Browsing by Author "Sztajnbok, Flavio"

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    Application and performance of disease activity indices proposed for patients with systemic sclerosis in an international cohort of patients with juvenile systemic sclerosis
    (2023-04-10) Klotsche, Jens; Torok, Kathryn S.; Kasapcopur, Ozgur; Adrovic, Amra; Terreri, Maria Teresa; Sakamoto, Ana Paula; Katsicas, Maria; Sztajnbok, Flavio; Marrani, Edoardo; Sifuentes-Giraldo, Alberto; Stanevicha, Valda; Anton, Jordi; Feldmann, Brian; Kostik, Mikhail; Nemcova, Dana; Santos, Maria Jose; Appenzeller, Simone; Avcin, Tadej; Battagliotti, Cristina; Berntson, Lillemor; Bica, Blanca; Brunner, Jürgen; Eleftheriou, Despina; Harel, Liora; Horneff, Gerd; Kallinich, Tilmann; Minden, Kirsten; Nielsen, Susan; Patwardhan, Anjali; Helmus, Nicola; Foeldvari, Ivan; Department of Paediatrics
    Objectives: Juvenile systemic sclerosis is a rare childhood disease. Three disease activity indices have been published for adult patients with systemic sclerosis: the European Scleroderma Study Group Index, a modified version of the European Scleroderma Study Group Index and the revised European Scleroderma Trials and Research index. The objective of this study was to determine the feasibility and performance of the three disease activity indices in a prospectively followed cohort of patients with juvenile systemic sclerosis. Methods: The analysis cohort was selected from the prospective international inception cohort enrolling juvenile systemic sclerosis patients. The correlation of the disease activity indices with the physicians’ and the patients’ global assessment of disease activity was determined. The disease activity indices were compared between patients with active and inactive disease. Sensitivity to change between 6- and 12-month follow-up was investigated by mixed models. Results: Eighty percent of the 70 patients had a diffuse cutaneous subtype. The revised European Scleroderma Trials and Research index was highly correlated with the physician-reported global disease activity/parents-reported global disease activity (r = 0.74/0.64), followed by the European Scleroderma Study Group activity index (r = 0.61/0.55) and the modified version of the European Scleroderma Study Group activity index (r = 0.51/0.43). The disease activity indices significantly differed between active and inactive patients. The disease activity indices showed sensitivity to change between 6- and 12-month follow-up among patients who improved or worsened according to the physician-reported global disease activity and the parents-reported global disease activity. Conclusion: Overall, no disease activity score is superior to the other, and all three scores have limitations in the application in juvenile systemic sclerosis patients. Furthermore, research on the concept of disease activity and suitable scores to measure disease activity in patients with juvenile systemic sclerosis is necessary in future.
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    Defining Criteria for Disease Activity States in Systemic Juvenile Idiopathic Arthritis Based on the Systemic Juvenile Arthritis Disease Activity Score
    (2024-09) Rosina, Silvia; Rebollo-Giménez, Ana I; Tarantola, Letizia; Pistorio, Angela; Vyzhga, Yulia; El Miedany, Yasser; Lotfy, Hala M; Abu-Shady, Hend; Eissa, Mervat; Osman, Naglaa S; Hassan, Waleed; Mahgoub, Marwa Y; Fouad, Nermeen A; Mosa, Doaa M; Adel, Yasmin; Mohamed, Sheren E M; Radwan, Ahmed R; Abu-Zaid, Mohamed H; Tabra, Samar A A; Shalaby, Radwa H; Nasef, Samah I; Khubchandani, Raju; Khan, Archana; Maldar, Naziya P; Ozen, Seza; Bayindir, Yagmur; Alsuweiti, Motasem; Alzyoud, Raed; Almaaitah, Hiba; Vilaiyuk, Soamarat; Lerkvaleekul, Butsabong; Alexeeva, Ekaterina; Dvoryakovskaya, Tatyana; Kriulin, Ivan; Bracaglia, Claudia; Pardeo, Manuela; De Benedetti, Fabrizio; Licciardi, Francesco; Montin, Davide; Robasto, Francesca; Minoia, Francesca; Filocamo, Giovanni; Rossano, Martina; Simonini, Gabriele; Marrani, Edoardo; Abu-Rumeileh, Sarah; Kostik, Mikhail M; Belozerov, Konstantin E; Pal, Priyankar; Bathia, Jigna N; Katsicas, María M; Villarreal, Giselle; Marino, Achille; Costi, Stefania; Sztajnbok, Flavio; Silva, Rodrigo M; Maggio, Maria C; El-Ghoneimy, Dalia H; El Owaidy, Rasha; Civino, Adele; Diomeda, Federico; Al-Mayouf, Sulaiman M; Al-Sofyani, Fuad; Dāvidsone, Zane; Patrone, Elisa; Saad-Magalhães, Claudia; Consolaro, Alessandro; Ravelli, Angelo
    OBJECTIVE: Our objective was to develop and validate cutoff values in the systemic Juvenile Arthritis Disease Activity Score 10 (sJADAS10) that distinguish the states of inactive disease (ID), minimal disease activity (MDA), moderate disease activity (MoDA), and high disease activity (HDA) in children with systemic juvenile idiopathic arthritis, based on subjective disease state assessment by the treating pediatric rheumatologist. METHODS: The cutoff definition cohort was composed of 400 patients enrolled at 30 pediatric rheumatology centers in 11 countries. Using the subjective physician rating as an external criterion, six methods were applied to identify the cutoffs: mapping, calculation of percentiles of cumulative score distribution, the Youden index, 90% specificity, maximum agreement, and receiver operating characteristic curve analysis. Sixty percent of the patients were assigned to the definition cohort, and 40% were assigned to the validation cohort. Cutoff validation was conducted by assessing discriminative ability. RESULTS: The sJADAS10 cutoffs that separated ID from MDA, MDA from MoDA, and MoDA from HDA were ≤2.9, ≤10, and >20.6, respectively. The cutoffs discriminated strongly among different levels of pain, between patients with and without morning stiffness, and among patients whose parents judged their disease status as remission or persistent activity or flare or were satisfied or not satisfied with current illness outcome. CONCLUSION: The sJADAS cutoffs revealed good metrologic properties in both definition and validation cohorts and are therefore suitable for use in clinical trials and routine practice.
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    Performance of current guidelines for diagnosis of macrophage activation syndrome complicating systemic juvenile idiopathic arthritis
    (2014-10-01) Davì, Sergio; Minoia, Francesca; Pistorio, Angela; Horne, Annacarin; Consolaro, Alessandro; Rosina, Silvia; Bovis, Francesca; Cimaz, Rolando; Gamir, Maria Luz; Ilowite, Norman T.; Kone-Paut, Isabelle; Feitosa De Oliveira, Sheila Knupp; McCurdy, Deborah; Silva, Clovis Artur; Sztajnbok, Flavio; Tsitsami, Elena; Unsal, Erbil; Weiss, Jennifer E.; Wulffraat, Nico; Abinun, Mario; Aggarwal, Amita; Apaz, Maria Teresa; Astigarraga, Itziar; Corona, Fabrizia; Cuttica, Ruben; D'Angelo, Gianfranco; Eisenstein, Eli M.; Hashad, Soad; Lepore, Loredana; Mulaosmanovic, Velma; Nielsen, Susan; Prahalad, Sampath; Rigante, Donato; Stanevicha, Valda; Sterba, Gary; Susic, Gordana; Takei, Syuji; Trauzeddel, Ralf; Zletni, Mabruka; Ruperto, Nicolino; Martini, Alberto; Cron, Randy Q.; Ravelli, Angelo; Rīga Stradiņš University
    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 enhanced their capacity to differentiate MAS from systemic infections. The HLH-2004 guidelines are likely not appropriate for identification of MAS in children with systemic JIA. 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.