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Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS)

BACKGROUND: End points used to determine treatment efficacy in eosinophilic esophagitis (EoE) have evolved over time. With multiple novel therapies in development for EoE, harmonization of outcomes measures will facilitate evidence synthesis and appraisal when comparing different treatments. OBJECTI...

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Autores principales: Ma, Christopher, Schoepfer, Alain M., Dellon, Evan S., Bredenoord, Albert J., Chehade, Mirna, Collins, Margaret H., Feagan, Brian G., Furuta, Glenn T., Gupta, Sandeep K., Hirano, Ikuo, Jairath, Vipul, Katzka, David A., Pai, Rish K., Rothenberg, Marc E., Straumann, Alex, Aceves, Seema S., Alexander, Jeffrey A., Arva, Nicoleta C., Atkins, Dan, Biedermann, Luc, Blanchard, Carine, Cianferoni, Antonella, de los Rios, Constanza Ciriza, Clayton, Frederic, Davis, Carla M., de Bortoli, Nicola, Dias, Jorge A., Falk, Gary W., Genta, Robert M., Ghaffari, Gisoo, Gonsalves, Nirmala, Greuter, Thomas, Hopp, Russell, Blatman, Karen S. Hsu, Jensen, Elizabeth T., Johnston, Doug, Kagalwalla, Amir F., Larsson, Helen M., Leung, John, Louis, Hubert, Masterson, Joanne C., Menard-Katcher, Calies, Menard-Katcher, Paul A., Moawad, Fouad J., Muir, Amanda B., Mukkada, Vincent A., Penagini, Roberto, Pesek, Robert D., Peterson, Kathryn, Putnam, Philip E., Ravelli, Alberto, Savarino, Edoardo V., Schlag, Christoph, Schreiner, Philipp, Simon, Dagmar, Smyrk, Thomas C., Spergel, Jonathan M., Taft, Tiffany H., Terreehorst, Ingrid, Vanuytsel, Tim, Venter, Carina, Vieira, Mario C., Vieth, Michael, Vlieg-Boerstra, Berber, von Arnim, Ulrike, Walker, Marjorie M., Wechsler, Joshua B., Woodland, Philip, Woosley, John T., Yang, Guang-Yu, Zevit, Noam, Safroneeva, Ekaterina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8733049/
https://www.ncbi.nlm.nih.gov/pubmed/34242635
http://dx.doi.org/10.1016/j.jaci.2021.07.001
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author Ma, Christopher
Schoepfer, Alain M.
Dellon, Evan S.
Bredenoord, Albert J.
Chehade, Mirna
Collins, Margaret H.
Feagan, Brian G.
Furuta, Glenn T.
Gupta, Sandeep K.
Hirano, Ikuo
Jairath, Vipul
Katzka, David A.
Pai, Rish K.
Rothenberg, Marc E.
Straumann, Alex
Aceves, Seema S.
Alexander, Jeffrey A.
Arva, Nicoleta C.
Atkins, Dan
Biedermann, Luc
Blanchard, Carine
Cianferoni, Antonella
de los Rios, Constanza Ciriza
Clayton, Frederic
Davis, Carla M.
de Bortoli, Nicola
Dias, Jorge A.
Falk, Gary W.
Genta, Robert M.
Ghaffari, Gisoo
Gonsalves, Nirmala
Greuter, Thomas
Hopp, Russell
Blatman, Karen S. Hsu
Jensen, Elizabeth T.
Johnston, Doug
Kagalwalla, Amir F.
Larsson, Helen M.
Leung, John
Louis, Hubert
Masterson, Joanne C.
Menard-Katcher, Calies
Menard-Katcher, Paul A.
Moawad, Fouad J.
Muir, Amanda B.
Mukkada, Vincent A.
Penagini, Roberto
Pesek, Robert D.
Peterson, Kathryn
Putnam, Philip E.
Ravelli, Alberto
Savarino, Edoardo V.
Schlag, Christoph
Schreiner, Philipp
Simon, Dagmar
Smyrk, Thomas C.
Spergel, Jonathan M.
Taft, Tiffany H.
Terreehorst, Ingrid
Vanuytsel, Tim
Venter, Carina
Vieira, Mario C.
Vieth, Michael
Vlieg-Boerstra, Berber
von Arnim, Ulrike
Walker, Marjorie M.
Wechsler, Joshua B.
Woodland, Philip
Woosley, John T.
Yang, Guang-Yu
Zevit, Noam
Safroneeva, Ekaterina
author_facet Ma, Christopher
Schoepfer, Alain M.
Dellon, Evan S.
Bredenoord, Albert J.
Chehade, Mirna
Collins, Margaret H.
Feagan, Brian G.
Furuta, Glenn T.
Gupta, Sandeep K.
Hirano, Ikuo
Jairath, Vipul
Katzka, David A.
Pai, Rish K.
Rothenberg, Marc E.
Straumann, Alex
Aceves, Seema S.
Alexander, Jeffrey A.
Arva, Nicoleta C.
Atkins, Dan
Biedermann, Luc
Blanchard, Carine
Cianferoni, Antonella
de los Rios, Constanza Ciriza
Clayton, Frederic
Davis, Carla M.
de Bortoli, Nicola
Dias, Jorge A.
Falk, Gary W.
Genta, Robert M.
Ghaffari, Gisoo
Gonsalves, Nirmala
Greuter, Thomas
Hopp, Russell
Blatman, Karen S. Hsu
Jensen, Elizabeth T.
Johnston, Doug
Kagalwalla, Amir F.
Larsson, Helen M.
Leung, John
Louis, Hubert
Masterson, Joanne C.
Menard-Katcher, Calies
Menard-Katcher, Paul A.
Moawad, Fouad J.
Muir, Amanda B.
Mukkada, Vincent A.
Penagini, Roberto
Pesek, Robert D.
Peterson, Kathryn
Putnam, Philip E.
Ravelli, Alberto
Savarino, Edoardo V.
Schlag, Christoph
Schreiner, Philipp
Simon, Dagmar
Smyrk, Thomas C.
Spergel, Jonathan M.
Taft, Tiffany H.
Terreehorst, Ingrid
Vanuytsel, Tim
Venter, Carina
Vieira, Mario C.
Vieth, Michael
Vlieg-Boerstra, Berber
von Arnim, Ulrike
Walker, Marjorie M.
Wechsler, Joshua B.
Woodland, Philip
Woosley, John T.
Yang, Guang-Yu
Zevit, Noam
Safroneeva, Ekaterina
author_sort Ma, Christopher
collection PubMed
description BACKGROUND: End points used to determine treatment efficacy in eosinophilic esophagitis (EoE) have evolved over time. With multiple novel therapies in development for EoE, harmonization of outcomes measures will facilitate evidence synthesis and appraisal when comparing different treatments. OBJECTIVE: We sought to develop a core outcome set (COS) for controlled and observational studies of pharmacologic and diet interventions in adult and pediatric patients with EoE. METHODS: Candidate outcomes were generated from systematic literature reviews and patient engagement interviews and surveys. Consensus was established using an iterative Delphi process, with items voted on using a 9-point Likert scale and with feedback from other participants to allow score refinement. Consensus meetings were held to ratify the outcome domains of importance and the core outcome measures. Stakeholders were recruited internationally and included adult and pediatric gastroenterologists, allergists, dieticians, pathologists, psychologists, researchers, and methodologists. RESULTS: The COS consists of 4 outcome domains for controlled and observational studies: histopathology, endoscopy, patient-reported symptoms, and EoE-specific quality of life. A total of 69 stakeholders (response rate 95.8%) prioritized 42 outcomes in a 2-round Delphi process, and the final ratification meeting generated consensus on 33 outcome measures. These included measurement of the peak eosinophil count, Eosinophilic Esophagitis Histology Scoring System, Eosinophilic Esophagitis Endoscopic Reference Score, and patient-reported measures of dysphagia and quality of life. CONCLUSIONS: This interdisciplinary collaboration involving global stakeholders has produced a COS that can be applied to adult and pediatric studies of pharmacologic and diet therapies for EoE and will facilitate meaningful treatment comparisons and improve the quality of data synthesis.
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spelling pubmed-87330492023-02-01 Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS) Ma, Christopher Schoepfer, Alain M. Dellon, Evan S. Bredenoord, Albert J. Chehade, Mirna Collins, Margaret H. Feagan, Brian G. Furuta, Glenn T. Gupta, Sandeep K. Hirano, Ikuo Jairath, Vipul Katzka, David A. Pai, Rish K. Rothenberg, Marc E. Straumann, Alex Aceves, Seema S. Alexander, Jeffrey A. Arva, Nicoleta C. Atkins, Dan Biedermann, Luc Blanchard, Carine Cianferoni, Antonella de los Rios, Constanza Ciriza Clayton, Frederic Davis, Carla M. de Bortoli, Nicola Dias, Jorge A. Falk, Gary W. Genta, Robert M. Ghaffari, Gisoo Gonsalves, Nirmala Greuter, Thomas Hopp, Russell Blatman, Karen S. Hsu Jensen, Elizabeth T. Johnston, Doug Kagalwalla, Amir F. Larsson, Helen M. Leung, John Louis, Hubert Masterson, Joanne C. Menard-Katcher, Calies Menard-Katcher, Paul A. Moawad, Fouad J. Muir, Amanda B. Mukkada, Vincent A. Penagini, Roberto Pesek, Robert D. Peterson, Kathryn Putnam, Philip E. Ravelli, Alberto Savarino, Edoardo V. Schlag, Christoph Schreiner, Philipp Simon, Dagmar Smyrk, Thomas C. Spergel, Jonathan M. Taft, Tiffany H. Terreehorst, Ingrid Vanuytsel, Tim Venter, Carina Vieira, Mario C. Vieth, Michael Vlieg-Boerstra, Berber von Arnim, Ulrike Walker, Marjorie M. Wechsler, Joshua B. Woodland, Philip Woosley, John T. Yang, Guang-Yu Zevit, Noam Safroneeva, Ekaterina J Allergy Clin Immunol Article BACKGROUND: End points used to determine treatment efficacy in eosinophilic esophagitis (EoE) have evolved over time. With multiple novel therapies in development for EoE, harmonization of outcomes measures will facilitate evidence synthesis and appraisal when comparing different treatments. OBJECTIVE: We sought to develop a core outcome set (COS) for controlled and observational studies of pharmacologic and diet interventions in adult and pediatric patients with EoE. METHODS: Candidate outcomes were generated from systematic literature reviews and patient engagement interviews and surveys. Consensus was established using an iterative Delphi process, with items voted on using a 9-point Likert scale and with feedback from other participants to allow score refinement. Consensus meetings were held to ratify the outcome domains of importance and the core outcome measures. Stakeholders were recruited internationally and included adult and pediatric gastroenterologists, allergists, dieticians, pathologists, psychologists, researchers, and methodologists. RESULTS: The COS consists of 4 outcome domains for controlled and observational studies: histopathology, endoscopy, patient-reported symptoms, and EoE-specific quality of life. A total of 69 stakeholders (response rate 95.8%) prioritized 42 outcomes in a 2-round Delphi process, and the final ratification meeting generated consensus on 33 outcome measures. These included measurement of the peak eosinophil count, Eosinophilic Esophagitis Histology Scoring System, Eosinophilic Esophagitis Endoscopic Reference Score, and patient-reported measures of dysphagia and quality of life. CONCLUSIONS: This interdisciplinary collaboration involving global stakeholders has produced a COS that can be applied to adult and pediatric studies of pharmacologic and diet therapies for EoE and will facilitate meaningful treatment comparisons and improve the quality of data synthesis. 2022-02 2021-07-06 /pmc/articles/PMC8733049/ /pubmed/34242635 http://dx.doi.org/10.1016/j.jaci.2021.07.001 Text en https://creativecommons.org/licenses/by/4.0/This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) ).
spellingShingle Article
Ma, Christopher
Schoepfer, Alain M.
Dellon, Evan S.
Bredenoord, Albert J.
Chehade, Mirna
Collins, Margaret H.
Feagan, Brian G.
Furuta, Glenn T.
Gupta, Sandeep K.
Hirano, Ikuo
Jairath, Vipul
Katzka, David A.
Pai, Rish K.
Rothenberg, Marc E.
Straumann, Alex
Aceves, Seema S.
Alexander, Jeffrey A.
Arva, Nicoleta C.
Atkins, Dan
Biedermann, Luc
Blanchard, Carine
Cianferoni, Antonella
de los Rios, Constanza Ciriza
Clayton, Frederic
Davis, Carla M.
de Bortoli, Nicola
Dias, Jorge A.
Falk, Gary W.
Genta, Robert M.
Ghaffari, Gisoo
Gonsalves, Nirmala
Greuter, Thomas
Hopp, Russell
Blatman, Karen S. Hsu
Jensen, Elizabeth T.
Johnston, Doug
Kagalwalla, Amir F.
Larsson, Helen M.
Leung, John
Louis, Hubert
Masterson, Joanne C.
Menard-Katcher, Calies
Menard-Katcher, Paul A.
Moawad, Fouad J.
Muir, Amanda B.
Mukkada, Vincent A.
Penagini, Roberto
Pesek, Robert D.
Peterson, Kathryn
Putnam, Philip E.
Ravelli, Alberto
Savarino, Edoardo V.
Schlag, Christoph
Schreiner, Philipp
Simon, Dagmar
Smyrk, Thomas C.
Spergel, Jonathan M.
Taft, Tiffany H.
Terreehorst, Ingrid
Vanuytsel, Tim
Venter, Carina
Vieira, Mario C.
Vieth, Michael
Vlieg-Boerstra, Berber
von Arnim, Ulrike
Walker, Marjorie M.
Wechsler, Joshua B.
Woodland, Philip
Woosley, John T.
Yang, Guang-Yu
Zevit, Noam
Safroneeva, Ekaterina
Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS)
title Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS)
title_full Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS)
title_fullStr Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS)
title_full_unstemmed Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS)
title_short Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS)
title_sort development of a core outcome set for therapeutic studies in eosinophilic esophagitis (coreos)
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8733049/
https://www.ncbi.nlm.nih.gov/pubmed/34242635
http://dx.doi.org/10.1016/j.jaci.2021.07.001
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