Minimum dataset for treatment effectiveness in pyoderma gangrenosum for an international registry: an international...

Pyoderma gangrenosum (PG) is a rare, painful neutrophilic dermatosis with a profound impact on patient quality of life. Its management is hindered by a lack of approved therapies, limited clinical trials, and low-quality evidence. The rarity of the disease and funding constraints have impeded research progress and the development of standardized outcome measures. Patient data registries offer a promising solution to these challenges, providing essential infrastructure to improve the generation of evidence and clinical care.

To develop a consensus-based minimum dataset for an international treatment effectiveness registry for PG, informed by real-world clinical data.

An initial list of candidate domain items was generated from a systematic literature review conducted according to a previously published protocol. An international, multistakeholder panel of 45 participants - including patients with PG, clinicians, researchers, methodologists and industry representatives - was convened from 97 invited experts. Through three rounds of modified Delphi surveys and a virtual consensus meeting, items were ranked using predefined criteria: 'consensus in' (≥ 70% scoring 7-9 and ≤ 15% scoring 1-3), 'consensus out' (≥ 70% scoring 1-3 and ≤ 15% scoring 7-9) and 'no consensus'. A final verification survey confirmed inclusion if < 30% of participants voted 'no'.

All 45 stakeholders completed all three Delphi rounds (0% dropout). Thirty-four (76%) participated in the consensus meeting, and 42 (93%) completed the final verification survey. Of 143 initial items across 24 domains, 118 items across 26 domains achieved consensus for inclusion in the minimum dataset.

This international consensus establishes a standardized framework for collecting real-world data on PG. The resulting registry will serve as a critical resource for evaluating treatment effectiveness, understanding disease progression, and improving patient outcomes. It will also support future clinical trials, guideline development, quality improvement initiatives, and global patient recruitment efforts.