Development and preliminary validation of the Sjögren's Tool for Assessing Response (STAR): a consensual composite score for assessing treatment effect in primary Sjögren's syndrome

Raphaele Seror; Gabriel Baron; Marine Camus; Divi Cornec; Elodie Perrodeau; Simon J. Bowman; Michele Bombardieri; Hendrika Bootsma; Jacques Eric Gottenberg; Benjamin Fisher; Wolfgang Hueber; Joel A van Roon; Valérie Devauchelle-Pensec; Peter Gergely; Xavier Mariette; Raphael Porcher

doi:10.1136/annrheumdis-2021-222054

Development and preliminary validation of the Sjögren's Tool for Assessing Response (STAR): a consensual composite score for assessing treatment effect in primary Sjögren's syndrome

Raphaele Seror^*, Gabriel Baron, Marine Camus, Divi Cornec, Elodie Perrodeau, Simon J. Bowman, Michele Bombardieri, Hendrika Bootsma, Jacques Eric Gottenberg, Benjamin Fisher, Wolfgang Hueber, Joel A van Roon, Valérie Devauchelle-Pensec, Peter Gergely, Xavier Mariette, Raphael Porcher

^*Corresponding author for this work

Inflammation and Ageing

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Abstract

Objective: To develop a composite responder index in primary Sjögren's syndrome (pSS): the Sjögren's Tool for Assessing Response (STAR).

Methods: To develop STAR, the NECESSITY (New clinical endpoints in primary Sjögren's syndrome: an interventional trial based on stratifying patients) consortium used data-driven methods based on nine randomised controlled trials (RCTs) and consensus techniques involving 78 experts and 20 patients. Based on reanalysis of rituximab trials and the literature, the Delphi panel identified a core set of domains with their respective outcome measures. STAR options combining these domains were proposed to the panel for selection and improvement. For each STAR option, sensitivity to change was estimated by the C-index in nine RCTs. Delphi rounds were run for selecting STAR. For the options remaining before the final vote, a meta-analysis of the RCTs was performed.

Results: The Delphi panel identified five core domains (systemic activity, patient symptoms, lachrymal gland function, salivary gland function and biological parameters), and 227 STAR options combining these domains were selected to be tested for sensitivity to change. After two Delphi rounds, a meta-analysis of the 20 remaining options was performed. The candidate STAR was then selected by a final vote based on metrological properties and clinical relevance.

Conclusion: The candidate STAR is a composite responder index that includes all main disease features in a single tool and is designed for use as a primary endpoint in pSS RCTs. The rigorous and consensual development process ensures its face and content validity. The candidate STAR showed good sensitivity to change and will be prospectively validated by the NECESSITY consortium in a dedicated RCT.

Original language	English
Pages (from-to)	979-989
Number of pages	11
Journal	Annals of the Rheumatic Diseases
Volume	81
Issue number	7
Early online date	7 Apr 2022
DOIs	https://doi.org/10.1136/annrheumdis-2021-222054
Publication status	Published - Jul 2022

Bibliographical note

Funding Information:
Funding This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking (JU) under grant agreement number 806975. JU receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA. The present article reflects only the authors’ view and JU is not responsible for any use that may be made of the information it contains.

Competing interests RS has received consulting fees from GlaxoSmithKline, Boehringer, Janssen and Novartis, participated in an advisory board for Janssen, and received support for attending meeting from GlaxoSmithKline and Amgen. DC has received consulting fees from GlaxoSmithKline, Bristol Myers Squibb, Janssen, Amgen, Pfizer and Roche. J-EG has received honoraria from AbbVie, Bristol Myers Squibb, Eli Lilly, Galapagos, Gilead, Pfizer, Roche, Sanofi, Novartis, MSD, CSL Behring and Genzyme and received grant from Bristol Myers Squibb. SJB and BF receive funding from the National Institute for Health Research (NIHR) Birmingham Biomedical Research Centre, Birmingham, UK. SJB has provided consultancy services in the field of clinical trial design for Sjögren’s syndrome for AbbVie, AstraZeneca, Galapagos and Novartis Pharmaceuticals in 2018–2021. BF has received grants from Servier, Galapagos and Janssen, provided consultancy services for Novartis, Bristol Myers Squibb, Janssen and Servier, and received honoraria from Bristol Myers Squibb and Novartis. MB has received grants from Amgen/ MedImmune, Janssen and GlaxoSmithKline, and personal fees from UCB, Amgen/ MedImmune, Janssen and GlaxoSmithKline. HB has received grants from Bristol Myers Squibb and Roche, and consulting fees from Bristol Myers Squibb, Roche, Novartis, MedImmune and Union Chimique Belge. WH and PG are employees of Novartis Pharma and recipients of Novartis stocks. XM has received a grant from Ose Pharmaceuticals and consultancy fees from Bristol Myers Squibb, Galapagos, GlaxoSmithKline, Janssen, Novartis, Pfizer and UCB. GB, MC, EP, JAvR, VD-P and RP declare no competing interests.

Publisher Copyright:
© Author(s) (or their employer(s)) 2022. Re-use permitted under CC BY. Published by BMJ.

Keywords

outcome assessment, health care
patient reported outcome measures
Sjogren's syndrome

ASJC Scopus subject areas

Rheumatology
Immunology and Allergy
Immunology
General Biochemistry,Genetics and Molecular Biology

Access to Document

10.1136/annrheumdis-2021-222054Licence: Creative Commons: Attribution (CC BY)

SerorR2022DevelopmentFinal published version, 1.7 MBLicence: Creative Commons: Attribution (CC BY)

H2020_RIA_NECESSITY_PARTNER
Fisher, B.
European Commission
1/01/19 → 31/12/24
Project: EU

Cite this

Seror, R., Baron, G., Camus, M., Cornec, D., Perrodeau, E., Bowman, S. J., Bombardieri, M., Bootsma, H., Gottenberg, J. E., Fisher, B., Hueber, W., van Roon, J. A., Devauchelle-Pensec, V., Gergely, P., Mariette, X., & Porcher, R. (2022). Development and preliminary validation of the Sjögren's Tool for Assessing Response (STAR): a consensual composite score for assessing treatment effect in primary Sjögren's syndrome. Annals of the Rheumatic Diseases, 81(7), 979-989. https://doi.org/10.1136/annrheumdis-2021-222054

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abstract = "Objective: To develop a composite responder index in primary Sj{\"o}gren's syndrome (pSS): the Sj{\"o}gren's Tool for Assessing Response (STAR). Methods: To develop STAR, the NECESSITY (New clinical endpoints in primary Sj{\"o}gren's syndrome: an interventional trial based on stratifying patients) consortium used data-driven methods based on nine randomised controlled trials (RCTs) and consensus techniques involving 78 experts and 20 patients. Based on reanalysis of rituximab trials and the literature, the Delphi panel identified a core set of domains with their respective outcome measures. STAR options combining these domains were proposed to the panel for selection and improvement. For each STAR option, sensitivity to change was estimated by the C-index in nine RCTs. Delphi rounds were run for selecting STAR. For the options remaining before the final vote, a meta-analysis of the RCTs was performed. Results: The Delphi panel identified five core domains (systemic activity, patient symptoms, lachrymal gland function, salivary gland function and biological parameters), and 227 STAR options combining these domains were selected to be tested for sensitivity to change. After two Delphi rounds, a meta-analysis of the 20 remaining options was performed. The candidate STAR was then selected by a final vote based on metrological properties and clinical relevance. Conclusion: The candidate STAR is a composite responder index that includes all main disease features in a single tool and is designed for use as a primary endpoint in pSS RCTs. The rigorous and consensual development process ensures its face and content validity. The candidate STAR showed good sensitivity to change and will be prospectively validated by the NECESSITY consortium in a dedicated RCT.",

keywords = "outcome assessment, health care, patient reported outcome measures, Sjogren's syndrome",

author = "Raphaele Seror and Gabriel Baron and Marine Camus and Divi Cornec and Elodie Perrodeau and Bowman, {Simon J.} and Michele Bombardieri and Hendrika Bootsma and Gottenberg, {Jacques Eric} and Benjamin Fisher and Wolfgang Hueber and {van Roon}, {Joel A} and Val{\'e}rie Devauchelle-Pensec and Peter Gergely and Xavier Mariette and Raphael Porcher",

note = "Funding Information: Funding This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking (JU) under grant agreement number 806975. JU receives support from the European Union{\textquoteright}s Horizon 2020 research and innovation programme and EFPIA. The present article reflects only the authors{\textquoteright} view and JU is not responsible for any use that may be made of the information it contains. Competing interests RS has received consulting fees from GlaxoSmithKline, Boehringer, Janssen and Novartis, participated in an advisory board for Janssen, and received support for attending meeting from GlaxoSmithKline and Amgen. DC has received consulting fees from GlaxoSmithKline, Bristol Myers Squibb, Janssen, Amgen, Pfizer and Roche. J-EG has received honoraria from AbbVie, Bristol Myers Squibb, Eli Lilly, Galapagos, Gilead, Pfizer, Roche, Sanofi, Novartis, MSD, CSL Behring and Genzyme and received grant from Bristol Myers Squibb. SJB and BF receive funding from the National Institute for Health Research (NIHR) Birmingham Biomedical Research Centre, Birmingham, UK. SJB has provided consultancy services in the field of clinical trial design for Sj{\"o}gren{\textquoteright}s syndrome for AbbVie, AstraZeneca, Galapagos and Novartis Pharmaceuticals in 2018–2021. BF has received grants from Servier, Galapagos and Janssen, provided consultancy services for Novartis, Bristol Myers Squibb, Janssen and Servier, and received honoraria from Bristol Myers Squibb and Novartis. MB has received grants from Amgen/ MedImmune, Janssen and GlaxoSmithKline, and personal fees from UCB, Amgen/ MedImmune, Janssen and GlaxoSmithKline. HB has received grants from Bristol Myers Squibb and Roche, and consulting fees from Bristol Myers Squibb, Roche, Novartis, MedImmune and Union Chimique Belge. WH and PG are employees of Novartis Pharma and recipients of Novartis stocks. XM has received a grant from Ose Pharmaceuticals and consultancy fees from Bristol Myers Squibb, Galapagos, GlaxoSmithKline, Janssen, Novartis, Pfizer and UCB. GB, MC, EP, JAvR, VD-P and RP declare no competing interests. Publisher Copyright: {\textcopyright} Author(s) (or their employer(s)) 2022. Re-use permitted under CC BY. Published by BMJ.",

year = "2022",

month = jul,

doi = "10.1136/annrheumdis-2021-222054",

language = "English",

volume = "81",

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Seror, R, Baron, G, Camus, M, Cornec, D, Perrodeau, E, Bowman, SJ, Bombardieri, M, Bootsma, H, Gottenberg, JE, Fisher, B, Hueber, W, van Roon, JA, Devauchelle-Pensec, V, Gergely, P, Mariette, X & Porcher, R 2022, 'Development and preliminary validation of the Sjögren's Tool for Assessing Response (STAR): a consensual composite score for assessing treatment effect in primary Sjögren's syndrome', Annals of the Rheumatic Diseases, vol. 81, no. 7, pp. 979-989. https://doi.org/10.1136/annrheumdis-2021-222054

Development and preliminary validation of the Sjögren's Tool for Assessing Response (STAR): a consensual composite score for assessing treatment effect in primary Sjögren's syndrome. / Seror, Raphaele; Baron, Gabriel; Camus, Marine et al.
In: Annals of the Rheumatic Diseases, Vol. 81, No. 7, 07.2022, p. 979-989.

Research output: Contribution to journal › Article › peer-review

TY - JOUR

T1 - Development and preliminary validation of the Sjögren's Tool for Assessing Response (STAR)

T2 - a consensual composite score for assessing treatment effect in primary Sjögren's syndrome

AU - Seror, Raphaele

AU - Baron, Gabriel

AU - Camus, Marine

AU - Cornec, Divi

AU - Perrodeau, Elodie

AU - Bowman, Simon J.

AU - Bombardieri, Michele

AU - Bootsma, Hendrika

AU - Gottenberg, Jacques Eric

AU - Fisher, Benjamin

AU - Hueber, Wolfgang

AU - van Roon, Joel A

AU - Devauchelle-Pensec, Valérie

AU - Gergely, Peter

AU - Mariette, Xavier

AU - Porcher, Raphael

N1 - Funding Information: Funding This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking (JU) under grant agreement number 806975. JU receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA. The present article reflects only the authors’ view and JU is not responsible for any use that may be made of the information it contains. Competing interests RS has received consulting fees from GlaxoSmithKline, Boehringer, Janssen and Novartis, participated in an advisory board for Janssen, and received support for attending meeting from GlaxoSmithKline and Amgen. DC has received consulting fees from GlaxoSmithKline, Bristol Myers Squibb, Janssen, Amgen, Pfizer and Roche. J-EG has received honoraria from AbbVie, Bristol Myers Squibb, Eli Lilly, Galapagos, Gilead, Pfizer, Roche, Sanofi, Novartis, MSD, CSL Behring and Genzyme and received grant from Bristol Myers Squibb. SJB and BF receive funding from the National Institute for Health Research (NIHR) Birmingham Biomedical Research Centre, Birmingham, UK. SJB has provided consultancy services in the field of clinical trial design for Sjögren’s syndrome for AbbVie, AstraZeneca, Galapagos and Novartis Pharmaceuticals in 2018–2021. BF has received grants from Servier, Galapagos and Janssen, provided consultancy services for Novartis, Bristol Myers Squibb, Janssen and Servier, and received honoraria from Bristol Myers Squibb and Novartis. MB has received grants from Amgen/ MedImmune, Janssen and GlaxoSmithKline, and personal fees from UCB, Amgen/ MedImmune, Janssen and GlaxoSmithKline. HB has received grants from Bristol Myers Squibb and Roche, and consulting fees from Bristol Myers Squibb, Roche, Novartis, MedImmune and Union Chimique Belge. WH and PG are employees of Novartis Pharma and recipients of Novartis stocks. XM has received a grant from Ose Pharmaceuticals and consultancy fees from Bristol Myers Squibb, Galapagos, GlaxoSmithKline, Janssen, Novartis, Pfizer and UCB. GB, MC, EP, JAvR, VD-P and RP declare no competing interests. Publisher Copyright: © Author(s) (or their employer(s)) 2022. Re-use permitted under CC BY. Published by BMJ.

PY - 2022/7

Y1 - 2022/7

N2 - Objective: To develop a composite responder index in primary Sjögren's syndrome (pSS): the Sjögren's Tool for Assessing Response (STAR). Methods: To develop STAR, the NECESSITY (New clinical endpoints in primary Sjögren's syndrome: an interventional trial based on stratifying patients) consortium used data-driven methods based on nine randomised controlled trials (RCTs) and consensus techniques involving 78 experts and 20 patients. Based on reanalysis of rituximab trials and the literature, the Delphi panel identified a core set of domains with their respective outcome measures. STAR options combining these domains were proposed to the panel for selection and improvement. For each STAR option, sensitivity to change was estimated by the C-index in nine RCTs. Delphi rounds were run for selecting STAR. For the options remaining before the final vote, a meta-analysis of the RCTs was performed. Results: The Delphi panel identified five core domains (systemic activity, patient symptoms, lachrymal gland function, salivary gland function and biological parameters), and 227 STAR options combining these domains were selected to be tested for sensitivity to change. After two Delphi rounds, a meta-analysis of the 20 remaining options was performed. The candidate STAR was then selected by a final vote based on metrological properties and clinical relevance. Conclusion: The candidate STAR is a composite responder index that includes all main disease features in a single tool and is designed for use as a primary endpoint in pSS RCTs. The rigorous and consensual development process ensures its face and content validity. The candidate STAR showed good sensitivity to change and will be prospectively validated by the NECESSITY consortium in a dedicated RCT.

AB - Objective: To develop a composite responder index in primary Sjögren's syndrome (pSS): the Sjögren's Tool for Assessing Response (STAR). Methods: To develop STAR, the NECESSITY (New clinical endpoints in primary Sjögren's syndrome: an interventional trial based on stratifying patients) consortium used data-driven methods based on nine randomised controlled trials (RCTs) and consensus techniques involving 78 experts and 20 patients. Based on reanalysis of rituximab trials and the literature, the Delphi panel identified a core set of domains with their respective outcome measures. STAR options combining these domains were proposed to the panel for selection and improvement. For each STAR option, sensitivity to change was estimated by the C-index in nine RCTs. Delphi rounds were run for selecting STAR. For the options remaining before the final vote, a meta-analysis of the RCTs was performed. Results: The Delphi panel identified five core domains (systemic activity, patient symptoms, lachrymal gland function, salivary gland function and biological parameters), and 227 STAR options combining these domains were selected to be tested for sensitivity to change. After two Delphi rounds, a meta-analysis of the 20 remaining options was performed. The candidate STAR was then selected by a final vote based on metrological properties and clinical relevance. Conclusion: The candidate STAR is a composite responder index that includes all main disease features in a single tool and is designed for use as a primary endpoint in pSS RCTs. The rigorous and consensual development process ensures its face and content validity. The candidate STAR showed good sensitivity to change and will be prospectively validated by the NECESSITY consortium in a dedicated RCT.

KW - outcome assessment, health care

KW - patient reported outcome measures

KW - Sjogren's syndrome

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U2 - 10.1136/annrheumdis-2021-222054

DO - 10.1136/annrheumdis-2021-222054

M3 - Article

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AN - SCOPUS:85128509402

SN - 0003-4967

VL - 81

SP - 979

EP - 989

JO - Annals of the Rheumatic Diseases

JF - Annals of the Rheumatic Diseases

IS - 7

ER -

Development and preliminary validation of the Sjögren's Tool for Assessing Response (STAR): a consensual composite score for assessing treatment effect in primary Sjögren's syndrome

Abstract

Bibliographical note

Keywords

ASJC Scopus subject areas

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