{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T17:38:36Z","timestamp":1787679516976,"version":"build-2784847793"},"reference-count":37,"publisher":"Oxford University Press (OUP)","issue":"2","license":[{"start":{"date-parts":[[2025,4,18]],"date-time":"2025-04-18T00:00:00Z","timestamp":1744934400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025,7,17]]},"abstract":"Abstract<\/jats:title>\n \n Background<\/jats:title>\n Pemphigus is an autoimmune bullous disease (AIBD) and has two main subtypes, pemphigus vulgaris (PV) and pemphigus foliaceus (PF). For adequate interpretation and comparison of clinical studies in pemphigus, it is essential to have outcomes and outcome measurement instruments (OMIs) that are well-defined, uniform and relevant.<\/jats:p>\n <\/jats:sec>\n \n Objectives<\/jats:title>\n To provide a comprehensive overview of previously reported outcomes and OMIs in pemphigus clinical research over the past two decades.<\/jats:p>\n <\/jats:sec>\n \n Methods<\/jats:title>\n A systematic literature search was performed in the databases MEDLINE, Embase, CINAHL, PsycINFO, Cochrane CENTRAL, Web of Science and trial registries covering the period between January 2002 and December 2023. Clinical trials, prospective cohort studies and systematic reviews were included. Retrospective studies were excluded. All identified outcomes and their respective OMIs were grouped into outcome domains and areas.<\/jats:p>\n <\/jats:sec>\n \n Results<\/jats:title>\n From all the included studies (n = 205), a total of 1502 outcomes were extracted verbatim, categorized in 55 outcome domains and 11 outcome areas. We found a gradual increase in the number of published trials over the past two decades. The included studies mainly focused on \u2018clinical response\u2019 (92%), \u2018safety monitoring\u2019 (75%) and \u2018immune response\u2019 (61%). \u2018Quality of life\u2019 was sparsely reported (12%). Heterogeneity of reported outcomes was observed across all study designs. Within the outcome area \u2018clinical response\u2019, the proportion of defined outcomes based on the 2008 consensus statement has not been consistent over time. However, four recent clinical trials used primary endpoints based on the 2008 consensus definitions. Moreover, a broad spectrum of outcome measurement instruments (n = 65) was reported.<\/jats:p>\n <\/jats:sec>\n \n Conclusions<\/jats:title>\n This review showed a wide variety in reported outcomes and OMIs in pemphigus clinical research over the past two decades. Despite standardization efforts, most studies still lack well-defined, consistent and relevant outcomes and OMIs. However, a positive trend is emerging as four recent trials did use consensus-based definitions and patient-focused OMIs. Nevertheless, the outcomes are overly stringent, lack patient-centeredness, and do not include intermediate endpoints. Therefore, refinement of the 2008 expert consensus statement through collaborative efforts of all stakeholders is essential to promote uniformity in future pemphigus outcome measurement and to advance pemphigus drug development.<\/jats:p>\n <\/jats:sec>","DOI":"10.1093\/bjd\/ljaf152","type":"journal-article","created":{"date-parts":[[2025,4,18]],"date-time":"2025-04-18T07:45:21Z","timestamp":1744962321000},"page":"221-231","source":"Crossref","is-referenced-by-count":5,"title":["Outcome measurement in pemphigus clinical research: a scoping review on heterogeneity in outcome reporting and definitions"],"prefix":"10.1093","volume":"193","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7715-1697","authenticated-orcid":false,"given":"Marjolein A J","family":"Hiel","sequence":"first","affiliation":[{"name":"Department of Dermatology, Centre of Expertise for Blistering Diseases, European Reference Network for Rare Skin Diseases (ERN SKIN), University Medical Centre Groningen, University of Groningen , Groningen ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9166-5654","authenticated-orcid":false,"given":"Eva W H","family":"Korte","sequence":"additional","affiliation":[{"name":"Department of Dermatology, Centre of Expertise for Blistering Diseases, European Reference Network for Rare Skin Diseases (ERN SKIN), University Medical Centre Groningen, University of Groningen , Groningen ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7404-010X","authenticated-orcid":false,"given":"Billal","family":"Tedbirt","sequence":"additional","affiliation":[{"name":"Department of Dermatology, Rouen University Hospital, Normandie University and INSERM U1234, European Reference Network for Rare Skin Diseases (ERN SKIN), Centre de R\u00e9f\u00e9rence National des Maladies Bulleuses Auto-Immunes , Rouen ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2086-9363","authenticated-orcid":false,"given":"Marieke C","family":"Bolling","sequence":"additional","affiliation":[{"name":"Department of Dermatology, Centre of Expertise for Blistering Diseases, European Reference Network for Rare Skin Diseases (ERN SKIN), University Medical Centre Groningen, University of Groningen , Groningen ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7654-3528","authenticated-orcid":false,"given":"Joost M","family":"Meijer","sequence":"additional","affiliation":[{"name":"Department of Dermatology, Centre of Expertise for Blistering Diseases, European Reference Network for Rare Skin Diseases (ERN SKIN), University Medical Centre Groningen, University of Groningen , Groningen ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6035-2863","authenticated-orcid":false,"given":"Phyllis I","family":"Spuls","sequence":"additional","affiliation":[{"name":"Department of Dermatology, Amsterdam UMC, Academic Medical Centre, University of Amsterdam , Amsterdam ,","place":["the Netherlands"]},{"name":"Amsterdam Public Health, Infection and Immunity, Academic Medical Centre, University of Amsterdam , Amsterdam ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5856-7657","authenticated-orcid":false,"given":"Sjoukje","family":"van der Werf","sequence":"additional","affiliation":[{"name":"Medical Library, University of Groningen, University Medical Centre Groningen , Groningen ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0006-6722-3569","authenticated-orcid":false,"given":"Marc","family":"Yale","sequence":"additional","affiliation":[{"name":"International Pemphigus and Pemphigoid Foundation , Roseville, CA ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8559-3674","authenticated-orcid":false,"given":"Barbara","family":"Horv\u00e1th","sequence":"additional","affiliation":[{"name":"Department of Dermatology, Centre of Expertise for Blistering Diseases, European Reference Network for Rare Skin Diseases (ERN SKIN), University Medical Centre Groningen, University of Groningen , Groningen ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5734-0255","authenticated-orcid":false,"given":"Pascal","family":"Joly","sequence":"additional","affiliation":[{"name":"Department 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