Development and Implementation of the AIDA International Registry for Patients With Still's Disease

Antonio Vitale, Francesca Della Casa, Giuseppe Lopalco, Rosa Maria Pereira, Piero Ruscitti, Roberto Giacomelli, Gaafar Ragab, Francesco La Torre, Elena Bartoloni, Emanuela Del Giudice, Claudia Lomater, Giacomo Emmi, Marcello Govoni, Maria Cristina Maggio, Armin Maier, Joanna Makowska, Benson Ogunjimi, Petros P Sfikakis, Paolo Sfriso, Carla Gaggiano, Florenzo Iannone, Marília A Dagostin, Ilenia Di Cola, Luca Navarini, Ayman Abdelmonem Ahmed Mahmoud, Fabio Cardinale, Ilenia Riccucci, Maria Pia Paroli, Elena Maria Marucco, Irene Mattioli, Jurgen Sota, Anna Abbruzzese, Isabele P B Antonelli, Paola Cipriani, Abdurrahman Tufan, Claudia Fabiani, Mustafa Mahmoud Ramadan, Marco Cattalini, Riza Can Kardas, Gian Domenico Sebastiani, Henrique A Mayrink Giardini, José Hernández-Rodríguez, Violetta Mastrorilli, Ewa Więsik-Szewczyk, Micol Frassi, Valeria Caggiano, Salvatore Telesca, Heitor F Giordano, Emmanuele Guadalupi, Teresa Giani, Alessandra Renieri, Sergio Colella, Giulia Cataldi, Martina Gentile, Alessandra Fabbiani, Ibrahim A Al-Maghlouth, Bruno Frediani, Alberto Balistreri, Donato Rigante, Luca Cantarini, Antonio Vitale, Francesca Della Casa, Giuseppe Lopalco, Rosa Maria Pereira, Piero Ruscitti, Roberto Giacomelli, Gaafar Ragab, Francesco La Torre, Elena Bartoloni, Emanuela Del Giudice, Claudia Lomater, Giacomo Emmi, Marcello Govoni, Maria Cristina Maggio, Armin Maier, Joanna Makowska, Benson Ogunjimi, Petros P Sfikakis, Paolo Sfriso, Carla Gaggiano, Florenzo Iannone, Marília A Dagostin, Ilenia Di Cola, Luca Navarini, Ayman Abdelmonem Ahmed Mahmoud, Fabio Cardinale, Ilenia Riccucci, Maria Pia Paroli, Elena Maria Marucco, Irene Mattioli, Jurgen Sota, Anna Abbruzzese, Isabele P B Antonelli, Paola Cipriani, Abdurrahman Tufan, Claudia Fabiani, Mustafa Mahmoud Ramadan, Marco Cattalini, Riza Can Kardas, Gian Domenico Sebastiani, Henrique A Mayrink Giardini, José Hernández-Rodríguez, Violetta Mastrorilli, Ewa Więsik-Szewczyk, Micol Frassi, Valeria Caggiano, Salvatore Telesca, Heitor F Giordano, Emmanuele Guadalupi, Teresa Giani, Alessandra Renieri, Sergio Colella, Giulia Cataldi, Martina Gentile, Alessandra Fabbiani, Ibrahim A Al-Maghlouth, Bruno Frediani, Alberto Balistreri, Donato Rigante, Luca Cantarini

Abstract

Objective: Aim of this paper is to present the design, construction, and modalities of dissemination of the AutoInflammatory Disease Alliance (AIDA) International Registry for patients with systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD), which are the pediatric and adult forms of the same autoinflammatory disorder.

Methods: This Registry is a clinical, physician-driven, population- and electronic-based instrument implemented for the retrospective and prospective collection of real-world data. The collection of data is based on the Research Electronic Data Capture (REDCap) tool and is intended to obtain evidence drawn from routine patients' management. The collection of standardized data is thought to bring knowledge about real-life clinical research and potentially communicate with other existing and future Registries dedicated to Still's disease. Moreover, it has been conceived to be flexible enough to easily change according to future scientific acquisitions.

Results: Starting from June 30th to February 7th, 2022, 110 Centers from 23 Countries in 4 continents have been involved. Fifty-four of these have already obtained the approval from their local Ethics Committees. Currently, the platform counts 290 users (111 Principal Investigators, 175 Site Investigators, 2 Lead Investigators, and 2 data managers). The Registry collects baseline and follow-up data using 4449 fields organized into 14 instruments, including patient's demographics, history, clinical manifestations and symptoms, trigger/risk factors, therapies and healthcare access.

Conclusions: This international Registry for patients with Still's disease will allow a robust clinical research through collection of standardized data, international consultation, dissemination of knowledge, and implementation of observational studies based on wide cohorts of patients followed-up for very long periods. Solid evidence drawn from "real-life" data represents the ultimate goal of this Registry, which has been implemented to significantly improve the overall management of patients with Still's disease. NCT05200715 available at https://clinicaltrials.gov/.

Keywords: autoinflammatory diseases; personalized medicine; precision medicine; rare diseases; research; treatment.

Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Copyright © 2022 Vitale, Della Casa, Lopalco, Pereira, Ruscitti, Giacomelli, Ragab, La Torre, Bartoloni, Del Giudice, Lomater, Emmi, Govoni, Maggio, Maier, Makowska, Ogunjimi, Sfikakis, Sfriso, Gaggiano, Iannone, Dagostin, Di Cola, Navarini, Ahmed Mahmoud, Cardinale, Riccucci, Paroli, Marucco, Mattioli, Sota, Abbruzzese, Antonelli, Cipriani, Tufan, Fabiani, Ramadan, Cattalini, Kardas, Sebastiani, Giardini, Hernández-Rodríguez, Mastrorilli, Więsik-Szewczyk, Frassi, Caggiano, Telesca, Giordano, Guadalupi, Giani, Renieri, Colella, Cataldi, Gentile, Fabbiani, Al-Maghlouth, Frediani, Balistreri, Rigante and Cantarini.

Figures

Figure 1
Figure 1
Worldwide distribution of the AIDA network in February 2022.

References

    1. Deliverska M. Patient registries for rare diseases. J IMAB Annu Proceeding Sci Pap. (2016) 22:1166–8. 10.5272/jimab.2016222.1166
    1. Jansen-van der Weide MC, Gaasterland CMW, Roes KCB, Pontes C, Vives R, Sancho A, et al. . Rare disease registries: potential applications towards impact on development of new drug treatments. Orphanet J Rare Dis. (2018) 13:154. 10.1186/s13023-018-0836-0
    1. Public Health - European Commission . Rare Diseases. (2016). Available online at: (accessed December 4, 2021).
    1. Boulanger V, Schlemmer M, Rossov S, Seebald A, Gavin P. Establishing patient registries for rare diseases: rationale and challenges. Pharmaceut Med. (2020) 34:185–90. 10.1007/s40290-020-00332-1
    1. Guidance for Industry . Clin Trials. (2011). p. 21. Available online at: (accessed December 4, 2021).
    1. Giacomelli R, Ruscitti P, Shoenfeld Y. A comprehensive review on adult onset Still's disease. J Autoimmun. (2018) 93:24–36. 10.1016/j.jaut.2018.07.018
    1. Lee JJY, Schneider R. Systemic juvenile idiopathic arthritis. Pediatr Clin North Am. (2018) 65:691–709. 10.1016/j.pcl.2018.04.005
    1. Ruscitti P, Rago C, Breda L, Cipriani P, Liakouli V, Berardicurti O, et al. . Macrophage activation syndrome in Still's disease: analysis of clinical characteristics and survival in paediatric and adult patients. Clin Rheumatol. (2017) 36:2839–45. 10.1007/s10067-017-3830-3
    1. Saper VE, Chen G, Deutsch GH, Guillerman RP, Birgmeier J, Jagadeesh K, et al. . Emergent high fatality lung disease in systemic juvenile arthritis. Ann Rheum Dis. (2019) 78:1722–31. 10.1136/annrheumdis-2019-216040
    1. Pouchot J, Sampalis JS, Beaudet F, Carette SI, Décary FR, Salusinsky-Sternbach MA, et al. . Adult Still's disease: manifestations, disease course, and outcome in 62 patients. Medicine. (1991) 70:118–36. 10.1097/00005792-199103000-00004
    1. Yamaguchi M, Ohta A, Tsunematsu T, Kasukawa R, Mizushima Y, Kashiwagi H, et al. . Preliminary criteria for classification of adult Still's disease. J Rheumatol. (1992) 19:424–30.
    1. Fautrel B, Zing E, Golmard JL, Le Moel G, Bissery A, Rioux C, et al. . Proposal for a new set of classification criteria for adult-onset still disease. Medicine. (2002) 81:194–200. 10.1097/00005792-200205000-00003
    1. Martini A, Ravelli A, Avcin T, Beresford MW, Burgos-Vargas R, Cuttica R, et al. . Toward new classification criteria for juvenile idiopathic arthritis: first steps, pediatric rheumatology international trials organization international consensus. J Rheumatol. (2019) 46:190–7. 10.3899/jrheum.180168
    1. Cush JJ, Medsger TA, Jr, Christy WC, Herbert DC, Cooperstein LA. Adult-onset Still's disease. Clinical course and outcome. Arthritis Rheum. (1987) 30:186–94. 10.1002/art.1780300209
    1. Petty RE, Southwood TR, Manners P, Baum J, Glass DN, Goldenberg J, et al. . League of Associations for Rheumatology classification of juvenile idiopathic arthritis: second revision, Edmonton, 2001. J Rheumatol Int. (2004) 31:390–2.
    1. Harris PA, Taylor R, Thielke R, Payne J, Gonzalez N, Conde JG. Research electronic data capture (REDCap) - A metadata-driven methodology and workflow process for providing translational research informatics support. J Biomed Inform. (2009) 42:377–81. 10.1016/j.jbi.2008.08.010
    1. Ali SR, Bryce J, Tan LE, Hiort O, Pereira AM, van den Akker EL, et al. . The EuRRECa project as a model for data access and governance policies for rare disease registries that collect clinical outcomes. Int J Environ Res Public Health. (2020) 17:8743. 10.3390/ijerph17238743
    1. EUR-Lex - 32016R0679 - EN - EUR-Lex. Available online at: (accessed December 21, 2021).
    1. Kang H. The prevention and handling of the missing data. Korean J Anesthesiol. (2013) 64:402–6. 10.4097/kjae.2013.64.5.402
    1. Rau M, Schiller M, Krienke S, Heyder P, Lorenz H, Blank N. Clinical manifestations but not cytokine profiles differentiate adult-onset Still's disease and sepsis. J Rheumatol. (2010) 37:2369–76. 10.3899/jrheum.100247
    1. Bhargava J, Panginikkod S. Still Disease. Treasure Island, FL: StatPearls Publishing; (2021).
    1. Janow G, Schanberg LE, Setoguchi S, Hasselblad V, Mellins ED, Schneider R, et al. . The systemic juvenile idiopathic arthritis cohort of the childhood arthritis and rheumatology research alliance registry: 2010-2013. J Rheumatol. (2016) 43:1755–62. 10.3899/jrheum.150997
    1. Hosoya S, Sadatsuki M, Izuka S, Yamashita H, Oishi H. A case of recurrence of adult-onset Still's disease in the third trimester: a case report and literature review. BMC Pregnancy Childbirth. (2021) 21:163. 10.1186/s12884-021-03648-1
    1. Kearsley-Fleet L, Klotsche J, van Straalen JW, Costello W, D'Angelo G, Giancane G, et al. . Burden of comorbid conditions in children and young people with juvenile idiopathic arthritis: a collaborative analysis of 3 JIA registries. Rheumatology. (2021) 6:keab641. 10.1093/rheumatology/keab641
    1. Lainka E, Baehr M, Raszka B, Haas JP, Hügle B, Fischer N, et al. . Experiences with IL-1 blockade in systemic juvenile idiopathic arthritis - data from the German AID-registry. Pediatr Rheumatol Online J. (2021) 19:38. 10.1186/s12969-021-00510-8
    1. Atemnkeng Ntam V, Klein A, Horneff G. Safety and efficacy of anakinra as first-line or second-line therapy for systemic onset juvenile idiopathic arthritis - data from the German BIKER registry. Expert Opin Drug Saf. (2021) 20:93–100. 10.1080/14740338.2021.1843631
    1. Beukelman T, Kimura Y, Ilowite NT, Mieszkalski K, Natter MD, Burrell G, et al. . The new Childhood Arthritis and Rheumatology Research Alliance (CARRA) registry: design, rationale, and characteristics of patients enrolled in the first 12 months. Pediatr Rheumatol Online J. (2017) 15:30. 10.1186/s12969-017-0160-6
    1. Demirkaya E, Ozen S, Bilginer Y, Ayaz NA, Makay BB, Unsal E, et al. . The distribution of juvenile idiopathic arthritis in the eastern Mediterranean: results from the registry of the Turkish Paediatric Rheumatology Association. Clin Exp Rheumatol. (2011) 29:111–6.
    1. European Medicines Agency . Patient Registries. (2018). Available online at: (accessed March 8, 2022).
    1. Kodra Y, Weinbach J, Posada-de-la-Paz M, Coi A, Lemonnier SL, Van Enckevort D, et al. . Recommendations for improving the quality of rare disease registries. Int J Environ Res Public Health. (2018) 15:1644. 10.3390/ijerph15081644

Source: PubMed

Sponsorzy i współpracownicy

Warunki medyczne

Interwencje lekowe

3
Subskrybuj