Evidence of prophylaxis treatment in adult haemophiliacs

Summary

In contrast to children with haemophilia the scientific evidence of prophylaxis treatment in adults is not yet proven. Existing studies are of observational character and mostly retrospectively designed. Therefore, opinion leaders in this field postulate prospectively designed, randomized, controlled and multicentric studies to set up urgently needed guidelines. Evidence according to the Canadian task force ranking is assessed as level III with a re- commendation grade C by the authors.

The recognition of benefits of health care providers in accordance with the German Federal Joint Committee generally demands a Grade- Ia to Ib evidence. As long as the actual evidence of prophylaxis in adult haemophiliacs does not meet the postulated criteria of the German Federal Joint Committee, prophylactic replacement therapy of the individual case has to be well documented and reasonably explained.

Zusammenfassung

Im Gegensatz zur Prophylaxe bei hämophilen Kindern fehlt bei den Erwachsenen der Nachweis der Überlegenheit einer prophylaktischen Gerinnungsfaktorsubstitution gegenüber der bei Bedarf. Vorliegende Arbeiten sind Beobachtungsstudien und meist retrospektiv. Anerkannte Wissenschaftler fordern daher prospektive, randomisierte, multizentrische Studien für dringend notwendige Leitlinien. Die aktuelle Evidenz zum Thema vorhandener Studien wird nach der Skala der Canadian Task Force auf Evidenz-Level III mit Empfehlungsgrad C von den Autoren eingeschätzt. Zur Leistungsanerkennung in der gesetzlichen Krankenversicherung fordert der gemeinsame Bundesausschuss in Deutschland grundsätzlich einen Evidenz-Level Ia bis Ib. Bis zum Nachweis der geforderten Evidenz ist die Anwendung der Prophylaxetherapie bei Erwachsenen im Einzelfall gut und nachvollziehbar zu begründen.

Keywords

Replacement therapy - adult haemophiliacs - primary and secondary prophylaxis

Schlüsselwörter

Substitutionstherapie - erwachsene Hämophile - primäre und sekundäre Prophylaxe

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