Zusammenfassung
Prospektive Kohortenstudien, in denen demografische und klinischen Daten neu erkrankter Patienten mittels standardisierter Fragebögen und anhand validierter Messinstrumente erfasst werden, stellen eine wertvolle Datenquelle dar, um den Krankheitsverlauf, Outcome-Parameter und Prädiktoren zu evaluieren. Im Folgenden wird eine Auswahl an Ergebnissen aus 4 Inzeptionskohorten zur JIA dargestellt. In allen Kohorten erreicht schon im ersten Jahr unter Beobachtung die Hälfte bis drei Viertel der Patienten eine inaktive Erkrankung, wobei es relevante Unterschiede zwischen den verschiedenen JIA(juvenile idiopathische Arthritis)-Kategorien gibt. Als wichtiger Prädiktor für dieses Outcome hat sich die Zeit vom Symptombeginn bis zur Diagnosestellung erwiesen. Daten aus der deutschen JIA-Kohorte zeigen, dass sich die gesundheitsbezogene Lebensqualität bei Patienten 3 Jahre nach Einschluss der von gesunden Kontrollpersonen angeglichen hat. Jung erkrankte Kinder mit Nachweis von antinukleären Antikörpern haben ein erhöhtes Risiko, eine JIA-assoziierte Uveitis zu entwickeln. Von diesen ist bei ca. 80 % nach 1 Jahr die Uveitis inaktiv; allerdings ist bereits bei Diagnosestellung bei knapp 30 % der Patienten eine uveitisbedingte Komplikation vorhanden. Als präventiv für die Entwicklung einer Uveitis hat sich die vorangehende Therapie mit Methotrexat herausgestellt. Das frühe Outcome der JIA-Patienten ist insgesamt gut. Die Unterschiede in den JIA-Kategorien deuten auf die Notwendigkeit hin, die Therapie weiter zu individualisieren und dem Risikoprofil des einzelnen Patienten besser anzupassen. Daten zum Langzeit-Outcome werden Aufschluss darüber geben können, welche Faktoren entscheidenden Einfluss auf den Krankheitsverlauf nehmen und wie die Versorgung von Kindern und Jugendlichen mit JIA weiter verbessert werden kann.
Abstract
Prospective cohort studies collect demographic and clinical data of newly diagnosed patients using standardized questionnaires and validated measuring instruments. Therefore, they are a valuable data source for evaluating disease progression, outcome parameters and predictors. In this article a selection of results from four inception cohorts on juvenile idiopathic arthritis (JIA) are presented. In all cohorts, one half to three quarters of the patients achieved an inactive disease within the first year under observation but there were relevant differences between the different JIA categories. The time from symptom onset to diagnosis could be identified as an important predictor of this outcome. Data from the German JIA cohort showed that the health-related quality of life of patients and healthy control subjects had largely converged 3 years after inclusion. Young children with JIA and the detection of antinuclear antibodies have an increased risk of developing JIA-associated uveitis. Of these, the uveitis was inactive in approximately 80% after 1 year; however, at the time of diagnosis, almost 30% of patients already had uveitis-related complications. The previous therapy with methotrexate proved to be preventive for the development of uveitis. The early outcome of JIA patients is generally good. The differences in the JIA categories indicate the need to further individualize the therapy and to adapt it better to the risk profile of the individual patient. Data on long-term outcomes will provide information on which factors have a decisive influence on the course of the disease and how the care of children and adolescents with JIA can be further improved.
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Danksagung
Ein großer Dank gilt allen Patientinnen und Patienten und deren Eltern, allen Ärztinnen und Ärzten sowie allen Mitarbeiterinnen und Mitarbeitern im Deutschen Rheumaforschungszentrum, die sich an der ICON-Studie beteiligen. Aus den Studienzentren: Tilmann Kallinich, Berlin; Hans-Iko Huppertz, Bremen; Johannes-Peter Haas, Garmisch-Partenkirchen; Ivan Foeldvari, Hamburg; Angelika Thon, Hannover; Kirsten Mönkemöller, Köln; Dirk Foell, Münster; Arnd Heiligenhaus, Münster; Gerd Ganser, Sendenhorst; Gerd Horneff, St. Augustin; Anton Hospach, Stuttgart; Jasmin Kümmerle-Deschner, Tübingen.
Funding
ICON wird gefördert durch das Bundesministerium für Bildung und Forschung (Förderkennzeichen 01 ER 1504A, 01 ER 1504B, 01 ER 1504C).
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C. Sengler gibt an, dass kein Interessenkonflikt besteht.
Das Studienprotokoll wurde von der Ethikkommission der Charité – Universitätsmedizin Berlin genehmigt. Eltern und Patienten/Patientinnen ab 8 Jahre erteilten schriftlich ihr Einverständnis zur Teilnahme.
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H.-I. Huppertz, Bremen
K. Minden, Berlin
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Sengler, C. Neue Therapiewege, bessere Outcomes?. Z Rheumatol 78, 610–619 (2019). https://doi.org/10.1007/s00393-019-0648-1
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DOI: https://doi.org/10.1007/s00393-019-0648-1