Objectives Autoinflammatory diseases cause systemic inflammation that can result in damage to multiple organs. A validated instrument is essential to quantify damage in individual patients and to compare disease outcomes in clinical studies. Currently, there is no such tool. Our objective was to develop a common autoinflammatory disease damage index (ADDI) for familial Mediterranean fever, cryopyrin-associated periodic syndromes, tumour necrosis factor receptor-associated periodic fever syndrome and mevalonate kinase deficiency.
Methods We developed the ADDI by consensus building. The top 40 enrollers of patients in the Eurofever Registry and 9 experts from the Americas participated in multiple rounds of online surveys to select items and definitions. Further, 22 (parents of) patients rated damage items and suggested new items. A consensus meeting was held to refine the items and definitions, which were then formally weighted in a scoring system derived using decision-making software, known as 1000minds.
Results More than 80% of the experts and patients completed the online surveys. The preliminary ADDI contains 18 items, categorised in the following eight organ systems: reproductive, renal/amyloidosis, developmental, serosal, neurological, ears, ocular and musculoskeletal damage. The categories renal/amyloidosis and neurological damage were assigned the highest number of points, serosal damage the lowest number of points. The involvement of (parents of) patients resulted in the inclusion of, for example, chronic musculoskeletal pain.
Conclusions An instrument to measure damage caused by autoinflammatory diseases is developed based on consensus building. Patients fulfilled a significant role in this process.
- Fever Syndromes
- Familial Mediterranean Fever
- Outcomes research
- Patient perspective
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Handling editor Tore K Kvien
NMtH and KVA are joint first authors and MG and JF are joint last authors.
Dr Ornella Della Casa Alberighi is deceased in the course of this project.
Twitter Follow Ricardo Russo at @el_reumatologo
Contributors NMtH and KVA are joint first authors. MG and JF are joint last authors. NMtH, KVA and JF designed the study and wrote the manuscript. ODCA was the principal investigator of RADICEA. KLD contacted patients for patient recruitment. The consensus meeting was prepared with and led by AR. KLD, JF and all other authors contributed to the online surveys and/or the consensus meeting, and attributed to and approved the manuscript.
Funding The project was supported by ERANET-PRIOMEDCHILD RaDiCEA Project No. 40-41800-98-007. The Eurofever Registry was funded by the Executive Agency for Health and Consumers (EAHC, Project No. 2007332). The work was supported by an unrestricted grant by Novartis Pharma AG.
Competing interests Novartis Pharma AG financially supported the final consensus meeting. They did not have any influence on the selection of participants or on the content of the ADDI/consensus meeting or the reporting of the findings. FdB: Novartis, Novimmune, Hoffmann-La Roche, SOBI, AbbVie. LC: speaker's fee for Novartis and SOBI. MC: consultancy fees for Novartis, SOBI and Abbvie. KLD: consultancy work for SOBI and Novartis, donations, honorariums and unrestricted grants have been received by the Autoinflammatory Alliance from SOBI, Novartis, and Regeneron. RG: consultant for Abbvie. RGM: study support from SOBI, Novartis, Regeneron. VH: honorariums and educational grants from Novartis, honorariums from SOBI. MH: consultant for Novartis. HMH: consultant for Novartis and SOBI, and speaker for Novartis. TK: research grant by Novartis, speaker's bureau by Roche, BMS, Novartis and SOBI. JKD: consultant/speaker for Novartis and SOBI and has received grant support from SOBI and Novartis. RML: ad board and consultant for Abbvie and Novartis. PQ: investigator, consultant and speaker's bureau for Novartis and SOBI. MG: consultant for and unrestricted grants to Eurofever and speaker's fee from SOBI and Novartis. YU: Y. Uziel Grant/Research Support from Novartis, Consultant for Novartis, Speaker Bureau of Abbvie, Neopharm, Novartis, Roche. JF: consultant for Novartis.
Ethics approval The Medical Ethical Committee of the University Medical Centre Utrecht.
Provenance and peer review Not commissioned; externally peer reviewed.