Patient-centred composite scores as tools for assesment of response to biological therapy for paediatric and adult severe asthma

Ekaterina Khaleva; Chris Brightling; Thomas Eiwegger; Alan Altraja; Philippe Bégin; Katharina Blumchen; Apostolos Bossios; Arnaud Bourdin; Anneke Ten Brinke; Guy Brusselle; Roxana Silvia Bumbacea; Andrew Bush; Thomas B Casale; Graham W Clarke; Rekha Chaudhuri; Kian Fan Chung; Courtney Coleman; Jonathan Corren; Sven-Erik Dahlén; Antoine Deschildre; Ratko Djukanovic; Katrien Eger; Andrew Exley; Louise Fleming; Stephen J Fowler; Erol A Gaillard; Monika Gappa; Atul Gupta; Hans Michael Haitchi; Simone Hashimoto; Liam G Heaney; Gunilla Hedlin; Markaya Henderson; Wen Hua; David J Jackson; Bülent Karadag; Constance Helen Katelaris; Mariko S Koh; Matthias Volkmar Kopp; Gerard H Koppelman; Inger Kull; Ramesh J Kurukulaaratchy; Ji-Hyang Lee; Vera Mahler; Mika Mäkelä; Matthew Masoli; Alexander G Mathioudakis; Angel Mazon; Erik Melén; Katrin Milger; Alexander Moeller; Clare S Murray; Prasad Nagakumar; Parameswaran Nair; Jenny Negus; Antonio Nieto; Nikolaos G Papadopoulos; James Paton; Mariëlle W Pijnenburg; Katharine C Pike; Celeste Porsbjerg; Anna Rattu; Hitasha Rupani; Franca Rusconi; Niels W Rutjes; Sejal Saglani; Paul Seddon; Salman Siddiqui; Florian Singer; Tomoko Tajiri; Steve Turner; John W Upham; Susanne J H Vijverberg; Peter A B Wark; Michael E Wechsler; Valentyna Yasinska; Graham Roberts

doi:10.1183/13993003.00691-2024

Patient-centred composite scores as tools for assesment of response to biological therapy for paediatric and adult severe asthma

Ekaterina Khaleva, Chris Brightling, Thomas Eiwegger, Alan Altraja, Philippe Bégin, Katharina Blumchen, Apostolos Bossios, Arnaud Bourdin, Anneke Ten Brinke, Guy Brusselle, Roxana Silvia Bumbacea, Andrew Bush, Thomas B Casale, Graham W Clarke, Rekha Chaudhuri, Kian Fan Chung, Courtney Coleman, Jonathan Corren, Sven-Erik Dahlén, Antoine DeschildreRatko Djukanovic, Katrien Eger, Andrew Exley, Louise Fleming, Stephen J Fowler, Erol A Gaillard, Monika Gappa, Atul Gupta, Hans Michael Haitchi, Simone Hashimoto, Liam G Heaney, Gunilla Hedlin, Markaya Henderson, Wen Hua, David J Jackson, Bülent Karadag, Constance Helen Katelaris, Mariko S Koh, Matthias Volkmar Kopp, Gerard H Koppelman, Inger Kull, Ramesh J Kurukulaaratchy, Ji-Hyang Lee, Vera Mahler, Mika Mäkelä, Matthew Masoli, Alexander G Mathioudakis, Angel Mazon, Erik Melén, Katrin Milger, Alexander Moeller, Clare S Murray, Prasad Nagakumar, Parameswaran Nair, Jenny Negus, Antonio Nieto, Nikolaos G Papadopoulos, James Paton, Mariëlle W Pijnenburg, Katharine C Pike, Celeste Porsbjerg, Anna Rattu, Hitasha Rupani, Franca Rusconi, Niels W Rutjes, Sejal Saglani, Paul Seddon, Salman Siddiqui, Florian Singer, Tomoko Tajiri, Steve Turner, John W Upham, Susanne J H Vijverberg, Peter A B Wark, Michael E Wechsler, Valentyna Yasinska, Graham Roberts

Research output: Journal article (peer-reviewed) › Journal article

Abstract

BACKGROUND: We have previously developed Core Outcome Measures sets for Severe Asthma (COMSA) by multi-stakeholder consensus. There are no patient-centred tools to quantify response to biologics for severe asthma. We aimed to develop paediatric and adult CompOsite iNdexes For Response in asthMa (CONFiRM) incorporating clinical parameters and patient-reported quality of life (QoL).

METHODS: International expert healthcare professionals (HCPs) and patients with severe asthma were invited to: 1) develop consensus levels of clinically relevant changes for each outcome measure within COMSA; 2) use multicriteria decision analysis to develop the CONFiRM scores and 3) assess their internal validity. A separate group of HCPs evaluated CONFiRM's external validity.

RESULTS: Five levels of change for each COMSA outcome were agreed. Severe exacerbations and maintenance oral corticosteroids use were rated as most important in determining both paediatric and adult CONFiRM scores. There was strong agreement between HCPs and patients, although patients assigned greater importance to QoL. The CONFiRM score quantified response to a biological from -31 (deterioration) to 69 (best possible response). Paediatric and adult CONFiRMs had good discriminative ability for a sufficient (AUC≥0.92) and a substantial (AUC≥0.95) response to biologics. Both CONFiRMs demonstrated excellent external validity (Spearman correlation coefficients 0.9 and 0.8 for paediatric and adult respectively (p<0.0001)).

CONCLUSIONS: We have developed novel patient-centred paediatric and adult CONFiRMs which include QoL measures. CONFiRMs should allow a more holistic understanding of response for the patient and a standardised assessment of the effectiveness of biologics between studies. Further research is needed to prospectively validate CONFiRM scores.

Original language	English
Journal	European Respiratory Journal
DOIs	https://doi.org/10.1183/13993003.00691-2024
Publication status	E-pub ahead of print - 21 Nov 2024

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10.1183/13993003.00691-2024

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Khaleva, E., Brightling, C., Eiwegger, T., Altraja, A., Bégin, P., Blumchen, K., Bossios, A., Bourdin, A., Ten Brinke, A., Brusselle, G., Silvia Bumbacea, R., Bush, A., Casale, T. B., Clarke, G. W., Chaudhuri, R., Fan Chung, K., Coleman, C., Corren, J., Dahlén, S.-E., ... Roberts, G. (2024). Patient-centred composite scores as tools for assesment of response to biological therapy for paediatric and adult severe asthma. European Respiratory Journal. Advance online publication. https://doi.org/10.1183/13993003.00691-2024

@article{84b634e0d43040eea1cd32c642a571b6,

title = "Patient-centred composite scores as tools for assesment of response to biological therapy for paediatric and adult severe asthma",

abstract = "BACKGROUND: We have previously developed Core Outcome Measures sets for Severe Asthma (COMSA) by multi-stakeholder consensus. There are no patient-centred tools to quantify response to biologics for severe asthma. We aimed to develop paediatric and adult CompOsite iNdexes For Response in asthMa (CONFiRM) incorporating clinical parameters and patient-reported quality of life (QoL).METHODS: International expert healthcare professionals (HCPs) and patients with severe asthma were invited to: 1) develop consensus levels of clinically relevant changes for each outcome measure within COMSA; 2) use multicriteria decision analysis to develop the CONFiRM scores and 3) assess their internal validity. A separate group of HCPs evaluated CONFiRM's external validity.RESULTS: Five levels of change for each COMSA outcome were agreed. Severe exacerbations and maintenance oral corticosteroids use were rated as most important in determining both paediatric and adult CONFiRM scores. There was strong agreement between HCPs and patients, although patients assigned greater importance to QoL. The CONFiRM score quantified response to a biological from -31 (deterioration) to 69 (best possible response). Paediatric and adult CONFiRMs had good discriminative ability for a sufficient (AUC≥0.92) and a substantial (AUC≥0.95) response to biologics. Both CONFiRMs demonstrated excellent external validity (Spearman correlation coefficients 0.9 and 0.8 for paediatric and adult respectively (p<0.0001)).CONCLUSIONS: We have developed novel patient-centred paediatric and adult CONFiRMs which include QoL measures. CONFiRMs should allow a more holistic understanding of response for the patient and a standardised assessment of the effectiveness of biologics between studies. Further research is needed to prospectively validate CONFiRM scores.",

author = "Ekaterina Khaleva and Chris Brightling and Thomas Eiwegger and Alan Altraja and Philippe B{\'e}gin and Katharina Blumchen and Apostolos Bossios and Arnaud Bourdin and {Ten Brinke}, Anneke and Guy Brusselle and {Silvia Bumbacea}, Roxana and Andrew Bush and Casale, {Thomas B} and Clarke, {Graham W} and Rekha Chaudhuri and {Fan Chung}, Kian and Courtney Coleman and Jonathan Corren and Sven-Erik Dahl{\'e}n and Antoine Deschildre and Ratko Djukanovic and Katrien Eger and Andrew Exley and Louise Fleming and Fowler, {Stephen J} and Gaillard, {Erol A} and Monika Gappa and Atul Gupta and {Michael Haitchi}, Hans and Simone Hashimoto and Heaney, {Liam G} and Gunilla Hedlin and Markaya Henderson and Wen Hua and Jackson, {David J} and B{\"u}lent Karadag and {Helen Katelaris}, Constance and Koh, {Mariko S} and {Volkmar Kopp}, Matthias and Koppelman, {Gerard H} and Inger Kull and Kurukulaaratchy, {Ramesh J} and Ji-Hyang Lee and Vera Mahler and Mika M{\"a}kel{\"a} and Matthew Masoli and Mathioudakis, {Alexander G} and Angel Mazon and Erik Mel{\'e}n and Katrin Milger and Alexander Moeller and Murray, {Clare S} and Prasad Nagakumar and Parameswaran Nair and Jenny Negus and Antonio Nieto and Papadopoulos, {Nikolaos G} and James Paton and Pijnenburg, {Mari{\"e}lle W} and Pike, {Katharine C} and Celeste Porsbjerg and Anna Rattu and Hitasha Rupani and Franca Rusconi and Rutjes, {Niels W} and Sejal Saglani and Paul Seddon and Salman Siddiqui and Florian Singer and Tomoko Tajiri and Steve Turner and Upham, {John W} and Vijverberg, {Susanne J H} and Wark, {Peter A B} and Wechsler, {Michael E} and Valentyna Yasinska and Graham Roberts",

note = "Copyright {\textcopyright}The authors 2024.",

year = "2024",

month = nov,

day = "21",

doi = "10.1183/13993003.00691-2024",

language = "English",

journal = "European Respiratory Journal",

issn = "0903-1936",

publisher = "European Respiratory Society",

}

Khaleva, E, Brightling, C, Eiwegger, T, Altraja, A, Bégin, P, Blumchen, K, Bossios, A, Bourdin, A, Ten Brinke, A, Brusselle, G, Silvia Bumbacea, R, Bush, A, Casale, TB, Clarke, GW, Chaudhuri, R, Fan Chung, K, Coleman, C, Corren, J, Dahlén, S-E, Deschildre, A, Djukanovic, R, Eger, K, Exley, A, Fleming, L, Fowler, SJ, Gaillard, EA, Gappa, M, Gupta, A, Michael Haitchi, H, Hashimoto, S, Heaney, LG, Hedlin, G, Henderson, M, Hua, W, Jackson, DJ, Karadag, B, Helen Katelaris, C, Koh, MS, Volkmar Kopp, M, Koppelman, GH, Kull, I, Kurukulaaratchy, RJ, Lee, J-H, Mahler, V, Mäkelä, M, Masoli, M, Mathioudakis, AG, Mazon, A, Melén, E, Milger, K, Moeller, A, Murray, CS, Nagakumar, P, Nair, P, Negus, J, Nieto, A, Papadopoulos, NG, Paton, J, Pijnenburg, MW, Pike, KC, Porsbjerg, C, Rattu, A, Rupani, H, Rusconi, F, Rutjes, NW, Saglani, S, Seddon, P, Siddiqui, S, Singer, F, Tajiri, T, Turner, S, Upham, JW, Vijverberg, SJH, Wark, PAB, Wechsler, ME, Yasinska, V & Roberts, G 2024, 'Patient-centred composite scores as tools for assesment of response to biological therapy for paediatric and adult severe asthma', European Respiratory Journal. https://doi.org/10.1183/13993003.00691-2024

TY - JOUR

T1 - Patient-centred composite scores as tools for assesment of response to biological therapy for paediatric and adult severe asthma

AU - Khaleva, Ekaterina

AU - Brightling, Chris

AU - Eiwegger, Thomas

AU - Altraja, Alan

AU - Bégin, Philippe

AU - Blumchen, Katharina

AU - Bossios, Apostolos

AU - Bourdin, Arnaud

AU - Ten Brinke, Anneke

AU - Brusselle, Guy

AU - Silvia Bumbacea, Roxana

AU - Bush, Andrew

AU - Casale, Thomas B

AU - Clarke, Graham W

AU - Chaudhuri, Rekha

AU - Fan Chung, Kian

AU - Coleman, Courtney

AU - Corren, Jonathan

AU - Dahlén, Sven-Erik

AU - Deschildre, Antoine

AU - Djukanovic, Ratko

AU - Eger, Katrien

AU - Exley, Andrew

AU - Fleming, Louise

AU - Fowler, Stephen J

AU - Gaillard, Erol A

AU - Gappa, Monika

AU - Gupta, Atul

AU - Michael Haitchi, Hans

AU - Hashimoto, Simone

AU - Heaney, Liam G

AU - Hedlin, Gunilla

AU - Henderson, Markaya

AU - Hua, Wen

AU - Jackson, David J

AU - Karadag, Bülent

AU - Helen Katelaris, Constance

AU - Koh, Mariko S

AU - Volkmar Kopp, Matthias

AU - Koppelman, Gerard H

AU - Kull, Inger

AU - Kurukulaaratchy, Ramesh J

AU - Lee, Ji-Hyang

AU - Mahler, Vera

AU - Mäkelä, Mika

AU - Masoli, Matthew

AU - Mathioudakis, Alexander G

AU - Mazon, Angel

AU - Melén, Erik

AU - Milger, Katrin

AU - Moeller, Alexander

AU - Murray, Clare S

AU - Nagakumar, Prasad

AU - Nair, Parameswaran

AU - Negus, Jenny

AU - Nieto, Antonio

AU - Papadopoulos, Nikolaos G

AU - Paton, James

AU - Pijnenburg, Mariëlle W

AU - Pike, Katharine C

AU - Porsbjerg, Celeste

AU - Rattu, Anna

AU - Rupani, Hitasha

AU - Rusconi, Franca

AU - Rutjes, Niels W

AU - Saglani, Sejal

AU - Seddon, Paul

AU - Siddiqui, Salman

AU - Singer, Florian

AU - Tajiri, Tomoko

AU - Turner, Steve

AU - Upham, John W

AU - Vijverberg, Susanne J H

AU - Wark, Peter A B

AU - Wechsler, Michael E

AU - Yasinska, Valentyna

AU - Roberts, Graham

PY - 2024/11/21

Y1 - 2024/11/21

N2 - BACKGROUND: We have previously developed Core Outcome Measures sets for Severe Asthma (COMSA) by multi-stakeholder consensus. There are no patient-centred tools to quantify response to biologics for severe asthma. We aimed to develop paediatric and adult CompOsite iNdexes For Response in asthMa (CONFiRM) incorporating clinical parameters and patient-reported quality of life (QoL).METHODS: International expert healthcare professionals (HCPs) and patients with severe asthma were invited to: 1) develop consensus levels of clinically relevant changes for each outcome measure within COMSA; 2) use multicriteria decision analysis to develop the CONFiRM scores and 3) assess their internal validity. A separate group of HCPs evaluated CONFiRM's external validity.RESULTS: Five levels of change for each COMSA outcome were agreed. Severe exacerbations and maintenance oral corticosteroids use were rated as most important in determining both paediatric and adult CONFiRM scores. There was strong agreement between HCPs and patients, although patients assigned greater importance to QoL. The CONFiRM score quantified response to a biological from -31 (deterioration) to 69 (best possible response). Paediatric and adult CONFiRMs had good discriminative ability for a sufficient (AUC≥0.92) and a substantial (AUC≥0.95) response to biologics. Both CONFiRMs demonstrated excellent external validity (Spearman correlation coefficients 0.9 and 0.8 for paediatric and adult respectively (p<0.0001)).CONCLUSIONS: We have developed novel patient-centred paediatric and adult CONFiRMs which include QoL measures. CONFiRMs should allow a more holistic understanding of response for the patient and a standardised assessment of the effectiveness of biologics between studies. Further research is needed to prospectively validate CONFiRM scores.

AB - BACKGROUND: We have previously developed Core Outcome Measures sets for Severe Asthma (COMSA) by multi-stakeholder consensus. There are no patient-centred tools to quantify response to biologics for severe asthma. We aimed to develop paediatric and adult CompOsite iNdexes For Response in asthMa (CONFiRM) incorporating clinical parameters and patient-reported quality of life (QoL).METHODS: International expert healthcare professionals (HCPs) and patients with severe asthma were invited to: 1) develop consensus levels of clinically relevant changes for each outcome measure within COMSA; 2) use multicriteria decision analysis to develop the CONFiRM scores and 3) assess their internal validity. A separate group of HCPs evaluated CONFiRM's external validity.RESULTS: Five levels of change for each COMSA outcome were agreed. Severe exacerbations and maintenance oral corticosteroids use were rated as most important in determining both paediatric and adult CONFiRM scores. There was strong agreement between HCPs and patients, although patients assigned greater importance to QoL. The CONFiRM score quantified response to a biological from -31 (deterioration) to 69 (best possible response). Paediatric and adult CONFiRMs had good discriminative ability for a sufficient (AUC≥0.92) and a substantial (AUC≥0.95) response to biologics. Both CONFiRMs demonstrated excellent external validity (Spearman correlation coefficients 0.9 and 0.8 for paediatric and adult respectively (p<0.0001)).CONCLUSIONS: We have developed novel patient-centred paediatric and adult CONFiRMs which include QoL measures. CONFiRMs should allow a more holistic understanding of response for the patient and a standardised assessment of the effectiveness of biologics between studies. Further research is needed to prospectively validate CONFiRM scores.

U2 - 10.1183/13993003.00691-2024

DO - 10.1183/13993003.00691-2024

M3 - Journal article

C2 - 39510551

SN - 0903-1936

JO - European Respiratory Journal

JF - European Respiratory Journal

ER -

Patient-centred composite scores as tools for assesment of response to biological therapy for paediatric and adult severe asthma

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