Prophylaktische Faktorsubstitution bei schwerer Hämophilie A

Zusammenfassung

Prophylaxe kann bei Erwachsenen mit schwerer Hämophilie A klinisch notwendig sein. Ziel war es, die prophylaktische Faktor-VIII-Substitution bei erwachsenen Patienten mit Hämophilie in Deutschland ökonomisch zu bewerten. Patienten, Methoden: Für die Analysen wurde ein Entscheidungsmodell erstellt: Zeithorizont ein Jahr; Perspektive gesetzliche Krankenkassen; Referenzpatient (RP) und zwei Patientenprofile. Die Berech-nungen basieren auf Daten aus einer struktu-rierten Literatursuche, -analyse und einer Pharmakovigilanzstudie mit Patienten zu Therapiewechsel On-demand/Prophylaxe (OD/Proph). Ergebnisse: RP: 45 Jahre, 20 Blutungen p.a. OD, 16 vermiedene Blutungen mit 8,5 I.U./kg/d Proph, Zusatzkosten 141 113 Euro p.a.; Profil 1: 50 Jahre, 55 Blutungen p.a. OD, Faktorverbrauch pro Blutung 20 I.U./kg höher als bei R P, 39 vermiedene Blutungen mit 8,5 I.U./kg/d Proph, Zusatzkosten 19 134 Euro p.a.; Profil 2: 60 Jahre, 35 Blutungen p.a. OD, Faktorverbrauch pro Blutung 40–80 I.U./kg höher als bei R P, 34 vermiedene Blutungen mit 11 I.U./kg/d Proph, Einsparung 660 Euro p.a. Schlussfolgerung: In der indivi-duellen Situation kann eine prophylaktische Faktorsubstitution bei erwachsenen Patienten auch ökonomisch sinnvoll sein. Um künftig diesen Effekt umfassend zu bewerten, werden longitudinale Daten aus dem Versorgungsalltag inklusive klinischer Outcomes, Lebensqua-lität und Adhärenz benötigt.

Summary

Prophylaxis in adults can be necessary and reasonable for clinical reasons. The aim was to evaluate from an economic viewpoint prophylactic factor VIII substitution in adult patients with haemophilia in Germany. Patients, methods: A decision model (time frame: one year; perspective: statutory health insurance; reference patient (RP) and 2 patient profiles) was developed. Calculations are based on data from a structured literature search and a pharmacovigilance study: therapy switch ondemand/prophylaxis (OD/Proph). Results: RP: 45 years, 20 bleeds p.a. OD, 16 bleeds avoided with 8.5 I.U./kg/d Proph, additional cost Euro 141 113 p.a.; profile 1: 50 years, 55 bleeds p.a. OD, factor consumption per bleed 20 I.U./kg higher than R P, 39 bleeds avoided with 8.5 I.U./kg/d Proph, additional cost Euro 19 134 p.a.; profile 2: 60 years, 35 bleeds p.a. OD, factor consumption per bleed 40–80 I.U./ kg higher than R P, 34 bleeds avoided with 11 I.U./kg/d Proph, cost reduction Euro 660 p.a. Conclusions: Prophylactic factor VIII substitution in adult haemophilia patients is depending on the individual clinical situation not only clinically but also economically reasonable. To evaluate this effects in the future comprehensively, longitudinal real-life data from patient-centered care are needed including clinical outcomes, quality of life and adherence.

Schlüsselwörter

Hämophilie A - Erwachsene - Prophylaxe - Wirt-schaftlichkeit

Keywords

Haemophilia A - adults - prophylaxis - economics

^* These authors contributed equally.

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