American College of Rheumatology Provisional Criteria for Clinically Relevant Improvement in Children and Adolescents With Childhood-Onset Systemic Lupus Erythematosus

the Paediatric Rheumatology International Trial Organisation and Pediatric Rheumatology Collaborative Study Group

doi:10.1002/acr.23834

American College of Rheumatology Provisional Criteria for Clinically Relevant Improvement in Children and Adolescents With Childhood-Onset Systemic Lupus Erythematosus

the Paediatric Rheumatology International Trial Organisation and Pediatric Rheumatology Collaborative Study Group

Pediatric Rheumatology, Allergy & Immunology

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

24 Scopus citations

Abstract

Objective: To develop a Childhood Lupus Improvement Index (CHILI) as a tool to measure response to therapy in childhood-onset systemic lupus erythematosus (cSLE), with a focus on clinically relevant improvement (CRI _c _SLE ). Methods: Pediatric nephrology and rheumatology subspecialists (n = 213) experienced in cSLE management were invited to define CRI _c _SLE and rate a total of 433 unique patient profiles for the presence/absence of CRI _c _SLE . Patient profiles included the following cSLE core response variables (CRVs): global assessment of patient well-being (patient-global), physician assessment of cSLE activity (MD-global), disease activity index score (here, we used the Systemic Lupus Erythematosus Disease Activity Index), urine protein-to-creatinine ratio, and Child Health Questionnaire physical summary score. Percentage and absolute changes in these cSLE-CRVs (baseline versus follow-up) were considered in order to develop candidate algorithms and validate their performance (sensitivity, specificity, area under the receiver operating characteristic curve [AUC]; range 0–1). Results: During an international consensus conference, unanimous agreement on a definition of CRI _c _SLE was achieved; cSLE experts (n = 13) concurred (100%) that the preferred CHILI algorithm considers absolute changes in the cSLE-CRVs. After transformation to a range of 0–100, a CHILI score of ≥54 had outstanding accuracy for identifying CRI _c _SLE (AUC 0.93, sensitivity 81.1%, and specificity 84.2%). CHILI scores also reflect minor, moderate, and major improvement for values exceeding 15, 68, and 92, respectively (all AUC ≥0.92, sensitivity ≥93.1%, and specificity ≥73.4%). Conclusion: The CHILI is a new, seemingly highly accurate index for measuring CRI in cSLE over time. This index is useful to categorize the degree of response to therapy in children and adolescents with cSLE.

Original language	English (US)
Pages (from-to)	579-590
Number of pages	12
Journal	Arthritis Care and Research
Volume	71
Issue number	5
DOIs	https://doi.org/10.1002/acr.23834
State	Published - May 2019

Bibliographical note

Funding Information:
We thank Kasha Wiley (overall study coordination), Susan Priest (consensus conference logistics), Carly Muller, Malea Rolfsen, Allen Watts, Gaurav Gulati, and Jamie Meyers-Eaton (patient profile testing) from Cincinnati Children Hospital Medical Center (CCHMC) as well as CCHMC Biomedical Informatics (web-based data management application development). We are indebted to the members of the External Scientific Advisory Committee of this study for their advice regarding study implementation, conduct, and statistical analysis: Drs. Tuhina Neogi, Ian Bruce, David Isenberg, Nicola Ruperto, and James Witter.

Funding Information:
12Marisa S? Klein-Gitelman, MD: Northwestern University Feinberg School of Medicine and Ann and Robert Lurie Children’s Hospital of Chicago, Chicago, 阀llinois13;J un Ying, PhD: University of Cincinnati, Cincinnati, Ohio? Dr? ?runner has received consulting fees from AbbVie, Ablynx, Amgen, AstraZeneca, ?axalta ?iosimilars, ?iogen 阀dec, ?oehringer 阀ngelheim, ?ristol-Myers Squibb, Celgene, Eli Lilly, EMD Serono, Gilead Sciences, Janssen, Med 阀mmune, Novartis, Pfizer, R-Pharm, Roche, Servier, Takeda (less than $10,000 each), speaking fees from Genentech and Novartis (more than $10,000 each), and Scientific grant support from Pfizer and ?ristol-Myers Squibb for research studies in adult lupus and juvenile idiopathic arthritis? Dr? ?runner is a full-time employee of Cincinnati Children’s Hospital, which has received contributions from ?ristol-Myers Squibb, Ho 贀man-La Roche, Janssen, Novartis, and Pfizer for the coordination activity of the Pediatric Rheumatology Collaborative Study Group network? Dr? Ravelli has received consulting and/or speaking fees from AbbVie, ?ristol-Myers Squibb, Pfizer, Ho 贀man-LaRoche, Novartis, Centocor, “Francesco Angelini,” and Reckitt ?enckiser (less than $10,000 each)? Dr? Avcin has received consulting and/or speaking fees from AbbVie and ?oehringer 阀ngelheim (less than

Funding Information:
Supported by the N 阀H (grants 5U01-AR-51868, P30-AR-AR47363, and 2UL1-RR-026314)? Dr? Silva’s work was supported by grants from Fundação de Amparo à Pesquisa do Estado de São Paulo (FAPESP 215/03756-4), Conselho Nacional de Desenvolvimento Científico e Tecnológico (CNPq 303422/2015-7 and CNPq 304255/2015-7), and by Núcleo de Apoio à Pesquisa “Saúde da Criança e do Adolescente” da USP (NAP-CriAd)? Dr? Ardoin’s work was supported by CNPq 7/2016-9?

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© 2019, American College of Rheumatology

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10.1002/acr.23834

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American College of Rheumatology Provisional Criteria for Clinically Relevant Improvement in Children and Adolescents With Childhood-Onset Systemic Lupus Erythematosus. / the Paediatric Rheumatology International Trial Organisation and Pediatric Rheumatology Collaborative Study Group.
In: Arthritis Care and Research, Vol. 71, No. 5, 05.2019, p. 579-590.

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

the Paediatric Rheumatology International Trial Organisation and Pediatric Rheumatology Collaborative Study Group 2019, 'American College of Rheumatology Provisional Criteria for Clinically Relevant Improvement in Children and Adolescents With Childhood-Onset Systemic Lupus Erythematosus', Arthritis Care and Research, vol. 71, no. 5, pp. 579-590. https://doi.org/10.1002/acr.23834

@article{e01a8e92bc514f8aa7a3e34e4ef27e96,

title = "American College of Rheumatology Provisional Criteria for Clinically Relevant Improvement in Children and Adolescents With Childhood-Onset Systemic Lupus Erythematosus",

abstract = " Objective: To develop a Childhood Lupus Improvement Index (CHILI) as a tool to measure response to therapy in childhood-onset systemic lupus erythematosus (cSLE), with a focus on clinically relevant improvement (CRI c SLE ). Methods: Pediatric nephrology and rheumatology subspecialists (n = 213) experienced in cSLE management were invited to define CRI c SLE and rate a total of 433 unique patient profiles for the presence/absence of CRI c SLE . Patient profiles included the following cSLE core response variables (CRVs): global assessment of patient well-being (patient-global), physician assessment of cSLE activity (MD-global), disease activity index score (here, we used the Systemic Lupus Erythematosus Disease Activity Index), urine protein-to-creatinine ratio, and Child Health Questionnaire physical summary score. Percentage and absolute changes in these cSLE-CRVs (baseline versus follow-up) were considered in order to develop candidate algorithms and validate their performance (sensitivity, specificity, area under the receiver operating characteristic curve [AUC]; range 0{\textendash}1). Results: During an international consensus conference, unanimous agreement on a definition of CRI c SLE was achieved; cSLE experts (n = 13) concurred (100\%) that the preferred CHILI algorithm considers absolute changes in the cSLE-CRVs. After transformation to a range of 0{\textendash}100, a CHILI score of ≥54 had outstanding accuracy for identifying CRI c SLE (AUC 0.93, sensitivity 81.1\%, and specificity 84.2\%). CHILI scores also reflect minor, moderate, and major improvement for values exceeding 15, 68, and 92, respectively (all AUC ≥0.92, sensitivity ≥93.1\%, and specificity ≥73.4\%). Conclusion: The CHILI is a new, seemingly highly accurate index for measuring CRI in cSLE over time. This index is useful to categorize the degree of response to therapy in children and adolescents with cSLE.",

author = "\{the Paediatric Rheumatology International Trial Organisation and Pediatric Rheumatology Collaborative Study Group\} and Brunner, \{Hermine I.\} and Holland, \{Michael J.\} and Beresford, \{Michael W.\} and Ardoin, \{Stacy P.\} and Simone Appenzeller and Silva, \{Clovis A.\} and Francisco Flores and Beatrice Goilav and \{Avar Aydin\}, \{Pinar Ozge\} and Wenderfer, \{Scott E.\} and Levy, \{Deborah M.\} and Angelo Ravelli and Raju Khubchandani and Tadej Avcin and Klein-Gitelman, \{Marisa S.\} and Nicolino Ruperto and Feldman, \{Brian M.\} and Jun Ying and Cristina Battagliotti and Brusco, \{Maria Isabel\} and Rub{\'e}n Cuttica and \{De Cunto\}, Carmen and Graciela Espada and Maximiliano Farfan and Stella Garay and Maria Marcantoni and Alvarez Marcela and Silvia Meiorin and Rama, \{Maria Elena\} and Ricardo Russo and \{Torre Walsh\}, Carolina and Celso Zamparo and Navid Adib and Jonathan Akikusa and Christina Boros and Lee, \{Senq J.\} and Damien Mckay and Susan Piper and Rik Joos and Blanca Bica and Leonardo Campos and Andr{\'e} Cavalcanti and \{do Prado\}, Rogerio and Amanda Donner-Maliki and Taciana Fernandes and Adriana Fonseca and \{Gasparello de Almeida\}, Rozana and Andressa Guariento and Bryce Binstadt and Richard Vehe",

note = "Funding Information: We thank Kasha Wiley (overall study coordination), Susan Priest (consensus conference logistics), Carly Muller, Malea Rolfsen, Allen Watts, Gaurav Gulati, and Jamie Meyers-Eaton (patient profile testing) from Cincinnati Children Hospital Medical Center (CCHMC) as well as CCHMC Biomedical Informatics (web-based data management application development). We are indebted to the members of the External Scientific Advisory Committee of this study for their advice regarding study implementation, conduct, and statistical analysis: Drs. Tuhina Neogi, Ian Bruce, David Isenberg, Nicola Ruperto, and James Witter. Funding Information: 12Marisa S? Klein-Gitelman, MD: Northwestern University Feinberg School of Medicine and Ann and Robert Lurie Children{\textquoteright}s Hospital of Chicago, Chicago, 阀llinois13;J un Ying, PhD: University of Cincinnati, Cincinnati, Ohio? Dr? ?runner has received consulting fees from AbbVie, Ablynx, Amgen, AstraZeneca, ?axalta ?iosimilars, ?iogen 阀dec, ?oehringer 阀ngelheim, ?ristol-Myers Squibb, Celgene, Eli Lilly, EMD Serono, Gilead Sciences, Janssen, Med 阀mmune, Novartis, Pfizer, R-Pharm, Roche, Servier, Takeda (less than \$10,000 each), speaking fees from Genentech and Novartis (more than \$10,000 each), and Scientific grant support from Pfizer and ?ristol-Myers Squibb for research studies in adult lupus and juvenile idiopathic arthritis? Dr? ?runner is a full-time employee of Cincinnati Children{\textquoteright}s Hospital, which has received contributions from ?ristol-Myers Squibb, Ho 贀man-La Roche, Janssen, Novartis, and Pfizer for the coordination activity of the Pediatric Rheumatology Collaborative Study Group network? Dr? Ravelli has received consulting and/or speaking fees from AbbVie, ?ristol-Myers Squibb, Pfizer, Ho 贀man-LaRoche, Novartis, Centocor, {\textquotedblleft}Francesco Angelini,{\textquotedblright} and Reckitt ?enckiser (less than \$10,000 each)? Dr? Avcin has received consulting and/or speaking fees from AbbVie and ?oehringer 阀ngelheim (less than Funding Information: Supported by the N 阀H (grants 5U01-AR-51868, P30-AR-AR47363, and 2UL1-RR-026314)? Dr? Silva{\textquoteright}s work was supported by grants from Funda{\c c}{\~a}o de Amparo {\`a} Pesquisa do Estado de S{\~a}o Paulo (FAPESP 215/03756-4), Conselho Nacional de Desenvolvimento Cient{\'i}fico e Tecnol{\'o}gico (CNPq 303422/2015-7 and CNPq 304255/2015-7), and by N{\'u}cleo de Apoio {\`a} Pesquisa {\textquotedblleft}Sa{\'u}de da Crian{\c c}a e do Adolescente{\textquotedblright} da USP (NAP-CriAd)? Dr? Ardoin{\textquoteright}s work was supported by CNPq 7/2016-9? Publisher Copyright: {\textcopyright} 2019, American College of Rheumatology",

year = "2019",

month = may,

doi = "10.1002/acr.23834",

language = "English (US)",

volume = "71",

pages = "579--590",

journal = "Arthritis Care and Research",

issn = "2151-464X",

number = "5",

}

TY - JOUR

T1 - American College of Rheumatology Provisional Criteria for Clinically Relevant Improvement in Children and Adolescents With Childhood-Onset Systemic Lupus Erythematosus

AU - the Paediatric Rheumatology International Trial Organisation and Pediatric Rheumatology Collaborative Study Group

AU - Brunner, Hermine I.

AU - Holland, Michael J.

AU - Beresford, Michael W.

AU - Ardoin, Stacy P.

AU - Appenzeller, Simone

AU - Silva, Clovis A.

AU - Flores, Francisco

AU - Goilav, Beatrice

AU - Avar Aydin, Pinar Ozge

AU - Wenderfer, Scott E.

AU - Levy, Deborah M.

AU - Ravelli, Angelo

AU - Khubchandani, Raju

AU - Avcin, Tadej

AU - Klein-Gitelman, Marisa S.

AU - Ruperto, Nicolino

AU - Feldman, Brian M.

AU - Ying, Jun

AU - Battagliotti, Cristina

AU - Brusco, Maria Isabel

AU - Cuttica, Rubén

AU - De Cunto, Carmen

AU - Espada, Graciela

AU - Farfan, Maximiliano

AU - Garay, Stella

AU - Marcantoni, Maria

AU - Marcela, Alvarez

AU - Meiorin, Silvia

AU - Rama, Maria Elena

AU - Russo, Ricardo

AU - Torre Walsh, Carolina

AU - Zamparo, Celso

AU - Adib, Navid

AU - Akikusa, Jonathan

AU - Boros, Christina

AU - Lee, Senq J.

AU - Mckay, Damien

AU - Piper, Susan

AU - Joos, Rik

AU - Bica, Blanca

AU - Campos, Leonardo

AU - Cavalcanti, André

AU - do Prado, Rogerio

AU - Donner-Maliki, Amanda

AU - Fernandes, Taciana

AU - Fonseca, Adriana

AU - Gasparello de Almeida, Rozana

AU - Guariento, Andressa

AU - Binstadt, Bryce

AU - Vehe, Richard

N1 - Funding Information: We thank Kasha Wiley (overall study coordination), Susan Priest (consensus conference logistics), Carly Muller, Malea Rolfsen, Allen Watts, Gaurav Gulati, and Jamie Meyers-Eaton (patient profile testing) from Cincinnati Children Hospital Medical Center (CCHMC) as well as CCHMC Biomedical Informatics (web-based data management application development). We are indebted to the members of the External Scientific Advisory Committee of this study for their advice regarding study implementation, conduct, and statistical analysis: Drs. Tuhina Neogi, Ian Bruce, David Isenberg, Nicola Ruperto, and James Witter. Funding Information: 12Marisa S? Klein-Gitelman, MD: Northwestern University Feinberg School of Medicine and Ann and Robert Lurie Children’s Hospital of Chicago, Chicago, 阀llinois13;J un Ying, PhD: University of Cincinnati, Cincinnati, Ohio? Dr? ?runner has received consulting fees from AbbVie, Ablynx, Amgen, AstraZeneca, ?axalta ?iosimilars, ?iogen 阀dec, ?oehringer 阀ngelheim, ?ristol-Myers Squibb, Celgene, Eli Lilly, EMD Serono, Gilead Sciences, Janssen, Med 阀mmune, Novartis, Pfizer, R-Pharm, Roche, Servier, Takeda (less than $10,000 each), speaking fees from Genentech and Novartis (more than $10,000 each), and Scientific grant support from Pfizer and ?ristol-Myers Squibb for research studies in adult lupus and juvenile idiopathic arthritis? Dr? ?runner is a full-time employee of Cincinnati Children’s Hospital, which has received contributions from ?ristol-Myers Squibb, Ho 贀man-La Roche, Janssen, Novartis, and Pfizer for the coordination activity of the Pediatric Rheumatology Collaborative Study Group network? Dr? Ravelli has received consulting and/or speaking fees from AbbVie, ?ristol-Myers Squibb, Pfizer, Ho 贀man-LaRoche, Novartis, Centocor, “Francesco Angelini,” and Reckitt ?enckiser (less than $10,000 each)? Dr? Avcin has received consulting and/or speaking fees from AbbVie and ?oehringer 阀ngelheim (less than Funding Information: Supported by the N 阀H (grants 5U01-AR-51868, P30-AR-AR47363, and 2UL1-RR-026314)? Dr? Silva’s work was supported by grants from Fundação de Amparo à Pesquisa do Estado de São Paulo (FAPESP 215/03756-4), Conselho Nacional de Desenvolvimento Científico e Tecnológico (CNPq 303422/2015-7 and CNPq 304255/2015-7), and by Núcleo de Apoio à Pesquisa “Saúde da Criança e do Adolescente” da USP (NAP-CriAd)? Dr? Ardoin’s work was supported by CNPq 7/2016-9? Publisher Copyright: © 2019, American College of Rheumatology

PY - 2019/5

Y1 - 2019/5

N2 - Objective: To develop a Childhood Lupus Improvement Index (CHILI) as a tool to measure response to therapy in childhood-onset systemic lupus erythematosus (cSLE), with a focus on clinically relevant improvement (CRI c SLE ). Methods: Pediatric nephrology and rheumatology subspecialists (n = 213) experienced in cSLE management were invited to define CRI c SLE and rate a total of 433 unique patient profiles for the presence/absence of CRI c SLE . Patient profiles included the following cSLE core response variables (CRVs): global assessment of patient well-being (patient-global), physician assessment of cSLE activity (MD-global), disease activity index score (here, we used the Systemic Lupus Erythematosus Disease Activity Index), urine protein-to-creatinine ratio, and Child Health Questionnaire physical summary score. Percentage and absolute changes in these cSLE-CRVs (baseline versus follow-up) were considered in order to develop candidate algorithms and validate their performance (sensitivity, specificity, area under the receiver operating characteristic curve [AUC]; range 0–1). Results: During an international consensus conference, unanimous agreement on a definition of CRI c SLE was achieved; cSLE experts (n = 13) concurred (100%) that the preferred CHILI algorithm considers absolute changes in the cSLE-CRVs. After transformation to a range of 0–100, a CHILI score of ≥54 had outstanding accuracy for identifying CRI c SLE (AUC 0.93, sensitivity 81.1%, and specificity 84.2%). CHILI scores also reflect minor, moderate, and major improvement for values exceeding 15, 68, and 92, respectively (all AUC ≥0.92, sensitivity ≥93.1%, and specificity ≥73.4%). Conclusion: The CHILI is a new, seemingly highly accurate index for measuring CRI in cSLE over time. This index is useful to categorize the degree of response to therapy in children and adolescents with cSLE.

AB - Objective: To develop a Childhood Lupus Improvement Index (CHILI) as a tool to measure response to therapy in childhood-onset systemic lupus erythematosus (cSLE), with a focus on clinically relevant improvement (CRI c SLE ). Methods: Pediatric nephrology and rheumatology subspecialists (n = 213) experienced in cSLE management were invited to define CRI c SLE and rate a total of 433 unique patient profiles for the presence/absence of CRI c SLE . Patient profiles included the following cSLE core response variables (CRVs): global assessment of patient well-being (patient-global), physician assessment of cSLE activity (MD-global), disease activity index score (here, we used the Systemic Lupus Erythematosus Disease Activity Index), urine protein-to-creatinine ratio, and Child Health Questionnaire physical summary score. Percentage and absolute changes in these cSLE-CRVs (baseline versus follow-up) were considered in order to develop candidate algorithms and validate their performance (sensitivity, specificity, area under the receiver operating characteristic curve [AUC]; range 0–1). Results: During an international consensus conference, unanimous agreement on a definition of CRI c SLE was achieved; cSLE experts (n = 13) concurred (100%) that the preferred CHILI algorithm considers absolute changes in the cSLE-CRVs. After transformation to a range of 0–100, a CHILI score of ≥54 had outstanding accuracy for identifying CRI c SLE (AUC 0.93, sensitivity 81.1%, and specificity 84.2%). CHILI scores also reflect minor, moderate, and major improvement for values exceeding 15, 68, and 92, respectively (all AUC ≥0.92, sensitivity ≥93.1%, and specificity ≥73.4%). Conclusion: The CHILI is a new, seemingly highly accurate index for measuring CRI in cSLE over time. This index is useful to categorize the degree of response to therapy in children and adolescents with cSLE.

UR - https://www.scopus.com/pages/publications/85064810180

UR - https://www.scopus.com/pages/publications/85064810180#tab=citedBy

U2 - 10.1002/acr.23834

DO - 10.1002/acr.23834

M3 - Article

C2 - 30680946

AN - SCOPUS:85064810180

SN - 2151-464X

VL - 71

SP - 579

EP - 590

JO - Arthritis Care and Research

JF - Arthritis Care and Research

IS - 5

ER -

American College of Rheumatology Provisional Criteria for Clinically Relevant Improvement in Children and Adolescents With Childhood-Onset Systemic Lupus Erythematosus

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