HENRIQUE AYRES MAYRINK GIARDINI

Índice h a partir de 2011
4
Projetos de Pesquisa
Unidades Organizacionais
P ICHC, Hospital das Clínicas, Faculdade de Medicina - Médico
LIM/17 - Laboratório de Investigação em Reumatologia, Hospital das Clínicas, Faculdade de Medicina

Filtros

Mostrar mais
Limpar filtros

Configurações

search.filters.applied.f.dateIssued: 2022 ×

Resultados de Busca

Agora exibindo 1 - 10 de 10
  • article 2 Citação(ões) na Scopus
    Development and implementation of the AIDA International Registry for patients with Periodic Fever, Aphthous stomatitis, Pharyngitis, and cervical Adenitis syndrome
    (2022) CASA, Francesca Della; VITALE, Antonio; CATTALINI, Marco; TORRE, Francesco La; CAPOZIO, Giovanna; GIUDICE, Emanuela Del; MAGGIO, Maria Cristina; CONTI, Giovanni; ALESSIO, Maria; OGUNJIMI, Benson; RAGAB, Gaafar; EMMI, Giacomo; ARAGONA, Emma; GIANI, Teresa; LOPALCO, Giuseppe; PARRONCHI, Paola; SHAHRAM, Farhad; VERRECCHIA, Elena; RICCI, Francesca; CARDINALE, Fabio; NOI, Silvia Di; NUZZOLESE, Rossana; LUBRANO, Riccardo; PATRONITI, Serena; NADDEI, Roberta; SABATO, Vito; HUSSEIN, Mohamed A.; DOTTA, Laura; MASTRORILLI, Violetta; GENTILESCHI, Stefano; TUFAN, Abdurrahman; CAGGIANO, Valeria; HEGAZY, Mohamed Tharwat; SOTA, Jurgen; ALMAGHLOUTH, Ibrahim A.; IBRAHIM, Amr; WIESIK-SZEWCZYK, Ewa; OZKIZILTAS, Burcugul; GROSSO, Salvatore; FRASSI, Micol; TARSIA, Maria; PEREIRA, Rosa Maria R.; TAYMOUR, Maged; GAGGIANO, Carla; COLELLA, Sergio; FABIANI, Claudia; MORRONE, Maria; RUSCITTI, Piero; FREDIANI, Bruno; SPEDICATO, Veronica; GIARDINI, Henrique A. Mayrink; BALISTRERI, Alberto; RIGANTE, Donato; CANTARINI, Luca
    Objective: Aim of this paper is to illustrate the methodology, design, and development of the AutoInflammatory Disease Alliance (AIDA) International Registry dedicated to patients with the Periodic Fever, Aphthous stomatitis, Pharyngitis, and cervical Adenitis (PFAPA) syndrome. Methods: This is a physician-driven, non-population- and electronic-based registry proposed to gather real-world demographics, clinical, laboratory, instrumental and socioeconomic data from PFAPA patients. Data recruitment is realized through the on-line Research Electronic Data Capture (REDCap) tool. This registry is thought to collect standardized information for clinical research leading to solid real-life evidence. The international scope and the flexibility of the registry will facilitate the realization of cutting-edge study projects through the constant updating of variables and the possible merging and transfer of data between current and future PFAPA registries. Results: A total of 112 centers have already been involved from 23 countries and 4 continents starting from August 24th, 2021, to April 6th, 2022. In total 56/112 have already obtained the formal approval from their local Ethics Committees. The platform counts 321 users (113 principal investigators, 203 site investigators, two lead investigators, and three data managers). The registry collects retrospective and prospective data using 3,856 fields organized into 25 instruments, including PFAPA patient's demographics, medical histories, symptoms, triggers/risk factors, therapies, and impact on the healthcare systems. Conclusions: The development of the AIDA International Registry for PFAPA patients will enable the on-line collection of standardized data prompting real-life studies through the connection of worldwide groups of physicians and researchers. This project can be found on NCT 05200715.
  • conferenceObject
    CLOSE TO THE BONE: A CASE OF CONCURRENTMULTIFOCAL OSTEONECROSIS AND PERIPHERAL SPONDYLOARTHROPATHY
    (2022) MACEDO, M. B.; GIARDINI, H. A. M.; GUEDES, L. K. N.; PEREIRA, R. M. R.
  • article 2 Citação(ões) na Scopus
    Development and implementation of the AIDA international registry for patients with Schnitzler's syndrome
    (2022) SOTA, Jurgen; VITALE, Antonio; WIESIK-SZEWCZYK, Ewa; FRASSI, Micol; LOPALCO, Giuseppe; EMMI, Giacomo; GOVONI, Marcello; PAULIS, Amato de; MARINO, Achille; GIDARO, Antonio; MONTI, Sara; OPRIS-BELINSKI, Daniela; PEREIRA, Rosa Maria R.; JAHNZ-ROZYK, Karina; GAGGIANO, Carla; CRISAFULLI, Francesca; IANNONE, Florenzo; MATTIOLI, Irene; RUFFILLI, Francesca; MORMILE, Ilaria; RYBAK, Katarzyna; CAGGIANO, Valeria; AIRO, Paolo; TUFAN, Abdurrahman; GENTILESCHI, Stefano; RAGAB, Gaafar; ALMAGHLOUTH, Ibrahim A.; KHALIL, Adham Aboul-Fotouh; CATTALINI, Marco; TORRE, Francesco La; TARSIA, Maria; GIARDINI, Henrique A. Mayrink; SAAD, Moustafa Ali; BOCCHIA, Monica; CARONI, Federico; GIANI, Teresa; CINOTTI, Elisa; RUSCITTI, Piero; RUBEGNI, Pietro; DAGOSTIN, Marilia A.; FREDIANI, Bruno; GULER, Aslihan Avanoglu; CASA, Francesca Della; MAGGIO, Maria Cristina; RECKE, Andreas; BUBNOFF, Dagmar von; KRAUSE, Karoline; BALISTRERI, Alberto; FABIANI, Claudia; RIGANTE, Donato; CANTARINI, Luca
    Objective: The present paper describes the design, development, and implementation of the AutoInflammatory Disease Alliance (AIDA) International Registry specifically dedicated to patients with Schnitzler's syndrome. Methods: This is a clinical physician-driven, population- and electronic-based registry implemented for the retrospective and prospective collection of real-life data from patients with Schnitzler's syndrome; the registry is based on the Research Electronic Data Capture (REDCap) tool, which is designed to collect standardized information for clinical research, and has been realized to change over time according to future scientific acquisitions and potentially communicate with other existing or future similar registries. Results: Since its launch, 113 centers from 23 countries in 4 continents have been involved. Fifty-seven have already obtained the approval from their local Ethics Committees. The platform counts 324 users (114 Principal Investigators, 205 Site Investigators, 2 Lead Investigators, and 3 data managers) at current (April 28th, 2022). The registry collects baseline and follow-up data using 3,924 fields organized into 25 instruments, including patient's demographics, history, clinical manifestations and symptoms, trigger/risk factors, laboratory, instrumental exams, therapies, socioeconomic information, and healthcare access. Conclusions: This International Registry for patients with Schnitzler's syndrome facilitates standardized data collection, enabling international collaborative projects through data sharing and dissemination of knowledge; in turn, it will shed light into many blind spots characterizing this complex autoinflammatory disorder.
  • article 4 Citação(ões) na Scopus
    Immunogenicity decay and case incidence six months post Sinovac-CoronaVac vaccine in autoimmune rheumatic diseases patients
    (2022) SILVA, Clovis A.; MEDEIROS-RIBEIRO, Ana C.; KUPA, Leonard V. K.; YUKI, Emily F. N.; PASOTO, Sandra G.; SAAD, Carla G. S.; FUSCO, Solange R. G.; PEREIRA, Rosa M. R.; SHINJO, Samuel K.; HALPERN, Ari S. R.; BORBA, Eduardo F.; SOUZA, Fernando H. C.; GUEDES, Lissiane K. N.; MIOSSI, Renata; BONFIGLIOLI, Karina R.; DOMICIANO, Diogo S.; SHIMABUCO, Andrea Y.; ANDRADE, Danieli C. O.; SEGURO, Luciana P. C.; FULLER, Ricardo; SAMPAIO-BARROS, Percival D.; ASSAD, Ana P. L.; MORAES, Julio C. B.; GOLDENSTEIN-SCHAINBERG, Claudia; GIARDINI, Henrique A. M.; SILVA, Henrique C.; MARTINS, Victor A. O.; VILLAMARIN, Lorena E. B.; NOVELLINO, Renata S.; SALES, Lucas P.; ARAUJO, Carlo S. R.; SILVA, Matheus S. R.; FILHO, Dilson M. N.; LOPES, Marta H.; DUARTE, Alberto J. S.; KALLAS, Esper G.; AIKAWA, Nadia E.; BONFA, Eloisa
    Characterising the response to SARS-CoV-2 post vaccination is critical in the appraisement of the induced immune response, performance and protective potential. Here the authors present data from a phase 4 clinical trial in autoimmune rheumatic disease patients 6 months post second dose of Sinovac-CoronaVac inactivated vaccine that show a marked reduction in antibody particularly in males or those under treatment with immune targeting therapies but saw no rise in COVID-19 disease. The determination of durability and vaccine-associated protection is essential for booster doses strategies, however data on the stability of SARS-CoV-2 immunity are scarce. Here we assess anti-SARS-CoV-2 immunogenicity decay and incident cases six months after the 2(nd) dose of Sinovac-CoronaVac inactivated vaccine (D210) in 828 autoimmune rheumatic diseases patients compared with 207 age/sex-balanced control individuals. The primary outcome is the presence of anti-S1/S2 SARS-CoV-2 IgG at 6 months compared to 6 weeks after 2nd vaccine dose for decay evaluation. Secondary outcomes are presence of neutralizing antibodies, percent inhibition by neutralizing, geometric mean titers and cumulative incident cases at 6 months after 2nd dose. Anti-S1/S2 IgG positivity and titers reduce to 23.8% and 38% in patients (p < 0.001) during the six-month follow up and 20% and 51% in controls (p < 0.001), respectively. Neutralizing antibodies positivity and percent inhibition declines 41% and 54% in patients (p < 0.001) and 39.7% and 47% in controls (p < 0.001). Multivariate logistic regression analysis show males (OR = 0.56;95% CI0.40-0.79), prednisone (OR = 0.56; 95% CI0.41-0.76), anti-TNF (OR = 0.66;95% CI0.45-0.96), abatacept (OR = 0.29; 95% CI0.15-0.56) and rituximab (OR = 0.32;95% CI0.11-0.90) associate with a substantial reduction in IgG response at day 210 in patients. Although cellular immunity was not assessed, a decrease of COVID-19 cases (from 27.5 to 8.1/100 person-years; p < 0.001) is observed despite the concomitant emergence and spread of the Delta variant. Altogether we show a reduction in immunity 6-months of Sinovac-CoronaVac 2nd dose, particularly in males and those under immunosuppressives therapies, without a concomitant rise in COVID-19 cases. (CoronavRheum clinicaltrials.gov:NCT04754698).
  • article 3 Citação(ões) na Scopus
    Development and Implementation of the AIDA International Registry for Patients With Undifferentiated Systemic AutoInflammatory Diseases
    (2022) CASA, Francesca Della; VITALE, Antonio; LOPALCO, Giuseppe; RUSCITTI, Piero; CICCIA, Francesco; EMMI, Giacomo; CATTALINI, Marco; WIESIK-SZEWCZYK, Ewa; MAGGIO, Maria Cristina; OGUNJIMI, Benson; SFIKAKIS, Petros P.; TUFAN, Abdurrahman; AL-MAYOUF, Sulaiman M.; GIUDICE, Emanuela Del; ARAGONA, Emma; TORRE, Francesco La; SOTA, Jurgen; COLELLA, Sergio; COLA, Ilenia Di; IACONO, Daniela; MATTIOLI, Irene; JAHNZ-ROZYK, Karina; JOOS, Rik; LASKARI, Katerina; GAGGIANO, Carla; ABBRUZZESE, Anna; CIPRIANI, Paola; ROZZA, Gelsomina; ALSALEEM, Alhanouf; YILDIRIM, Derya; TARSIA, Maria; RAGAB, Gaafar; RICCI, Francesca; CARDINALE, Fabio; KORZENIOWSKA, Marcelina; FRASSI, Micol; CAGGIANO, Valeria; SAAD, Moustafa Ali; PEREIRA, Rosa Maria; BERLENGIERO, Virginia; GENTILESCHI, Stefano; GUERRIERO, Silvana; GIANI, Teresa; GELARDI, Viviana; IANNONE, Florenzo; GIARDINI, Henrique Ayres Mayrink; ALMAGHLOUTH, Ibrahim A.; KARDAS, Riza Can; AIT-IDIR, Djouher; FREDIANI, Bruno; BALISTRERI, Alberto; FABIANI, Claudia; RIGANTE, Donato; CANTARINI, Luca
    Objective: This paper points out the design, development and deployment of the AutoInflammatory Disease Alliance (AIDA) International Registry dedicated to pediatric and adult patients affected by Undifferentiated Systemic AutoInflammatory Diseases (USAIDs). Methods: This is an electronic registry employed for real-world data collection about demographics, clinical, laboratory, instrumental and socioeconomic data of USAIDs patients. Data recruitment, based on the Research Electronic Data Capture (REDCap) tool, is designed to obtain standardized information for real-life research. The instrument is endowed with flexibility, and it could change over time according to the scientific acquisitions and potentially communicate with other similar tools; this platform ensures security, data quality and data governance. Results: The focus of the AIDA project is connecting physicians and researchers from all over the world to shed a new light on heterogeneous rare diseases. Since its birth, 110 centers from 23 countries and 4 continents have joined the AIDA project. Fifty-four centers have already obtained the approval from their local Ethics Committees. Currently, the platform counts 290 users (111 Principal Investigators, 179 Site Investigators, 2 Lead Investigators, and 2 data managers). The Registry is collecting baseline and follow-up data using 3,769 fields organized into 23 instruments, which include demographics, history, symptoms, trigger/risk factors, therapies, and healthcare information access for USAIDs patients. Conclusions: The development of the AIDA International Registry for USAIDs patients will facilitate the online collection of real standardized data, connecting a worldwide group of researchers: the Registry constitutes an international multicentre observational groundwork aimed at increasing the patient cohort of USAIDs in order to improve our knowledge of this peculiar cluster of autoinflammatory diseases.
  • article 13 Citação(ões) na Scopus
    Development and Implementation of the AIDA International Registry for Patients With Still's Disease
    (2022) VITALE, Antonio; CASA, Francesca Della; LOPALCO, Giuseppe; PEREIRA, Rosa Maria; RUSCITTI, Piero; GIACOMELLI, Roberto; RAGAB, Gaafar; TORRE, Francesco La; BARTOLONI, Elena; GIUDICE, Emanuela Del; LOMATER, Claudia; EMMI, Giacomo; GOVONI, Marcello; MAGGIO, Maria Cristina; MAIER, Armin; MAKOWSKA, Joanna; OGUNJIMI, Benson; SFIKAKIS, Petros P.; SFRISO, Paolo; GAGGIANO, Carla; IANNONE, Florenzo; DAGOSTIN, Marilia A.; COLA, Ilenia Di; NAVARINI, Luca; MAHMOUD, Ayman Abdelmonem Ahmed; CARDINALE, Fabio; RICCUCCI, Ilenia; PAROLI, Maria Pia; MARUCCO, Elena Maria; MATTIOLI, Irene; SOTA, Jurgen; ABBRUZZESE, Anna; ANTONELLI, Isabele P. B.; CIPRIANI, Paola; TUFAN, Abdurrahman; FABIANI, Claudia; RAMADAN, Mustafa Mahmoud; CATTALINI, Marco; KARDAS, Riza Can; SEBASTIANI, Gian Domenico; GIARDINI, Henrique A. Mayrink; HERNANDEZ-RODRIGUEZ, Jose; MASTRORILLI, Violetta; WIESIK-SZEWCZYK, Ewa; FRASSI, Micol; CAGGIANO, Valeria; TELESCA, Salvatore; GIORDANO, Heitor F.; GUADALUPI, Emmanuele; GIANI, Teresa; RENIERI, Alessandra; COLELLA, Sergio; CATALDI, Giulia; GENTILE, Martina; FABBIANI, Alessandra; AL-MAGHLOUTH, Ibrahim A.; FREDIANI, Bruno; BALISTRERI, Alberto; RIGANTE, Donato; CANTARINI, Luca
    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. NCT 05200715 available at .
  • article 12 Citação(ões) na Scopus
    Development and implementation of the AIDA International Registry for patients with Behcet's disease
    (2022) VITALE, Antonio; CASA, Francesca Della; RAGAB, Gaafar; ALMAGHLOUTH, Ibrahim A.; LOPALCO, Giuseppe; PEREIRA, Rosa Maria; GUERRIERO, Silvana; GOVONI, Marcello; SFIKAKIS, Petros P.; GIACOMELLI, Roberto; CICCIA, Francesco; MONTI, Sara; RUSCITTI, Piero; PIGA, Matteo; LOMATER, Claudia; TUFAN, Abdurrahman; OPRIS-BELINSKI, Daniela; EMMI, Giacomo; HERNANDEZ-RODRIGUEZ, Jose; KARKAS, Burak; SEBASTIANI, Gian Domenico; BARTOLONI, Elena; AKKOC, Nurullah; CATTALINI, Marco; CONTI, Giovanni; HATEMI, Gulen; MAIER, Armin; PARRONCHI, Paola; GIUDICE, Emanuela Del; ERTEN, Sukran; INSALACO, Antonella; GOBBI, Francesca Li; MAGGIO, Maria Cristina; SHAHRAM, Farhad; CAGGIANO, Valeria; HEGAZY, Mohamed Tharwat; ASFINA, Kazi Nur; MORRONE, Maria; PRADO, Leandro L.; DAMMACCO, Rosanna; RUFFILLI, Francesca; ARIDA, Aikaterini; NAVARINI, Luca; PANTANO, Ilenia; CAVAGNA, Lorenzo; CONFORTI, Alessandro; CAULI, Alberto; MARUCCO, Elena Maria; KUCUK, Hamit; IONESCU, Ruxandra; MATTIOLI, Irene; ESPINOSA, Gerard; ARAUJO, Olga; CANOFARI, Claudia; SOTA, Jurgen; LAYMOUNA, Ahmed Hatem; BEDAIWI, Asma A.; COLELLA, Sergio; GIARDINI, Henrique Ayres M.; ALBANO, Valeria; MONACO, Andrea Lo; FRAGOULIS, George E.; KARDAS, Riza Can; BERLENGIERO, Virginia; HUSSEIN, Mohamed A.; RICCI, Francesca; TORRE, Francesco La; RIGANTE, Donato; WIESIK-SZEWCZYK, Ewa; FRASSI, Micol; GENTILESCHI, Stefano; TOSI, Gian Marco; DAGOSTIN, Marilia Ambiel; MAHMOUD, Ayman Abdel-Monem Ahmed; TARSIA, Maria; ALESSIO, Giovanni; CIMAZ, Rolando; GIANI, Teresa; GAGGIANO, Carla; IANNONE, Florenzo; CIPRIANI, Paola; MOURABI, Mariam; SPEDICATO, Veronica; BARNESCHI, Sara; ARAGONA, Emma; BALISTRERI, Alberto; FREDIANI, Bruno; FABIANI, Claudia; CANTARINI, Luca
    Purpose of the present paper is to point out the design, development and deployment of the AutoInflammatory Disease Alliance (AIDA) International Registry dedicated to pediatric and adult patients with Behcet's disease (BD). The Registry is a clinical physician-driven non-population- and electronic-based instrument implemented for the retrospective and prospective collection of real-life data about demographics, clinical, therapeutic, laboratory, instrumental and socioeconomic information from BD patients; the Registry is based on the Research Electronic Data Capture (REDCap) tool, which is thought to collect standardised information for clinical real-life research, and has been realised to change over time according to future scientific acquisitions and potentially communicate with other existing and future Registries dedicated to BD. Starting from January 31st, 2021, to February 7th, 2022, 110 centres 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 5993 fields organised into 16 instruments, including patient's demographics, history, clinical manifestations and symptoms, trigger/risk factors, therapies and healthcare access. The development of the AIDA International Registry for BD patients will facilitate the collection of standardised data leading to real-world evidence, enabling international multicentre collaborative research through data sharing, international consultation, dissemination of knowledge, inclusion of patients and families, and ultimately optimisation of scientific efforts and implementation of standardised care. Trial registration NCT05200715 in 21/01/2022.
  • article 0 Citação(ões) na Scopus

    A retracted diagnosis-when liver capsule retraction is not what it seems

    (2022) MACEDO, Marina Bargui; GIARDINI, Henrique Ayres Mayrink; PEREIRA, Rosa Maria Rodrigues
  • article 13 Citação(ões) na Scopus
    Development and Implementation of the AIDA International Registry for Patients With VEXAS Syndrome
    (2022) VITALE, Antonio; CAGGIANO, Valeria; CASA, Francesca Della; HERNANDEZ-RODRIGUEZ, Jose; FRASSI, Micol; MONTI, Sara; TUFAN, Abdurrahman; TELESCA, Salvatore; CONTICINI, Edoardo; RAGAB, Gaafar; LOPALCO, Giuseppe; ALMAGHLOUTH, Ibrahim; PEREIRA, Rosa Maria R.; YILDIRIM, Derya; CATTALINI, Marco; MARINO, Achille; GIANI, Teresa; TORRE, Francesco La; RUSCITTI, Piero; ARAGONA, Emma; WIESIK-SZEWCZYK, Ewa; GIUDICE, Emanuela Del; SFIKAKIS, Petros P.; GOVONI, Marcello; EMMI, Giacomo; MAGGIO, Maria Cristina; GIACOMELLI, Roberto; CICCIA, Francesco; CONTI, Giovanni; AIT-IDIR, Djouher; LOMATER, Claudia; SABATO, Vito; PIGA, Matteo; SAHIN, Ali; OPRIS-BELINSKI, Daniela; IONESCU, Ruxandra; BARTOLONI, Elena; FRANCESCHINI, Franco; PARRONCHI, Paola; PAULIS, Amato de; ESPINOSA, Gerard; MAIER, Armin; SEBASTIANI, Gian Domenico; INSALACO, Antonella; SHAHRAM, Farhad; SFRISO, Paolo; MINOIA, Francesca; ALESSIO, Maria; MAKOWSKA, Joanna; HATEMI, Gulen; AKKOC, Nurullah; GOBBI, Francesca Li; GIDARO, Antonio; OLIVIERI, Alma Nunzia; AL-MAYOUF, Sulaiman M.; ERTEN, Sukran; GENTILESCHI, Stefano; VASI, Ibrahim; TARSIA, Maria; MAHMOUD, Ayman Abdel-Monem Ahmed; FREDIANI, Bruno; ALZAHRANI, Musa Fares; LAYMOUNA, Ahmed Hatem; RICCI, Francesca; CARDINALE, Fabio; JAHNZ-ROZYK, Karina; TOSI, Gian Marco; CRISAFULLI, Francesca; BALISTRERI, Alberto; DAGOSTIN, Marilia A.; GHANEMA, Mahmoud; GAGGIANO, Carla; SOTA, Jurgen; COLA, Ilenia Di; FABIANI, Claudia; GIARDINI, Henrique A. Mayrink; RENIERI, Alessandra; FABBIANI, Alessandra; CARRER, Anna; BOCCHIA, Monica; CARONI, Federico; RIGANTE, Donato; CANTARINI, Luca
    Objective: The aim of this paper is to present the AutoInflammatory Disease Alliance (AIDA) international Registry dedicated to Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) syndrome, describing its design, construction, and modalities of dissemination. Methods: This Registry is a clinical, physician-driven, population- and electronic-based instrument designed for the retrospective and prospective collection of real-life data. Data gathering is based on the Research Electronic Data Capture (REDCap) tool and is intended to obtain real-world evidence for daily patients' management. The Registry may potentially communicate with other on-line tools dedicated to VEXAS syndrome, thus enhancing international collaboration and data sharing for research purposes. The Registry is practical enough to be easily modified to meet future needs regarding VEXAS syndrome. Results: To date (April 22(nd), 2022), 113 Centers from 23 Countries in 4 continents have been involved; 324 users (114 Principal Investigators, 205 Site Investigators, 2 Lead Investigators, and 3 data managers) are currently able to access the registry for data entry (or data sharing) and collection. The Registry includes 4,952 fields organized into 18 instruments designed to fully describe patient's details about demographics, clinical manifestations, symptoms, histologic details about skin and bone marrow biopsies and aspirate, laboratory features, complications, comorbidities, therapies, and healthcare access. Conclusion: This international Registry for patients with VEXAS syndrome will allow the achievement of a comprehensive knowledge about this new disease, with the final goal to obtain real-world evidence for daily clinical practice, especially in relation to the comprehension of this disease about the natural history and the possible therapeutic approaches.
  • article 10 Citação(ões) na Scopus
    The Autoinflammatory Diseases Alliance Registry of monogenic autoinflammatory diseases
    (2022) GAGGIANO, Carla; VITALE, Antonio; TUFAN, Abdurrahman; RAGAB, Gaafar; ARAGONA, Emma; WIESIK-SZEWCZYK, Ewa; AIT-IDIR, Djouher; CONTI, Giovanni; IEZZI, Ludovica; MAGGIO, Maria Cristina; CATTALINI, Marco; TORRE, Francesco La; LOPALCO, Giuseppe; VERRECCHIA, Elena; PAULIS, Amato de; SAHIN, Ali; INSALACO, Antonella; SFIKAKIS, Petros P.; MARINO, Achille; FRASSI, Micol; OGUNJIMI, Benson; OPRIS-BELINSKI, Daniela; PARRONCHI, Paola; EMMI, Giacomo; SHAHRAM, Farhad; CICCIA, Francesco; PIGA, Matteo; HERNANDEZ-RODRIGUEZ, Jose; PEREIRA, Rosa Maria R.; ALESSIO, Maria; NADDEI, Roberta; OLIVIERI, Alma Nunzia; GIUDICE, Emanuela Del; SFRISO, Paolo; RUSCITTI, Piero; GOBBI, Francesca Li; KUCUK, Hamit; SOTA, Jurgen; HUSSEIN, Mohamed A.; MALIZIA, Giuseppe; JAHNZ-ROZYK, Karina; SARI-HAMIDOU, Rawda; ROMEO, Mery; RICCI, Francesca; CARDINALE, Fabio; IANNONE, Florenzo; CASA, Francesca Della; NATALE, Marco Francesco; LASKARI, Katerina; GIANI, Teresa; FRANCESCHINI, Franco; SABATO, Vito; YILDIRIM, Derya; CAGGIANO, Valeria; HEGAZY, Mohamed Tharwat; MARZO, Rosalba Di; KUCHARCZYK, Aleksandra; KHELLAF, Ghalia; TARSIA, Maria; ALMAGHLOUTH, Ibrahim A.; LAYMOUNA, Ahmed Hatem; MASTRORILLI, Violetta; DOTTA, Laura; BENACQUISTA, Luca; GROSSO, Salvatore; CRISAFULLI, Francesca; PARRETTI, Veronica; GIORDANO, Heitor F.; MAHMOUD, Ayman Abdel-Monem Ahmed; NUZZOLESE, Rossana; MUSSO, Marta De; CHIGHIZOLA, Cecilia Beatrice; GENTILESCHI, Stefano; MORRONE, Mirella; COLA, Ilenia Di; SPEDICATO, Veronica; GIARDINI, Henrique A. Mayrink; VASI, Ibrahim; RENIERI, Alessandra; FABBIANI, Alessandra; MENCARELLI, Maria Antonietta; FREDIANI, Bruno; BALISTRERI, Alberto; TOSI, Gian Marco; FABIANI, Claudia; LIDAR, Merav; RIGANTE, Donato; CANTARINI, Luca
    ObjectiveThe present manuscript aims to describe an international, electronic-based, user-friendly and interoperable patient registry for monogenic autoinflammatory diseases (mAIDs), developed in the contest of the Autoinflammatory Diseases Alliance (AIDA) Network.MethodsThis is an electronic platform, based on the Research Electronic Data Capture (REDCap) tool, used for real-world data collection of demographics, clinical, laboratory, instrumental and socioeconomic data of mAIDs patients. The instrument has flexibility, may change over time based on new scientific acquisitions, and communicate potentially with other similar registries; security, data quality and data governance are corner stones of the platform.ResultsAIDA project will share knowledge and expertise on mAIDs. Since its start, 118 centers from 24 countries and 4 continents have joined the AIDA project. Fifty-nine centers have already obtained the approval from their local Ethics Committees. Currently, the platform counts 337 users (122 Principal Investigators, 210 Site Investigators, 2 Lead Investigators, and 3 data managers). The Registry collects baseline and follow-up data using 3,748 fields organized into 21 instruments, which include demographics, patient history, symptoms, trigger/risk factors, therapies, and healthcare information for mAIDs patients.ConclusionsThe AIDA mAIDs Registry, acts both as a research tool for future collaborative real-life studies on mAIDs and as a service to connect all the figures called to participate. On this basis, the registry is expected to play a pivotal role in generating new scientific evidence on this group of rare diseases, substantially improving the management of patients, and optimizing the impact on the healthcare system. NCT 05200715 available at .