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Hamostaseologie 2017; 37(S 01): S32-S41
DOI: 10.5482/HAMO-16-02-0003
Originalarbeit
Schattauer GmbH

Rapid Report Hämophilie

Evidenzlage zur Therapie aus Sicht des IQWiGRapid Report HemophiliaEvidence in treatment of hemophilia from the IQWiG’s perspective
Hans-Holger Bleß
1   IGES Institut GmbH, Berlin
,
Tonio Schönfelder
1   IGES Institut GmbH, Berlin
,
Anne Talaschus
1   IGES Institut GmbH, Berlin
› Institutsangaben
Weitere Informationen

Publikationsverlauf

received: 08. Februar 2016

accepted in revised form: 25. Mai 2016

Publikationsdatum:
28. Dezember 2017 (online)

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Zusammenfassung

Das IQWiG nahm im Rahmen eines Rapid Reports eine Nutzenbewertung der Therapie von Hämophilie-Patienten vor. Durch eine systematische Recherche wurden 16 Studien identifiziert, die zeigen, dass die Evidenzlücken bei wichtigen Fragen zur Behandlungen von Hämophilie-Patienten teilweise geschlossen sind. Der Vergleich der Behandlungsergebnisse einer prophylaktischen gegenüber einer On-Demand-Therapie zeigte die Überlegenheit der Prophylaxe bei schweren Blutungen für alle Altersgruppen. Bei Jugendlichen und Erwachsenen wurde zudem ein Zusatznutzen bezüglich Gesundheitszustand und Schmerzen festgestellt. Der Abgleich von Behandlungsempfehlungen mit der identifizierten Evidenz zeigte eine geringe Übereinstimmung zwischen den ermittelten Studien und den in den Empfehlungen verwendeten Referenzen, die Empfehlungen stimmten inhaltlich jedoch überwiegend mit den Ergebnissen der Nutzenbewertung überein. Schlussfolgerung: Die Nutzenbewertung zeigt die deutliche Überlegenheit der Prophylaxe gegenüber der On-Demand-Therapie auf und stellt ein deutliches Signal an die Behandelnden und Kostenträger dar, die prophylaktische Therapie der schweren Hämophilie A regelhaft in Erwägung zu ziehen.

Summary

The German Institute for Quality and Efficiency in Health Care (IQWiG) conducted a rapid report to assess the therapy of hemophilia patients. Based on a systematic literature search the IQWiG identified 16 studies which show that there is now sufficient information regarding questions that previously lacked data. A benefit assessment of prophylactic and on-demand-treatment concerning different treatment outcomes shows the superiority of the prophylactic therapy regarding major bleedings for all age groups. For the group of youths and adults the analysis shows additional benefits concerning health status and pain within the preceding four weeks. The alignment of guidelines with the identified evidence shows minor correspondence between the guidelines’ references and included studies. However, the guidelines’ statements primarily correspond with the results of the conducted benefit assessment. Conclusion: The IQWiG’s rapid report shows prophylactic therapy to be superior to on-demand therapy. These findings represent a clear indication for attending physicians and payers for prophylaxis in patients with severe hemophilia A.

Schlüsselwörter

Faktor VIII - Faktor IX - Hämophilie - Nutzenbewertung

Keywords

Factor VIII - factor IX - hemophilia - benefit assessment
 

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