Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis

Alain M. Schoepfer, Alex Straumann, Radoslaw Panczak, Michael Coslovsky, Claudia E. Kuehni, Elisabeth Maurer, Nadine A. Haas, Yvonne Romero, Ikuo Hirano, Jeffrey A. Alexander, Nirmala Gonsalves, Glenn T. Furuta, Evan S. Dellon, John Leung, Margaret H. Collins, Christian Bussmann, Peter Netzer, Sandeep Gupta, Seema S. Aceves, Mirna ChehadeFouad J. Moawad, Felicity T. Enders, Kathleen J. Yost, Tiffany H. Taft, Emily Kern, Marcel Zwahlen, Ekaterina Safroneeva, Sami R. Achem, Amindra S. Arora, Oral Alpan, David Armstrong, Stephen E. Attwood, Joseph H. Butterfield, Michael D. Crowell, Giovanni De Petris, Kenneth R. DeVault, Eric Drouin, Benjamin Enav, David E. Fleischer, Amy Foxx-Orenstein, Dawn L. Francis, Gordon H. Guyatt, Lucinda A. Harris, Amir F. Kagalwalla, David A. Katzka, Hirohito Kita, Murli Krishna, James J. Lee, John C. Lewis, Kaiser Lim, G. Richard Locke, Joseph A. Murray, Cuong C. Nguyen, Diana M. Orbelo, Shabana F. Pasha, Francisco C. Ramirez, Javed Sheikh, Thomas C. Smyrk, Jonathan M. Spergel, Sarah B. Umar, Catherine R. Weiler, John Wo, John T. Woosley, Tsung Teh Wu, Pu Yan, Guang Yu Yang

Research output: Contribution to journalArticle

101 Citations (Scopus)

Abstract

BACKGROUND & AIMS: Standardized instruments are needed to assess the activity of eosinophilic esophagitis (EoE) and to provide end points for clinical trials and observational studies. We aimed to develop and validate a patient-reported outcome (PRO) instrument and score, based on items that could account for variations in patient assessments of disease severity. We also evaluated relationships between patient assessment of disease severity and EoE-associated endoscopic, histologic, and laboratory findings.

METHODS: We collected information from 186 patients with EoE in Switzerland and the United States (69.4% male; median age, 43 y) via surveys (n = 135), focus groups (n = 27), and semistructured interviews (n = 24). Items were generated for the instruments to assess biologic activity based on physician input. Linear regression was used to quantify the extent to which variations in patient-reported disease characteristics could account for variations in patient assessment of EoE severity. The PRO instrument was used prospectively in 153 adult patients with EoE (72.5% male; median age, 38 y), and validated in an independent group of 120 patients with EoE (60.8% male; median age, 40.5 y).

RESULTS: Seven PRO factors that are used to assess characteristics of dysphagia, behavioral adaptations to living with dysphagia, and pain while swallowing accounted for 67% of the variation in patient assessment of disease severity. Based on statistical consideration and patient input, a 7-day recall period was selected. Highly active EoE, based on endoscopic and histologic findings, was associated with an increase in patient-assessed disease severity. In the validation study, the mean difference between patient assessment of EoE severity (range, 0-10) and PRO score (range, 0-8.52) was 0.15.

CONCLUSIONS: We developed and validated an EoE scoring system based on 7 PRO items that assess symptoms over a 7-day recall period.

Original languageEnglish
Pages (from-to)1255-1266.e21
JournalGastroenterology
Volume147
Issue number6
DOIs
StatePublished - Dec 1 2014

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Eosinophilic Esophagitis
Deglutition Disorders
Validation Studies
Deglutition
Focus Groups
Switzerland
Observational Studies
Linear Models

Keywords

  • Disease Activity Measurement
  • Esophagus
  • Marker
  • Patient-Reported Outcome

ASJC Scopus subject areas

  • Gastroenterology
  • Medicine(all)

Cite this

Schoepfer, A. M., Straumann, A., Panczak, R., Coslovsky, M., Kuehni, C. E., Maurer, E., ... Yang, G. Y. (2014). Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis. Gastroenterology, 147(6), 1255-1266.e21. https://doi.org/10.1053/j.gastro.2014.08.028

Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis. / Schoepfer, Alain M.; Straumann, Alex; Panczak, Radoslaw; Coslovsky, Michael; Kuehni, Claudia E.; Maurer, Elisabeth; Haas, Nadine A.; Romero, Yvonne; Hirano, Ikuo; Alexander, Jeffrey A.; Gonsalves, Nirmala; Furuta, Glenn T.; Dellon, Evan S.; Leung, John; Collins, Margaret H.; Bussmann, Christian; Netzer, Peter; Gupta, Sandeep; Aceves, Seema S.; Chehade, Mirna; Moawad, Fouad J.; Enders, Felicity T.; Yost, Kathleen J.; Taft, Tiffany H.; Kern, Emily; Zwahlen, Marcel; Safroneeva, Ekaterina; Achem, Sami R.; Arora, Amindra S.; Alpan, Oral; Armstrong, David; Attwood, Stephen E.; Butterfield, Joseph H.; Crowell, Michael D.; De Petris, Giovanni; DeVault, Kenneth R.; Drouin, Eric; Enav, Benjamin; Fleischer, David E.; Foxx-Orenstein, Amy; Francis, Dawn L.; Guyatt, Gordon H.; Harris, Lucinda A.; Kagalwalla, Amir F.; Katzka, David A.; Kita, Hirohito; Krishna, Murli; Lee, James J.; Lewis, John C.; Lim, Kaiser; Locke, G. Richard; Murray, Joseph A.; Nguyen, Cuong C.; Orbelo, Diana M.; Pasha, Shabana F.; Ramirez, Francisco C.; Sheikh, Javed; Smyrk, Thomas C.; Spergel, Jonathan M.; Umar, Sarah B.; Weiler, Catherine R.; Wo, John; Woosley, John T.; Wu, Tsung Teh; Yan, Pu; Yang, Guang Yu.

In: Gastroenterology, Vol. 147, No. 6, 01.12.2014, p. 1255-1266.e21.

Research output: Contribution to journalArticle

Schoepfer, AM, Straumann, A, Panczak, R, Coslovsky, M, Kuehni, CE, Maurer, E, Haas, NA, Romero, Y, Hirano, I, Alexander, JA, Gonsalves, N, Furuta, GT, Dellon, ES, Leung, J, Collins, MH, Bussmann, C, Netzer, P, Gupta, S, Aceves, SS, Chehade, M, Moawad, FJ, Enders, FT, Yost, KJ, Taft, TH, Kern, E, Zwahlen, M, Safroneeva, E, Achem, SR, Arora, AS, Alpan, O, Armstrong, D, Attwood, SE, Butterfield, JH, Crowell, MD, De Petris, G, DeVault, KR, Drouin, E, Enav, B, Fleischer, DE, Foxx-Orenstein, A, Francis, DL, Guyatt, GH, Harris, LA, Kagalwalla, AF, Katzka, DA, Kita, H, Krishna, M, Lee, JJ, Lewis, JC, Lim, K, Locke, GR, Murray, JA, Nguyen, CC, Orbelo, DM, Pasha, SF, Ramirez, FC, Sheikh, J, Smyrk, TC, Spergel, JM, Umar, SB, Weiler, CR, Wo, J, Woosley, JT, Wu, TT, Yan, P & Yang, GY 2014, 'Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis', Gastroenterology, vol. 147, no. 6, pp. 1255-1266.e21. https://doi.org/10.1053/j.gastro.2014.08.028
Schoepfer AM, Straumann A, Panczak R, Coslovsky M, Kuehni CE, Maurer E et al. Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis. Gastroenterology. 2014 Dec 1;147(6):1255-1266.e21. https://doi.org/10.1053/j.gastro.2014.08.028
Schoepfer, Alain M. ; Straumann, Alex ; Panczak, Radoslaw ; Coslovsky, Michael ; Kuehni, Claudia E. ; Maurer, Elisabeth ; Haas, Nadine A. ; Romero, Yvonne ; Hirano, Ikuo ; Alexander, Jeffrey A. ; Gonsalves, Nirmala ; Furuta, Glenn T. ; Dellon, Evan S. ; Leung, John ; Collins, Margaret H. ; Bussmann, Christian ; Netzer, Peter ; Gupta, Sandeep ; Aceves, Seema S. ; Chehade, Mirna ; Moawad, Fouad J. ; Enders, Felicity T. ; Yost, Kathleen J. ; Taft, Tiffany H. ; Kern, Emily ; Zwahlen, Marcel ; Safroneeva, Ekaterina ; Achem, Sami R. ; Arora, Amindra S. ; Alpan, Oral ; Armstrong, David ; Attwood, Stephen E. ; Butterfield, Joseph H. ; Crowell, Michael D. ; De Petris, Giovanni ; DeVault, Kenneth R. ; Drouin, Eric ; Enav, Benjamin ; Fleischer, David E. ; Foxx-Orenstein, Amy ; Francis, Dawn L. ; Guyatt, Gordon H. ; Harris, Lucinda A. ; Kagalwalla, Amir F. ; Katzka, David A. ; Kita, Hirohito ; Krishna, Murli ; Lee, James J. ; Lewis, John C. ; Lim, Kaiser ; Locke, G. Richard ; Murray, Joseph A. ; Nguyen, Cuong C. ; Orbelo, Diana M. ; Pasha, Shabana F. ; Ramirez, Francisco C. ; Sheikh, Javed ; Smyrk, Thomas C. ; Spergel, Jonathan M. ; Umar, Sarah B. ; Weiler, Catherine R. ; Wo, John ; Woosley, John T. ; Wu, Tsung Teh ; Yan, Pu ; Yang, Guang Yu. / Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis. In: Gastroenterology. 2014 ; Vol. 147, No. 6. pp. 1255-1266.e21.
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abstract = "BACKGROUND & AIMS: Standardized instruments are needed to assess the activity of eosinophilic esophagitis (EoE) and to provide end points for clinical trials and observational studies. We aimed to develop and validate a patient-reported outcome (PRO) instrument and score, based on items that could account for variations in patient assessments of disease severity. We also evaluated relationships between patient assessment of disease severity and EoE-associated endoscopic, histologic, and laboratory findings.METHODS: We collected information from 186 patients with EoE in Switzerland and the United States (69.4{\%} male; median age, 43 y) via surveys (n = 135), focus groups (n = 27), and semistructured interviews (n = 24). Items were generated for the instruments to assess biologic activity based on physician input. Linear regression was used to quantify the extent to which variations in patient-reported disease characteristics could account for variations in patient assessment of EoE severity. The PRO instrument was used prospectively in 153 adult patients with EoE (72.5{\%} male; median age, 38 y), and validated in an independent group of 120 patients with EoE (60.8{\%} male; median age, 40.5 y).RESULTS: Seven PRO factors that are used to assess characteristics of dysphagia, behavioral adaptations to living with dysphagia, and pain while swallowing accounted for 67{\%} of the variation in patient assessment of disease severity. Based on statistical consideration and patient input, a 7-day recall period was selected. Highly active EoE, based on endoscopic and histologic findings, was associated with an increase in patient-assessed disease severity. In the validation study, the mean difference between patient assessment of EoE severity (range, 0-10) and PRO score (range, 0-8.52) was 0.15.CONCLUSIONS: We developed and validated an EoE scoring system based on 7 PRO items that assess symptoms over a 7-day recall period.",
keywords = "Disease Activity Measurement, Esophagus, Marker, Patient-Reported Outcome",
author = "Schoepfer, {Alain M.} and Alex Straumann and Radoslaw Panczak and Michael Coslovsky and Kuehni, {Claudia E.} and Elisabeth Maurer and Haas, {Nadine A.} and Yvonne Romero and Ikuo Hirano and Alexander, {Jeffrey A.} and Nirmala Gonsalves and Furuta, {Glenn T.} and Dellon, {Evan S.} and John Leung and Collins, {Margaret H.} and Christian Bussmann and Peter Netzer and Sandeep Gupta and Aceves, {Seema S.} and Mirna Chehade and Moawad, {Fouad J.} and Enders, {Felicity T.} and Yost, {Kathleen J.} and Taft, {Tiffany H.} and Emily Kern and Marcel Zwahlen and Ekaterina Safroneeva and Achem, {Sami R.} and Arora, {Amindra S.} and Oral Alpan and David Armstrong and Attwood, {Stephen E.} and Butterfield, {Joseph H.} and Crowell, {Michael D.} and {De Petris}, Giovanni and DeVault, {Kenneth R.} and Eric Drouin and Benjamin Enav and Fleischer, {David E.} and Amy Foxx-Orenstein and Francis, {Dawn L.} and Guyatt, {Gordon H.} and Harris, {Lucinda A.} and Kagalwalla, {Amir F.} and Katzka, {David A.} and Hirohito Kita and Murli Krishna and Lee, {James J.} and Lewis, {John C.} and Kaiser Lim and Locke, {G. Richard} and Murray, {Joseph A.} and Nguyen, {Cuong C.} and Orbelo, {Diana M.} and Pasha, {Shabana F.} and Ramirez, {Francisco C.} and Javed Sheikh and Smyrk, {Thomas C.} and Spergel, {Jonathan M.} and Umar, {Sarah B.} and Weiler, {Catherine R.} and John Wo and Woosley, {John T.} and Wu, {Tsung Teh} and Pu Yan and Yang, {Guang Yu}",
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TY - JOUR

T1 - Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis

AU - Schoepfer, Alain M.

AU - Straumann, Alex

AU - Panczak, Radoslaw

AU - Coslovsky, Michael

AU - Kuehni, Claudia E.

AU - Maurer, Elisabeth

AU - Haas, Nadine A.

AU - Romero, Yvonne

AU - Hirano, Ikuo

AU - Alexander, Jeffrey A.

AU - Gonsalves, Nirmala

AU - Furuta, Glenn T.

AU - Dellon, Evan S.

AU - Leung, John

AU - Collins, Margaret H.

AU - Bussmann, Christian

AU - Netzer, Peter

AU - Gupta, Sandeep

AU - Aceves, Seema S.

AU - Chehade, Mirna

AU - Moawad, Fouad J.

AU - Enders, Felicity T.

AU - Yost, Kathleen J.

AU - Taft, Tiffany H.

AU - Kern, Emily

AU - Zwahlen, Marcel

AU - Safroneeva, Ekaterina

AU - Achem, Sami R.

AU - Arora, Amindra S.

AU - Alpan, Oral

AU - Armstrong, David

AU - Attwood, Stephen E.

AU - Butterfield, Joseph H.

AU - Crowell, Michael D.

AU - De Petris, Giovanni

AU - DeVault, Kenneth R.

AU - Drouin, Eric

AU - Enav, Benjamin

AU - Fleischer, David E.

AU - Foxx-Orenstein, Amy

AU - Francis, Dawn L.

AU - Guyatt, Gordon H.

AU - Harris, Lucinda A.

AU - Kagalwalla, Amir F.

AU - Katzka, David A.

AU - Kita, Hirohito

AU - Krishna, Murli

AU - Lee, James J.

AU - Lewis, John C.

AU - Lim, Kaiser

AU - Locke, G. Richard

AU - Murray, Joseph A.

AU - Nguyen, Cuong C.

AU - Orbelo, Diana M.

AU - Pasha, Shabana F.

AU - Ramirez, Francisco C.

AU - Sheikh, Javed

AU - Smyrk, Thomas C.

AU - Spergel, Jonathan M.

AU - Umar, Sarah B.

AU - Weiler, Catherine R.

AU - Wo, John

AU - Woosley, John T.

AU - Wu, Tsung Teh

AU - Yan, Pu

AU - Yang, Guang Yu

PY - 2014/12/1

Y1 - 2014/12/1

N2 - BACKGROUND & AIMS: Standardized instruments are needed to assess the activity of eosinophilic esophagitis (EoE) and to provide end points for clinical trials and observational studies. We aimed to develop and validate a patient-reported outcome (PRO) instrument and score, based on items that could account for variations in patient assessments of disease severity. We also evaluated relationships between patient assessment of disease severity and EoE-associated endoscopic, histologic, and laboratory findings.METHODS: We collected information from 186 patients with EoE in Switzerland and the United States (69.4% male; median age, 43 y) via surveys (n = 135), focus groups (n = 27), and semistructured interviews (n = 24). Items were generated for the instruments to assess biologic activity based on physician input. Linear regression was used to quantify the extent to which variations in patient-reported disease characteristics could account for variations in patient assessment of EoE severity. The PRO instrument was used prospectively in 153 adult patients with EoE (72.5% male; median age, 38 y), and validated in an independent group of 120 patients with EoE (60.8% male; median age, 40.5 y).RESULTS: Seven PRO factors that are used to assess characteristics of dysphagia, behavioral adaptations to living with dysphagia, and pain while swallowing accounted for 67% of the variation in patient assessment of disease severity. Based on statistical consideration and patient input, a 7-day recall period was selected. Highly active EoE, based on endoscopic and histologic findings, was associated with an increase in patient-assessed disease severity. In the validation study, the mean difference between patient assessment of EoE severity (range, 0-10) and PRO score (range, 0-8.52) was 0.15.CONCLUSIONS: We developed and validated an EoE scoring system based on 7 PRO items that assess symptoms over a 7-day recall period.

AB - BACKGROUND & AIMS: Standardized instruments are needed to assess the activity of eosinophilic esophagitis (EoE) and to provide end points for clinical trials and observational studies. We aimed to develop and validate a patient-reported outcome (PRO) instrument and score, based on items that could account for variations in patient assessments of disease severity. We also evaluated relationships between patient assessment of disease severity and EoE-associated endoscopic, histologic, and laboratory findings.METHODS: We collected information from 186 patients with EoE in Switzerland and the United States (69.4% male; median age, 43 y) via surveys (n = 135), focus groups (n = 27), and semistructured interviews (n = 24). Items were generated for the instruments to assess biologic activity based on physician input. Linear regression was used to quantify the extent to which variations in patient-reported disease characteristics could account for variations in patient assessment of EoE severity. The PRO instrument was used prospectively in 153 adult patients with EoE (72.5% male; median age, 38 y), and validated in an independent group of 120 patients with EoE (60.8% male; median age, 40.5 y).RESULTS: Seven PRO factors that are used to assess characteristics of dysphagia, behavioral adaptations to living with dysphagia, and pain while swallowing accounted for 67% of the variation in patient assessment of disease severity. Based on statistical consideration and patient input, a 7-day recall period was selected. Highly active EoE, based on endoscopic and histologic findings, was associated with an increase in patient-assessed disease severity. In the validation study, the mean difference between patient assessment of EoE severity (range, 0-10) and PRO score (range, 0-8.52) was 0.15.CONCLUSIONS: We developed and validated an EoE scoring system based on 7 PRO items that assess symptoms over a 7-day recall period.

KW - Disease Activity Measurement

KW - Esophagus

KW - Marker

KW - Patient-Reported Outcome

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