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1. AB1697 INCREASED INCIDENCE OF ADVERSE EVENTS AND EVENTS OF SPECIAL INTEREST WITH TREATMENT INTENSIFICATION IN NON-SYSTEMIC JUVENILE IDIOPATHIC ARTHRITIS

Author

Miettunen, P., primary, Rebollo Gimenez, A., additional, Carlini, L., additional, Pistorio, A., additional, Panaviene, V., additional, Anton, J., additional, Kamphuis, S., additional, Herlin, T., additional, Dolezalova, P., additional, Cattalini, M., additional, Susic, G., additional, Sanner, H., additional, Maggio, M. C., additional, Hashad, S., additional, Abdwani, R., additional, Rigante, D., additional, Rodriquez Lorzano, A., additional, Pallotti, C., additional, Swart, J. F., additional, and Ruperto, N., additional

Published
2024
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2. Health-related quality of life, anxiety, and depressive symptoms in children with primary immunodeficiencies

Author

Kuburovic NB, Pasic S, Susic G, Stevanovic D, Kuburovic V, Zdravkovic S, Janicijevic Petrovic M, and Pekmezovic T

Subjects
Medicine (General), R5-920
Abstract

Nina B Kuburovic,1 Srdjan Pasic,2 Gordana Susic,3 Dejan Stevanovic,4 Vladimir Kuburovic,5 Slavisa Zdravkovic,6 Mirjana Janicijevic Petrovic,7 Tatjana Pekmezovic8 1Department of Public Health, Mother and Child Health Care Institute of Serbia, 2Department of Immunology, Pediatric Clinic, Faculty of Medicine, University of Belgrade, 3Institute for Rheumatology, Clinical Center of Serbia, 4Clinic for Neurology and Psychiatry for Children and Adolescents, 5Pediatric Clinic, Mother and Child Health Care Institute of Serbia, 6Pediatric Day Hospital, Mother and Child Health Care Institute of Serbia, Belgrade, 7Faculty of Medical Sciences, University of Kragujevac, Kragujevac, 8Serbia Institute of Epidemiology, Faculty of Medicine, University of Belgrade, Belgrade, Serbia Introduction: The aims of this study were to evaluate levels of health-related quality of life (HRQOL) and the presence of anxiety and depressive symptoms in children with primary immunodeficiency disease (PID) in Serbia. Materials and methods: Self- and parent-rated data from 25 children with PID were available. As controls, data from 50 children with juvenile idiopathic arthritis (JIA) and 89 healthy children were included. The Pediatric Quality of Life Inventory was used for HRQOL assessments. Anxiety symptoms were identified using the Screen for Child Anxiety-Related Emotional Disorders questionnaire, while depressive symptoms were identified using the Mood and Feeling Questionnaire. Results: Children with PID had significantly lower Pediatric Quality of Life Inventory total scores compared to children with JIA and healthy children as child-rated (P=0.02) and parent-rated (P

Published
2014

3. POS0169 OPEN-LABEL, LONG-TERM (10-YEAR) STUDY OF THE SAFETY OF ETANERCEPT IN CHILDREN AND YOUNG ADULTS WITH EXTENDED OLIGOARTICULAR, ENTHESITIS-RELATED, OR PSORIATIC JUVENILE IDIOPATHIC ARTHRITIS

Author

Vojinovic, J., primary, Dehoorne, J., additional, Panaviene, V., additional, Susic, G., additional, Horneff, G., additional, Stanevicha, V., additional, Kobusinska, K., additional, Żuber, Z., additional, Dobrzyniecka, B., additional, Akikusa, J., additional, Avcin, T., additional, Martini, A., additional, Borlenghi, C., additional, Arthur, E., additional, Tatulych, S. Y., additional, Zang, C., additional, Vlahos, B., additional, and Ruperto, N., additional

Published
2022
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4. POS1293 TEN-YEAR EFFICACY DATA FROM THE CLIPPER STUDIES: OPEN-LABEL, LONG-TERM ETANERCEPT TREATMENT IN CHILDREN AND YOUNG ADULTS WITH EXTENDED OLIGOARTICULAR, ENTHESITIS-RELATED, OR PSORIATIC JUVENILE IDIOPATHIC ARTHRITIS

Author

Vojinovic, J., primary, Dehoorne, J., additional, Panaviene, V., additional, Susic, G., additional, Horneff, G., additional, Stanevicha, V., additional, Kobusinska, K., additional, Żuber, Z., additional, Dobrzyniecka, B., additional, Akikusa, J., additional, Avcin, T., additional, Martini, A., additional, Borlenghi, C., additional, Arthur, E., additional, Tatulych, S. Y., additional, Zang, C., additional, Tsekouras, V., additional, Vlahos, B., additional, and Ruperto, N., additional

Published
2022
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5. Validity and reliability of four parent/patient reported outcome measures for juvenile idiopathic arthritis remote monitoring

Author

van Dijkhuizen EHP, Ridella F, Naddei R, Trincianti C, Avrusin I, Mazzoni M, Sutera D, Ayaz NA, Penades IC, Constantin T, Herlin T, Oliveira SK, Rygg M, Sanner H, Susic G, Sztajnbok F, Varbanova B, Ruperto N, Ravelli A, Consolaro A, and Paediatric Rheumatology International Trials Organisation (PRINTO).

Abstract

OBJECTIVE: Aim of this work is to provide evidence of validity and reliability for four parent/child reported outcomes (PCROs) measures included in the OMERACT JIA core domain set: the evaluation of the child's pain and level of disease activity, the assessment of the morning stiffness (MS) duration, and an active joint count for proxy/self-assessment. METHODS: Patients were included in the multinational study Epidemiology Treatment and Outcome of Childhood Arthritis. Criterion validity was assessed by examining the correlation of the four tested measures with physician measures and the clinical Juvenile Arthritis Disease Activity Score (cJADAS)10 in the whole sample and after grouping patients by ILAR category, geographic area, and education level. Reliability was assessed comparing two visits 7-14 days apart with intraclass correlation coefficients (ICC). RESULTS: A total of 8,848 parents and 6,204 patients had all the evaluations available. Correlations of tested measures were moderate (0.4-0-7) with physician reported measures. The level of correlation with the cJADAS10 remained stable after grouping patients by ILAR category, geographic areas, and level of education of the parent filling the questionnaire. In 442 parents and 344 children, ICC ranged between 0.79 and 0.87 for parents and 0.81 and 0.88 for children. CONCLUSION: The four tested PCROs showed good criterion validity and excellent reliability. These tools can be considered for remote patient assessment, when in person evaluation might not be possible.

Published
2022

6. International Consensus for the Dosing of Corticosteroids in Childhood-Onset Systemic Lupus Erythematosus With Proliferative Lupus Nephritis

Author

Chalhoub, N.E. Wenderfer, S.E. Levy, D.M. Rouster-Stevens, K. Aggarwal, A. Savani, S.I. Ruth, N.M. Arkachaisri, T. Qiu, T. Merritt, A. Onel, K. Goilav, B. Khubchandani, R.P. Deng, J. Fonseca, A.R. Ardoin, S.P. Ciurtin, C. Kasapcopur, O. Jelusic, M. Huber, A.M. Ozen, S. Klein-Gitelman, M.S. Appenzeller, S. Cavalcanti, A. Fotis, L. Lim, S.C. Silva, R.M. Miramontes, J.R. Rosenwasser, N.L. Saad-Magalhaes, C. Schonenberg-Meinema, D. Scott, C. Silva, C.A. Enciso, S. Terreri, M.T. Torres-Jimenez, A.-R. Trachana, M. Al-Mayouf, S.M. Devarajan, P. Huang, B. Brunner, H.I. Abulaban, K. Aguiar, C. Ahn, S.-Y. Akoghlanian, S. Al-Abrawi, S. Aljaberi, N. Alperin, R. Angeles-Han, S. Ardalan, K. Bader-Meunier, B. Balboni, I. Barbar-Smiley, F. Baxter, S. Beary, J. Boneparth, A. Brakeman, P. Bridges, J. Burgos-Vargas, R. Cabral, D.A. Cameto, J. Carter, C. Chang, J. Chédeville, G. Chhakchhuak, C. Chiraseveenuprapund, P. Cifuentes Alvarado, M. Concannon, A. Cooper, J. Cron, R. De Carvalho, L.M. De Quattro, K. De Ranieri, D. Dizon, B. Donnelly Wrigley, C. Duong, M.D. Eberhard, A. Ede, K. Edelheit, B. Edens, C. Espada, G. Farhey, Y. Flores, F. Fritz, D. Ganguli, S. Gilbert, M. Gittar, P. Greenbaum, L. Grom, A. Gulati, G. Harry, O. Hayward, K. Henrickson, M. Hersh, A. Hiraki, L. Hiskey, M. Hoffmann, S. Hollander, M. Hom, C. Houk, L. Houk, J.B. Hsieh, E.W.Y. Hsu, J. Jensen, P. Joos, R. Jurado, R. Jusan Fiorot, F. Kallash, M. Kamphuis, S. Keltsev, V., (deceased) Khanna, S. Kim, S. Kimseng, K.J. Knight, A. Kunder, R. Lai, J. Laskin, B. Lewandowski, L. Lim, L. Linda, W.-W. Lo, M. Lovell, D. Luggen, M. Madison, J. Mansuri, A. Martin, L. Mason, S. Miller, M. Mina, R. Mohammed, A. Moncrieffe, H. Moorthy, L. Morgan, E. Mosquera, A. Muntel, E. Muscal, E. Myones, B. Nocton, J. Ogbu, E. Okamura, D. Olson, J. Orrock, J. Paim-Marques, L. Pain, C. Park, C. Patel, P. Pereira, M. Prado, R.D. Radhakrishna, S. Rheault, M. Ridgway, W. Riskalla, M. Ronis, T. Sadun, R. Sagcal-Gironella, A.C. Santos, M.C. Schikler, K. AL Suwairi, W. Siddiqi, N. Silva, M.F. Singh-Grewal, D. Smitherman, E. Smolewska, E. Son, M.B. Srinivasalu, H. Sule, S. Susic, G. Syed, R. Thatayatikom, A. Ting, T. Toth, M. Turnier, J. Vashisht, P. Vega Fernandez, P. Velasquez, M. von Scheven, E. Wahezi, D. Ware, A. Wu, E. Yan, J. Yildirim-Toruner, C. Zamparo, C. Zhang, Y. Lawson, E. for the Childhood Arthritis Rheumatology Research Alliance Lupus Nephritis Work Group the Pediatric Rheumatology European Society Lupus Working Party

Abstract

Objective: To develop a standardized steroid dosing regimen (SSR) for physicians treating childhood-onset systemic lupus erythematosus (SLE) complicated by lupus nephritis (LN), using consensus formation methodology. Methods: Parameters influencing corticosteroid (CS) dosing were identified (step 1). Data from children with proliferative LN were used to generate patient profiles (step 2). Physicians rated changes in renal and extrarenal childhood-onset SLE activity between 2 consecutive visits and proposed CS dosing (step 3). The SSR was developed using patient profile ratings (step 4), with refinements achieved in a physician focus group (step 5). A second type of patient profile describing the course of childhood-onset SLE for ≥4 months since kidney biopsy was rated to validate the SSR-recommended oral and intravenous (IV) CS dosages (step 6). Patient profile adjudication was based on majority ratings for both renal and extrarenal disease courses, and consensus level was set at 80%. Results: Degree of proteinuria, estimated glomerular filtration rate, changes in renal and extrarenal disease activity, and time since kidney biopsy influenced CS dosing (steps 1 and 2). Considering these parameters in 5,056 patient profile ratings from 103 raters, and renal and extrarenal course definitions, CS dosing rules of the SSR were developed (steps 3–5). Validation of the SSR for up to 6 months post–kidney biopsy was achieved with 1,838 patient profile ratings from 60 raters who achieved consensus for oral and IV CS dosage in accordance with the SSR (step 6). Conclusion: The SSR represents an international consensus on CS dosing for use in patients with childhood-onset SLE and proliferative LN. The SSR is anticipated to be used for clinical care and to standardize CS dosage during clinical trials. © 2021, American College of Rheumatology

Published
2022

8. Opportunistic infections in immunosuppressed patients with juvenile idiopathic arthritis: analysis by the Pharmachild Safety Adjudication Committee

Author

Giancane, G, Swart, Jf, Castagnola, E, Groll, Ah, Horneff, G, Huppertz, Hi, Lovell, Dj, Wolfs, T, Herlin, T, Dolezalova, P, Sanner, H, Susic, G, Sztajnbok, F, Maritsi, D, Constantin, T, Vargova, V, Sawhney, S, Rygg, M, K Oliveira, S, Cattalini, M, Bovis, F, Bagnasco, F, Pistorio, A, Martini, A, Wulffraat, N, Ruperto, N, Cuttica, R, Garay, Sm, Brunner, J, Emminger, W, Appenzeller, S, Len, C, Saad Magalhaes, C, Telcharova-Mihaylovska, A, Harjacek, M, Jelusic, M, Estmann, A, Nielsen, S, Herrera Mora, C, Gervais, E, Koné-Paut, I, Quartier, P, Foeldvari, I, Lutz, T, Minden, K, Tzaribachev, N, Trachana, M, Tsitsami, E, Vougiouka, O, Orban, I, Harel, L, Hashkes, P, Uziel, Y, Cimaz, R, Civino, A, Consolini, R, D'Angelo, G, De Benedetti, F, Filocamo, G, Fueri, E, Gallizzi, R, Maggio, Mc, Magnolia, Mg, Miniaci, A, Montin, D, Olivieri, An, Pastore, S, Rigante, Donato, Zulian, F, Rumba-Rozenfelde, I, Stanevicha, V, Panaviene, V, Rodriguez Lozano, Al, Rubio-Perez, N, Vega Cornejo, G, Hoppenreijs, E, Kamphuis, S, Flato, B, Nordal, Eb, Abdwani, R, Miraval, T, Paz Gastanaga, Me, Smolewska, E, Ailioaie, C, Cochino, Av, Laday, M, Lazar, C, Alexeeva, E, Chasnyk, V, Keltsev, V, Suwairi, Wm, Vijatov-Djuric, G, Vojinovic, J, Arkachaisri, T, Koskova, E, Avcin, T, Ally, M, Van Rensburg, Cj, Louw, I, Lopez, Ja, Boteanu, Al, Calvo Penades, I, De Inocencio, J, Mesa-Del-Castillo, P, Moreno, E, Remesal, A, Hofer, M, Gok, F, Ozen, S, Ramanan, A, Pallotti, C, Villa, L &amp, Paediatric Rheumatology International Trials Organisation, (PRINTO), Rigante D (ORCID:0000-0001-7032-7779), Giancane, G, Swart, Jf, Castagnola, E, Groll, Ah, Horneff, G, Huppertz, Hi, Lovell, Dj, Wolfs, T, Herlin, T, Dolezalova, P, Sanner, H, Susic, G, Sztajnbok, F, Maritsi, D, Constantin, T, Vargova, V, Sawhney, S, Rygg, M, K Oliveira, S, Cattalini, M, Bovis, F, Bagnasco, F, Pistorio, A, Martini, A, Wulffraat, N, Ruperto, N, Cuttica, R, Garay, Sm, Brunner, J, Emminger, W, Appenzeller, S, Len, C, Saad Magalhaes, C, Telcharova-Mihaylovska, A, Harjacek, M, Jelusic, M, Estmann, A, Nielsen, S, Herrera Mora, C, Gervais, E, Koné-Paut, I, Quartier, P, Foeldvari, I, Lutz, T, Minden, K, Tzaribachev, N, Trachana, M, Tsitsami, E, Vougiouka, O, Orban, I, Harel, L, Hashkes, P, Uziel, Y, Cimaz, R, Civino, A, Consolini, R, D'Angelo, G, De Benedetti, F, Filocamo, G, Fueri, E, Gallizzi, R, Maggio, Mc, Magnolia, Mg, Miniaci, A, Montin, D, Olivieri, An, Pastore, S, Rigante, Donato, Zulian, F, Rumba-Rozenfelde, I, Stanevicha, V, Panaviene, V, Rodriguez Lozano, Al, Rubio-Perez, N, Vega Cornejo, G, Hoppenreijs, E, Kamphuis, S, Flato, B, Nordal, Eb, Abdwani, R, Miraval, T, Paz Gastanaga, Me, Smolewska, E, Ailioaie, C, Cochino, Av, Laday, M, Lazar, C, Alexeeva, E, Chasnyk, V, Keltsev, V, Suwairi, Wm, Vijatov-Djuric, G, Vojinovic, J, Arkachaisri, T, Koskova, E, Avcin, T, Ally, M, Van Rensburg, Cj, Louw, I, Lopez, Ja, Boteanu, Al, Calvo Penades, I, De Inocencio, J, Mesa-Del-Castillo, P, Moreno, E, Remesal, A, Hofer, M, Gok, F, Ozen, S, Ramanan, A, Pallotti, C, Villa, L &amp, Paediatric Rheumatology International Trials Organisation, (PRINTO), and Rigante D (ORCID:0000-0001-7032-7779)

Abstract

BACKGROUND: To derive a list of opportunistic infections (OI) through the analysis of the juvenile idiopathic arthritis (JIA) patients in the Pharmachild registry by an independent Safety Adjudication Committee (SAC). METHODS: The SAC (3 pediatric rheumatologists and 2 pediatric infectious disease specialists) elaborated and approved by consensus a provisional list of OI for use in JIA. Through a 5 step-procedure, all the severe and serious infections, classified as per MedDRA dictionary and retrieved in the Pharmachild registry, were evaluated by the SAC by answering six questions and adjudicated with the agreement of 3/5 specialists. A final evidence-based list of OI resulted by matching the adjudicated infections with the provisional list of OI. RESULTS: A total of 772 infectious events in 572 eligible patients, of which 335 serious/severe/very severe non-OI and 437 OI (any intensity/severity), according to the provisional list, were retrieved. Six hundred eighty-two of 772 (88.3%) were adjudicated as infections, of them 603/682 (88.4%) as common and 119/682 (17.4%) as OI by the SAC. Matching these 119 opportunistic events with the provisional list, 106 were confirmed by the SAC as OI, and among them infections by herpes viruses were the most frequent (68%), followed by tuberculosis (27.4%). The remaining events were divided in the groups of non-OI and possible/patient and/or pathogen-related OI. CONCLUSIONS: We found a significant number of OI in JIA patients on immunosuppressive therapy. The proposed list of OI, created by consensus and validated in the Pharmachild cohort, could facilitate comparison among future pharmacovigilance studies.

Published
2020

11. Health-related quality of life of patients with juvenile idiopathic arthritis coming from 3 different geographic areas. The PRINTO multinational quality of life cohort study

Author

Gutiérrez-Suárez, R., Pistorio, A., Cruz, A. Cespedes, Norambuena, X., Flato, B., Rumba, I., Harjacek, M., Nielsen, S., Susic, G., Mihaylova, D., Huemer, C., Melo-Gomes, J., Andersson-Gare, B., Balogh, Z., De Cunto, C., Vesely, R., Pagava, K., Romicka, A. M., Burgos-Vargas, R., Martini, A., and Ruperto, N.

Published
2007

12. PRINTO/PRES international website for families of children with rheumatic diseases: www.pediatric-rheumatology.printo.it

Author

Ruperto, N, Garcia-Munitis, P, Villa, L, Pesce, M, Aggarwal, A, Fasth, A, Avcin, T, Bae, S-C, Balogh, Z, Li, C, De Inocencio, J, Dibra, M, Dolezalova, P, Miedany, Y El, Flato, B, Harjacek, M, Huppertz, H-I, Kanakoudi-Tsakalidou, F, Wulffraat, N, Lahdenne, P, Melo-Gomes, J A, Mihaylova, D, Nielsen, S, Nikishina, I, Ozdogan, H, Pagava, K, Panaviene, V, Prieur, A-M, Romicka, A-M, Rumba, I, Shafaie, N, Susic, G, Takei, S, Uziel, Y, Vesely, R, Woo, P, and Martini, A

Published
2005

13. Phenotypic variability and disparities in treatment and outcomes of childhood arthritis throughout the world: an observational cohort study

Author

Consolaro, A, Giancane, G, Alongi, A, van Dijkhuizen, Ehp, Aggarwal, A, Al-Mayouf, Sm, Bovis, F, De Inocencio, J, Demirkaya, E, Flato, B, Foell, D, Garay, Sm, Lazăr, C, Lovell, Dj, Montobbio, C, Miettunen, P, Mihaylova, D, Nielsen, S, Orban, I, Rumba-Rozenfelde, I, Magalhães, C, Shafaie, N, Susic, G, Trachana, M, Wulffraat, N, Pistorio, A, Martini, A, Ruperto, N, Ravelli, A &amp, Abdwani R, Aghighi, Y, Aiche, Mf, Ailioaie, C, Aktay Ayaz, N, Al-Abrawi, S, Alexeeva, E, Anton, J, Apostol, A, Arguedas, O, Avcin, T, Barone, P, Berntson, L, Boteanu, Al, Boyko, Y, Burgos-Vargas, R, Calvo Penades, I, Chédeville, G, Cimaz, R, Civino, A, Consolini, R, Constantin, T, Cuttica, R, Dallos, T, Martin, N, Magni-Manzoni, S, De Cunto, C, Dolezalova, P, Ekelund, M, El Miedany, Y, Espada, G, Estmann Christensen, A, Foeldvari, I, Gallizzi, R, Ganser, G, Gerloni, V, Haas, Jp, Harel, L, Harjacek, M, Hashad, S, Herlin, T, Herrera, C, Hofer, M, Holzinger, D, Horneff, G, Huppertz, Hi, Iagăru, N, Ibanez Estrella, A, Ioseliani, M, Joos, R, Knupp Oliveira, S, Kamphuis, S, Kasapcopur, O, Katsicas, Mm, Khubchandani, R, Kondi, A, Kröger, L, La Torre, F, Laday, M, Lahdenne, P, Maggio, Mc, Magnolia, Mg, Malagon, C, Malin, M, Martino, S, Melo-Gomes, Ja, Mesa-Del-Castillo, P, Militaru, A, Minden, K, Miniaci, A, Moradinejad, Mh, Morel Ayala, Z, Nikishina, I, Norambuena, X, Nordal, Eb, Pagava, K, Panaviene, V, Pastore, S, Pieropan, S, Podda, Ra, Pruunsild, C, Putto-Laurila, A, Quartier, P, Remesal, A, Rigante, Donato, Ringold, S, Rutkowska-Sak, L, Rygg, M, Saurenmann, Rk, Sawhney, S, Scott, C, Shiari, R, Smolewska, E, Sozeri, B, Swart, Jf, Sztajnbok, F, Torcoletti, M, Tsitsami, E, Tzaribachev, N, Unsal, E, Uziel, Y, Vähäsalo, P, Varbanova, B, Vargova, V, Vesely, R, Vijatov-Djuric, G, Vilaiyuk, S, Vojinovic, J, Vougiouka, O, Weiss, P, Wouters, C, Rigante D (ORCID:0000-0001-7032-7779), Consolaro, A, Giancane, G, Alongi, A, van Dijkhuizen, Ehp, Aggarwal, A, Al-Mayouf, Sm, Bovis, F, De Inocencio, J, Demirkaya, E, Flato, B, Foell, D, Garay, Sm, Lazăr, C, Lovell, Dj, Montobbio, C, Miettunen, P, Mihaylova, D, Nielsen, S, Orban, I, Rumba-Rozenfelde, I, Magalhães, C, Shafaie, N, Susic, G, Trachana, M, Wulffraat, N, Pistorio, A, Martini, A, Ruperto, N, Ravelli, A &amp, Abdwani R, Aghighi, Y, Aiche, Mf, Ailioaie, C, Aktay Ayaz, N, Al-Abrawi, S, Alexeeva, E, Anton, J, Apostol, A, Arguedas, O, Avcin, T, Barone, P, Berntson, L, Boteanu, Al, Boyko, Y, Burgos-Vargas, R, Calvo Penades, I, Chédeville, G, Cimaz, R, Civino, A, Consolini, R, Constantin, T, Cuttica, R, Dallos, T, Martin, N, Magni-Manzoni, S, De Cunto, C, Dolezalova, P, Ekelund, M, El Miedany, Y, Espada, G, Estmann Christensen, A, Foeldvari, I, Gallizzi, R, Ganser, G, Gerloni, V, Haas, Jp, Harel, L, Harjacek, M, Hashad, S, Herlin, T, Herrera, C, Hofer, M, Holzinger, D, Horneff, G, Huppertz, Hi, Iagăru, N, Ibanez Estrella, A, Ioseliani, M, Joos, R, Knupp Oliveira, S, Kamphuis, S, Kasapcopur, O, Katsicas, Mm, Khubchandani, R, Kondi, A, Kröger, L, La Torre, F, Laday, M, Lahdenne, P, Maggio, Mc, Magnolia, Mg, Malagon, C, Malin, M, Martino, S, Melo-Gomes, Ja, Mesa-Del-Castillo, P, Militaru, A, Minden, K, Miniaci, A, Moradinejad, Mh, Morel Ayala, Z, Nikishina, I, Norambuena, X, Nordal, Eb, Pagava, K, Panaviene, V, Pastore, S, Pieropan, S, Podda, Ra, Pruunsild, C, Putto-Laurila, A, Quartier, P, Remesal, A, Rigante, Donato, Ringold, S, Rutkowska-Sak, L, Rygg, M, Saurenmann, Rk, Sawhney, S, Scott, C, Shiari, R, Smolewska, E, Sozeri, B, Swart, Jf, Sztajnbok, F, Torcoletti, M, Tsitsami, E, Tzaribachev, N, Unsal, E, Uziel, Y, Vähäsalo, P, Varbanova, B, Vargova, V, Vesely, R, Vijatov-Djuric, G, Vilaiyuk, S, Vojinovic, J, Vougiouka, O, Weiss, P, Wouters, C, and Rigante D (ORCID:0000-0001-7032-7779)

Abstract

BACKGROUND: To our knowledge, the characteristics and burden of childhood arthritis have never been studied on a worldwide basis. We aimed to investigate, with a cross-sectional study, the prevalence of disease categories, treatment methods, and disease status in patients from across different geographical areas and from countries with diverse wealth status. METHODS: In this multinational, cross-sectional, observational cohort study, we asked international paediatric rheumatologists from specialised centres to enrol children with a diagnosis of juvenile idiopathic arthritis, according to International League of Associations for Rheumatology criteria, who were seen consecutively for a period of 6 months. Each patient underwent retrospective and cross-sectional assessments, including measures of disease activity and damage and questionnaires on the wellbeing and quality of life of the children. We qualitatively compared the collected data across eight geographical areas, and we explored an association between disease activity and damage and a country's gross domestic product (GDP) with a multiple logistic regression analysis. FINDINGS: Between April 4, 2011, and Nov 21, 2016, 9081 patients were enrolled at 130 centres in 49 countries, grouped into eight geographical areas. Systemic arthritis (125 [33·0%] of 379 patients) and enthesitis-related arthritis (113 [29·8%] of 379) were more common in southeast Asia, whereas oligoarthritis was more prevalent in southern Europe (1360 [56·7%] of 2400) and rheumatoid factor-negative polyarthritis was more frequent in North America (165 [31·5%] of 523) than in the other areas. Prevalence of uveitis was highest in northern Europe (161 [19·1%] of 845 patients) and southern Europe (450 [18·8%] of 2400) and lowest in Latin America (54 [6·4%] of 849), Africa and Middle East (71 [5·9%] of 1209), and southeast Asia (19 [5·0%] of 379). Median age at disease onset was lower in southern Europe (3·5 years, IQR 1·9-7·3) than in other regions

Published
2019

14. Standardised procedures for ultrasound imaging in paediatric rheumatology: progress of EULAR/PRES task force

Author

Vojinovic, Jelena, Collado, Paz, Janta, I, Carmona, Loreto, Malattia, Clara, Magni-Manzoni, Silvia, Susic, G., Tzaribachev, Nikolay, Ravagnani, Viviana, Rossi-Semerano, Linda, Kljucevsek, D, Lanni, Stefano, Sudoł-Szopińska, Iwona, Windschall, Daniel, Balint, P, Jousse-Joulin, Sandrine, Modesto, Consuelo, Boutry, N, Iagnocco, Annamaria, Naredo, Esperanza, University Clinical Center, Nis, Severo Ochoa Hospital, University of Barcelona, Instituto de Salud Musculoesqueletica (InMusc), IRCCS G. Gaslini, Pediatria II, Reumatologia, Pædiatric Rheumatology International Trials Organisation (PRINTO), IRCCS Ospedale Pediatrico Bambino Gesù [Roma], Institute for Rheumatology, Belgrade, Pediatric Rheumatology Research Institute, Bad Bramstedt, ASST Mantova, Service de Pédiatrie Générale et Rhumatologie Pédiatrique, Université Paris-Sud - Paris 11 (UP11)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Bicêtre, Children's Hospital, Ljubljana, Instituto Giannina Gaslini, Genoa, Department of Diagnostic Imaging, Medical University, Asklepios Hospital Weissenfels, National Institute of Rheumatology and Physiotherapy, Budapest, CHRU Brest - Service de Rhumatologie (CHU - BREST - Rhumato), Centre Hospitalier Régional Universitaire de Brest (CHRU Brest), Lymphocytes B, Autoimmunité et Immunothérapies (LBAI), Université de Brest (UBO)-Institut National de la Santé et de la Recherche Médicale (INSERM)-LabEX IGO Immunothérapie Grand Ouest-Institut Brestois Santé Agro Matière (IBSAM), Université de Brest (UBO), Vall d'Hebron Institute of Oncology [Barcelone] (VHIO), Vall d'Hebron University Hospital [Barcelona], Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille), Università degli studi di Torino = University of Turin (UNITO), Fundación Jiménez Díaz, Fundacion Jimenez Diaz [Madrid] (FJD), Michel, Geneviève, Lymphocyte B et Auto-immunité (LBAI), Université de Brest (UBO)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut Brestois Santé Agro Matière (IBSAM), Università degli studi di Torino (UNITO), Université de Brest (UBO)-Institut Brestois Santé Agro Matière (IBSAM), and Université de Brest (UBO)-Institut National de la Santé et de la Recherche Médicale (INSERM)

Subjects
[SDV] Life Sciences [q-bio], [SDV]Life Sciences [q-bio], ComputingMilieux_MISCELLANEOUS
Abstract

International audience

Published
2018

17. Expert consensus on dynamics of laboratory tests for diagnosis of macrophage activation syndrome complicating systemic juvenile idiopathic arthritis

Author

Ravelli, A, Minoia, F, Davì, S, Horne, A, Bovis, F, Pistorio, A, Aricò, M, Avcin, T, Behrens, Em, De Benedetti, F, Filipovic, A, Grom, Aa, Henter, J-i, Ilowite, Nte, Jordan, Mb, Khubchandani, R, Kitoh, T, Lehmberg, K, Lovell, Dj, Miettunen, P, Nichols, Ke, Ozen, S, Schmid, Jp, Ramanan, Av, Russo, R, Schneider, R, Sterba, G, Uziel, Y, Wallace, C, Wouters, C, Wulffraat, N, Demirkaya, E, Brunner, Hi, Martini, A, Ruperto, N, Cron, Rq, Angioloni, S, Pallotti, C, Pesce, M, Rinaldi, M, Villa, L, Abinun, M, Aggarwal, A, Akikusa, J, Al-mayouf, Sm, Alessio, M, Anton, J, Apaz, Mt, Astigarraga, I, Ayaz, Na, Barone, P, Bica, B, Bolt, I, Breda, L, Chasnyk, V, Cimaz, R, Corona, F, Cuttica, R, D'Angelo, G, Davidsone, Z, De Cunto, C, De Inocencio, J, Eisenstein, E, Enciso, S, Espada, G, Fischbach, M, Frosch, M, Gallizzi, R, Gamir, Ml, Gao, Y-j, Griffin, T, Hashad, S, Hennon, T, Horneff, G, Huasong, Z, Huber, A, Insalaco, A, Ioseliani, M, Jelusic-drazic, M, Jeng, M, Kapovic, A, Kasapcopur, O, Kone-paut, I, De Oliveira Skf, Lattanzi, B, Lepore, L, Li, C, Lipton, Jm, Magni-manzoni, S, Maritsi, D, Mccurdy, D, Merino, R, Mulaosmanovic, V, Nielsen, S, Pal, P, Prahalad, S, Rigante, Donato, Rumba-rozenfelde, I, Magalhaes, Cs, Sanner, H, Sawhney, S, Sewairi, Wm, Shakoory, B, Shenoi, S, Clovis, As, Stanevicha, V, Stine, Kc, Susic, G, Sztajnbok, F, Takei, S, Tezer, H, Trauzeddel, R, Tsitsami, E, Unsal, E, Vougiouka, O, Weaver, Lk, Weiss, J, Weitzman, S, On Behalf Of The Pediatric Rheumatology International Trials Organization, Zletni M., The Childhood Arthritis & Rheumatology Research Alliance, The Pediatric Rheumatology Collaborative Study Group And The Histiocyte Society, Ravelli, A., Minoia, F., Davi, S., Horne, A., Bovis, F., Pistorio, A., Arico, M., Avcin, T., Behrens, E. M., De Benedetti, F., Filipovic, A., Grom, A. A., Henter, J. -I., Ilowite, N. T., Jordan, M. B., Khubchandani, R., Kitoh, T., Lehmberg, K., Lovell, D. J., Miettunen, P., Nichols, K. E., Ozen, S., Schmid, J. P., Ramanan, A. V., Russo, R., Schneider, R., Sterba, G., Uziel, Y., Wallace, C., Wouters, C., Wulffraat, N., Demirkaya, E., Brunner, H. I., Martini, A., Ruperto, N., Cron, R. Q., Angioloni, S., Pallotti, C., Pesce, M., Rinaldi, M., Villa, L., Abinun, M., Aggarwal, A., Akikusa, J., Al-Mayouf, S. M., Alessio, M., Anton, J., Apaz, M. T., Astigarraga, I., Ayaz, N. A., Barone, P., Bica, B., Bolt, I., Breda, L., Chasnyk, V., Cimaz, R., Corona, F., Cuttica, R., D'Angelo, G., Davidsone, Z., De Cunto, C., De Inocencio, J., Eisenstein, E., Enciso, S., Espada, G., Fischbach, M., Frosch, M., Gallizzi, R., Gamir, M. L., Gao, Y. -J., Griffin, T., Hashad, S., Hennon, T., Horneff, G., Huasong, Z., Huber, A., Insalaco, A., Ioseliani, M., Jelusic-Drazic, M., Jeng, M., Kapovic, A., Kasapcopur, O., Kone-Paut, I., De Oliveira, S. K. F., Lattanzi, B., Lepore, L., Li, C., Lipton, J. M., Magni-Manzoni, S., Maritsi, D., Mccurdy, D., Merino, R., Mulaosmanovic, V., Nielsen, S., Pal, P., Prahalad, S., Rigante, D., Rumba-Rozenfelde, I., Magalhaes, C. S., Sanner, H., Sawhney, S., Sewairi, W. M., Shakoory, B., Shenoi, S., Clovis, A. S., Stanevicha, V., Stine, K. C., Susic, G., Sztajnbok, F., Takei, S., Tezer, H., Trauzeddel, R., Tsitsami, E., Unsal, E., Vougiouka, O., Weaver, L. K., Weiss, J., Weitzman, S., Zletni, M., and Çocuk Sağlığı ve Hastalıkları

Subjects
medicine.medical_specialty, systemic juvenile idiopathic arthritis, Epidemiology, Immunology, Arthritis, Bioinformatics, 03 medical and health sciences, 0302 clinical medicine, Rheumatology, Internal medicine, medicine, Journal Article, Immunology and Allergy, 030212 general & internal medicine, Juvenile Idiopathic Arthritis, Prospective cohort study, 030203 arthritis & rheumatology, medicine.diagnostic_test, business.industry, Paediatric Rheumatology, medicine.disease, Outcomes research, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Macrophage activation syndrome, Erythrocyte sedimentation rate, Absolute neutrophil count, sense organs, business
Abstract

OBJECTIVE: To identify which laboratory tests that change over time are most valuable for the timely diagnosis of macrophage activation syndrome (MAS) complicating systemic juvenile idiopathic arthritis (sJIA).METHODS: A multistep process, based on a combination of expert consensus and analysis of real patient data, was conducted. A panel of experts was first asked to evaluate 115 profiles of patients with MAS, which included the values of laboratory tests at the pre-MAS visit and at MAS onset, and the change in values between the two time points. The experts were asked to choose the 5 laboratory tests in which change was most important for the diagnosis of MAS and to rank the 5 selected tests in order of importance. The relevance of change in laboratory parameters was further discussed and ranked by the same experts at a consensus conference.RESULTS: Platelet count was the most frequently selected test, followed by ferritin level, aspartate aminotransferase (AST), white cell count, neutrophil count, and fibrinogen and erythrocyte sedimentation rate. Ferritin was most frequently assigned the highest score. At the end of the process, platelet count, ferritin level and AST were the laboratory tests in which the experts found change over time to be most important.CONCLUSIONS: We identified the laboratory tests in which change over time is most valuable for the early diagnosis of MAS in sJIA. The dynamics of laboratory values during the course of MAS should be further scrutinised in a prospective study in order to establish the optimal cut-off values for their variation.

Published
2016
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18. Evidence-based provisional clinical classification criteria for autoinflammatory periodic fevers

Author

Federici S., Sormani M. P., Ozen S., Lachmann H. J., Amaryan G., Woo P., Kone-Paut I., Dewarrat N., Cantarini L., Insalaco A., Uziel Y., Rigante D., Quartier P., Demirkaya E., Herlin T., Meini A., Fabio G., Kallinich T., Martino S., Butbul A. Y., Olivieri A., Kuemmerle-Deschner J., Neven B., Simon A., Ozdogan H., Touitou I., Frenkel J., Hofer M., Martini A., Ruperto N., Gattorno M., Espada G., Russo R., De Cunto C., Boros C., Borzutzky A., Jelusic-Drazic M., Dolezalova P., Nielsen S., Hentgen V., Schwarz T., Berendes R., Jansson A., Horneff G., Papadopoulou-Alataki E., Tsitsami E., Tsakalidou F. K., Gallizzi R., Obici L., Barone P., Cimaz R., Alessio M., Nishikomori R., Stanevicha V., Hoppenreijs E., Wolska-Kusnierz B., Iagaru N., Nikishina I., Al-Mayouf S. M., Sewairi, Susic G., Toplak N., Modesto C., Elorduy M. J. R., Anton J., Bou R., Federici, S., Sormani, M. P., Ozen, S., Lachmann, H. J., Amaryan, G., Woo, P., Kone-Paut, I., Dewarrat, N., Cantarini, L., Insalaco, A., Uziel, Y., Rigante, D., Quartier, P., Demirkaya, E., Herlin, T., Meini, A., Fabio, G., Kallinich, T., Martino, S., Butbul, A. Y., Olivieri, A., Kuemmerle-Deschner, J., Neven, B., Simon, A., Ozdogan, H., Touitou, I., Frenkel, J., Hofer, M., Martini, A., Ruperto, N., Gattorno, M., Espada, G., Russo, R., De Cunto, C., Boros, C., Borzutzky, A., Jelusic-Drazic, M., Dolezalova, P., Nielsen, S., Hentgen, V., Schwarz, T., Berendes, R., Jansson, A., Horneff, G., Papadopoulou-Alataki, E., Tsitsami, E., Tsakalidou, F. K., Gallizzi, R., Obici, L., Barone, P., Cimaz, R., Alessio, M., Nishikomori, R., Stanevicha, V., Hoppenreijs, E., Wolska-Kusnierz, B., Iagaru, N., Nikishina, I., Al-Mayouf, S. M., Sewairi, Susic, G., Toplak, N., Modesto, C., Elorduy, M. J. R., Anton, J., Bou, R., Istituto Giannina Gaslini, Genova, Immunologia Clinica e Sperimentale, University of Genoa (UNIGE), Hacettepe University = Hacettepe Üniversitesi, National Amyloidosis Centre, University College London Medical School, University College of London [London] (UCL), 'ARABKIR' JOINT MEDICAL CENTER & INSTITUTE OF CHILD AND ADOLESCENT HEALTH, Cardiac Unit, Institute of Child Health (UCL), Centre de Référence des Maladies Auto-Inflammatoires et des Amyloses [CH Versailles] (CeRéMAIA - Hôpital André Mignot), Centre Hospitalier de Versailles André Mignot (CHV), University Hospital Center (CHUV) and University of Lausanne (UNIL), Lausanne, Università degli Studi di Siena = University of Siena (UNISI), Children's Hospital Bambino Gesù IRCCS [Rome], Meir Medical Centre, Università cattolica del Sacro Cuore [Roma] (Unicatt), Imagine - Institut des maladies génétiques (IHU) (Imagine - U1163), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Paris (UP), Ankara University School of Medicine [Turkey], Aarhus University Hospital, Università degli Studi di Brescia [Brescia], IRCCS Istituto Nazionale dei Tumori [Milano], Charité - UniversitätsMedizin = Charité - University Hospital [Berlin], Università degli studi di Torino (UNITO), Rambam Medical Health Center, Israel., Universita degli studi di Napoli 'Parthenope' [Napoli], Universitätsklinikum Tübingen - University Hospital of Tübingen, Eberhard Karls Universität Tübingen = Eberhard Karls University of Tuebingen, Radboud University Medical Centre [Nijmegen, The Netherlands], Cerrahpasa Faculty of Medicine, Istanbul University, CHU Montpellier, Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier), Cellules Souches, Plasticité Cellulaire, Médecine Régénératrice et Immunothérapies (IRMB), Université de Montpellier (UM)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier), University Medical Center [Utrecht], Geneva University Hospital (HUG), Universita degli studi di Genova, and Olivieri, Alma Nunzia

Subjects
Male, Pediatrics, Multivariate analysis, [SDV]Life Sciences [q-bio], Fever Syndromes, Familial Mediterranean fever, Immunology and Allergy, Mevalonate Kinase Deficiency/classification/diagnosis, Registries, Child, ddc:618, Evidence-Based Medicine, Middle Aged, Pharyngitis, 3. Good health, Familial Mediterranean Fever, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Child, Preschool, Autoinflammation, Familial Mediterranean Fever/classification/diagnosis, Female, medicine.symptom, Periodic fever syndrome, Inflammatory diseases Radboud Institute for Molecular Life Sciences [Radboudumc 5], Adult, medicine.medical_specialty, Fever, Adolescent, Immunology, Cryopyrin-Associated Periodic Syndromes/classification/diagnosis, Sensitivity and Specificity, General Biochemistry, Genetics and Molecular Biology, Young Adult, Inflammation, Rheumatology, medicine, Humans, Preschool, Hereditary Autoinflammatory Diseases/classification/diagnosis, Receiver operating characteristic, business.industry, Hereditary Autoinflammatory Diseases, Case-control study, Cryopyrin-associated periodic syndrome, Infant, Gold standard (test), medicine.disease, Case-Control Studies, Cryopyrin-Associated Periodic Syndromes, Mevalonate Kinase Deficiency, ROC Curve, business
Abstract

Item does not contain fulltext The objective of this work was to develop and validate a set of clinical criteria for the classification of patients affected by periodic fevers. Patients with inherited periodic fevers (familial Mediterranean fever (FMF); mevalonate kinase deficiency (MKD); tumour necrosis factor receptor-associated periodic fever syndrome (TRAPS); cryopyrin-associated periodic syndromes (CAPS)) enrolled in the Eurofever Registry up until March 2013 were evaluated. Patients with periodic fever, aphthosis, pharyngitis and adenitis (PFAPA) syndrome were used as negative controls. For each genetic disease, patients were considered to be 'gold standard' on the basis of the presence of a confirmatory genetic analysis. Clinical criteria were formulated on the basis of univariate and multivariate analysis in an initial group of patients (training set) and validated in an independent set of patients (validation set). A total of 1215 consecutive patients with periodic fevers were identified, and 518 gold standard patients (291 FMF, 74 MKD, 86 TRAPS, 67 CAPS) and 199 patients with PFAPA as disease controls were evaluated. The univariate and multivariate analyses identified a number of clinical variables that correlated independently with each disease, and four provisional classification scores were created. Cut-off values of the classification scores were chosen using receiver operating characteristic curve analysis as those giving the highest sensitivity and specificity. The classification scores were then tested in an independent set of patients (validation set) with an area under the curve of 0.98 for FMF, 0.95 for TRAPS, 0.96 for MKD, and 0.99 for CAPS. In conclusion, evidence-based provisional clinical criteria with high sensitivity and specificity for the clinical classification of patients with inherited periodic fevers have been developed.

Published
2015
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19. THU0548 Standardised procedures for ultrasound imaging in paediatric rheumatology: progress of eular/pres task force

Author

Vojinovic, J., primary, Collado, P., additional, Janta, I., additional, Carmona, L., additional, Malattia, C., additional, Magni-Manzoni, S., additional, Susic, G., additional, Tzaribachev, N., additional, Ravagnani, V., additional, Rossi-Semerano, L., additional, Kljucevsek, D., additional, Lanni, S., additional, Sudoł-Szopinska, I., additional, Windschall, D., additional, Balint, P., additional, Jousse-Joulin, S., additional, Modesto, C., additional, Boutry, N., additional, Iagnocco, A., additional, and Naredo, E., additional

Published
2018
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20. Expert consensus on dynamics of laboratory tests for diagnosis of macrophage activation syndrome complicating systemic juvenile idiopathic arthritis

Author

Ravelli, A, Minoia, F, Davì, S, Horne, A, Bovis, F, Pistorio, A, Aricò, M, Avcin, T, Behrens, Em, De Benedetti, F, Filipovic, A, Grom, Aa, Henter, J-i, Ilowite, Nte, Jordan, Mb, Khubchandani, R, Kitoh, T, Lehmberg, K, Lovell, Dj, Miettunen, P, Nichols, Ke, Ozen, S, Schmid, Jp, Ramanan, Av, Russo, R, Schneider, R, Sterba, G, Uziel, Y, Wallace, C, Wouters, C, Wulffraat, N, Demirkaya, E, Brunner, Hi, Martini, A, Ruperto, N, Cron, Rq, Angioloni, S, Pallotti, C, Pesce, M, Rinaldi, M, Villa, L, Abinun, M, Aggarwal, A, Akikusa, J, Al-mayouf, Sm, Alessio, M, Anton, J, Apaz, Mt, Astigarraga, I, Ayaz, Na, Barone, P, Bica, B, Bolt, I, Breda, L, Chasnyk, V, Cimaz, R, Corona, F, Cuttica, R, D'Angelo, G, Davidsone, Z, De Cunto, C, De Inocencio, J, Eisenstein, E, Enciso, S, Espada, G, Fischbach, M, Frosch, M, Gallizzi, R, Gamir, Ml, Gao, Y-j, Griffin, T, Hashad, S, Hennon, T, Horneff, G, Huasong, Z, Huber, A, Insalaco, A, Ioseliani, M, Jelusic-drazic, M, Jeng, M, Kapovic, A, Kasapcopur, O, Kone-paut, I, De Oliveira, Skf, Lattanzi, B, Lepore, L, Li, C, Lipton, Jm, Magni-manzoni, S, Maritsi, D, Mccurdy, D, Merino, R, Mulaosmanovic, V, Nielsen, S, Pal, P, Prahalad, S, Rigante, Donato, Rumba-rozenfelde, I, Magalhaes, C, Sanner, H, Sawhney, S, Sewairi, Wm, Shakoory, B, Shenoi, S, Clovis, A, Stanevicha, V, Stine, Kc, Susic, G, Sztajnbok, F, Takei, S, Tezer, H, Trauzeddel, R, Tsitsami, E, Unsal, E, Vougiouka, O, Weaver, Lk, Weiss, J, Weitzman, S, Zletni M., On Behalf Of The Pediatric Rheumatology International Trials Organization, The Childhood Arthritis &amp, Rheumatology Research, Alliance, The Pediatric Rheumatology Collaborative Study Group And The Histiocyte, Society, Rigante D (ORCID:0000-0001-7032-7779), Ravelli, A, Minoia, F, Davì, S, Horne, A, Bovis, F, Pistorio, A, Aricò, M, Avcin, T, Behrens, Em, De Benedetti, F, Filipovic, A, Grom, Aa, Henter, J-i, Ilowite, Nte, Jordan, Mb, Khubchandani, R, Kitoh, T, Lehmberg, K, Lovell, Dj, Miettunen, P, Nichols, Ke, Ozen, S, Schmid, Jp, Ramanan, Av, Russo, R, Schneider, R, Sterba, G, Uziel, Y, Wallace, C, Wouters, C, Wulffraat, N, Demirkaya, E, Brunner, Hi, Martini, A, Ruperto, N, Cron, Rq, Angioloni, S, Pallotti, C, Pesce, M, Rinaldi, M, Villa, L, Abinun, M, Aggarwal, A, Akikusa, J, Al-mayouf, Sm, Alessio, M, Anton, J, Apaz, Mt, Astigarraga, I, Ayaz, Na, Barone, P, Bica, B, Bolt, I, Breda, L, Chasnyk, V, Cimaz, R, Corona, F, Cuttica, R, D'Angelo, G, Davidsone, Z, De Cunto, C, De Inocencio, J, Eisenstein, E, Enciso, S, Espada, G, Fischbach, M, Frosch, M, Gallizzi, R, Gamir, Ml, Gao, Y-j, Griffin, T, Hashad, S, Hennon, T, Horneff, G, Huasong, Z, Huber, A, Insalaco, A, Ioseliani, M, Jelusic-drazic, M, Jeng, M, Kapovic, A, Kasapcopur, O, Kone-paut, I, De Oliveira, Skf, Lattanzi, B, Lepore, L, Li, C, Lipton, Jm, Magni-manzoni, S, Maritsi, D, Mccurdy, D, Merino, R, Mulaosmanovic, V, Nielsen, S, Pal, P, Prahalad, S, Rigante, Donato, Rumba-rozenfelde, I, Magalhaes, C, Sanner, H, Sawhney, S, Sewairi, Wm, Shakoory, B, Shenoi, S, Clovis, A, Stanevicha, V, Stine, Kc, Susic, G, Sztajnbok, F, Takei, S, Tezer, H, Trauzeddel, R, Tsitsami, E, Unsal, E, Vougiouka, O, Weaver, Lk, Weiss, J, Weitzman, S, Zletni M., On Behalf Of The Pediatric Rheumatology International Trials Organization, The Childhood Arthritis &amp, Rheumatology Research, Alliance, The Pediatric Rheumatology Collaborative Study Group And The Histiocyte, Society, and Rigante D (ORCID:0000-0001-7032-7779)

Abstract

Objective: To identify which laboratory tests that change over time are most valuable for the timely diagnosis of macrophage activation syndrome (MAS) complicating systemic juvenile idiopathic arthritis (sJIA). Methods: A multistep process, based on a combination of expert consensus and analysis of real patient data, was conducted. A panel of experts was first asked to evaluate 115 profiles of patients with MAS, which included the values of laboratory tests at the pre-MAS visit and at MAS onset, and the change in values between the two time points. The experts were asked to choose the 5 laboratory tests in which change was most important for the diagnosis of MAS and to rank the 5 selected tests in order of importance. The relevance of change in laboratory parameters was further discussed and ranked by the same experts at a consensus conference. Results: Platelet count was the most frequently selected test, followed by ferritin level, aspartate aminotransferase (AST), white cell count, neutrophil count, and fibrinogen and erythrocyte sedimentation rate. Ferritin was most frequently assigned the highest score. At the end of the process, platelet count, ferritin level and AST were the laboratory tests in which the experts found change over time to be most important. Conclusions: We identified the laboratory tests in which change over time is most valuable for the early diagnosis of MAS in sJIA. The dynamics of laboratory values during the course of MAS should be further scrutinised in a prospective study in order to establish the optimal cut-off values for their variation.

Published
2017

21. Development and initial validation of the macrophage activation syndrome/primary hemophagocytic lymphohistiocytosis score, a diagnostic tool that differentiates primary hemophagocytic lymphohistiocytosis from macrophage activation syndrome

Author

Minoia, F, Bovis, F, Davì, S, Insalaco, A, Lehmberg, K, Shenoi, S, Weitzman, S, Espada, G, Gao, Yj, Anton, J, Kitoh, T, Kasapcopur, O, Sanner, H, Merino, R, Astigarraga, I, Alessio, M, Jeng, M, Chasnyk, V, Nichols, Ke, Huasong, Z, Li, C, Micalizzi, C, Ruperto, N, Martini, A, Cron, Rq, Ravelli, A, Horne, A, Abinun, M, Aggarwal, A, Akikusa, J, Al Mayouf, S, Apaz, Mt, Avcin, T, Ayaz, Na, Barone, P, Bica, B, Bolt, I, Breda, L, Cimaz, R, Corona, F, Cuttica, R, Davidsone, Z, De Cunto, C, De Inocencio, J, Demirkaya, E, Eisenstein, Em, Enciso, S, Fischbach, M, Frosch, M, Gallizzi, R, Gamir, Ml, Griffin, T, Grom, A, Hashad, S, Hennon, T, Henter, Ji, Horneff, G, Huber, A, Ilowite, N, Ioseliani, M, Kapović, Am, Khubchandani, R, Koné Paut, I, de Oliveira, Skf, Lattanzi, B, Lepore, L, Lipton, Jm, Magni Manzoni, S, Maritsi, D, Mccurdy, D, Miettunen, P, Mulaosmanovic, V, Nielsen, S, Ozen, S, Pal, P, Prahalad, S, Rigante, Donato, Rumba Rozenfelde, I, Russo, R, Magalhães, C, Sewairi, Wm, Artur Silva, C, Stanevicha, V, Sterba, G, Stine, Kc, Susic, G, Sztajnbok, F, Takei, S, Trauzeddel, R, Tsitsami, E, Unsal, E, Uziel, Y, Vougiouka, O, Wallace, Ca, Weaver, L, Weiss J, E, Wouters, C, Wulffraat, N, Zletni, M, Aricò, M, Egeler, Rm, Filipovich, Ah, Gadner, H, Imashuku, S, Janka, G, Ladisch, S, Mcclain, Kl, Webb, D., Rigante, Donato (ORCID:0000-0001-7032-7779), Minoia, F, Bovis, F, Davì, S, Insalaco, A, Lehmberg, K, Shenoi, S, Weitzman, S, Espada, G, Gao, Yj, Anton, J, Kitoh, T, Kasapcopur, O, Sanner, H, Merino, R, Astigarraga, I, Alessio, M, Jeng, M, Chasnyk, V, Nichols, Ke, Huasong, Z, Li, C, Micalizzi, C, Ruperto, N, Martini, A, Cron, Rq, Ravelli, A, Horne, A, Abinun, M, Aggarwal, A, Akikusa, J, Al Mayouf, S, Apaz, Mt, Avcin, T, Ayaz, Na, Barone, P, Bica, B, Bolt, I, Breda, L, Cimaz, R, Corona, F, Cuttica, R, Davidsone, Z, De Cunto, C, De Inocencio, J, Demirkaya, E, Eisenstein, Em, Enciso, S, Fischbach, M, Frosch, M, Gallizzi, R, Gamir, Ml, Griffin, T, Grom, A, Hashad, S, Hennon, T, Henter, Ji, Horneff, G, Huber, A, Ilowite, N, Ioseliani, M, Kapović, Am, Khubchandani, R, Koné Paut, I, de Oliveira, Skf, Lattanzi, B, Lepore, L, Lipton, Jm, Magni Manzoni, S, Maritsi, D, Mccurdy, D, Miettunen, P, Mulaosmanovic, V, Nielsen, S, Ozen, S, Pal, P, Prahalad, S, Rigante, Donato, Rumba Rozenfelde, I, Russo, R, Magalhães, C, Sewairi, Wm, Artur Silva, C, Stanevicha, V, Sterba, G, Stine, Kc, Susic, G, Sztajnbok, F, Takei, S, Trauzeddel, R, Tsitsami, E, Unsal, E, Uziel, Y, Vougiouka, O, Wallace, Ca, Weaver, L, Weiss J, E, Wouters, C, Wulffraat, N, Zletni, M, Aricò, M, Egeler, Rm, Filipovich, Ah, Gadner, H, Imashuku, S, Janka, G, Ladisch, S, Mcclain, Kl, Webb, D., and Rigante, Donato (ORCID:0000-0001-7032-7779)

Abstract

OBJECTIVE: To develop and validate a diagnostic score that assists in discriminating primary hemophagocytic lymphohistiocytosis (pHLH) from macrophage activation syndrome (MAS) related to systemic juvenile idiopathic arthritis. STUDY DESIGN: The clinical, laboratory, and histopathologic features of 362 patients with MAS and 258 patients with pHLH were collected in a multinational collaborative study. Eighty percent of the population was assessed to develop the score and the remaining 20% constituted the validation sample. Variables that entered the best fitted model of logistic regression were assigned a score, based on their statistical weight. The MAS/HLH (MH) score was made up with the individual scores of selected variables. The cutoff in the MH score that discriminated pHLH from MAS best was calculated by means of receiver operating characteristic curve analysis. Score performance was examined in both developmental and validation samples. RESULTS: Six variables composed the MH score: age at onset, neutrophil count, fibrinogen, splenomegaly, platelet count, and hemoglobin. The MH score ranged from 0 to 123, and its median value was 97 (1st-3rd quartile 75-123) and 12 (1st-3rd quartile 11-34) in pHLH and MAS, respectively. The probability of a diagnosis of pHLH ranged from <1% for a score of <11 to >99% for a score of ≥123. A cutoff value of ≥60 revealed the best performance in discriminating pHLH from MAS. CONCLUSION: The MH score is a powerful tool that may aid practitioners to identify patients who are more likely to have pHLH and, thus, could be prioritized for functional and genetic testing.

Published
2017

22. SAT0636 Ultrasonography definitions for synovitis grading in children: the omeract pediatric ultrasound task force

Author

Vojinovic, J, primary, Magni-Manzoni, S, additional, Collado, P, additional, Windschall, D, additional, Ravagnani, V, additional, Hernandez-Diaz, C, additional, Gonzales, JC Nieto, additional, Malattia, C, additional, Tzaribachev, N, additional, Susic, G, additional, Damjanov, N, additional, Czitrom, S Guillaume, additional, Herlin, T, additional, Lanni, S, additional, Bruyn, G, additional, Iagnocco, A, additional, Terslev, L, additional, D'Agostino, M-A, additional, and Naredo, E, additional

Published
2017
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25. OP0217 Adjudication of Infections in The Pharmacovigilance in Juvenile Idiopathic Arthritis Patients (Pharmachild) Treated with Biologic Agents and/or Methotrexate

Author

Swart, J., primary, Giancane, G., additional, Bovis, F., additional, Castagnola, E., additional, Groll, A., additional, Horneff, G., additional, Huppertz, H.-I., additional, Lovell, D., additional, Wolfs, T., additional, Hofer, M., additional, Alekseeva, E., additional, Panaviene, V., additional, Nielsen, S., additional, Anton, J., additional, Uettwiller, F., additional, Stanevicha, V., additional, Trachana, M., additional, De Benedetti, F., additional, Ailioaie, L.M., additional, Tsitsami, E., additional, Kamphuis, S., additional, Herlin, T., additional, Dolezalova, P., additional, Susic, G., additional, Flato, B., additional, Sztajnbok, F., additional, Pistorio, A., additional, Martini, A., additional, Wulffraat, N., additional, and Ruperto, N., additional

Published
2016
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26. The validated printo core set and definition of improvement for juvenile myositis

Author

Ruperto N, Woo P, Cuttica R, Cortis E, Porras O, Garay S, Sztajnbok F, Ozdogan H, Wulffraat N, Kanakoudi F, Quezada A, Gerloni V, Falcini F, Venning H, Susic G, Prieur AM, Ferriani V, Hofer M, De Conto C, Rider L, Trail L, Pistorio A, Giannini EH, Martini A., ALESSIO, MARIA, Ruperto, N, Woo, P, Cuttica, R, Cortis, E, Alessio, Maria, Porras, O, Garay, S, Sztajnbok, F, Ozdogan, H, Wulffraat, N, Kanakoudi, F, Quezada, A, Gerloni, V, Falcini, F, Venning, H, Susic, G, Prieur, Am, Ferriani, V, Hofer, M, De Conto, C, Rider, L, Trail, L, Pistorio, A, Giannini, Eh, and Martini, A.

Published
2004

27. Treatment of autoinflammatory diseases: results from the Eurofever Registry and a literature review

Author

Haar, Nienke Ter, Lachmann, Helen, Özen, Seza, Woo, Pat, Uziel, Yosef, Modesto, Consuelo, Koné Paut, Isabelle, Cantarini, Luca, Insalaco, Antonella, Neven, Bénédicte, Hofer, Michael, Rigante, Donato, Al Mayouf, Sulaiman, Touitou, Isabelle, Gallizzi, Romina, Papadopoulou Alataki, Efimia, Martino, Silvana, Kuemmerle Deschner, Jasmin, Obici, Laura, Iagaru, Nicolae, Simon, Anna, Nielsen, Susan, Martini, Alberto, Ruperto, Nicolino, Gattorno, Marco, Frenkel, Joost, Kondi, A, De Cunto, C, Espada, G, Russo, R, Amaryan, G, Boros, C, Wouters, C, de Oliveira SK, Borzutzky, A, Jelusic Drazic, M, Dolezalova, P, Herlin, T, Desjonqueres, M, Djeddi, D, Hentgen, V, Darce, M, Ioseliani, M, Berendes, R, Horneff, G, Jansson, A, Minden, K, Schwarz, T, Trauzeddel, R, Kanakoudi Tsakalidou, F, Vougiouka, O, Constantin, T, Rao, Ap, Brik, R, Harel, L, Alessio, M, Breda, L, Cimaz, R, Consolini, Rita, Fabio, G, Garozzo, R, Lepore, L, Manna, R, Meini, A, Olivieri, An, Stanevicha, V, Rusoniene, S, Hoppenreijs, E, Al Abrawi Sy, Nikishina, I, Sewairi, Wm, Susic, G, Ciznar, P, Avcin, T, Anton, J, Bou, R, Merino, R, Elorduy, Mj, Fasth, A, Aksu, G, Demirkaya, E., Ter Haar, N., Lachmann, H., Özen, S., Woo, P., Uziel, Y., Modesto, C., Koné Paut, I., Cantarini, L., Insalaco, A., Neven, B., Hofer, M., Rigante, D., Al Mayouf, S., Touitou, I., Gallizzi, R., Papadopoulou Alataki, E., Martino, S., Kuemmerle Deschner, J., Obici, L., Iagaru, N., Simon, A., Nielsen, S., Martini, A., Ruperto, N., Gattorno, M., Frenkel, J., and Olivieri, Alma Nunzia

Subjects
Genetics and Molecular Biology (all), medicine.medical_specialty, PFAPA syndrome, Immunology, autoinflammatory diseases, Eurofever, Registry, Familial Mediterranean fever, Disease, Biochemistry, General Biochemistry, Genetics and Molecular Biology, 03 medical and health sciences, 0302 clinical medicine, Rheumatology, autoinflammatory disease, Internal medicine, Acne Vulgaris, medicine, Immunology and Allergy, Humans, Registries, 030304 developmental biology, 030203 arthritis & rheumatology, 0303 health sciences, Arthritis, Infectious, Mevalonate kinase deficiency, business.industry, Hyper-IgD syndrome, Arthritis, Settore MED/09 - MEDICINA INTERNA, Infectious, Cryopyrin-associated periodic syndrome, medicine.disease, Cryopyrin-Associated Periodic Syndromes, Pyoderma Gangrenosum, 3. Good health, Familial Mediterranean Fever, Pathogenesis and modulation of inflammation [N4i 1], Europe, TNF receptor associated periodic syndrome, Mevalonate Kinase Deficiency, Biochemistry, Genetics and Molecular Biology (all), business
Abstract

Item does not contain fulltext OBJECTIVE: To evaluate the response to treatment of autoinflammatory diseases from an international registry and an up-to-date literature review. METHODS: The response to treatment was studied in a web-based registry in which clinical information on anonymised patients with autoinflammatory diseases was collected retrospectively as part of the Eurofever initiative. Participating hospitals included paediatric rheumatology centres of the Paediatric Rheumatology International Trial Organisation network and adult centres with a specific interest in autoinflammatory diseases. The following diseases were included: familial Mediterranean fever (FMF), cryopyrin-associated periodic syndromes (CAPS), tumour necrosis factor (TNF)-receptor associated periodic syndrome (TRAPS), mevalonate kinase deficiency (MKD), pyogenic arthritis pustulosis acne (PAPA) syndrome, deficiency of interleukin-1 receptor antagonist (DIRA), NLRP12-related periodic fever and periodic fever aphthosis pharyngitis adenitis (PFAPA) syndrome. Cases were independently validated by experts for each disease. A literature search regarding treatment of the abovementioned diseases was also performed using Medline and Embase. RESULTS: 22 months from the beginning of the enrolment, complete information on 496 validated patients was available. Data from the registry in combination with evidence from the literature confirmed that colchicine is the treatment of choice for FMF and IL-1 blockade for DIRA and CAPS. Corticosteroids on demand probably represent a valid therapeutic strategy for PFAPA, but also for MKD and TRAPS. Patients with poorly controlled MKD, TRAPS, PAPA or FMF may benefit from IL-1 blockade; anti-TNF treatment may represent a possible valuable alternative. CONCLUSIONS: In the absence of high-grade evidence, these results could serve as a basis for therapeutic guidelines and to identify candidate drugs for future therapeutic trials.

Published
2013

29. Health related quality of life measure in systemic pediatric rheumatic diseases and its translation to different languages: An international collaboration

Author

Moorthy, L. N., Roy, E., Kurra, V., Peterson, M. G. E., Hassett, A. L., Lehman, T. J. A., Scott, C., El-Ghoneimy, D., Saad, S., El Feky, R., Al-Mayouf, S., Dolezalova, P., Malcova, H., Herlin, T., Nielsen, S., Wulffraat, N., van Royen, A., Marks, S. D., Belot, A., Brunner, J., Huemer, C., Foeldvari, I., Horneff, G., Saurenman, T., Schroeder, S., Pratsidou-Gertsi, P., Trachana, M., Uziel, Y., Aggarwal, A., Constantin, T., Cimaz, R., Giani, T., Cantarini, L., Falcini, F., Manzoni, S. M., Ravelli, A., Rigante, Donato, Zulian, F., Miyamae, T., Yokota, S., Sato, J., Magalhaes, C. S., Len, C. A., Appenzeller, S., Knupp, S. O., Rodrigues, M. C., Sztajnbok, F., de Almeida, R. G., de Jesus, A. A., de Arruda Campos, L. M., Silva, C., Lazar, C., Susic, G., Avcin, T., Cuttica, R., Burgos-Vargas, R., Faugier, E., Anton, J., Modesto, C., Vazquez, L., Barillas, L., Barinstein, L., Sterba, G., Maldonado, I., Ozen, S., Kasapcopur, O., Demirkaya, E., Benseler, S., Rigante D. (ORCID:0000-0001-7032-7779), Moorthy, L. N., Roy, E., Kurra, V., Peterson, M. G. E., Hassett, A. L., Lehman, T. J. A., Scott, C., El-Ghoneimy, D., Saad, S., El Feky, R., Al-Mayouf, S., Dolezalova, P., Malcova, H., Herlin, T., Nielsen, S., Wulffraat, N., van Royen, A., Marks, S. D., Belot, A., Brunner, J., Huemer, C., Foeldvari, I., Horneff, G., Saurenman, T., Schroeder, S., Pratsidou-Gertsi, P., Trachana, M., Uziel, Y., Aggarwal, A., Constantin, T., Cimaz, R., Giani, T., Cantarini, L., Falcini, F., Manzoni, S. M., Ravelli, A., Rigante, Donato, Zulian, F., Miyamae, T., Yokota, S., Sato, J., Magalhaes, C. S., Len, C. A., Appenzeller, S., Knupp, S. O., Rodrigues, M. C., Sztajnbok, F., de Almeida, R. G., de Jesus, A. A., de Arruda Campos, L. M., Silva, C., Lazar, C., Susic, G., Avcin, T., Cuttica, R., Burgos-Vargas, R., Faugier, E., Anton, J., Modesto, C., Vazquez, L., Barillas, L., Barinstein, L., Sterba, G., Maldonado, I., Ozen, S., Kasapcopur, O., Demirkaya, E., Benseler, S., and Rigante D. (ORCID:0000-0001-7032-7779)

Abstract

Background: Rheumatic diseases in children are associated with significant morbidity and poor health-related quality of life (HRQOL). There is no health-related quality of life (HRQOL) scale available specifically for children with less common rheumatic diseases. These diseases share several features with systemic lupus erythematosus (SLE) such as their chronic episodic nature, multi-systemic involvement, and the need for immunosuppressive medications. HRQOL scale developed for pediatric SLE will likely be applicable to children with systemic inflammatory diseases. Findings: We adapted Simple Measure of Impact of Lupus Erythematosus in Youngsters (SMILEY) to Simple Measure of Impact of Illness in Youngsters (SMILY-Illness) and had it reviewed by pediatric rheumatologists for its appropriateness and cultural suitability. We tested SMILY-Illness in patients with inflammatory rheumatic diseases and then translated it into 28 languages. Conclusion: SMILY-Illness is a brief, easy to administer and score HRQOL scale for children with systemic rheumatic diseases. It is suitable for use across different age groups and literacy levels. SMILY-Illness with its available translations may be used as useful adjuncts to clinical practice and research.

Published
2014

30. Performance of current guidelines for diagnosis of macrophage activation syndrome complicating systemic juvenile idiopathic arthritis

Author

Davì, S, Minoia, F, Pistorio, A, Horne, A, Consolaro, A, Rosina, S, Bovis, F, Cimaz, R, Gamir, Ml, Ilowite, N, Kone Paut, I, Feitosa De Oliveira, Sk, Mccurdy, D, Silva, Ca, Sztajnbok, F, Tsitsami, E, Unsal, E, Weiss, Je, Wulffraat, N, Abinun, M, Aggarwal, A, Apaz, Mt, Astigarraga, I, Corona, F, Cuttica, R, D'Angelo, G, Eisenstein, Em, Hashad, S, Lepore, L, Mulaosmanovic, V, Nielsen, S, Prahalad, S, Rigante, Donato, Stanevicha, V, Sterba, G, Susic, G, Takei, S, Trauzeddel, R, Zletni, M, Ruperto, N, Martini, A, Cron, Rq, Ravelli, A., Rigante, Donato (ORCID:0000-0001-7032-7779), Davì, S, Minoia, F, Pistorio, A, Horne, A, Consolaro, A, Rosina, S, Bovis, F, Cimaz, R, Gamir, Ml, Ilowite, N, Kone Paut, I, Feitosa De Oliveira, Sk, Mccurdy, D, Silva, Ca, Sztajnbok, F, Tsitsami, E, Unsal, E, Weiss, Je, Wulffraat, N, Abinun, M, Aggarwal, A, Apaz, Mt, Astigarraga, I, Corona, F, Cuttica, R, D'Angelo, G, Eisenstein, Em, Hashad, S, Lepore, L, Mulaosmanovic, V, Nielsen, S, Prahalad, S, Rigante, Donato, Stanevicha, V, Sterba, G, Susic, G, Takei, S, Trauzeddel, R, Zletni, M, Ruperto, N, Martini, A, Cron, Rq, Ravelli, A., and Rigante, Donato (ORCID:0000-0001-7032-7779)

Abstract

OBJECTIVE: To compare the capacity of the 2004 diagnostic guidelines for hemophagocytic lymphohistiocytosis (HLH-2004) with the capacity of the preliminary diagnostic guidelines for systemic juvenile idiopathic arthritis (JIA)-associated macrophage activation syndrome (MAS) to discriminate MAS complicating systemic JIA from 2 potentially confusable conditions, represented by active systemic JIA without MAS and systemic infection. METHODS: International pediatric rheumatologists and hemato-oncologists were asked to retrospectively collect clinical information from patients with systemic JIA-associated MAS and confusable conditions. The ability of the guidelines to differentiate MAS from the control diseases was evaluated by calculating the sensitivity and specificity of each set of guidelines and the kappa statistics for concordance with the physician's diagnosis. Owing to the fact that not all patients were assessed for hemophagocytosis on bone marrow aspirates and given the lack of data on natural killer cell activity and soluble CD25 levels, the HLH-2004 guidelines were adapted to enable the diagnosis of MAS when 3 of 5 of the remaining items (3/5-adapted) or 4 of 5 of the remaining items (4/5-adapted) were present. RESULTS: The study sample included 362 patients with systemic JIA and MAS, 404 patients with active systemic JIA without MAS, and 345 patients with systemic infection. The best capacity to differentiate MAS from systemic JIA without MAS was found when the preliminary MAS guidelines were applied. The 3/5-adapted HLH-2004 guidelines performed better than the 4/5-adapted guidelines in distinguishing MAS from active systemic JIA without MAS. The 3/5-adapted HLH-2004 guidelines and the preliminary MAS guidelines with the addition of ferritin levels ≥500 ng/ml discriminated best between MAS and systemic infections. CONCLUSION: The preliminary MAS guidelines showed the strongest ability to identify MAS in systemic JIA. The addition of hyperferritinemia enhanc

Published
2014

31. Genetic diversity in connected reintroduced population: long term data on Griffon vulture (Gyps fulvus) in Europe

Author

Le Gouar, P., Rigal, F., Marie-Catherine Boisselier-Dubayle, Sarrazin, F., Arthur, C., Choisy, J. P., Hatzofe, O., Henriquet, S., Lécuyer, P., Tessier, C., Susic, G., Sarah Samadi, Systématique, adaptation, évolution (SAE), and Université Pierre et Marie Curie - Paris 6 (UPMC)-Centre National de la Recherche Scientifique (CNRS)

Subjects
[SDV.BBM]Life Sciences [q-bio]/Biochemistry, Molecular Biology, ComputingMilieux_MISCELLANEOUS
Abstract

International audience

Published
2008

32. Safety and efficacy of an oral histone deacetylase inhibitor in systemic-onset juvenile idiopathic arthritis.

Author

Vojinovic, J., Damjanov, N., D'Urzo, C., Furlan, A., Susic, G., Pasic, S., Iagaru, N., Stefan, M., Dinarello, C.A., Vojinovic, J., Damjanov, N., D'Urzo, C., Furlan, A., Susic, G., Pasic, S., Iagaru, N., Stefan, M., and Dinarello, C.A.

Abstract

1 mei 2011, Item does not contain fulltext, OBJECTIVE: The current treatment options for systemic-onset juvenile idiopathic arthritis (JIA) are methotrexate, steroids, and biologic agents. This study was undertaken to evaluate the safety of the orally active histone deacetylase inhibitor givinostat (ITF2357) and its ability to affect the disease. METHODS: Givinostat was administered orally, for up to 12 weeks at a dosage of 1.5 mg/kg/day, to 17 patients with systemic-onset JIA who had had active disease for >/=1 month. Disease activity was clinically assessed using the American College of Rheumatology Pediatric 30 (ACR Pedi 30), ACR Pedi 50, or ACR Pedi 70 criteria for improvement and a systemic feature score. The primary goal was safety and the primary efficacy end point was the number of patients completing 12 weeks of treatment who were responders. RESULTS: Givinostat was safe and well tolerated, with adverse events (AEs) being mild or moderate, of short duration, and self-limited. The 17 patients from the intent-to-treat population reported a total of 44 AEs, and the 9 patients in the per-protocol population reported a total of 25. Six AEs in 3 patients (nausea, vomiting, and fatigue) were related to the study drug, but each resolved spontaneously and no patient was withdrawn from the study due to drug-related AEs. In the per-protocol population at week 4, the improvement as measured by the ACR Pedi 30, ACR Pedi 50, and ACR Pedi 70, respectively, was 77.8%, 55.6%, and 22.2%, and this increased further to 77.8%, 77.8%, and 66.7% at week 12. The most consistent finding was the reduction in the number of joints with active disease or with limited range of motion. CONCLUSION: After 12 weeks, givinostat exhibited significant therapeutic benefit in patients with systemic-onset JIA, particularly with regard to the arthritic component of the disease, and showed an excellent safety profile.

Published
2011

33. The Ped-APS Registry: the antiphospholipid syndrome in childhood

Author

Avcin, T., Cimaz, R., Rozman, B., Cervera, R., Ravelli, A., Martini, A., Meroni, P. l., Garay, S., Sztajnbok, F. r., Silva, C. a., Campos, L. m., Saad Magalhaes, C., de Oliveira, S. k., Silverman, E. d., Nielsen, S., Pruunsild, C., Dressler, F., Berkun, Y., Padeh, S., Barash, J., Uziel, Y., Harel, L., Mukamel, M., Revel Vilk, S., Kenet, G., Gattorno, M., Rigante, Donato, Zulian, F., Falcini, F., Kuzmanovska, D. b., Susic, G., Buyukgebiz, A., Ozisik, K., Gozdasoglu, S., Rodriguez, V., Butani, L., Rigante, Donato (ORCID:0000-0001-7032-7779), Avcin, T., Cimaz, R., Rozman, B., Cervera, R., Ravelli, A., Martini, A., Meroni, P. l., Garay, S., Sztajnbok, F. r., Silva, C. a., Campos, L. m., Saad Magalhaes, C., de Oliveira, S. k., Silverman, E. d., Nielsen, S., Pruunsild, C., Dressler, F., Berkun, Y., Padeh, S., Barash, J., Uziel, Y., Harel, L., Mukamel, M., Revel Vilk, S., Kenet, G., Gattorno, M., Rigante, Donato, Zulian, F., Falcini, F., Kuzmanovska, D. b., Susic, G., Buyukgebiz, A., Ozisik, K., Gozdasoglu, S., Rodriguez, V., Butani, L., and Rigante, Donato (ORCID:0000-0001-7032-7779)

Abstract

In recent years, antiphospholipid syndrome (APS) has been increasingly recognised in various paediatric autoimmune and nonautoimmune diseases, but the relatively low prevalence and heterogeneity of APS in childhood made it very difficult to study in a systematic way. The project of an international registry of paediatric patients with APS (the Ped-APS Registry) was initiated in 2004 to foster and conduct multicentre, controlled studies with large number of paediatric APS patients. The Ped-APS Registry is organised as a collaborative project of the European Forum on Antiphospholipid Antibodies and Juvenile Systemic Lupus Erythematosus Working Group of the Paediatric Rheumatology European Society. Currently, it documents a standardised clinical, laboratory and therapeutic data of 133 children with antiphospholipid antibodies (aPL)-related thrombosis from 14 countries. The priority projects for future research of the Ped-APS Registry include prospective enrollment of new patients with aPL-related thrombosis, assessment of differences between the paediatric and adult APS, evaluation of proinflammatory genotype as a risk factor for APS manifestations in childhood and evaluation of patients with isolated nonthrombotic aPL-related manifestations.

Published
2009

47. Systemic sclerosis in childhood: Clinical and immunologic features of 153 patients in an international database.

Author

Martini G, Foeldvari I, Russo R, Cuttica R, Eberhard A, Ravelli A, Lehman TJ, de Oliveira SK, Susic G, Lyskina G, Nemcova D, Sundel R, Falcini F, Girschick H, Lotito AP, Buoncompagni A, Sztajnbok F, Al-Mayouf SM, Orbàn I, and Ferri C

Abstract

OBJECTIVE: To determine the clinical and immunologic features of systemic sclerosis (SSc) in a large group of children and describe the clinical evolution of the disease and compare it with the adult form. METHODS: Data on 153 patients with juvenile SSc collected from 55 pediatric rheumatology centers in Europe, Asia, and South and North America were analyzed. Demographic, clinical, and immunologic characteristics of children with juvenile SSc at the onset, at diagnosis, and during the disease course were evaluated. RESULTS: Raynaud's phenomenon was the most frequent symptom, followed by skin induration in approximately 75% of patients. Musculoskeletal symptoms were present in one-third of patients, and the most frequently involved internal organs were respiratory and gastrointestinal, while involvement of renal, cerebral, and cardiovascular systems was extremely rare. Antinuclear antibodies were present in the sera of 81% of patients. Anti-topoisomerase I (Scl-70) and anticentromere antibodies were found to be positive in 34% and 7.1% of patients, respectively. Involvement of the respiratory, gastrointestinal, and cardiovascular systems was more frequent and occurred earlier in patients who died than in those who survived. Compared with the adult form, juvenile SSc appears to be less severe, with the involvement of fewer internal organs, particularly at the time of diagnosis, and has a less characterized immunologic profile. CONCLUSION: This study provides information on the largest collection of patients with juvenile SSc ever reported. Juvenile SSc appears to be less severe than in adults because children have less internal organ involvement, a less specific autoantibody profile, and a better long-term outcome. [ABSTRACT FROM AUTHOR]

Published
2006
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48. The Pediatric Rheumatology International Trials Organization criteria for the evaluation of response to therapy in juvenile systemic lupus erythematosus: prospective validation of the disease activity core set.

Author

Ruperto N, Ravelli A, Cuttica R, Espada G, Ozen S, Porras O, Sztajnbok F, Falcini F, Kasapcopur O, Venning H, Bica B, Merino R, Coto C, Ros J, Susic G, Gamir ML, Minden K, See Y, Uziel Y, and Mukamel M

Abstract

OBJECTIVE: To validate and promulgate a core set of outcome measures for the evaluation of response to treatment in patients with juvenile systemic lupus erythematosus (SLE). METHODS: In 2001, a preliminary consensus-derived core set of measures for evaluating the response to therapy in juvenile SLE was established. In the present study, the core set was validated through an evidence-based, large-scale data collection process that led to the enrollment of 557 patients from 39 different countries. Consecutive patients with active disease were assessed at baseline and after 6 months. The validation procedures included assessment of feasibility, responsiveness, discriminant and construct ability, agreement in the evaluation of response to therapy between physicians and parents, redundancy, internal consistency, and ability to predict a therapeutic response. RESULTS: The following clinical measures were found to be feasible and to have good construct validity, discriminative ability, and internal consistency; furthermore, they were not redundant, proved responsive to clinically important changes in disease activity, and were associated strongly with treatment outcome and thus were included in the final core set: 1) physician's global assessment of disease activity, 2) global disease activity measure, 3) 24-hour proteinuria, 4) parent's global assessment of the patient's overall well-being, and 5) health-related quality of life assessment. CONCLUSION: The members of PRINTO propose a core set of criteria for the evaluation of response to therapy that is scientifically and clinically relevant and statistically validated. The core set will help standardize the conduct and reporting of clinical trials and assist practitioners in deciding whether a patient with juvenile SLE has responded adequately to therapy. [ABSTRACT FROM AUTHOR]

Published
2005
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50. Clinical characteristics and genetic analyses of 187 patients with undefined autoinflammatory diseases

Author

Joost Frenkel, Valda Stanevica, Michael Hofer, Francesco Licciardi, Antonella Insalaco, Rolando Cimaz, Riccardo Papa, Alma Nunzia Olivieri, Nienke M. ter Haar, Nicolino Ruperto, Susan Nielsen, Paul A. Brogan, Consuelo Modesto, Nicolae Iagaru, Marco Gattorno, Sarka Fingerhutova, Charlotte Eijkelboom, Antonio Vitale, Marielle E. van Gijn, Luca Cantarini, Marija Jelušić, Yosef Uziel, Gordana Susic, Efimia Papadopoulou-Alataki, Isabelle Koné-Paut, Irina Nikishina, Ter Haar, Nm, Eijkelboom, C, Cantarini, L, Papa, R, Brogan, Pa, Kone-Paut, I, Modesto, C, Hofer, M, Iagaru, N, Fingerhutová, S, Insalaco, A, Licciardi, F, Uziel, Y, Jelusic, M, Nikishina, I, Nielsen, S, Papadopoulou-Alataki, E, Olivieri, An, Cimaz, R, Susic, G, Stanevica, V, van Gijn, M, Vitale, A, Ruperto, N, Frenkel, J, and Gattorno, M

Subjects
Male, myalgia, Abdominal pain, Biochemistry, 0302 clinical medicine, eurofever, Adrenal Cortex Hormones, autoinflammatory diseases, inflammation, recurrent fever, Immunology and Allergy, Registries, Age of Onset, Child, 0303 health sciences, Pedigree, 3. Good health, Europe, Child, Preschool, Antirheumatic Agents, Adolescent, Adult, Chronic Disease, Colchicine, Female, Genetic Variation, Hereditary Autoinflammatory Diseases, Humans, Interleukin 1 Receptor Antagonist Protein, Retrospective Studies, Young Adult, medicine.symptom, medicine.drug, medicine.medical_specialty, Immunology, Mucocutaneous zone, General Biochemistry, Genetics and Molecular Biology, Malaise, 03 medical and health sciences, Pericarditis, Rheumatology, Internal medicine, Journal Article, medicine, Recurrent disease, Preschool, 030304 developmental biology, 030203 arthritis & rheumatology, Anakinra, Biochemistry, Genetics and Molecular Biology(all), business.industry, medicine.disease, business, Genetics and Molecular Biology(all)
Abstract

ObjectivesTo describe the clinical characteristics, treatment response and genetic findings in a large cohort of patients with undefined systemic autoinflammatory diseases (SAIDs).MethodsClinical and genetic data from patients with undefined SAIDs were extracted from the Eurofever registry, an international web-based registry that retrospectively collects clinical information on patients with autoinflammatory diseases.ResultsThis study included 187 patients. Seven patients had a chronic disease course, 180 patients had a recurrent disease course. The median age at disease onset was 4.3 years. Patients had a median of 12 episodes per year, with a median duration of 4 days. Most commonly reported symptoms were arthralgia (n=113), myalgia (n=86), abdominal pain (n=89), fatigue (n=111), malaise (n=104) and mucocutaneous manifestations (n=128). In 24 patients, relatives were affected as well. In 15 patients, genetic variants were found in autoinflammatory genes. Patients with genetic variants more often had affected relatives compared with patients without genetic variants (p=0.005). Most patients responded well to non-steroidal anti-inflammatory drugs (NSAIDs), corticosteroids, colchicine and anakinra. Complete remission was rarely achieved with NSAIDs alone. Notable patterns were found in patients with distinctive symptoms. Patients with pericarditis (n=11) were older at disease onset (33.8 years) and had fewer episodes per year (3.0/year) compared with other patients. Patients with an intellectual impairment (n=8) were younger at disease onset (2.2 years) and often had relatives affected (28.6%).ConclusionThis study describes the clinical characteristics of a large cohort of patients with undefined SAIDs. Among these, patients with pericarditis and intellectual impairment appear to comprise distinct subsets.

Published
2019

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