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Thromb Haemost 2008; 99(06): 1060-1067
DOI: 10.1160/TH07-11-0698
Blood Coagulation, Fibrinolysis and Cellular Haemostasis
Schattauer GmbH

Cost and outcome: Comparisons of two alternative bypassing agents for persons with haemophilia A complicated by an inhibitor

Katarina Steen Carlsson
1   Lund University Centre for Health Economics, LUCHE; and the Vårdal Institute for Health Sciences, Lund, Sweden
,
Jan Astermark
2   Department of Haematology and Coagulation Disorders, Malmö University Hospital, Malmö, Sweden
,
Sharyne Donfield
3   Department of Biostatistics, Rho Inc., Chapel Hill, North Carolina, USA
,
Erik Berntorp
2   Department of Haematology and Coagulation Disorders, Malmö University Hospital, Malmö, Sweden
› Author AffiliationsFinancial support:: The FENOC study was supported by an investigator initiated unrestricted grant from Baxter BioScience. All collection, management and analysis of study data were completed independently by the investigators.
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Publication History

Received 21 November 2007

Accepted after minor revision 20 April 2008

Publication Date:
27 November 2017 (online)

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Summary

The development of inhibitory antibodies to factor VIII is a serious complication of haemophilia. Two haemostatic agents with different bypassing mechanisms have been used in the treatment of patients with inhibitors: activated prothrombin complex concentrate (aPCC) and recombinant factor VIIa (rFVIIa). The objective was to compare cost and outcome of aPCC and rFVIIa in the treatment of joint bleeds. The analyses were based on the FENOC (FEIBA NovoSeven Comparative Study) crossover study where 48 patients used aPCC and rFVIIa to treat two joint bleeds. Incremental cost-effectiveness ratios were calculated for three outcome measures and the variation in cost was analyzed using two alternative regression methods. Results were subjected to sensitivity analyses. Key determinants of cost were prescribed dose, bodyweight and treatment in addition to protocol.The cost of aPCC was on average lower than rFVIIa. At all but one time point, patients rated slightly higher (but not statistically significantly) percentages of treatment efficacy and stopping of the bleed by aPCC. The reported reduction in pain from start of treatment up to 48 hours varied considerably among individuals. The different relative prices in the US, Turkey and Sweden mattered, but did not reverse the main results. In conclusion, the cost per episode was significantly lower for aPCC. The large individual-level variation in reduction of pain supports decisions that consider the individual patient’s experience and that accept trade-offs between cost and reduction in pain rather than focusing on cost only.

Keywords

Haemophilia A/B - factor VIII inhibitors - costs and cost analysis - bypassing agents - incremental cost-effectiveness

Institution where research was carried out: Lund University Centre for Health Economics, LUCHE, Lund, Sweden.


 

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