International survey of attitudes towards secondary prophylaxis with recombinant factor VIIa in haemophilia A patients with inhibitors

Mannucci, Pier Mannuccio; Miranda, Paulo

doi:10.1111/j.1365-2516.2008.01900.x

Cited by 3 publications

(3 citation statements)

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“…Infine, alcuni attuano una profilassi intermittente di pochi mesi con intervalli di durata variabile. In presenza di inibitori, dopo il fallimento di un trattamento ITI, alcuni pazienti vengono trattati con protocolli di profilassi a base di fattori bypassanti (APCC o rFVII) [30][31]. Le scelte tra le diverse strategie di gestione dipendono dalla gravità clinica dell'emofilia, dall'età del paziente, dalle condizioni socio-economico-assistenziali in cui vive e dai costi, ma talvolta sono anche condizionate dall'incertezza e scarsità delle evidenze scientifiche e dall'accettabilità dei protocolli di trattamento.…”

Section: Struttura Del Modello Decisionaleunclassified

Costs of treatment of haemophilia A in Italy: comparison of the use of plasma-derived and recombinant FVIII using a discrete event simulation (DES) model

Eandi¹,

Pradelli²,

Povero³

2013

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OBJECTIVE: To simulate haemophilia A (HA) real-life management and compare the cost of different treatment strategies, both with plasma-derived and recombinant factor VIII (pdFVIII and rFVIII, respectively), from the perspective of the Italian NHS.
METHODS: A discrete event (micro-)simulation (DES) model was developed to reproduce every possible HA patient clinical pathway: on-demand (OD) treatment of bleeding, continuous or discontinuous prophylaxis (PRO) with FVIII, inhibitors-tolerance-induction treatment (ITI), surgery in case of severe disability. Patient characteristics, treatment indications and disease evolution were modeled basing on data available in clinical literature in order to represent the actual state of art of HA management. In addition to the baseline scenario, reproducing current HA management, alternative strategies were simulated to explore the impact on the cost borne by the Italian NHS for these patients. Only differential direct sanitary costs were considered in the simulation, with a 3.5% discount rate.
RESULTS: Baseline scenario results show difference between patients treated with pdFVIII and those treated with rFVIII: mean lifetime HA patient management cost was estimated at € 1,332,373 with pdFVIII treatment, compared with € 2,013,222 for rFVIII. The saving is due mainly to the lower acquisition cost of pdFVIII. Total medical costs are strongly and positively correlated with HA severity: cost per patient increases from € 86,269 (mild HA) to € 1,509,231 (severe HA) for patients treated with pdFVIII and from € 147,900 to € 2,621,540 in patients treated with rFVIII. All analyses conducted in the study lead to the conclusion that the use of pdFVIII is much less expensive than rFVIII, but therapeutically equivalent.
CONCLUSION: Management of HA patients is complex and difficult to optimize; although involving a limited number of patients, lifetime management costs for the Italian NHS are extremely high. The main advantage of this model lies in the capability of estimating the economic impact of different strategic choices and economic/regulatory constraints.

http://dx.doi.org/10.7175/fe.v14i2.651

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Section: Struttura Del Modello Decisionaleunclassified

Costs of treatment of haemophilia A in Italy: comparison of the use of plasma-derived and recombinant FVIII using a discrete event simulation (DES) model

Eandi¹,

Pradelli²,

Povero³

2013

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http://dx.doi.org/10.7175/fe.v14i2.651

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“…Moreover, an international survey of attitudes towards secondary prophylaxis with rFVIIa in hemophilia A patients with inhibitors was conducted by interviewing 31 physicians from hemophilia centers and 20 health care providers in Europe and the US 78. The results revealed a positive attitude and more support for secondary prophylaxis in children than in adults.…”

Section: Independent Published Reportsmentioning

confidence: 99%

Critical appraisal of the role of recombinant activated factor VII in the treatment of hemophilia patients with inhibitors

Chuansumrit¹

2010

JBM

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Hemophilia patients with inhibitors faced the constraint of inadequate treatment for several years before the era of recombinant factor VIIa (rFVII). Initially, rFVIIa was used in the compassionate-use programs. After a worldwide license was issued, more than 1.5 million doses were administered. Bleeding of joints and muscles was controlled effectively by means of an early home treatment program, with either a standard dose of 90 μg/kg every 2 to 3 hours for a few doses or a single dose of 270 μg/kg. For more serious bleeding episodes or minor surgery, an initial dose of 90 μg/kg was given every 2 hours for 24 to 48 hours followed by increased intervals of 3 to 6 hours according to the severity of bleeding and efficacy of bleeding control. In cases of major surgery such as orthopedic procedures, the same regimen can be applied except for a higher initial dose of 120 to 180 μg/kg. However, increasing the dose should be considered if there are unexpected bleeding complications since the half-life and clearance of rFVIIa differ between individuals. In addition, prophylaxis is administered to a small number of patients. Finally, the reported thromboembolic events found in hemophilia patients with inhibitors receiving rFVIIa are extremely low, much less than 1%.

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“…On the whole, there is interest to use bypassing agents in inhibitor patients for regular prophylaxis, a method of treatment delivery that is so successful in children with uncomplicated haemophilia. Unfortunately the costs of secondary prophylaxis are huge [1], so there is a need to investigate further cost‐effectiveness of this therapeutic strategy before its use can become widespread [43].…”

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confidence: 99%

Inhibitors of propagation of coagulation (factors VIII, IX and XI): a review of current therapeutic practice

Franchini¹,

Mannucci

2011

Brit J Clinical Pharma

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The management of patients with congenital haemophilia who develop alloantibodies against factors of the propagation phase of blood coagulation, commonly known as inhibitors, is the most important challenge facing haemophilia caregivers at present, as this complication not only compromises the efficacy of replacement therapy but also consumes an enormous amount of economic resources. Development of inhibitors further complicates the clinical course of severe haemophilia, with a prevalence of up to 30% in patients with haemophilia A (factor VIII deficiency) and up to 5% in those with haemophilia B (factor IX deficiency) and haemophilia C (factor XI deficiency). While the short-term goal of treatment of patients who develop alloantibodies is the control of bleeding, the eradication of the inhibitor is the main long-term goal. The management of severe bleeding episodes and the eradication of the autoantibody are also the mainstays of treatment of patients with acquired haemophilia, a rare but life-threatening haemorrhagic condition characterized by the development of inhibitory autoantibodies against coagulation factor VIII. The most recent options available for treating patients with congenital haemophilia complicated by inhibitors and acquired haemophilia because of autoantibodies against factor VIII are summarized in this review article.

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International survey of attitudes towards secondary prophylaxis with recombinant factor VIIa in haemophilia A patients with inhibitors

Cited by 3 publications

References 4 publications

Costs of treatment of haemophilia A in Italy: comparison of the use of plasma-derived and recombinant FVIII using a discrete event simulation (DES) model

Costs of treatment of haemophilia A in Italy: comparison of the use of plasma-derived and recombinant FVIII using a discrete event simulation (DES) model

Critical appraisal of the role of recombinant activated factor VII in the treatment of hemophilia patients with inhibitors

Inhibitors of propagation of coagulation (factors VIII, IX and XI): a review of current therapeutic practice

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