-410ef95de46e
Crazzolara, Roman
2ae764da-3ca6-41ca-959d-6a71411289d7
de Rojas, Teresa
799aa1fb-52f8-4ecf-973a-632cfce7ad2f
Riedl, David
dd01d4ed-3140-4f77-9923-883a04a4c136
Rothmund, Maria
874c229d-a404-4eff-9281-d9ad7eb2c902
Darlington, Anne-Sophie
472fcfc9-160b-4344-8113-8dd8760ff962
Sodergren, Samantha
d66fc3fa-2c98-403d-8ae5-410ef95de46e
Crazzolara, Roman
2ae764da-3ca6-41ca-959d-6a71411289d7
de Rojas, Teresa
799aa1fb-52f8-4ecf-973a-632cfce7ad2f

Riedl, David, Rothmund, Maria, Darlington, Anne-Sophie, Sodergren, Samantha, Crazzolara, Roman and de Rojas, Teresa (2021) Rare use of Patient Reported Outcomes (PROs) in childhood cancer clinical trials – a systematic review of clinical trial registries. European Journal of Cancer. (In Press)

Record type: Article

Abstract

Background: patient-reported outcomes (PROs) are the gold standard to assess the patients’ subjective health-status. While both the FDA and EMA recommend the use of PROs as endpoints in paediatric clinical trials to support claims for medical product labelling, it is not known how often PROs are actually used. The aim of this study was to assess the usage of PRO instruments in childhood cancer clinical trials investigating anti-cancer medication. Methods : in June 2020 ClinicalTrials and EudraCT were systematically searched for all trials including children and adolescents (21 years) with cancer registered between 2007-2020. The use of PRO measures and trials characteristics were analysed. To investigate which trial characteristics are associated with the use of PROs, a binary logistic regression was calculated. Results: of 4789 identified trials, 711 were included. The most frequent reason for exclusion was age limitation (age >21 years). Of all included trials, only 8.2% used PROs as endpoints; 0.6% as the primary endpoint. The most commonly used questionnaire was the PedsQLTM (32.8%), followed by the PROMIS scales (12.1%). No association was observed between the use of PROs and trial region, number of centres, trial phase, time period or intervention type (all p > .05). The use of PROs did not substantially increase over time. Only 20.3% of the closed studies had published their results. Conclusion: despite recommendations of regulatory agencies, PRO assessment is extremely rare in paediatric oncology clinical trials. More efforts should be undertaken to facilitate implementation of PRO in paediatric trials to guarantee patient-centred research and treatments.

Text
Rare use of Patient Reported Outcomes (PROs) in childhood cancer clinical trials – a systematic review of clinical trial registries - Accepted Manuscript
Restricted to Repository staff only until 29 April 2022.
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Accepted/In Press date: 29 April 2021

Identifiers

Local EPrints ID: 449202
URI: http://eprints.soton.ac.uk/id/eprint/449202
ISSN: 0959-8049
PURE UUID: 77d1a46d-99d6-4f1b-8f5f-8f7987bacaac
ORCID for Samantha Sodergren: ORCID iD orcid.org/0000-0001-8755-146X

Catalogue record

Date deposited: 19 May 2021 18:18
Last modified: 20 May 2021 01:45

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Contributors

Author: David Riedl
Author: Maria Rothmund
Author: Roman Crazzolara
Author: Teresa de Rojas

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