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1. Clinical Determinants of Successful Omalizumab Therapy in Severe Allergic Asthma Patients: 4-Year-Long, Real-Life Observation

Author

Kucharczyk A, Więsik-Szewczyk E, Poznańska A, and Jahnz-Różyk K

Subjects
allergic asthma, anti-ige, biologics, personalized therapy, Immunologic diseases. Allergy, RC581-607
Abstract

Aleksandra Kucharczyk,1 Ewa Więsik-Szewczyk,1 Anna Poznańska,2 Karina Jahnz-Różyk1 1Department of Internal Medicine, Pulmonology, Allergy and Clinical Immunology, Central Clinical Hospital of the Ministry of National Defence, Military Institute of Medicine, Warsaw, Poland; 2Department of Population Health Monitoring and Analysis, National Institute of Public Health – National Institute of Hygiene, Warsaw, PolandCorrespondence: Ewa Więsik-SzewczykDepartment of Internal Medicine, Pulmonology, Allergy and Clinical Immunology, Central Clinical Hospital of the Ministry of National Defence, Military Institute of Medicine, Szaserów 128, Warsaw 01-141, PolandTel/ Fax +48 261 816 581Email ewa.w.szewczyk@gmail.comIntroduction: Omalizumab is a high-cost therapy recommended for the treatment of severe allergic asthma.Objective: To find clinical parameters that are related to the sustained response to omalizumab.Patients and Methods: This retrospective, real-life, 4-year follow-up was provided in Poland between March 2013 and May 2019. The success of omalizumab was assessed based on composed subjective and objective criteria. Simple/multiple regression analyses were performed to search for predictors of the response to omalizumab.Results: A total of 989 severe allergic asthma patients were referred for omalizumab therapy, of whom 854 patients were considered eligible for treatment. At weeks 16 and 52, omalizumab was successful in 84% and 91% of patients, respectively. Treatment effectiveness was maintained up to the 4-year follow-up. Four predictors of the response to omalizumab were found at week 16 and two at week 52. The results at week 16 may be used as predictors of success at week 52 based on the model including baseline FEV1% and change in ACQ-7 and miniAQLQ score at week 16: the area under the ROC curve equals 0.746 [95% CI: 0.672– 0.820].Conclusion: Omalizumab therapy is very effective, with this efficacy sustained after 4 years of treatment. Success of the therapy can be predicted from the baseline FEV1% and clinical improvement (based on ACQ-7 and miniAQLQ scores) at week 16.Keywords: allergic asthma, anti-IgE, biologics, personalized therapy

Published
2020

2. AB0431 CANAKINUMAB AS FIRST-LINE BIOLOGICAL THERAPY IN STILL’S DISEASE AND DIFFERENCES BETWEEN THE SYSTEMIC AND THE CHRONIC-ARTICULAR COURSES: REAL-LIFE EXPERIENCE FROM THE INTERNATIONAL AIDA REGISTRY

Author

Vitale, A., primary, Caggiano, V., additional, Maggio, M. C., additional, Lopalco, G., additional, Emmi, G., additional, Sota, J., additional, La Torre, F., additional, Ruscitti, P., additional, Bartoloni, E., additional, Conti, G., additional, Fabiani, C., additional, Mattioli, I., additional, Gaggiano, C., additional, Cardinale, F., additional, Dagna, L., additional, Campochiaro, C., additional, Giacomelli, R., additional, Balistreri, A., additional, Laskari, K., additional, Tufan, A., additional, Ragab, G., additional, Almaghlouth, I., additional, Więsik-Szewczyk, E., additional, Pereira, R. M., additional, Frediani, B., additional, Iannone, F., additional, Sfikakis, P., additional, and Cantarini, L., additional

Published
2023
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3. Axial spondyloarthritis in patients with recurrent fever attacks: data from the AIDA network registry for undifferentiated autoinflammatory diseases (USAIDs)

Author

Vitale, A, Caggiano, V, Silva, I, Oliveira, Dg, Ruscitti, P, Ciccia, F, Vasi, I, Tufan, A, Lopalco, G, Almaghlouth, Ia, Sota, J, Wiesik-Szewczyk, E, Gaggiano, C, Giardini, Ham, Spedicato, V, Ragab, G, Iannone, F, Balistreri, A, Frassi, M, Hernández-Rodríguez, J, Fabiani, C, Falsetti, P, Di Meglio, N, Frediani, B, Mazzei, Ma, Rigante, Donato, Faria, R, and Cantarini, L

Subjects
Uveitis, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Adalimumab
Published
2023

4. Preliminary data revealing efficacy of Streptococcus salivarius K12 (SSK12) in Periodic Fever, Aphthous stomatitis, Pharyngitis, and cervical Adenitis (PFAPA) syndrome : a multicenter study from the AIDA Network PFAPA syndrome registry

Author

La Torre, F, Sota, J, Insalaco, A, Conti, G, Del Giudice, E, Lubrano, R, Breda, L, Maggio, Mc, Civino, A, Mastrorilli, V, Loconte, R, Natale, Mf, Celani, C, Romeo, M, Patroniti, S, Gentile, C, Vitale, A, Caggiano, V, Gaggiano, C, Diomeda, F, Cattalini, M, Lopalco, G, Emmi, G, Parronchi, P, Gentileschi, S, Cardinale, F, Aragona, E, Shahram, F, Marino, A, Barone, P, Moscheo, C, Ozkiziltas, B, Carubbi, F, Alahmed, O, Iezzi, Ludovica, Ogunjimi, B, Mauro, A, Tarsia, M, Mahmoud, Aaa, Mayrink Giardini HAM, Sfikakis, Pp, Laskari, K, Więsik-Szewczyk, E, Hernàndez-Rodriguez, J, Frediani, B, Gòmez-Caverzaschi, V, Tufan, A, Almaghlouth, Ia, Balistreri, A, Ragab, G, Fabiani, C, Cantarini, L, Rigante, Donato, AIDA Network, La Torre, Francesco, Sota, Jurgen, Insalaco, Antonella, Conti, Giovanni, Del Giudice, Emanuela, Lubrano, Riccardo, Breda, Luciana, Maggio, Maria Cristina, Civino, Adele, Mastrorilli, Violetta, Loconte, Roberta, Natale, Marco Francesco, Celani, Camilla, Romeo, Mery, Patroniti, Serena, Gentile, Cristina, Vitale, Antonio, Caggiano, Valeria, Gaggiano, Carla, Diomeda, Federico, Cattalini, Marco, Lopalco, Giuseppe, Emmi, Giacomo, Parronchi, Paola, Gentileschi, Stefano, Cardinale, Fabio, Aragona, Emma, Shahram, Farhad, Marino, Achille, Barone, Patrizia, Moscheo, Carla, Ozkiziltas, Burcugul, Carubbi, Francesco, Alahmed, Ohoud, Iezzi, Ludovica, Ogunjimi, Benson, Mauro, Angela, Tarsia, Maria, Mahmoud, Ayman Abdel-Monem Ahmed, Giardini, Henrique Ayres Mayrink, Sfikakis, Petros P, Laskari, Katerina, Więsik-Szewczyk, Ewa, Hernández-Rodríguez, José, Frediani, Bruno, Gómez-Caverzaschi, Verónica, Tufan, Abdurrahman, Almaghlouth, Ibrahim A, Balistreri, Alberto, Ragab, Gaafar, Fabiani, Claudia, Cantarini, Luca, and Rigante, Donato

Subjects
International Registry, PFAPA syndrome, Streptococcus salivarius K12, autoinflammatory disease, probiotic, prophylaxis, tonsillitis, Settore MED/38 - Pediatria Generale E Specialistica, Autoinflammation, General Medicine, Human medicine, PFAPA syndrome
Abstract

ObjectiveTo evaluate the potential role of Streptococcus salivarius K12 (SSK12) in controlling febrile flares in patients with Periodic Fever, Aphthous stomatitis, Pharyngitis, and cervical Adenitis (PFAPA) syndrome. Further aims were to assess the impact of SSK12 on (i) flare duration, (ii) variation in the degree of the highest body temperature during flares, (iii) steroid-sparing effect, and (iv) change of PFAPA accompanying symptoms before and after SSK12 introduction.Patients and methodsThe medical charts from 85 pediatric patients with PFAPA syndrome (49 males and 36 females) enrolled in the AIDA registry and treated with SSK12 for a median period of 6.00 ± 7.00 months in the period between September 2017 and May 2022 were examined. Children recruited had a median time of disease duration of 19.00 ± 28.00 months.ResultsThe number of febrile flares significantly decreased comparing the 12 months before [median (IQR), 13.00 (6.00)] and after SSK12 initiation [median (IQR), 5.50 (8.00), p < 0.001]. The duration of fever was significantly reduced from 4.00 (2.00) days to 2.00 (2.00) days [p < 0.001]. Similarly, the highest temperature in°C was found significantly lower in the last follow-up assessment [median (IQR), 39.00 (1.00)] compared to the period prior to SSK12 start [median (IQR), 40.00 (1.00), p < 0.001]. Steroid load (mg/year) of betamethasone (or any equivalent steroid) significantly decreased between 12 months before treatment with SSK12 [median (IQR), 5.00 (8.00) mg/year] and the last follow-up visit [median (IQR), 2.00 (4.00) mg/year, p < 0.001]. The number of patients experiencing symptoms including pharyngitis/tonsillitis (p < 0.001), oral aphthae (p < 0.001) and cervical lymphadenopathy (p < 0.001) significantly decreased following SSK12.ConclusionSSK12 prophylaxis given for at least 6.00 months was found to reduce febrile flares of PFAPA syndrome: in particular, it halved the total number per year of fever flares, shortened the duration of the single febrile episode, lowered body temperature by 1°C in the febrile flare, provided a steroid-sparing effect, and significantly reduced the accompanying symptoms related to the syndrome.

Published
2023

5. Development and implementation of the AIDA International Registry for patients with Behçet's disease

Author

Vitale, A, Della Casa, F, Ragab, G, Almaghlouth, Ia, Lopalco, G, Pereira, Rm, Guerriero, S, Govoni, M, Sfikakis, Pp, Giacomelli, R, Ciccia, F, Monti, S, Ruscitti, P, Piga, M, Lomater, C, Tufan, A, Opris-Belinski, D, Emmi, G, Hernández-Rodríguez, J, Şahin, A, Sebastiani, Gd, Bartoloni, E, Akkoç, N, Gündüz, Ös, Cattalini, M, Conti, Giorgio, Hatemi, G, Maier, A, Parronchi, P, Del Giudice, E, Erten, S, Insalaco, A, Li Gobbi, F, Maggio, Mc, Shahram, F, Caggiano, V, Hegazy, Mt, Asfina, Kn, Morrone, M, Prado, Ll, Dammacco, R, Ruffilli, F, Arida, A, Navarini, L, Pantano, I, Cavagna, L, Conforti, A, Cauli, A, Marucco, Em, Kucuk, H, Ionescu, R, Mattioli, I, Espinosa, G, Araújo, O, Karkaş, B, Canofari, C, Sota, J, Laymouna, Ah, Bedaiwi, Aa, Colella, S, Giardini, Ham, Albano, V, Lo Monaco, A, Fragoulis, Ge, Kardas, Rc, Berlengiero, V, Hussein, Ma, Ricci, F, La Torre, F, Rigante, Donato, Więsik-Szewczyk, E, Frassi, M, Gentileschi, S, Tosi, Gm, Dagostin, Ma, Mahmoud, Aaa, Tarsia, M, Alessio, G, Cimaz, R, Giani, T, Gaggiano, C, Iannone, F, Cipriani, P, Mourabi, M, Spedicato, V, Barneschi, S, Aragona, E, Balistreri, A, Frediani, B, Fabiani, C, Cantarini, L, Autoinflammatory Diseases Alliance (AIDA) Network, Vitale, Antonio, Della Casa, Francesca, 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, Hernández-Rodríguez, José, Şahin, Ali, Sebastiani, Gian Domenico, Bartoloni, Elena, Akkoç, Nurullah, Gündüz, Özgül Soysal, Cattalini, Marco, Conti, Giovanni, Hatemi, Gulen, Maier, Armin, Parronchi, Paola, Del Giudice, Emanuela, Erten, Sukran, Insalaco, Antonella, Li Gobbi, Francesca, 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, Araújo, Olga, Karkaş, Burak, Canofari, Claudia, Sota, Jurgen, Laymouna, Ahmed Hatem, Bedaiwi, Asma A, Colella, Sergio, Giardini, Henrique Ayres M, Albano, Valeria, Lo Monaco, Andrea, Fragoulis, George E, Kardas, Riza Can, Berlengiero, Virginia, Hussein, Mohamed A, Ricci, Francesca, La Torre, Francesco, Rigante, Donato, Więsik-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, and Cantarini, Luca

Subjects
Adult, Registrie, Autoinflammatory disease, Registry, Settore MED/16 - REUMATOLOGIA, precision medicine, behçet’s disease, Settore MED/38 - Pediatria Generale E Specialistica, Retrospective Studie, Internal Medicine, Humans, Prospective Studies, Registries, Child, international registry, Retrospective Studies, Behçet's disease, autoinflammatory diseases, rare diseases, uveitis, Behcet Syndrome, Prospective Studie, Uveiti, Emergency Medicine, Rare disease, Human
Abstract

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 Behçet’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.

Published
2022

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

Author

Vitale, A. Della Casa, F. Lopalco, G. Pereira, R.M. Ruscitti, P. Giacomelli, R. Ragab, G. La Torre, F. Bartoloni, E. Del Giudice, E. Lomater, C. Emmi, G. Govoni, M. Maggio, M.C. Maier, A. Makowska, J. Ogunjimi, B. Sfikakis, P.P. Sfriso, P. Gaggiano, C. Iannone, F. Dagostin, M.A. Di Cola, I. Navarini, L. Ahmed Mahmoud, A.A. Cardinale, F. Riccucci, I. Paroli, M.P. Marucco, E.M. Mattioli, I. Sota, J. Abbruzzese, A. Antonelli, I.P.B. Cipriani, P. Tufan, A. Fabiani, C. Ramadan, M.M. Cattalini, M. Kardas, R.C. Sebastiani, G.D. Giardini, H.A.M. Hernández-Rodríguez, J. Mastrorilli, V. Więsik-Szewczyk, E. Frassi, M. Caggiano, V. Telesca, S. Giordano, H.F. Guadalupi, E. Giani, T. Renieri, A. Colella, S. Cataldi, G. Gentile, M. Fabbiani, A. Al-Maghlouth, I.A. Frediani, B. Balistreri, A. Rigante, D. Cantarini, L. The Autoinflammatory Diseases Alliance (AIDA) Network

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. NCT 05200715 available at https://clinicaltrials.gov/. 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.

Published
2022

7. Schnitzler Syndrome in a 27-Year-Old Man: Diagnostic and Therapeutic Dilemma in Adult Auto-Inflammatory Syndromes A Case Report and Literature Review

Author

Więsik-Szewczyk E, Felis-Giemza A, Dziuk M, and Jahnz-Różyk K

Subjects
tocilizumab, lcsh:R5-920, monoclonal gammopathy, autoinflammation, lcsh:Medicine (General), chronic urticaria, anakinra
Abstract

Ewa Więsik-Szewczyk,1 Anna Felis-Giemza,2 Mirosław Dziuk,3 Karina Jahnz-Różyk1 1Department of Internal Medicine, Pulmonology, Allergy and Clinical Immunology, Central Clinical Hospital of the Ministry of National Defense, Military Institute of Medicine in Warsaw, Warsaw, Poland; 2Department of Connective Tissue Diseases, National Institute of Geriatrics, Rheumatology and Rehabilitation, Warsaw, Poland; 3Department of Nuclear Medicine, Military Institute of Medicine in Warsaw, Warsaw, PolandCorrespondence: Ewa Więsik-SzewczykDepartment of Internal Medicine, Pulmonology, Allergy and Clinical Immunology, Military Institute of Medicine in Warsaw, Szaserów 128 01-141, Warsaw, PolandTel/Fax +48 261 816 581Email ewa.w.szewczyk@gmail.comAbstract: A 32-year-old-man, with a history of chronic urticaria from the age of 27, diagnosed with an adult-onset Still’s disease and received a low dose of glucocorticoids, methotrexate and tocilizumab. Despite the long-term combined treatments, he suffered from chronic urticaria, low-grade fever and bone pain. He was found to have high inflammatory markers, hypogammaglobulinemia, monoclonal IgM – kappa light chain in serum and increased radiotracer uptake in the whole bone scintigraphy. No pathological variants for monogenic autoinflammatory diseases were present in the genome exome sequencing. These investigations confirmed the diagnosis of Schnitzler syndrome, which is an exception before the age of 35. Switching from tocilizumab to interleukin 1 receptor inhibitor, anakinra led to a full clinical response and normalisation of inflammatory markers. Patients with a history of fever and chronic urticaria are routinely tested for monoclonal gammopathy in the context of malignancy, but it should also be considered as a sign of the autoinflammatory syndrome. The Schnitzler syndrome and the adult-onset Still’s disease share common features, so the diagnosis requires a thorough investigation to establish an optimal treatment. In the diagnostic algorithm, monoclonal gammopathy is usually considered red flag for malignancy but might be overlooked as a criterion of Schnitzler syndrome, particularly in young adults. We confirm that the interleukin 1 inhibitor should be the first line of therapy in Schnitzler syndrome, and in the presented case we found it more effective than the interleukin 6 blockade. The main goal of this paper is to increase awareness of Schnitzler syndrome among health care professionals. We aim to present features which can be helpful in differential diagnosis.Keywords: chronic urticaria, monoclonal gammopathy, tocilizumab, anakinra, autoinflammation

Published
2020

8. B-cell targeted therapy in systemic lupus erythematosus: potential of rituximab

Author

Wiesik-Szewczyk E and Olesinska M

Subjects
Medicine (General), R5-920
Abstract

E Wiesik-Szewczyk, M OlesinskaInstitute of Rheumatology, Department of Connective Tissue Diseases, Warsaw, PolandAbstract: Systemic lupus erythematosus (SLE) is a complex autoimmune disease of unknown etiology, and the limited available therapeutic options for this disease, are frustrating to both clinicians and patients. However, recent advances in the understanding of disease mechanisms have given rise to numerous studies on specific approaches to SLE treatment. Rituximab, the first chimeric, mouse-human monoclonal antibody which is directed against CD20, seems to be a new therapeutic option. The purpose of this review is to explain the current clinical evidence on the therapeutic use of rituximab in adult SLE patients. Two randomized clinical trials with rituximab (the EXPLORER and LUNAR studies) failed to prove efficacy of this drug on SLE. Ongoing data analysis continues to explain the reasons behind why this treatment fails to work. However data from open source and observational studies contrast with clinical trials results. The global analysis of this data supports the off-label use of rituximab in subsets of SLE that are refractory to standard treatment.Keywords: B cells, systemic lupus erythematosus, rituximab, off-label use, clinical trials

Published
2012

12. Hypofractionated Radiotherapy with Sequential Anthracycline-based Chemotherapy in Marginally Resectable Soft Tissue Sarcomas: Final Results of a Phase II Clinical Trial

Author

Spalek, M., primary, Koseła-Paterczyk, H., additional, Borkowska, A., additional, Wągrodzki, M., additional, Szumera-Ciećkiewicz, A., additional, Czarnecka, A.M., additional, Castaneda-Wysocka, P., additional, Dąbrowska-Szewczyk, E., additional, Cieszanowski, A., additional, and Rutkowski, P., additional

Published
2020
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17. Update of EULAR recommendations for the treatment of systemic sclerosis

Author

Kowal-Bielecka O., Fransen J., Avouac J., Becker M., Kulak A., Allanore Y., Distler O., Clements P., Cutolo M., Czirjak L., Damjanov N., Del Galdo F., Denton C. P., Distler J. H. W., Foeldvari I., Figelstone K., Frerix M., Furst D. E., Guiducci S., Hunzelmann N., Khanna D., Matucci-Cerinic M., Herrick A. L., Van Den Hoogen F., Van Laar J. M., Riemekasten G., Silver R., Smith V., Sulli A., Tarner I., Tyndall A., Welling J., Wigley F., Valentini G., Walker U. A., Zulian F., Muller-Ladner U., Daikeler T., Lanciano E., Becvar R., Tomcik M., Gindzienska-Sieskiewicz E., Cuomo G., Iudici M., Rednic S., Vlachoyiannopoulos P. G., Caporali R., Carreira P. E., Novak S., Minier T., Kucharz E. J., Gabrielli A., Moroncini G., Airo' P., Hesselstrand R., Martinovic D., Radic M., Marasovic-Krstulovic D., Braun-Moscovici Y., Balbir-Gurman A., Lo Monaco A., Caramaschi P., Morovic-Vergles J., Henes J., Ortiz Santamaria V., Heitmann S., Krasowska D., Seidel M. F., Hasler P., Pereira Da Silva J. A., Salvador M. J., Stamenkovic B., Stankovic A., Tikly M., Ananieva L. P., Beretta L., Szucs G., Szamosi S., de la Puente Bujidos C., Midtvedt O., Hoffmann-Vold A. -M., Launay D., Hachulla E., Riccieri V., Ionescu R., Opris D., Mihai C., Herrgott I., Beyer C., Ingegnoli F., von Muhlen C. A., Alegre-Sancho J. J., Beltran-Catalan E., Aringer M., Fantana J., Leuchten N., Tausche A. -K., De Langhe E., Vanthuyne M., Anic B., Baresic M., Mayer M., Uprus M., Otsa K., Yavuz S., Granel B., Azevedo V. F., Muller C., Jimenez S. A., Popa S., Agachi S., Zenone T., Stebbings S., Dockerty J., Vacca A., Schollum J., Veale D. J., Toloza S., Xu D., Olas J., Rosato E., Foti R., Adler S., Dan D., Wiesik-Szewczyk E., Olesinska M., Kayser C., Fathi N., de la Pena Lefebvre P. G., Imbert B., Kowal-Bielecka, O., Fransen, J., Avouac, J., Becker, M., Kulak, A., Allanore, Y., Distler, O., Clements, P., Cutolo, M., Czirjak, L., Damjanov, N., Del Galdo, F., Denton, C. P., Distler, J. H. W., Foeldvari, I., Figelstone, K., Frerix, M., Furst, D. E., Guiducci, S., Hunzelmann, N., Khanna, D., Matucci-Cerinic, M., Herrick, A. L., Van Den Hoogen, F., Van Laar, J. M., Riemekasten, G., Silver, R., Smith, V., Sulli, A., Tarner, I., Tyndall, A., Welling, J., Wigley, F., Valentini, G., Walker, U. A., Zulian, F., Muller-Ladner, U., Daikeler, T., Lanciano, E., Becvar, R., Tomcik, M., Gindzienska-Sieskiewicz, E., Cuomo, G., Iudici, M., Rednic, S., Vlachoyiannopoulos, P. G., Caporali, R., Carreira, P. E., Novak, S., Minier, T., Kucharz, E. J., Gabrielli, A., Moroncini, G., Airo', P., Hesselstrand, R., Martinovic, D., Radic, M., Marasovic-Krstulovic, D., Braun-Moscovici, Y., Balbir-Gurman, A., Lo Monaco, A., Caramaschi, P., Morovic-Vergles, J., Henes, J., Ortiz Santamaria, V., Heitmann, S., Krasowska, D., Seidel, M. F., Hasler, P., Pereira Da Silva, J. A., Salvador, M. J., Stamenkovic, B., Stankovic, A., Tikly, M., Ananieva, L. P., Beretta, L., Szucs, G., Szamosi, S., de la Puente Bujidos, C., Midtvedt, O., Hoffmann-Vold, A. -M., Launay, D., Hachulla, E., Riccieri, V., Ionescu, R., Opris, D., Mihai, C., Herrgott, I., Beyer, C., Ingegnoli, F., von Muhlen, C. A., Alegre-Sancho, J. J., Beltran-Catalan, E., Aringer, M., Fantana, J., Leuchten, N., Tausche, A. -K., De Langhe, E., Vanthuyne, M., Anic, B., Baresic, M., Mayer, M., Uprus, M., Otsa, K., Yavuz, S., Granel, B., Azevedo, V. F., Muller, C., Jimenez, S. A., Popa, S., Agachi, S., Zenone, T., Stebbings, S., Dockerty, J., Vacca, A., Schollum, J., Veale, D. J., Toloza, S., Xu, D., Olas, J., Rosato, E., Foti, R., Adler, S., Dan, D., Wiesik-Szewczyk, E., Olesinska, M., Kayser, C., Fathi, N., de la Pena Lefebvre, P. G., Imbert, B., UCL - SSS/IREC/SLUC - Pôle St.-Luc, UCL - (MGD) Service de rhumatologie, Kowal Bielecka, Otylia, Fransen, Jaap, Avouac, Jerome, Becker, Mike, Kulak, Agnieszka, Allanore, Yannick, Distler, Oliver, Clements, Philip, Cutolo, Maurizio, Czirjak, Laszlo, Damjanov, Nemanja, del Galdo, Francesco, Denton, Christopher P., Distler, Jörg H. W., Foeldvari, Ivan, Figelstone, Kim, Frerix, Marc, Furst, Daniel E., Guiducci, Serena, Hunzelmann, Nicola, Khanna, Dinesh, Matucci Cerinic, Marco, Herrick, Ariane L., van den Hoogen, Frank, van Laar, Jacob M., Riemekasten, Gabriela, Silver, Richard, Smith, Vanessa, Sulli, Alberto, Tarner, Ingo, Tyndall, Alan, Welling, Joep, Wigley, Frederic, Valentini, Gabriele, Walker, Ulrich A., Zulian, Francesco, Müller Ladner, Ulf, Daikeler, Thoma, Lanciano, Elisabetta, Becvã¡r, Radim, Tomcik, Michal, Gindzienska Sieskiewicz, Ewa, Iudici, Michele, Rednic, Simona, Vlachoyiannopoulos, Panayiotis G., Caporali, Roberto, Carreira, Patricia E., Novak, Srdan, Minier, Tã¼nde, Kucharz, Eugene J., Gabrielli, Armando, Moroncini, Gianluca, Airo, Paolo, Hesselstrand, Roger, Martinovic, Duska, Radic, Mislav, Marasovic Krstulovic, Daniela, Braun Moscovici, Yolanda, Monaco, Andrea Lo, Morovic Vergles, Jadranka, Culo, Melanie I., Henes, Jã¶rg, Santamaria, Vera Ortiz, Heitmann, Stefan, Krasowska, Dorota, Michalska Jakubus, Malgorzata, Seidel, Matthias F., Klinik III, Medizinische, Hasler, Paul, Da Silva, José A. Pereira, Salvador, Maria J., Stamenkovic, Bojana, Stankovic, Aleksandra, Tikly, Mohammed, Ananieva, Lidia P., Beretta, Lorenzo, Szucs, Gabriella, Szamosi, Szilvia, de la Puente Bujidos, Carlo, Midtvedt, Øyvind, Hoffmann Vold, Anna Maria, Launay, David, Hachulla, Eric, Riccieri, Valeria, Ionescu, Ruxandra, Opris, Daniela, Mihai, Carina, Herrgott, Ilka, Beyer, Christian, Ingegnoli, Francesca, von Mühlen, Carlos Alberto, Alegre Sancho, Juan José, Beltran Catalan, Emma, Aringer, Martin, Fantana, Julia, Leuchten, Nicolai, Tausche, Anne Kathrin, Langhe, Ellen De, Vanthuyne, Marie, Anic, Branimir, Bareå¡ic, Marko, Mayer, Miroslav, Ãœprus, Maria, Otsa, Kati, Yavuz, Sule, Granel, Brigitte, Jimenez, Sergio A., Popa, Serghei, Agachi, Svetlana, Zenone, Thierry, Stebbings, Simon, Dockerty, Joanne, Vacca, Alessandra, Schollum, Joanna, Veale, Douglas J., Toloza, Sergio, Xu, Dong, Olas, Jacek, Rosato, Edoardo, Foti, Rosario, Adler, Sabine, Dan, Diana, Wiesik Szewczyk, Ewa, Olesinska, Marzena, Kayser, Cristiane, Fathi, Nihal, de la Peña Lefebvre, Paloma García, Imbert, Bernard, and Cuomo, Giovanna

Subjects
Endothelin Receptor Antagonists, Lung Diseases, Kidney Disease, Delphi Technique, Gastrointestinal Diseases, systemic sclerosis, Scleroderma Renal Crisis, Placebo-controlled study, Angiotensin-Converting Enzyme Inhibitors, Lung Disease, Scleroderma, 0302 clinical medicine, Glucocorticoid, Phosphodiesterase 5 Inhibitor, Immunology and Allergy, skin and connective tissue diseases, BIOMEDICINA I ZDRAVSTVO. Kliničke medicinske znanosti. Interna medicina, integumentary system, treatment, genetics and molecular biology (all), Hematopoietic Stem Cell Transplantation, cyclophosphamide, methotrexate, Pulmonary, Orvostudományok, Serotonin Uptake Inhibitor, 3. Good health, Europe, Systematic review, Hypertension, Serotonin Uptake Inhibitors, Cyclophosphamide, Methotrexate, Systemic Sclerosis, Treatment, Fingers, Fluoxetine, Glucocorticoids, Humans, Hypertension, Pulmonary, Kidney Diseases, Phosphodiesterase 5 Inhibitors, Prostaglandins I, Pyrazoles, Pyrimidines, Raynaud Disease, Rheumatology, Scleroderma, Systemic, Ulcer, Immunology, Biochemistry, Genetics and Molecular Biology (all), 030211 gastroenterology & hepatology, Endothelin Receptor Antagonist, Selective Serotonin Reuptake Inhibitors, medicine.drug, Human, medicine.medical_specialty, Gastrointestinal Disease, Klinikai orvostudományok, Riociguat, General Biochemistry, Genetics and Molecular Biology, 03 medical and health sciences, medicine, Finger, biochemistry, Intensive care medicine, BIOMEDICINE AND HEALTHCARE. Clinical Medical Sciences. Internal Medicine, Systemic Sclerosi, 030203 arthritis & rheumatology, business.industry, Systemic, Angiotensin-Converting Enzyme Inhibitor, medicine.disease, Transplantation, Clinical research, Pyrimidine, immunology and allergy, rheumatology, immunology, Pyrazole, Physical therapy, business, Rheumatism
Abstract

The aim was to update the 2009 European League against Rheumatism (EULAR) recommendations for the treatment of systemic sclerosis (SSc), with attention to new therapeutic questions. Update of the previous treatment recommendations was performed according to EULAR standard operating procedures. The task force consisted of 32 SSc clinical experts from Europe and the USA, 2 patients nominated by the pan-European patient association for SSc (Federation of European Scleroderma Associations (FESCA)), a clinical epidemiologist and 2 research fellows. All centres from the EULAR Scleroderma Trials and Research group were invited to submit and select clinical questions concerning SSc treatment using a Delphi approach. Accordingly, 46 clinical questions addressing 26 different interventions were selected for systematic literature review. The new recommendations were based on the available evidence and developed in a consensus meeting with clinical experts and patients. The procedure resulted in 16 recommendations being developed (instead of 14 in 2009) that address treatment of several SSc-related organ complications: Raynaud's phenomenon (RP), digital ulcers (DUs), pulmonary arterial hypertension (PAH), skin and lung disease, scleroderma renal crisis and gastrointestinal involvement. Compared with the 2009 recommendations, the 2016 recommendations include phosphodiesterase type 5 (PDE-5) inhibitors for the treatment of SSc-related RP and DUs, riociguat, new aspects for endothelin receptor antagonists, prostacyclin analogues and PDE-5 inhibitors for SSc-related PAH. New recommendations regarding the use of fluoxetine for SSc-related RP and haematopoietic stem cell transplantation for selected patients with rapidly progressive SSc were also added. In addition, several comments regarding other treatments addressed in clinical questions and suggestions for the SSc research agenda were formulated. These updated data-derived and consensus-derived recommendations will help rheumatologists to manage patients with SSc in an evidence-based way. These recommendations also give directions for future clinical research in SSc.

Published
2017

18. OC-0069 5x5 Gy with chemotherapy in borderline resectable soft tissue sarcomas: early results of a trial

Author

Spalek, M., primary, Koseła-Paterczyk, H., additional, Borkowska, A., additional, Wągrodzki, M., additional, Szumera-Ciećkiewicz, A., additional, Cieszanowski, A., additional, Castaneda-Wysocka, P., additional, Świtaj, T., additional, Dudzisz-Śledź, M., additional, Czarnecka, A., additional, Dąbrowska-Szewczyk, E., additional, and Rutkowski, P., additional

Published
2019
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20. Preoperative hypofractionated radiotherapy (RT) combined with chemotherapy in primary marginally resectable high grade soft tissue sarcomas (STS) of extremities or trunk wall: Interim analysis of prospective phase II clinical trial

Author

Spalek, M., primary, Kosela Paterczyk, H.M., additional, Borkowska, A., additional, Wagrodzki, M., additional, Cieszanowski, A., additional, Castaneda-Wysocka, P., additional, Switaj, T., additional, Dudzisz-Sledz, M.E., additional, Czarnecka, A.M., additional, Dabrowska-Szewczyk, E., additional, and Rutkowski, P.L., additional

Published
2018
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21. Update of EULAR recommendations for the treatment of systemic sclerosis

Author

Kowal-Bielecka, O. Fransen, J. Avouac, J. Becker, M. Kulak, A. Allanore, Y. Distler, O. Clements, P. Cutolo, M. Czirjak, L. Damjanov, N. Del Galdo, F. Denton, C.P. Distler, J.H.W. Foeldvari, I. Figelstone, K. Frerix, M. Furst, D.E. Guiducci, S. Hunzelmann, N. Khanna, D. Matucci-Cerinic, M. Herrick, A.L. Van Den Hoogen, F. Van Laar, J.M. Riemekasten, G. Silver, R. Smith, V. Sulli, A. Tarner, I. Tyndall, A. Welling, J. Wigley, F. Valentini, G. Walker, U.A. Zulian, F. Müller-Ladner, U. EUSTAR Coauthors Daikeler, T. Lanciano, E. Becvár, R. Tomcik, M. Gińdzieńska-Sieskiewicz, E. Cuomo, G. Iudici, M. Rednic, S. Vlachoyiannopoulos, P.G. Caporali, R. Carreira, P.E. Novak, S. Minier, T. Kucharz, E.J. Gabrielli, A. Moroncini, G. Airo', P. Hesselstrand, R. Martinovic, D. Radic, M. Marasovic-Krstulovic, D. Braun-Moscovici, Y. Balbir-Gurman, A. Lo Monaco, A. Caramaschi, P. Morovic-Vergles, J. Henes, J. Ortiz Santamaria, V. Heitmann, S. Krasowska, D. Seidel, M.F. Hasler, P. Pereira Da Silva, J.A. Salvador, M.J. Stamenkovic, B. Stankovic, A. Tikly, M. Ananieva, L.P. Beretta, L. Szucs, G. Szamosi, S. de la Puente Bujidos, C. Midtvedt, Ø. Hoffmann-Vold, A.-M. Launay, D. Hachulla, E. Riccieri, V. Ionescu, R. Opris, D. Mihai, C. Herrgott, I. Beyer, C. Ingegnoli, F. von Mühlen, C.A. Alegre-Sancho, J.J. Beltrán-Catalán, E. Aringer, M. Fantana, J. Leuchten, N. Tausche, A.-K. De Langhe, E. Vanthuyne, M. Anic, B. Barešic, M. Mayer, M. Üprus, M. Otsa, K. Yavuz, S. Granel, B. Azevedo, V.F. Muller, C. Jimenez, S.A. Popa, S. Agachi, S. Zenone, T. Stebbings, S. Dockerty, J. Vacca, A. Schollum, J. Veale, D.J. Toloza, S. Xu, D. Olas, J. Rosato, E. Foti, R. Adler, S. Dan, D. Wiesik-Szewczyk, E. Olesińska, M. Kayser, C. Fathi, N. de la Peña Lefebvre, P.G. Imbert, B.

Subjects
integumentary system, skin and connective tissue diseases
Abstract

The aim was to update the 2009 European League against Rheumatism (EULAR) recommendations for the treatment of systemic sclerosis (SSc), with attention to new therapeutic questions. Update of the previous treatment recommendations was performed according to EULAR standard operating procedures. The task force consisted of 32 SSc clinical experts from Europe and the USA, 2 patients nominated by the pan-European patient association for SSc (Federation of European Scleroderma Associations (FESCA)), a clinical epidemiologist and 2 research fellows. All centres from the EULAR Scleroderma Trials and Research group were invited to submit and select clinical questions concerning SSc treatment using a Delphi approach. Accordingly, 46 clinical questions addressing 26 different interventions were selected for systematic literature review. The new recommendations were based on the available evidence and developed in a consensus meeting with clinical experts and patients. The procedure resulted in 16 recommendations being developed (instead of 14 in 2009) that address treatment of several SSc-related organ complications: Raynaud's phenomenon (RP), digital ulcers (DUs), pulmonary arterial hypertension (PAH), skin and lung disease, scleroderma renal crisis and gastrointestinal involvement. Compared with the 2009 recommendations, the 2016 recommendations include phosphodiesterase type 5 (PDE-5) inhibitors for the treatment of SSc-related RP and DUs, riociguat, new aspects for endothelin receptor antagonists, prostacyclin analogues and PDE-5 inhibitors for SSc-related PAH. New recommendations regarding the use of fluoxetine for SSc-related RP and haematopoietic stem cell transplantation for selected patients with rapidly progressive SSc were also added. In addition, several comments regarding other treatments addressed in clinical questions and suggestions for the SSc research agenda were formulated. These updated data-derived and consensus-derived recommendations will help rheumatologists to manage patients with SSc in an evidence-based way. These recommendations also give directions for future clinical research in SSc. © Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/.

Published
2017

24. The Effect of Fragmentation and Packaging of Dried Parsley Leaves on Selected Chemical and Microbiological Parameters

Author

Sikorska-Zimny Kalina, Wójcicka Karolina, Rochmińska Anita, Rutkowski Krzysztof P., Szewczyk Eligia M., and Lisiecki Paweł

Subjects
parsley leaves, drying, microbial analysis, chemical parameters, storage, Plant culture, SB1-1110
Abstract

Parsley leaves (Petroselinum crispum) have long been known for their organoleptic properties. They are widely used in cuisine all over the world in fresh and dried form and also as pharmaceutical raw material. The presented work assessed if the storage of parsley leaves (as whole leaves or leave pieces) and packaging with PE or Xtend® foils influence the content of selected chemical compounds and the microbiological quality of the product. For this purpose, the leaves were dried, packaged and analyzed after 3 weeks’ storage under room temperature. Neither the degree of fragmentation nor the type of packaging foil affected the content of vit. C and total sugars. Higher content of reducing sugars was obtained in the samples packed in PE foil. Number of detected bacteria did not exceed the safety border. Less bacterial colonies were detected in the material packed as fragmented in the PE foil. No fungal colonies were detected in the leaves packed in the Xtend® foil.

Published
2019
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25. AB0819 Clinical course and nailfold capillaroscopy patterns of 69 patients with mixed connective tissue disease (MCTD)

Author

Olesinska, M., primary, Czuszynska, Z., additional, Felis-Giemza, A., additional, Walkiewicz-Pielaszek, K., additional, Wiesik-Szewczyk, E., additional, Wierzba, K., additional, Paradowska-Gorycka, A., additional, Rutkowska-Sak, L., additional, Szczygielska, I., additional, Zabek, J., additional, and Zdrojewski, Z., additional

Published
2013
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28. AB0820 Physical disability in mixed connective tissue disease

Author

Olesinska, M., primary, Czuszynska, Z., additional, Felis-Giemza, A., additional, Walkiewicz-Pielaszek, K., additional, Paradowska-Gorycka, A., additional, Rutkowska-Sak, L., additional, Szczygielska, I., additional, Wiesik-Szewczyk, E., additional, and Zdrojewski, Z., additional

Published
2013
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35. Plasmids of Staphylococcus cohnii isolated from the intensive-care unit.

Author

Szewczyk, E., Różalska, M., Cieślikowski, T., and Nowak, T.

Abstract

Numerous isolates of both subspecies of Staphylococcus cohnii were found in the environment of the intensive-care unit of a pediatric hospital. These isolates carried in their cells many plasmids, up to fourteen, of a wide range of sizes (<2 to >56 kb). Striking was the occurrence of large plasmids not very common in staphylococci. These were present in >80 % of S. cohnii isolates. Fifty-two different plasmid profiles were found in 79 investigated isolates belonging to S. cohnii ssp. cohnii and S. cohnii ssp. urealyticus. Isolates similar in plasmid profiles were grouped in antibiotic-resistance clusters established for 9 antibiotics (gentamicin, ciprofloxacin, clindamycin, erythromycin, tetracycline, chloramphenicol, mupirocin, trimethoprim-sulfamethoxazole, vancomycin) using the method of unweighted pair group mathematical averages (UPGMA). Many isolates were multiresistant to antibiotics and produced bacteriocins. [ABSTRACT FROM AUTHOR]

Published
2004
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36. Evaluation of selected features of Staphylococcus cohnii enabling colonization of humans.

Author

Waldon, E., Sobiś-Glinkowska, M., and Szewczyk, E.

Abstract

Based on iron utilization, sensitivity to skin fatty acids, lipolytic and proteolytic activity the potential abilities of Staphylococcus cohnii strains to colonize humans were evaluated. The investigation included 60 strains that belong to both subspecies, viz. S. cohnii ssp. cohnii and S. cohnii ssp. urealyticus. Strains were isolated from different sources of the Intensive Care Unit and from non-hospital environment. Most of the strains were multiple antibiotic-resistant. Strains of both subspecies revealed a relatively low iron requirement. These strains were capable of utilizing iron bound in oxo acids and from host iron-binding proteins. S. cohnii ssp. urealyticus were more effective in iron uptake than S. cohnii ssp. cohnii. All investigated strains revealed sensitivity to skin fatty acids, but S. cohnii ssp. urealyticus strains were more resistant. Special features of strains of this subspecies promote colonization of humans. [ABSTRACT FROM AUTHOR]

Published
2002
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45. Initial presenting manifestations in 16,486 patients with inborn errors of immunity include infections and noninfectious manifestations

Author

Tim Niehues, Catherine Waruiru, Conleth Feighery, Uwe Schauer, Virginie Courteille, Kai Lehmberg, Ingo Müller, I. Esteves, Henner Morbach, Michael Borte, Patrick Hundsdoerfer, Klaus Schwarz, Ewelina Gowin, Alessandro Aiuti, Andreas Holbro, Federica Barzaghi, João Farela Neves, Dagmar Graf, Hannah Tamary, Veneta Milenova, Benedikt Boetticher, Eleonora Gambineri, Vera Goda, Alia Eldash, Jan-Christian Wasmuth, Fabio Candotti, Svetlana O. Sharapova, Markus Metzler, Juergen Brunner, Anna Hilfanova, Brindusa Ruxandra Capilna, Pere Soler-Palacín, Arnau Antolí, Horst von Bernuth, Vassilios Lougaris, Maria Carrabba, Bernd H. Belohradsky, Julian Thalhammer, Nathalie de Vergnes, Peter Olbrich, Peter Kopač, Leif G. Hanitsch, Alexandra Nieters, Filomeen Haerynck, Juliana Gabzdilova, Sezin Aydemir, Rabab El Hawary, Patrick F.K. Yong, Maria Giovanna Danieli, Alberto Tommasini, Sandra Steinmann, Ulrich Baumann, Figen Dogu, Elisabeth Förster-Waldl, Carolina Marasco, Donato Amodio, Lorenzo Lodi, Xavier Solanich, Caterina Cancrini, Brigita Sitkauskiene, Torsten Witte, Clementina Vanessa, Nima Rezaei, Jean-Christophe Goffard, Kirsten Wittke, Emmanouil Liatsis, Helen Baxendale, Susana L. Silva, Bodo Grimbacher, Henrike Ritterbusch, Evangelia Farmaki, Safa Meshaal, Sujal Ghosh, Larysa Kostyuchenko, David Edgar, Simone Cesaro, R Zeuner, Nerea Salmón Rodríguez, Isabella Quinti, Stephan Ehl, Pauline Brosselin, Joerg C. Henes, Pilar Llobet Agulló, Rosa Maria Dellepiane, Andrea Meinhardt, Marina Kojić, Georgios Sogkas, Stephan Borte, Catharina Schuetz, Suheyla Ocak, Karin Marschall, Lukas M. Gasteiger, Stefan Raffac, Sofia Tantou, Sadia Noorani, Matthaios Speletas, Philippe Randrianomenjanahary, Ursula Holzer, Ayca Kiykim, Johannes G. Liese, Angelo Vacca, Gisela Fecker, Ekrem Unal, Koen J. van Aerde, Alba Parra-Martínez, Kaan Boztug, Sophie Stiehler, Sybille Landwehr-Kenzel, Claudio Pignata, Jennifer Neubert, Janine Reichenbach, Shahnaz Parvin, Sarah Goddard, Andrea Schroll, Dirk Holzinger, Asghar Aghamohammadi, Hassan Abolhassani, Johannes Trück, Estela Paz-Artal, Shereen M. Reda, Anna Shcherbina, Maria Raptaki, Jaroslava Orosova, Beata Wolska-Kuśnierz, Tessa Kerre, Gerrit Ahrenstorf, Ben Zion Garty, Dirk Foell, Benjamin Becker, Ulrike F. Demel, Androniki Kapousouzi, Abraham Rutgers, Klaus Warnatz, Gemma Rocamora Blanch, Stephan Rusch, Luis M. Allende, Dalia Abd Elaziz, Safa Baris, Jorisvan Montfrans, Dominik T. Schneider, Raphael Scheible, Juana Gil-Herrera, Gerhard Kindle, Annarosa Soresina, Giovanna Fabio, Uwe Wintergerst, Emilia Faria, Maria Fasshauer, Silvia Ricci, Aisha Elmarsafy, Barbara Pietrucha, Carsten Speckmann, Nizar Mahlaoui, Ulrich Heininger, Isabelle Meyts, Matthew Buckland, Efimia Papadopoulou-Alataki, Robin Kobbe, A Herwadkar, Sebastian F. N. Bode, Ali Sobh, László Maródi, Baldassarre Martire, Chiara Azzari, Maximilian Heeg, Katja Masjosthusmann, Michael H. Albert, Matteo Chinello, Juan Luis Santos-Pérez, Aarnoud Huissoon, Tanya I. Coulter, Hendrik Schulze-Koops, Norbert Graf, Radwa Alkady, Jolanta Bernatoniene, Seraina Prader, Alenka Gagro, Joachim Roesler, Taco W. Kuijpers, Ewa Więsik-Szewczyk, Maria Elena Maccari, Conrad Ferdinand Lippert, Miriam González-Amores, Johannes Dirks, Daniel E Pleguezuelo, Christof M. Kramm, Anders Fasth, Volker Schuster, Olov Ekwall, Nikolaus Rieber, Javier Carbone, Petra Kaiser-Labusch, Diana Ernst, Lucia Augusta Baselli, Luis Ignacio Gonzalez-Granado, Maria Kanariou, Stefanie S. V. Henriet, Sigune Goldacker, Kerstin Felgentreff, Oana Joean, Fine Roosens, Fabian Hauck, Eva C. Schwaneck, Milos Jesenak, Manfred Hoenig, Lenka Kapustova, Christoph Boesecke, Alain Fischer, Sara Pereira da Silva, Julia Körholz, Ansgar Schulz, Carolynne Schwarze-Zander, Mikko Seppänen, Nermeen Galal, Nora Naumann-Bartsch, Tomaz Garcez, Peter Ciznar, Klara M. Posfay-Barbe, Zelimir Pavle Eric, Reinhold E. Schmidt, Hermann J. Girschick, Sabine Heine, Anika-Kerstin Biegner, Annick A. J. M. van de Ven, Stefan Schreiber, J. Merlijn van den Berg, Nurit Assia Batzir, Alexandra Jablonka, Kim Stol, Gregor Dückers, Antonios G.A. Kolios, Ioannis Kakkas, Christian Klemann, Marina N. Guseva, Sofia Grigoriadou, Elif Karakoc-Aydiner, Antonio Marzollo, Peter D. Arkwright, Urs C. Steiner, Sara Sebnem Kilic, Romina Dieli-Crimi, Gergely Kriván, Monika Sparber-Sauer, Marco Cazzaniga, Fulvio Porta, Paraskevi Maggina, Tomas Milota, Robbert G. M. Bredius, Martine Pergent, Klaus Tenbrock, Jana Pachlopnik Schmid, Florentia Dimitriou, Cathal Laurence Steele, Helen Bourne, Anna Bobcakova, Gerd Horneff, Judith Potjewijd, Marc Schmalzing, Tobias Ankermann, Paul Ryan, Oksana Boyarchuk, Necil Kutukculer, Carl Friedrich Classen, Zita Chovancová, Moira Thomas, Cinzia Milito, Michaela Bitzenhofer-Grüber, Faranaz Atschekzei, Eva Hlaváčková, Viviana Moschese, Julie Smet, Hans-Hartmut Peter, Carla Teixeira, Sabine M El-Helou, Suzanne de Kruijf Bazen, Helmut Wittkowski, Donate Jakoby, Marina Garcia-Prat, Esther de Vries, Richard Herriot, Sven Kracker, Alessandro Plebani, Lisa Göschl, Laura Hora Marques, Anna Sediva, Jiri Litzman, Mark M. Gompels, Renate Krüger, Şefika İlknur Kökçü Karadağ, Nadine Binder, Anna Szaflarska, Peter Jandus, Lisa Ibberson, Johann Greil, Ulf Schulze-Sturm, Mehtap Sirin, Aydan Ikinciogullari, Edyta Heropolitańska-Pliszka, Michael E. Weiss, Alla Skapenko, Lukas Wisgrill, Hana Alachkar, Uta Behrends, Silvia Sánchez-Ramón, Maria N. Hatzistilianou, Otilia Petrovicova, Darko Richter, Zoreh Nademi, Jürgen K. Rockstroh, Sohilla Lotfy, Markus G. Seidel, Timothy Ronan Leahy, Audra Blažienė, Translational Immunology Groningen (TRIGR), Paediatric Infectious Diseases / Rheumatology / Immunology, AII - Inflammatory diseases, ARD - Amsterdam Reproduction and Development, University of Zurich, Ehl, Stephan, Thalhammer, J., Kindle, G., Nieters, A., Rusch, S., Seppanen, M. R. J., Fischer, A., Grimbacher, B., Edgar, D., Buckland, M., Mahlaoui, N., Ehl, S., Boztug, K., Brunner, J., Demel, U. F., Forster-Waldl, E., Gasteiger, L. M., Goschl, L., Kojic, M., Schroll, A., Seidel, M. G., Wintergerst, U., Wisgrill, L., Sharapova, S. O., Goffard, J. -C., Kerre, T., Meyts, I., Roosens, F., Smet, J., Haerynck, F., Eric, Z. P., Milenova, V., Gagro, A., Richter, D., Chovancova, Z., Hlavackova, E., Litzman, J., Milota, T., Sediva, A., Elaziz, D. A., Alkady, R. S., El Sayed El Hawary, R., Eldash, A. S., Galal, N., Lotfy, S., Meshaal, S. S., Reda, S. M., Sobh, A., Elmarsafy, A., Brosselin, P., Courteille, V., De Vergnes, N., Kracker, S., Pergent, M., Randrianomenjanahary, P., Ahrenstorf, G., Albert, M. H., Ankermann, T., Atschekzei, F., Baumann, U., Becker, B. C., Behrends, U., Belohradsky, B. H., Biegner, A. -K., Binder, N., Bode, S. F. N., Boesecke, C., Boetticher, B., Borte, M., Borte, S., Classen, C. F., Dirks, J., Duckers, G., El-Helou, S., Ernst, D., Fasshauer, M., Fecker, G., Felgentreff, K., Foell, D., Ghosh, S., Girschick, H. J., Goldacker, S., Graf, N., Graf, D., Greil, J., Hanitsch, L. G., Hauck, F., Heeg, M., Heine, S. I., Henes, J. C., Hoenig, M., Holzer, U., Holzinger, D., Horneff, G., Hundsdoerfer, P., Jablonka, A., Jakoby, D., Joean, O., Kaiser-Labusch, P., Klemann, C., Kobbe, R., Korholz, J., Kramm, C. M., Kruger, R., Landwehr-Kenzel, S., Lehmberg, K., Liese, J. G., Lippert, C. F., Maccari, M. E., Masjosthusmann, K., Meinhardt, A., Metzler, M., Morbach, H., Muller, I., Naumann-Bartsch, N., Neubert, J., Niehues, T., Peter, H. -H., Rieber, N., Ritterbusch, H., Rockstroh, J. K., Roesler, J., Schauer, U., Scheible, R., Schmalzing, M., Schmidt, R. E., Schneider, D. T., Schreiber, S., Schuetz, C., Schulz, A., Schulze-Koops, H., Schulze-Sturm, U., Schuster, V., Schwaneck, E. C., Schwarz, K., Schwarze-Zander, C., Sirin, M., Skapenko, A., Sogkas, G., Sparber-Sauer, M., Speckmann, C., Steinmann, S., Stiehler, S., Tenbrock, K., von Bernuth, H., Warnatz, K., Wasmuth, J. -C., Weiss, M., Witte, T., Wittke, K., Wittkowski, H., Zeuner, R. A., Farmaki, E., Hatzistilianou, M. N., Kakkas, I., Kanariou, M. G., Kapousouzi, A., Liatsis, E., Maggina, P., Papadopoulou-Alataki, E., Raptaki, M., Speletas, M., Tantou, S., Goda, V., Krivan, G., Marodi, L., Abolhassani, H., Aghamohammadi, A., Rezaei, N., Feighery, C., Leahy, T. R., Ryan, P., Batzir, N. A., Garty, B. Z., Tamary, H., Aiuti, A., Amodio, D., Azzari, C., Barzaghi, F., Baselli, L. A., Cancrini, C., Carrabba, M., Cazzaniga, M., Cesaro, S., Chinello, M., Danieli, M. G., Dellepiane, R. M., Fabio, G., Gambineri, E., Lodi, L., Lougaris, V., Marasco, C., Martire, B., Marzollo, A., Milito, C., Moschese, V., Pignata, C., Plebani, A., Porta, F., Quinti, I., Ricci, S., Soresina, A., Tommasini, A., Vacca, A., Vanessa, C., Blaziene, A., Sitkauskiene, B., Gowin, E., Heropolitanska-Pliszka, E., Pietrucha, B., Szaflarska, A., Wiesik-Szewczyk, E., Wolska-Kusnierz, B., Esteves, I., Faria, E., Marques, L. H., Neves, J. F., Silva, S. L., Teixeira, C., Pereira da Silva, S., Capilna, B. R., Guseva, M. N., Shcherbina, A., Bobcakova, A., Ciznar, P., Gabzdilova, J., Jesenak, M., Kapustova, L., Orosova, J., Petrovicova, O., Raffac, S., Kopac, P., Allende, L. M., Antoli, A., Blanch, G. R., Carbone, J., Dieli-Crimi, R., Garcia-Prat, M., Gil-Herrera, J., Gonzalez-Granado, L. I., Agullo, P. L., Olbrich, P., Parra-Martinez, A., Paz-Artal, E., Pleguezuelo, D. E., Rodriguez, N. S., Sanchez-Ramon, S., Santos-Perez, J. L., Solanich, X., Soler-Palacin, P., Gonzalez-Amores, M., Ekwall, O., Fasth, A., Bitzenhofer-Gruber, M., Candotti, F., Dimitriou, F., Heininger, U., Holbro, A., Jandus, P., Kolios, A. G. A., Marschall, K., Schmid, J. P., Posfay-Barbe, K. M., Prader, S., Reichenbach, J., Steiner, U. C., Truck, J., Bredius, R. G., de Kruijf- Bazen, S., de Vries, E., Henriet, S. S. V., Kuijpers, T. W., Potjewijd, J., Rutgers, A., Stol, K., van Aerde, K. J., Van den Berg, J. M., van de Ven, A. A. J. M., Montfrans, J., Aydemir, S., Baris, S., Dogu, F., Ikinciogullari, A., Karakoc-Aydiner, E., Kilic, S. S., Kiykim, A., Kokcu Karadag, S. I., Kutukculer, N., Ocak, S., Unal, E., Boyarchuk, O., Hilfanova, A., Kostyuchenko, L. V., Alachkar, H., Arkwright, P. D., Baxendale, H. E., Bernatoniene, J., Coulter, T. I., Garcez, T., Goddard, S., Gompels, M. M., Grigoriadou, S., Herriot, R., Herwadkar, A., Huissoon, A., Ibberson, L., Nademi, Z., Noorani, S., Parvin, S., Steele, C. L., Thomas, M., Waruiru, C., Yong, P. F. K., and Bourne, H.

Subjects
0301 basic medicine, Male, Pediatrics, syndromic, Sex Factor, Disease, registry, medicine.disease_cause, Cohort Studies, 0302 clinical medicine, Primary Immunodeficiency Disease, inborn error of immunity, Immunology and Allergy, warning signs, Age Factor, Registries, Family history, presenting symptom, Child, Primary immunodeficiency, Granuloma, autoimmune, immune dysregulation, inflammatory, Adult, Autoimmune Diseases, Female, Humans, Infections, Lymphoproliferative Disorders, Middle Aged, Primary Immunodeficiency Diseases, Sex Factors, Age Factors, 10177 Dermatology Clinic, Infections/epidemiology, 3. Good health, Settore MED/02, Warning signs, Lymphoproliferative Disorder, 2723 Immunology and Allergy, Infection, Human, medicine.medical_specialty, Immunology, 610 Medicine & health, Malignancy, primary immunodeficiency, Autoimmune Disease, 03 medical and health sciences, Immunity, Autoimmune Diseases/epidemiology, medicine, 2403 Immunology, business.industry, warning sign, Common variable immunodeficiency, Granuloma/epidemiology, Immune dysregulation, medicine.disease, Primary Immunodeficiency Diseases/epidemiology, 030104 developmental biology, Lymphoproliferative Disorders/epidemiology, Cohort Studie, business, 030215 immunology
Abstract

BACKGROUND: Inborn errors of immunity (IEI) are rare diseases, which makes diagnosis a challenge. A better description of the initial presenting manifestations should improve awareness and avoid diagnostic delay. Although increased infection susceptibility is a well-known initial IEI manifestation, less is known about the frequency of other presenting manifestations.OBJECTIVE: We sought to analyze age-related initial presenting manifestations of IEI including different IEI disease cohorts.METHODS: We analyzed data on 16,486 patients of the European Society for Immunodeficiencies Registry. Patients with autoinflammatory diseases were excluded because of the limited number registered.RESULTS: Overall, 68% of patients initially presented with infections only, 9% with immune dysregulation only, and 9% with a combination of both. Syndromic features were the presenting feature in 12%, 4% had laboratory abnormalities only, 1.5% were diagnosed because of family history only, and 0.8% presented with malignancy. Two-third of patients with IEI presented before the age of 6 years, but a quarter of patients developed initial symptoms only as adults. Immune dysregulation was most frequently recognized as an initial IEI manifestation between age 6 and 25 years, with male predominance until age 10 years, shifting to female predominance after age 40 years. Infections were most prevalent as a first manifestation in patients presenting after age 30 years.CONCLUSIONS: An exclusive focus on infection-centered warning signs would have missed around 25% of patients with IEI who initially present with other manifestations.

Published
2021

46. Efficacy and safety of tocilizumab in adult-onset Still's disease: Real-life experience from the international AIDA registry

Author

Jurgen Sota, Antonio Vitale, Giuseppe Lopalco, Rosa Maria R. Pereira, Heitor F. Giordano, Isabele P.B. Antonelli, Joanna Makowska, Olga Brzezińska, Anna Lewandowska-Polak, Piero Ruscitti, Paola Cipriani, Ilenia Di Cola, Marcello Govoni, Francesca Ruffili, Petros P. Sfikakis, Katerina Laskari, Gaafar Ragab, Mohamed A. Hussein, Stefano Gentileschi, Carla Gaggiano, Francesco La Torre, Armin Maier, Giacomo Emmi, Achille Marino, Francesco Ciccia, Paolo Sfriso, Maria Cristina Maggio, Elena Bartoloni, Claudia Lomater, Mohamed Tharwat Hegazy, Maria Tektonidou, Marília A. Dagostin, Aleksandra Opinc, Gian Domenico Sebastiani, Roberto Giacomelli, Emanuela Del Giudice, Alma Nunzia Olivieri, Abdurrahman Tufan, Riza Kan Kardas, Rossana Nuzzolese, Fabio Cardinale, Ewa Więsik-Szewczyk, Parretti Veronica, Maria Tarsia, Florenzo Iannone, Francesca Della Casa, Claudia Fabiani, Bruno Frediani, Alberto Balistreri, Donato Rigante, Luca Cantarini, Sota, Jurgen, Vitale, Antonio, Lopalco, Giuseppe, Pereira, Rosa Maria R, Giordano, Heitor F, Antonelli, Isabele P B, Makowska, Joanna, Brzezińska, Olga, Lewandowska-Polak, Anna, Ruscitti, Piero, Cipriani, Paola, Cola, Ilenia Di, Govoni, Marcello, Ruffili, Francesca, Sfikakis, Petros P, Laskari, Katerina, Ragab, Gaafar, Hussein, Mohamed A, Gentileschi, Stefano, Gaggiano, Carla, La Torre, Francesco, Maier, Armin, Emmi, Giacomo, Marino, Achille, Ciccia, Francesco, Sfriso, Paolo, Maggio, Maria Cristina, Bartoloni, Elena, Lomater, Claudia, Hegazy, Mohamed Tharwat, Tektonidou, Maria, Dagostin, Marília A, Opinc, Aleksandra, Sebastiani, Gian Domenico, Giacomelli, Roberto, Giudice, Emanuela Del, Olivieri, Alma Nunzia, Tufan, Abdurrahman, Kardas, Riza Kan, Nuzzolese, Rossana, Cardinale, Fabio, Więsik-Szewczyk, Ewa, Veronica, Parretti, Tarsia, Maria, Iannone, Florenzo, Della Casa, Francesca, Fabiani, Claudia, Frediani, Bruno, Balistreri, Alberto, Rigante, Donato, Cantarini, Luca, Sota J., Vitale A., Lopalco G., Pereira R.M.R., Giordano H.F., Antonelli I.P.B., Makowska J., Brzezinska O., Lewandowska-Polak A., Ruscitti P., Cipriani P., Cola I.D., Govoni M., Ruffili F., Sfikakis P.P., Laskari K., Ragab G., Hussein M.A., Gentileschi S., Gaggiano C., La Torre F., Maier A., Emmi G., Marino A., Ciccia F., Sfriso P., Maggio M.C., Bartoloni E., Lomater C., Hegazy M.T., Tektonidou M., Dagostin M.A., Opinc A., Sebastiani G.D., Giacomelli R., Giudice E.D., Olivieri A.N., Tufan A., Kardas R.K., Nuzzolese R., Cardinale F., Wiesik-Szewczyk E., Veronica P., Tarsia M., Iannone F., Della Casa F., Fabiani C., Frediani B., Balistreri A., Rigante D., and Cantarini L.

Subjects
Registrie, Adult, Male, Settore MED/16 - REUMATOLOGIA, Interleukin-6, Innovative biotechnologies, Tocilizumab, Adult-onset Still's disease, Antibodies, Monoclonal, Humanized, Personalized medicine, Settore MED/38 - Pediatria Generale E Specialistica, Anesthesiology and Pain Medicine, Rheumatology, Innovative biotechnologie, Still's disease, Humans, Female, Registries, Immunotherapy, Still's Disease, Adult-Onset, Human
Abstract

© 2022 Elsevier Inc.Background/objectives: Long-term efficacy and safety of tocilizumab (TCZ) in adult-onset Still's disease (AOSD) mostly derive from small case series. Herein we report a registry-based study investigating TCZ efficacy and safety in a cohort of patients with AOSD evaluated by clinical and serum inflammatory markers as well as drug retention rate analysis. Methods: This is an international multicentre study analyzing data from patients with AOSD regularly enrolled in the AIDA registry. TCZ efficacy was evaluated between baseline and last follow-up assessment in terms of changes in the Pouchot score and laboratory findings. Drug-retention rate was estimated by the Kaplan-Meier method, while Cox-regression analysis was employed to detect potential predictive factors of treatment withdrawal. Results: Data from 31 patients (15 men, 16 women) refractory to the conventional therapies and treated with TCZ were extracted from the AIDA registry. Mean ± SD time of treatment duration with TCZ was 24.32 ± 20.57 months. Median (IRQ) Pouchot score significantly decreased throughout the study period (p=0.001) with a significant difference between baseline [2.00 (4.00)] and 6 month-follow-up [0.00 (0.00)] (p=0.003) and between baseline and last follow-up assessment [0.00 (0.00)] (p=0.032), while no differences were observed between 6 month-evaluation and last follow-up assessment (p=0.823). Similarly, laboratory parameters significantly decreased from baseline to the last follow-up visit. With regard to drug survival, cumulative TCZ retention rate at 12-, 24-, and 36-month follow-up visit were 83.1%, 71.7% and 63.7%, respectively, without significant differences between biologic naïve patients and those previously treated with other biologics (p=0.329). Likewise, no significant differences were observed between chronic articular course of AOSD and other types of disease course (p=0.938) or between patients co-administered with conventional immunosuppressants and patients receiving TCZ as monotherapy (p=0.778). Cox-regression analysis identified no variable associated with a higher hazard of treatment withdrawal. Treatment was discontinued in 9 patients due to long-term remission (n=4), adverse events (n=2), loss of efficacy (n=1), non-medical reason (n=1) and unspecified cause (n=1). Mean glucocorticosteroids daily dose significantly decreased from baseline (18.36 ± 24.72 mg) to the last follow-up assessment (4.02 ± 4.99 mg, p=0.003). Conclusions: TCZ allows control of disease activity as well as normalization of serum inflammatory markers in both systemic and chronic articular form of AOSD. Additionally, TCZ displays an excellent drug retention rate while minimizing the risk of long-term exposure to corticosteroids.

Published
2022

47. Development and implementation of the AIDA International Registry for patients with Periodic Fever, Aphthous stomatitis, Pharyngitis, and cervical Adenitis syndrome

Author

Francesca Della Casa, Antonio Vitale, Marco Cattalini, Francesco La Torre, Giovanna Capozio, Emanuela Del Giudice, Maria Cristina Maggio, Giovanni Conti, Maria Alessio, Benson Ogunjimi, Gaafar Ragab, Giacomo Emmi, Emma Aragona, Teresa Giani, Giuseppe Lopalco, Paola Parronchi, Farhad Shahram, Elena Verrecchia, Francesca Ricci, Fabio Cardinale, Silvia Di Noi, Rossana Nuzzolese, Riccardo Lubrano, Serena Patroniti, Roberta Naddei, Vito Sabato, Mohamed A. Hussein, Laura Dotta, Violetta Mastrorilli, Stefano Gentileschi, Abdurrahman Tufan, Valeria Caggiano, Mohamed Tharwat Hegazy, Jurgen Sota, Ibrahim A. Almaghlouth, Amr Ibrahim, Ewa Wiȩsik-Szewczyk, Burcugul Ozkiziltas, Salvatore Grosso, Micol Frassi, Maria Tarsia, Rosa Maria R. Pereira, Maged Taymour, Carla Gaggiano, Sergio Colella, Claudia Fabiani, Maria Morrone, Piero Ruscitti, Bruno Frediani, Veronica Spedicato, Henrique A. Mayrink Giardini, Alberto Balistreri, Donato Rigante, Luca Cantarini, Della Casa, F, Vitale, A, Cattalini, M, La Torre, F, Capozio, G, Del Giudice, E, Maggio, Mc, Conti, G, Alessio, M, Ogunjimi, B, Ragab, G, Emmi, G, Aragona, E, Giani, T, Lopalco, G, Parronchi, P, Shahram, F, Verrecchia, E, Ricci, F, Cardinale, F, Di Noi, S, Nuzzolese, R, Lubrano, R, Patroniti, S, Naddei, R, Sabato, V, Hussein, Ma, Dotta, L, Mastrorilli, V, Gentileschi, S, Tufan, A, Caggiano, V, Hegazy, Mt, Sota, J, Almaghlouth, Ia, Ibrahim, A, Wiȩsik-Szewczyk, E, Ozkiziltas, B, Grosso, S, Frassi, M, Tarsia, M, Pereira, Rmr, Taymour, M, Gaggiano, C, Colella, S, Fabiani, C, Morrone, M, Ruscitti, P, Frediani, B, Spedicato, V, Giardini, Ham, Balistreri, A, Rigante, D, Cantarini, L., Della Casa, Francesca, Vitale, Antonio, Cattalini, Marco, La Torre, Francesco, Capozio, Giovanna, Del Giudice, Emanuela, 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, Di Noi, Silvia, 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, Wiȩsik-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, and Cantarini, Luca

Subjects
Registry, rare disease, PFAPA syndrome, autoinflammatory diseases, international registry, personalized medicine, precision medicine, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, autoinflammatory disease, Pediatrics, Perinatology and Child Health, Human medicine
Abstract

ObjectiveAim 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.MethodsThis 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.ResultsA 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.ConclusionsThe 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 https://clinicaltrials.gov NCT 05200715.

Published
2022

48. Development and implementation of the AIDA international registry for patients with Still's disease

Author

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, Autoinflammatory Diseases Alliance (AIDA) Network, Vitale A., Della Casa F., Lopalco G., Pereira R.M., Ruscitti P., Giacomelli R., Ragab G., La Torre F., Bartoloni E., Del Giudice E., Lomater C., Emmi G., Govoni M., Maggio M.C., Maier A., Makowska J., Ogunjimi B., Sfikakis P.P., Sfriso P., Gaggiano C., Iannone F., Dagostin M.A., Di Cola I., Navarini L., Ahmed Mahmoud A.A., Cardinale F., Riccucci I., Paroli M.P., Marucco E.M., Mattioli I., Sota J., Abbruzzese A., Antonelli I.P.B., Cipriani P., Tufan A., Fabiani C., Ramadan M.M., Cattalini M., Kardas R.C., Sebastiani G.D., Giardini H.A.M., Hernandez-Rodriguez J., Mastrorilli V., Wiesik-Szewczyk E., Frassi M., Caggiano V., Telesca S., Giordano H.F., Guadalupi E., Giani T., Renieri A., Colella S., Cataldi G., Gentile M., Fabbiani A., Al-Maghlouth I.A., Frediani B., Balistreri A., Rigante D., and Cantarini L.

Subjects
Registry, Settore MED/16 - REUMATOLOGIA, research, treatment, precision medicine, rare diseases, General Medicine, personalized medicine, autoinflammatory diseases, Settore MED/38 - Pediatria Generale E Specialistica, autoinflammatory diseases, personalized medicine, precision medicine, rare diseases, research treatment, Still's disease, Human medicine
Abstract

ObjectiveAim 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.MethodsThis 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.ResultsStarting 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.ConclusionsThis 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 https://clinicaltrials.gov/.

Published
2022

49. Development and Implementation of the AIDA International Registry for Patients With VEXAS Syndrome

Author

Antonio Vitale, Valeria Caggiano, Francesca Della Casa, José Hernández-Rodríguez, Micol Frassi, Sara Monti, Abdurrahman Tufan, Salvatore Telesca, Edoardo Conticini, Gaafar Ragab, Giuseppe Lopalco, Ibrahim Almaghlouth, Rosa Maria R. Pereira, Derya Yildirim, Marco Cattalini, Achille Marino, Teresa Giani, Francesco La Torre, Piero Ruscitti, Emma Aragona, Ewa Wiesik-Szewczyk, Emanuela Del Giudice, Petros P. Sfikakis, Marcello Govoni, Giacomo Emmi, Maria Cristina Maggio, Roberto Giacomelli, Francesco Ciccia, Giovanni Conti, Djouher Ait-Idir, Claudia Lomater, Vito Sabato, Matteo Piga, Ali Sahin, Daniela Opris-Belinski, Ruxandra Ionescu, Elena Bartoloni, Franco Franceschini, Paola Parronchi, Amato de Paulis, Gerard Espinosa, Armin Maier, Gian Domenico Sebastiani, Antonella Insalaco, Farhad Shahram, Paolo Sfriso, Francesca Minoia, Maria Alessio, Joanna Makowska, Gülen Hatemi, Nurullah Akkoç, Francesca Li Gobbi, Antonio Gidaro, Alma Nunzia Olivieri, Sulaiman M. Al-Mayouf, Sükran Erten, Stefano Gentileschi, Ibrahim Vasi, Maria Tarsia, Ayman Abdel-Monem Ahmed Mahmoud, Bruno Frediani, Musa Fares Alzahrani, Ahmed Hatem Laymouna, Francesca Ricci, Fabio Cardinale, Karina Jahnz-Rózyk, Gian Marco Tosi, Francesca Crisafulli, Alberto Balistreri, Marília A. Dagostin, Mahmoud Ghanema, Carla Gaggiano, Jurgen Sota, Ilenia Di Cola, Claudia Fabiani, Henrique A. Mayrink Giardini, Alessandra Renieri, Alessandra Fabbiani, Anna Carrer, Monica Bocchia, Federico Caroni, Donato Rigante, Luca Cantarini, Vitale, Antonio, Caggiano, Valeria, Della Casa, Francesca, Hernández-Rodríguez, José, 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, La Torre, Francesco, Ruscitti, Piero, Aragona, Emma, Wiesik-Szewczyk, Ewa, Del Giudice, Emanuela, 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, de Paulis, Amato, Espinosa, Gerard, Maier, Armin, Sebastiani, Gian Domenico, Insalaco, Antonella, Shahram, Farhad, Sfriso, Paolo, Minoia, Francesca, Alessio, Maria, Makowska, Joanna, Hatemi, Gülen, Akkoç, Nurullah, Li Gobbi, Francesca, Gidaro, Antonio, Olivieri, Alma Nunzia, Al-Mayouf, Sulaiman M, Erten, Sükran, Gentileschi, Stefano, Vasi, Ibrahim, Tarsia, Maria, Mahmoud, Ayman Abdel-Monem Ahmed, Frediani, Bruno, Fares Alzahrani, Musa, Laymouna, Ahmed Hatem, Ricci, Francesca, Cardinale, Fabio, Jahnz-Rózyk, Karina, Tosi, Gian Marco, Crisafulli, Francesca, Balistreri, Alberto, Dagostin, Marília A, Ghanema, Mahmoud, Gaggiano, Carla, Sota, Jurgen, Di Cola, Ilenia, Fabiani, Claudia, Giardini, Henrique A Mayrink, Renieri, Alessandra, Fabbiani, Alessandra, Carrer, Anna, Bocchia, Monica, Caroni, Federico, Rigante, Donato, Cantarini, Luca, Vitale, A, Caggiano, V, Della Casa, F, Hernández-Rodríguez, J, Frassi, M, Monti, S, Tufan, A, Telesca, S, Conticini, E, Ragab, G, Lopalco, G, Almaghlouth, I, Pereira, Rmr, Yildirim, D, Cattalini, M, Marino, A, Giani, T, La Torre, F, Ruscitti, P, Aragona, E, Wiesik-Szewczyk, E, Del Giudice, E, Sfikakis, Pp, Govoni, M, Emmi, G, Maggio, Mc, Giacomelli, R, Ciccia, F, Conti, G, Ait-Idir, D, Lomater, C, Sabato, V, Piga, M, Sahin, A, Opris-Belinski, D, Ionescu, R, Bartoloni, E, Franceschini, F, Parronchi, P, de Paulis, A, Espinosa, G, Maier, A, Sebastiani, Gd, Insalaco, A, Shahram, F, Sfriso, P, Minoia, F, Alessio, M, Makowska, J, Hatemi, G, Akkoç, N, Li Gobbi, F, Gidaro, A, Olivieri, An, Al-Mayouf, Sm, Erten, S, Gentileschi, S, Vasi, I, Tarsia, M, Mahmoud, Aaa, Frediani, B, Fares Alzahrani, M, Laymouna, Ah, Ricci, F, Cardinale, F, Jahnz-Rózyk, K, Tosi, Gm, Crisafulli, F, Balistreri, A, Dagostin, Ma, Ghanema, M, Gaggiano, C, Sota, J, Di Cola, I, Fabiani, C, Giardini, Ham, Renieri, A, Fabbiani, A, Carrer, A, Bocchia, M, Caroni, F, Rigante, D, and Cantarini, L.

Subjects
Registry, Keywords: autoinflammatory diseases, clinical management, precision medicine, rare diseases, research, treatment, Settore MED/16 - REUMATOLOGIA, rare disease, General Medicine, autoinflammatory diseases, Settore MED/38 - Pediatria Generale E Specialistica, autoinflammatory disease, VEXAS syndrome, Human medicine
Abstract

ObjectiveThe 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.MethodsThis 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.ResultsTo date (April 22nd, 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.ConclusionThis 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. This Project can be found on https://clinicaltrials.gov NCT05200715.

Published
2022

50. The Systemic Score May Identify Life-Threatening Evolution in Still Disease: Data from the GIRRCS AOSD-Study Group and the AIDA Network Still Disease Registry.

Author

Ruscitti P, Masedu F, Vitale A, Caggiano V, Di Cola I, Cipriani P, Valenti M, Mayrink Giardini HA, de Brito Antonelli IP, Dagostin MA, Lopalco G, Iannone F, Maria M, Almaghlouth IA, Asfina KN, Ali HH, Ciccia F, Iacono D, Pantano I, Mauro D, Sfikakis PP, Tektonidou M, Laskari K, Berardicurti O, Dagna L, Tomelleri A, Tufan A, Can Kardas R, Hinojosa-Azaola A, Martín-Nares E, Kawakami-Campos PA, Ragab G, Hegazy MT, Direskeneli H, Alibaz-Oner F, Fotis L, Sfriso P, Govoni M, La Torre F, Cristina Maggio M, Montecucco C, De Stefano L, Bugatti S, Rossi S, Makowska J, Del Giudice E, Emmi G, Bartoloni E, Hernández-Rodríguez J, Conti G, Nunzia Olivieri A, Lo Gullo A, Simonini G, Viapiana O, Wiesik-Szewczyk E, Erten S, Carubbi F, De Paulis A, Maier A, Tharwat S, Costi S, Iagnocco A, Sebastiani GD, Gidaro A, Brucato AL, Karamanakos A, Akkoç N, Caso F, Costa L, Prete M, Perosa F, Atzeni F, Guggino G, Fabiani C, Frediani B, Giacomelli R, and Cantarini L

Subjects
Humans, Male, Female, Adult, Prospective Studies, Middle Aged, Young Adult, Severity of Illness Index, Prognosis, Disease Progression, Macrophage Activation Syndrome diagnosis, Registries, Still's Disease, Adult-Onset diagnosis, Still's Disease, Adult-Onset complications
Abstract

Objective: We aimed to evaluate the clinical usefulness of the systemic score in the prediction of life-threatening evolution in Still disease. We also aimed to assess the clinical relevance of each component of the systemic score in predicting life-threatening evolution and to derive patient subsets accordingly., Methods: A multicenter, observational, prospective study was designed including patients included in the Gruppo Italiano Di Ricerca in Reumatologia Clinica e Sperimentale Adult-Onset Still Disease Study Group and the Autoinflammatory Disease Alliance Network Still Disease Registry. Patients were assessed to see if the variables to derive the systemic score were available. The life-threatening evolution was defined as mortality, whatever the clinical course, and/or macrophage activation syndrome, a secondary hemophagocytic lymphohistiocytosis associated with a poor prognosis., Results: A total of 597 patients with Still disease were assessed (mean ± SD age 36.6 ± 17.3 years; male 44.4%). The systemic score, assessed as a continuous variable, significantly predicted the life-threatening evolution (odds ratio [OR] 1.24; 95% confidence interval [CI] 1.07-1.42; P = 0.004). A systemic score ≥7 also significantly predicted the likelihood of a patient experiencing life-threatening evolution (OR 3.36; 95% CI 1.81-6.25; P < 0.001). Assessing the clinical relevance of each component of the systemic score, liver involvement (OR 1.68; 95% CI 1.48-2.67; P = 0.031) and lung disease (OR 2.12; 95% CI 1.14-4.49; P = 0.042) both significantly predicted life-threatening evolution. The clinical characteristics of patients with liver involvement and lung disease were derived, highlighting their relevance in multiorgan disease manifestations., Conclusion: The clinical utility of the systemic score was shown in identifying Still disease at a higher risk of life-threatening evolution in a large cohort. Furthermore, the clinical relevance of liver involvement and lung disease was highlighted., (© 2024 American College of Rheumatology.)

Published
2024
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