In silico validation of the Autoinflammatory Disease Damage Index

Nienke M Ter Haar, Amber Laetitia Justine van Delft, Kim Valerie Annink, Henk van Stel, Sulaiman M Al-Mayouf, Gayane Amaryan, Jordi Anton, Karyl S Barron, Susanne Benseler, Paul A Brogan, Luca Cantarini, Marco Cattalini, Alexis-Virgil Cochino, Fabrizio de Benedetti, Fatma Dedeoglu, Adriana Almeida de Jesus, Erkan Demirkaya, Pavla Dolezalova, Karen L Durrant, Giovanna FabioRomina Gallizzi, Raphaela Goldbach-Mansky, Eric Hachulla, Veronique Hentgen, Troels Herlin, Michaël Hofer, Hal M Hoffman, Antonella Insalaco, Annette F Jansson, Tilmann Kallinich, Isabelle Kone-Paut, Anna Kozlova, Jasmin Beate Kuemmerle-Deschner, Helen J Lachmann, Ronald M Laxer, Alberto Martini, Susan Nielsen, Irina Nikishina, Amanda K Ombrello, Seza Özen, Efimia Papadopoulou-Alataki, Pierre Quartier, Donato Rigante, Ricardo Russo, Anna Simon, Maria Trachana, Yosef Uziel, Angelo Ravelli, Grant Schulert, Marco Gattorno, Joost Frenkel

Research output: Contribution to journalArticle

Abstract

INTRODUCTION: Autoinflammatory diseases can cause irreversible tissue damage due to systemic inflammation. Recently, the Autoinflammatory Disease Damage Index (ADDI) was developed. The ADDI is the first instrument to quantify damage in familial Mediterranean fever, cryopyrin-associated periodic syndromes, mevalonate kinase deficiency and tumour necrosis factor receptor-associated periodic syndrome. The aim of this study was to validate this tool for its intended use in a clinical/research setting.

METHODS: The ADDI was scored on paper clinical cases by at least three physicians per case, independently of each other. Face and content validity were assessed by requesting comments on the ADDI. Reliability was tested by calculating the intraclass correlation coefficient (ICC) using an 'observer-nested-within-subject' design. Construct validity was determined by correlating the ADDI score to the Physician Global Assessment (PGA) of damage and disease activity. Redundancy of individual items was determined with Cronbach's alpha.

RESULTS: The ADDI was validated on a total of 110 paper clinical cases by 37 experts in autoinflammatory diseases. This yielded an ICC of 0.84 (95% CI 0.78 to 0.89). The ADDI score correlated strongly with PGA-damage (r=0.92, 95% CI 0.88 to 0.95) and was not strongly influenced by disease activity (r=0.395, 95% CI 0.21 to 0.55). After comments from disease experts, some item definitions were refined. The interitem correlation in all different categories was lower than 0.7, indicating that there was no redundancy between individual damage items.

CONCLUSION: The ADDI is a reliable and valid instrument to quantify damage in individual patients and can be used to compare disease outcomes in clinical studies.

Original languageEnglish
Pages (from-to)1599-1605
Number of pages7
JournalAnnals of the Rheumatic Diseases
Volume77
Issue number11
DOIs
Publication statusPublished - Nov 2018

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Computer Simulation
mevalonate kinase
Physicians
Redundancy
Mevalonate Kinase Deficiency
Cryopyrin-Associated Periodic Syndromes
Familial Mediterranean Fever
Tumor Necrosis Factor Receptors
Reproducibility of Results
Tissue
Inflammation

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Ter Haar, N. M., van Delft, A. L. J., Annink, K. V., van Stel, H., Al-Mayouf, S. M., Amaryan, G., ... Frenkel, J. (2018). In silico validation of the Autoinflammatory Disease Damage Index. Annals of the Rheumatic Diseases, 77(11), 1599-1605. https://doi.org/10.1136/annrheumdis-2018-213725

In silico validation of the Autoinflammatory Disease Damage Index. / Ter Haar, Nienke M; van Delft, Amber Laetitia Justine; Annink, Kim Valerie; van Stel, Henk; Al-Mayouf, Sulaiman M; Amaryan, Gayane; Anton, Jordi; Barron, Karyl S; Benseler, Susanne; Brogan, Paul A; Cantarini, Luca; Cattalini, Marco; Cochino, Alexis-Virgil; de Benedetti, Fabrizio; Dedeoglu, Fatma; de Jesus, Adriana Almeida; Demirkaya, Erkan; Dolezalova, Pavla; Durrant, Karen L; Fabio, Giovanna; Gallizzi, Romina; Goldbach-Mansky, Raphaela; Hachulla, Eric; Hentgen, Veronique; Herlin, Troels; Hofer, Michaël; Hoffman, Hal M; Insalaco, Antonella; Jansson, Annette F; Kallinich, Tilmann; Kone-Paut, Isabelle; Kozlova, Anna; Kuemmerle-Deschner, Jasmin Beate; Lachmann, Helen J; Laxer, Ronald M; Martini, Alberto; Nielsen, Susan; Nikishina, Irina; Ombrello, Amanda K; Özen, Seza; Papadopoulou-Alataki, Efimia; Quartier, Pierre; Rigante, Donato; Russo, Ricardo; Simon, Anna; Trachana, Maria; Uziel, Yosef; Ravelli, Angelo; Schulert, Grant; Gattorno, Marco; Frenkel, Joost.

In: Annals of the Rheumatic Diseases, Vol. 77, No. 11, 11.2018, p. 1599-1605.

Research output: Contribution to journalArticle

Ter Haar, NM, van Delft, ALJ, Annink, KV, van Stel, H, Al-Mayouf, SM, Amaryan, G, Anton, J, Barron, KS, Benseler, S, Brogan, PA, Cantarini, L, Cattalini, M, Cochino, A-V, de Benedetti, F, Dedeoglu, F, de Jesus, AA, Demirkaya, E, Dolezalova, P, Durrant, KL, Fabio, G, Gallizzi, R, Goldbach-Mansky, R, Hachulla, E, Hentgen, V, Herlin, T, Hofer, M, Hoffman, HM, Insalaco, A, Jansson, AF, Kallinich, T, Kone-Paut, I, Kozlova, A, Kuemmerle-Deschner, JB, Lachmann, HJ, Laxer, RM, Martini, A, Nielsen, S, Nikishina, I, Ombrello, AK, Özen, S, Papadopoulou-Alataki, E, Quartier, P, Rigante, D, Russo, R, Simon, A, Trachana, M, Uziel, Y, Ravelli, A, Schulert, G, Gattorno, M & Frenkel, J 2018, 'In silico validation of the Autoinflammatory Disease Damage Index', Annals of the Rheumatic Diseases, vol. 77, no. 11, pp. 1599-1605. https://doi.org/10.1136/annrheumdis-2018-213725
Ter Haar NM, van Delft ALJ, Annink KV, van Stel H, Al-Mayouf SM, Amaryan G et al. In silico validation of the Autoinflammatory Disease Damage Index. Annals of the Rheumatic Diseases. 2018 Nov;77(11):1599-1605. https://doi.org/10.1136/annrheumdis-2018-213725
Ter Haar, Nienke M ; van Delft, Amber Laetitia Justine ; Annink, Kim Valerie ; van Stel, Henk ; Al-Mayouf, Sulaiman M ; Amaryan, Gayane ; Anton, Jordi ; Barron, Karyl S ; Benseler, Susanne ; Brogan, Paul A ; Cantarini, Luca ; Cattalini, Marco ; Cochino, Alexis-Virgil ; de Benedetti, Fabrizio ; Dedeoglu, Fatma ; de Jesus, Adriana Almeida ; Demirkaya, Erkan ; Dolezalova, Pavla ; Durrant, Karen L ; Fabio, Giovanna ; Gallizzi, Romina ; Goldbach-Mansky, Raphaela ; Hachulla, Eric ; Hentgen, Veronique ; Herlin, Troels ; Hofer, Michaël ; Hoffman, Hal M ; Insalaco, Antonella ; Jansson, Annette F ; Kallinich, Tilmann ; Kone-Paut, Isabelle ; Kozlova, Anna ; Kuemmerle-Deschner, Jasmin Beate ; Lachmann, Helen J ; Laxer, Ronald M ; Martini, Alberto ; Nielsen, Susan ; Nikishina, Irina ; Ombrello, Amanda K ; Özen, Seza ; Papadopoulou-Alataki, Efimia ; Quartier, Pierre ; Rigante, Donato ; Russo, Ricardo ; Simon, Anna ; Trachana, Maria ; Uziel, Yosef ; Ravelli, Angelo ; Schulert, Grant ; Gattorno, Marco ; Frenkel, Joost. / In silico validation of the Autoinflammatory Disease Damage Index. In: Annals of the Rheumatic Diseases. 2018 ; Vol. 77, No. 11. pp. 1599-1605.
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abstract = "INTRODUCTION: Autoinflammatory diseases can cause irreversible tissue damage due to systemic inflammation. Recently, the Autoinflammatory Disease Damage Index (ADDI) was developed. The ADDI is the first instrument to quantify damage in familial Mediterranean fever, cryopyrin-associated periodic syndromes, mevalonate kinase deficiency and tumour necrosis factor receptor-associated periodic syndrome. The aim of this study was to validate this tool for its intended use in a clinical/research setting.METHODS: The ADDI was scored on paper clinical cases by at least three physicians per case, independently of each other. Face and content validity were assessed by requesting comments on the ADDI. Reliability was tested by calculating the intraclass correlation coefficient (ICC) using an 'observer-nested-within-subject' design. Construct validity was determined by correlating the ADDI score to the Physician Global Assessment (PGA) of damage and disease activity. Redundancy of individual items was determined with Cronbach's alpha.RESULTS: The ADDI was validated on a total of 110 paper clinical cases by 37 experts in autoinflammatory diseases. This yielded an ICC of 0.84 (95{\%} CI 0.78 to 0.89). The ADDI score correlated strongly with PGA-damage (r=0.92, 95{\%} CI 0.88 to 0.95) and was not strongly influenced by disease activity (r=0.395, 95{\%} CI 0.21 to 0.55). After comments from disease experts, some item definitions were refined. The interitem correlation in all different categories was lower than 0.7, indicating that there was no redundancy between individual damage items.CONCLUSION: The ADDI is a reliable and valid instrument to quantify damage in individual patients and can be used to compare disease outcomes in clinical studies.",
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T1 - In silico validation of the Autoinflammatory Disease Damage Index

AU - Ter Haar, Nienke M

AU - van Delft, Amber Laetitia Justine

AU - Annink, Kim Valerie

AU - van Stel, Henk

AU - Al-Mayouf, Sulaiman M

AU - Amaryan, Gayane

AU - Anton, Jordi

AU - Barron, Karyl S

AU - Benseler, Susanne

AU - Brogan, Paul A

AU - Cantarini, Luca

AU - Cattalini, Marco

AU - Cochino, Alexis-Virgil

AU - de Benedetti, Fabrizio

AU - Dedeoglu, Fatma

AU - de Jesus, Adriana Almeida

AU - Demirkaya, Erkan

AU - Dolezalova, Pavla

AU - Durrant, Karen L

AU - Fabio, Giovanna

AU - Gallizzi, Romina

AU - Goldbach-Mansky, Raphaela

AU - Hachulla, Eric

AU - Hentgen, Veronique

AU - Herlin, Troels

AU - Hofer, Michaël

AU - Hoffman, Hal M

AU - Insalaco, Antonella

AU - Jansson, Annette F

AU - Kallinich, Tilmann

AU - Kone-Paut, Isabelle

AU - Kozlova, Anna

AU - Kuemmerle-Deschner, Jasmin Beate

AU - Lachmann, Helen J

AU - Laxer, Ronald M

AU - Martini, Alberto

AU - Nielsen, Susan

AU - Nikishina, Irina

AU - Ombrello, Amanda K

AU - Özen, Seza

AU - Papadopoulou-Alataki, Efimia

AU - Quartier, Pierre

AU - Rigante, Donato

AU - Russo, Ricardo

AU - Simon, Anna

AU - Trachana, Maria

AU - Uziel, Yosef

AU - Ravelli, Angelo

AU - Schulert, Grant

AU - Gattorno, Marco

AU - Frenkel, Joost

N1 - © Author(s) (or their employer(s)) 2018. No commercial re-use. See rights and permissions. Published by BMJ.

PY - 2018/11

Y1 - 2018/11

N2 - INTRODUCTION: Autoinflammatory diseases can cause irreversible tissue damage due to systemic inflammation. Recently, the Autoinflammatory Disease Damage Index (ADDI) was developed. The ADDI is the first instrument to quantify damage in familial Mediterranean fever, cryopyrin-associated periodic syndromes, mevalonate kinase deficiency and tumour necrosis factor receptor-associated periodic syndrome. The aim of this study was to validate this tool for its intended use in a clinical/research setting.METHODS: The ADDI was scored on paper clinical cases by at least three physicians per case, independently of each other. Face and content validity were assessed by requesting comments on the ADDI. Reliability was tested by calculating the intraclass correlation coefficient (ICC) using an 'observer-nested-within-subject' design. Construct validity was determined by correlating the ADDI score to the Physician Global Assessment (PGA) of damage and disease activity. Redundancy of individual items was determined with Cronbach's alpha.RESULTS: The ADDI was validated on a total of 110 paper clinical cases by 37 experts in autoinflammatory diseases. This yielded an ICC of 0.84 (95% CI 0.78 to 0.89). The ADDI score correlated strongly with PGA-damage (r=0.92, 95% CI 0.88 to 0.95) and was not strongly influenced by disease activity (r=0.395, 95% CI 0.21 to 0.55). After comments from disease experts, some item definitions were refined. The interitem correlation in all different categories was lower than 0.7, indicating that there was no redundancy between individual damage items.CONCLUSION: The ADDI is a reliable and valid instrument to quantify damage in individual patients and can be used to compare disease outcomes in clinical studies.

AB - INTRODUCTION: Autoinflammatory diseases can cause irreversible tissue damage due to systemic inflammation. Recently, the Autoinflammatory Disease Damage Index (ADDI) was developed. The ADDI is the first instrument to quantify damage in familial Mediterranean fever, cryopyrin-associated periodic syndromes, mevalonate kinase deficiency and tumour necrosis factor receptor-associated periodic syndrome. The aim of this study was to validate this tool for its intended use in a clinical/research setting.METHODS: The ADDI was scored on paper clinical cases by at least three physicians per case, independently of each other. Face and content validity were assessed by requesting comments on the ADDI. Reliability was tested by calculating the intraclass correlation coefficient (ICC) using an 'observer-nested-within-subject' design. Construct validity was determined by correlating the ADDI score to the Physician Global Assessment (PGA) of damage and disease activity. Redundancy of individual items was determined with Cronbach's alpha.RESULTS: The ADDI was validated on a total of 110 paper clinical cases by 37 experts in autoinflammatory diseases. This yielded an ICC of 0.84 (95% CI 0.78 to 0.89). The ADDI score correlated strongly with PGA-damage (r=0.92, 95% CI 0.88 to 0.95) and was not strongly influenced by disease activity (r=0.395, 95% CI 0.21 to 0.55). After comments from disease experts, some item definitions were refined. The interitem correlation in all different categories was lower than 0.7, indicating that there was no redundancy between individual damage items.CONCLUSION: The ADDI is a reliable and valid instrument to quantify damage in individual patients and can be used to compare disease outcomes in clinical studies.

U2 - 10.1136/annrheumdis-2018-213725

DO - 10.1136/annrheumdis-2018-213725

M3 - Article

C2 - 30077992

VL - 77

SP - 1599

EP - 1605

JO - Annals of the Rheumatic Diseases

JF - Annals of the Rheumatic Diseases

SN - 0003-4967

IS - 11

ER -