Zusammenfassung
Hintergrund
Patienten im rheumatologischen Versorgungsalltag unterscheiden sich in ihrem individuellen Risikoprofil entscheidend von Teilnehmern an klinischen Zulassungsstudien, z. B. durch Begleiterkrankungen oder Alter. Die Übertragung der Ergebnisse aus klinischen Studien in den Praxisalltag ist daher nur bedingt möglich.
Fragestellung
Welchen Beitrag leisten Beobachtungsstudien für Entscheidungen in der rheumatologischen Praxis?
Material und Methoden
Datengrundlage ist das Biologikaregister RABBIT, in dem Patienten mit einer rheumatoiden Arthritis (RA) mit Beginn einer synthetischen (s) oder biologischen (b) DMARD („disease-modifying antirheumatic drugs“)-Therapie eingeschlossen und mindestens 5 Jahre beobachtet werden. Komorbiditäten und wichtige anamnestische Angaben (wie z. B. Tumorhistorie) werden bei Einschluss erhoben, im Verlauf werden unerwünschte Ereignisse regelmäßig dokumentiert.
Ergebnisse
Nur etwa ein Drittel der in RABBIT mit bDMARDs behandelten Patienten erfüllte die Einschlusskriterien entsprechender Zulassungsstudien. Anhand der Registerdaten wurde ein Modell erstellt, mit dem das individuelle Risiko eines Patienten für schwerwiegende Infektionen unter verschiedenen Therapiekombinationen berechnet werden kann. Mithilfe eines Online-Risikorechners kann dies vom behandelnden Rheumatologen einfach ermittelt werden. Weitere Ergebnisse zeigen, dass vor allem eine hohe Krankheitsaktivität ein bedeutender Risikofaktor für die Verschlechterung bestehender Komorbiditäten ist. Für Patienten mit einer Herzinsuffizienz scheint die Behandlung mit TNF-Inhibitoren wegen der effektiven Kontrolle der Krankheitsaktivität eher protektiv als schädlich.
Schlussfolgerungen
Auswertungen von Beobachtungsstudien tragen heutzutage bedeutend zur Beurteilung des individuellen Risikos eines Patienten bei. Ergebnisse der Biologikaregister stellen nicht nur eine Ergänzung zu klinischen Studien dar, sondern sind eine wichtige Grundlage für Entscheidungen im Praxisalltag und erhöhen die Handlungssicherheit bei der Behandlung von Patienten mit Komorbiditäten oder in höherem Alter.
Abstract
Background
Patients in daily rheumatological care differ in their individual risk profiles from participants in randomized controlled trials (RCT), e.g. due to comorbidities and age. Transferring results from RCTs into routine daily practice is therefore limited.
Objective
The aim of this study was to evaluate the contribution of observational studies for decision-making in routine rheumatology practice.
Material and methods
We used data from the German biologics register RABBIT which includes patients with rheumatoid arthritis (RA) when starting synthetic (s) or biologic (b) disease-modifying antirheumatic drugs (DMARD). They are observed for at least 5 years. Comorbidities and clinically relevant aspects (e.g. history of malignancies) are reported at baseline and adverse events at regular follow-up.
Results
Only one out of three patients treated with bDMARDs in RABBIT would have fulfilled the inclusion criteria of the respective pivotal study. Register data enabled developing a risk scoring model which evaluates the individual risk of a patient for serious infections depending on different risk factors and the respective DMARD treatment. Open online access to the score provides the possibility of risk estimation for all rheumatologists. Further results identified long-standing high disease activity as a dominant risk factor for a worsening of prevalent comorbidities. In patients with heart failure it was shown that effective treatment and control of disease activity with tumor necrosis factor (TNF) inhibitors was more likely to be protective than harmful.
Conclusion
Observational studies contribute essentially to the assessment of individual risks of patients. The results provide valuable information to support clinical decision-making and therefore strengthen the evidence when treating patients of higher age or with existing comorbidities.
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Einhaltung ethischer Richtlinien
Interessenkonflikt. A. Strangfeld: Referententätigkeit für AbbVie, BMS, Merck-Sharp & Dohme, Pfizer, Roche, Sanofi-Aventis, UCB. A. Richter: kein Interessenkonflikt.
Alle im vorliegenden Manuskript beschriebenen Untersuchungen am Menschen wurden mit Zustimmung der zuständigen Ethik-Kommission, im Einklang mit nationalem Recht sowie gemäß der Deklaration von Helsinki von 1975 (in der aktuellen, überarbeiteten Fassung) durchgeführt. Von allen beteiligten Patienten liegt eine Einverständniserklärung vor.
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Strangfeld, A., Richter, A. Wie unterstützen Registerdaten die klinische Entscheidungsfindung?. Z. Rheumatol. 74, 119–124 (2015). https://doi.org/10.1007/s00393-014-1449-1
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DOI: https://doi.org/10.1007/s00393-014-1449-1