Abstract
Pyoderma gangrenosum (PG) is a rare inflammatory condition with an immense disease burden that remains understudied. With limited approved treatments and low-quality clinical evidence, PG continues to have poor patient outcomes. Unfortunately, improvement in PG treatments and patient care is based on additional research endeavors that can only be developed from existing high-quality data. The following protocol outlines the development of the Minimum Data Set for Treatment Effectiveness in Pyoderma gangrenosum (MIDSTEP), a core set of domains and domain items for the Pyoderma Gangrenosum Treatment Effectiveness (PyGaTE) international registry. The outcomes and benefits are focused on providing real-world data for physicians to improve their clinical decisions on PG treatment and inform clinical trial design, promoting clinical research among the international scientific community. MIDSTEP is a multi-phase project. The first phase will produce a domain item list from a literature review to take into the second phase which would finalize the core data set by an e-Delphi exercise. There will be a single stakeholder group participating together in the e-Delphi consisting of PG experts (healthcare providers, researchers, methodologists, industry representatives, and regulators), ulcerative PG patients, and PG patient advocates. The methodology outlined in the protocol is a systematic method based on several guidelines through COMET and established dermatologic registries and outcome sets with systematic methodologies of their own. The third phase will identify the instruments for the items, the ‘when to measure’ the items, and the platform for the registry. The last phase is the implementation and continued maintenance of the international registry PyGaTE. By solidifying a consensus on standardized outcomes and collecting information on PG treatment effectiveness in a centralized database, existing treatments can be compared more systematically and analyzed with increased evidence. MIDSTEP and the PyGaTE international registry will have the ambitious goal to generate and disseminate real-world data that can be used by all stakeholders to improve health outcomes for PG patients. Future potential for the outcome of this project includes the development of a gold-standard PG treatment.
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Author OMH wrote the original draft, prepared figure 1, led investigation and participated in project administration and methodology. Author MEJ participated in review & editing and investigation and contributed to methodology. Authors AGOL and LAAG participated in review and editing and were responsible for the project conceptualization and contributed to methodology. DC, DOC, JD, JRVG, PJH, RIK AVM, and YT participated in review and editing.
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Dr. Diana Chen is an employee of Genentech, a member of the Roche Group. Dr. David Croitoru worked as a consultant for UCB, Bausche, Novartis, Abbvie and Sanofi. Dr. Angelo Marzano receives consultation and advisory boards disease-relevant honoraria from AbbVie, Boehringer-Ingelheim, Novartis, Pfizer, Sanofi, and UCB. Dr. Yayoi Tada receives research grants and honoraria from Eisai, AbbVie, and Novartis. Dr. Alex Ortega-Loayza is president-elect of Pacific Dermatologic Association, an associate editor of Dermatology (Karger), a consultant for Genentech, Guidepoint, Corvus Pharmaceuticals, and Castle Biosciences, on the advisory board of Bristol Meyer Squibb, Boehringer Ingelheim, and Janssen, and receives research grants from AbbVie, Lilly, Janssen, Pfizer, and InflaRx GmbH. All other authors declare no competing interests.
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Appendix 1. Literature search strategy
Appendix 1. Literature search strategy
Database: Ovid MEDLINE(R) ALL < 1946 to May 23, 2023 >
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1.
exp Pyoderma Gangrenosum/ (2571)
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2.
(pyoderm* adj3 gangren*).ab,hw,kf,ot,sy,ti,fx,nm,ox,px,rx,ui. (4098)
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3.
1 or 2 (4098)
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4.
limit 3 to (meta analysis or "systematic review") (53)
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5.
limit 3 to (controlled clinical trial or randomized controlled trial) (9)
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6.
exp epidemiologic studies/ (3125284)
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7.
3 and 6 (355)
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8.
4 or 5 or 7 (414)
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9.
limit 8 to english language (382)
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10.
limit 8 to abstracts (346)
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11.
9 or 10 (413)
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Haddadin, O.M., Jacobson, M.E., Chen, D.M. et al. Minimum data set for treatment effectiveness in pyoderma gangrenosum (MIDSTEP): an international protocol of an e-Delphi study to develop a clinical physician-driven treatment effectiveness registry on behalf of the UPGRADE initiative. Arch Dermatol Res 315, 2913–2919 (2023). https://doi.org/10.1007/s00403-023-02729-9
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DOI: https://doi.org/10.1007/s00403-023-02729-9