Economic analysis of recombinant activated factor VII versus plasma-derived activated prothrombin complex concentrate in mild to moderate bleeds: Haemophilia registry data from the Czech Republic

Salaj, Peter; Penka, Miroslav; Šmejkal, Petr; Geierová, Véra; Ovesná, Petra; Brabec, Petr; Cetkovský, Petr; Kubeš, R; Mesterton, Johan; Lindgren, Peter

doi:10.1016/j.thromres.2012.02.005

Cited by 13 publications

(11 citation statements)

References 16 publications

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“…The keywords that we used for our search were inhibitor, haemophilia A, costs, cost analysis and ITI. We found a total of 63 studies of which 40 were excluded at title screening, six were excluded after a review of the abstracts, and five were excluded after full text review, leaving 12 [eight for bypassing agents and four for ITI ] that were suitable for our research (Tables and ).…”

Section: Methodsmentioning

confidence: 99%

Strategies for inhibitor treatment and costs in the short and long term: a critical evaluation of recent clinical studies

Baghaipour

Carlsson

2015

European J of Haematology

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One important complication of patients with severe haemophilia A is the formation of inhibitory antibodies to factor VIII (FVIII). Immune tolerance induction (ITI) is the treatment of choice for patients with inhibitors, but this approach is successful in about 60% of patients. Treatment of acute bleeding in patients with inhibitors is one of the greatest challenges in haemophilia management and is costly. Bypassing agents are the mainstay of treatment in these patients. The aims of this study were to review the most recent publications concerning the costs of inhibitor treatment. We conducted a literature review using PubMed which yielded 63 papers analysing the costs of inhibitor management of which 12 were suitable for our study. Four of eight studies supported the use of activated prothrombin complex concentrate (aPCC) with lower costs, but the remaining four studies showed that recombinant factor VIIa (rFVIIa) had a lower average treatment cost. Of four ITI studies, two supported lifelong cost-effectiveness of ITI vs. bypassing agents and the remaining two papers showed a high cost of inhibitor treatment. Dosages, time between onset of bleeding and treatment, patient characteristics and the price of drugs are some of the important issues that should be considered for further studies. Haemophilia A and B is an X-linked disorder with an incidence of 1/5000 and 1/20 000 males, respectively (1). It results from low or absent factor VIII (FVIII) and factor IX (FIX), respectively. The mainstay of treatment is factor replacement. Between 20 and 35% of patients with haemophilia A and between 4 and 6% of those with haemophilia B develop inhibitory antibodies (2). Treatment of acute bleeding in patients with inhibitors is one of the greatest challenges in haemophilia management and is costly. These are treated by bypassing haemostatic agents, such as porcine FVIII, prothrombin complex concentrates (PCC), activated prothrombin complex concentrates (aPCC) or recombinant factor VIIa (rFVIIa). Of these, PCC are not used so much today, having been largely superseded by aPCC or rFVIIa. Different regimens of immune tolerance induction (ITI) are used: high dose (Bonn protocol), with additional drugs or procedures (Malm€ o protocol), and a low-dose regimen.Efficacy, costs, adverse effects and other features of ITI and bypassing agents have been evaluated in numerous studies, but many questions still remain. The aims of this study were to reassess and review the most recent publications concerning the costs of inhibitor treatment by focusing on the outcome, success rate, the duration of studies and other parameters such as number of doses, method of investigation and time elapsed before initiation of treatment. MethodsAs a part of the Advanced International Haemophilia course which was held in Malm€ o, Sweden in 2013-2014, we conducted a literature review using PubMed. The keywords that we used for our search were inhibitor, haemophilia A, costs, cost analysis and ITI. We found a total of 63 studies of which 40 were ex...

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Section: Methodsmentioning

confidence: 99%

Strategies for inhibitor treatment and costs in the short and long term: a critical evaluation of recent clinical studies

Baghaipour

Carlsson

2015

European J of Haematology

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“…The HemoRec registry also reported on outcomes of treatment of bleeding events. Measures including time to resolution [27][28][29], effectiveness of treatment [27] and rebleeding [27,28,30] were introduced to evaluate treatment efficiency. The PedNet registry introduced the extent of bleed as an outcome measure [31].…”

Section: Bleeding Specifications and Outcomesmentioning

confidence: 99%

“…Of these, four registries included the number of joint bleeding events whilst only three noted TJ. In three of the four papers reported on TJ, the definition used was missing [28,30,43]. Arthropathy and ROM were each reported by two registries.…”

Section: Joint Outcomesmentioning

confidence: 99%

“…Outcome of treatment of a bleeding event is one of the most important aspects of haemophilia care, although there is no consensus as to how to report it. The HemoRec registry reported on different indexes of bleeding treatment outcome [27][28][29][30]. Because bleeding events are generally patientreported outcomes, efforts should be made to reduce the amount of information by focusing on the most important questions.…”

Section: Bleeding Specifications and Outcomesmentioning

confidence: 99%

“…Many of them suffered from some design issues resulting in huge variations of their results [47]. Two economic evaluation papers published using HemoRec registry based data were enrolled in this study [27,28]. Clotting factor consumption, as one of the main determinants of haemophilia treatment cost, has been reported more frequently than other, less direct outcomes such as loss of days at school/work or hospitalization due to haemophilia.…”

Section: Burden Of Disease and 'Cost Of Treatment'mentioning

confidence: 99%

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Registry‐based outcome assessment in haemophilia: a scoping study to explore the available evidence

Osooli

Berntorp

2016

J Intern Med

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Content List -Read more articles from the symposium: Future of haemophilia outcome assessment.Abstract. Osooli M, Berntorp E (Lund University and Sk ane University Hospital, Malm€ o, Sweden). Registry-based outcome assessment in haemophilia: a scoping study to explore the available evidence. (Review Symposium). J Intern Med 2016; 279: 502-514.Haemophilia is a congenital disorder with bleeding episodes as its primary symptom. These episodes can result in negative outcomes including joint damage, loss of active days due to hospitalization and reduced quality of life. Effective treatment, however, can improve the outcome. Registries have been used as a valuable source of information regarding the monitoring of treatment and outcome. The two main aims of this exploratory study were to establish which haemophilia registries publish peer-reviewed outcome assessment research and then to extract, classify and report the treatment outcomes and their extent of use in the retrieved registries. Using relevant keywords, we searched PubMed and Web of Science databases for publications during the period 1990-2015. Retrieved references were screened in a stepwise process. Eligible papers were original full articles on haemophilia outcomes that used data from a computerized patient database. Descriptive results were summarized. Of 2352 references reviewed, 25 full texts were eligible for inclusion in the study. These papers were published by 11 registries ranging from local to international in coverage. It is still relatively rare for registries to produce peer-reviewed publications about outcomes, and most that currently do produce such papers are located in Europe and North America. More information is available on traditional outcomes such as comorbidities and arthropathy than on health-related quality of life or the social and developmental impact of haemophilia on patients. Inhibitors, HIV and viral hepatitis are amongst the most commonly reported comorbidities. Research has focused more on factor consumption and less on hospitalization or time lost at school or work due to haemophilia. Haemophilia registries, especially those at the national level, are valuable resources for the delivery of effective health care to patients. Validated outcome measurement instruments are essential for the production of reliable and accurate evidence. Finally, such evidence should be communicated to physicians, patients, the public and health policymakers.

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Modern Treatments of Haemophilia: Review of Cost-Effectiveness Analyses and Future Directions

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Economic analysis of recombinant activated factor VII versus plasma-derived activated prothrombin complex concentrate in mild to moderate bleeds: Haemophilia registry data from the Czech Republic

Cited by 13 publications

References 16 publications

Strategies for inhibitor treatment and costs in the short and long term: a critical evaluation of recent clinical studies

Strategies for inhibitor treatment and costs in the short and long term: a critical evaluation of recent clinical studies

Registry‐based outcome assessment in haemophilia: a scoping study to explore the available evidence

Modern Treatments of Haemophilia: Review of Cost-Effectiveness Analyses and Future Directions

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