A cost evaluation of treatment alternatives in mild-to-moderate bleeding episodes in haemophilia patients with inhibitors in Turkey

Dündar, Semra; Zülfikar, Bulent; Kavaklı, Kaan; Gönen, Can; Zülfikar, Haluk; Yılmaz, Deniz; Hart, WM; Sumen, Anita P.; Tuna, Süleyman Hakan; Karamalis, M.

doi:10.3111/200508046054

Cited by 22 publications

(45 citation statements)

References 13 publications

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“…Buscando validar os resultados encontrados e minimizar o impacto das limitações acima expostas, foi realizada uma busca por estudos farmacoeconômicos que avaliaram o rFVIIa em diferentes cenários e países. Sete estudos foram avaliados visando demonstrar e comparar os principais aspectos econô-micos do tratamento em outras populações e comprovaram os resultados encontrados neste estudo (Dundar et al, 2005;Ozelo et al, 2007;Putnam et al, 2005;Seremetis et al, 2006;Lyseng & Plosker, 2007;Treur et al, 2009;Knight et al, 2003). De maneira geral, os estudos concluíram que o rFVIIa foi mais eficaz e mais economicamente viável comparado ao CCPa, quando utilizado como tratamento de primeira linha dos episódios hemorrágicos leves a moderados.…”

Section: Discussionunclassified

“…Assim, para a estimativa dos custos e desfechos dos tratamentos, foram reproduzidas as estruturas das árvores de decisão de dois modelos internacionais já publicados, que acompanharam pacientes com hemofilia complicada por inibidores no tratamento de episódios de sangramento leve a moderado. Esses modelos foram extraídos de uma busca na literatura por estudos econômicos (You et al, 2009;Dundar et al, 2005;Ozelo et al, 2007;Putnam et al, 2005;Seremetis et al, 2006;Lyseng & Plosker, 2007;Treur et al, 2009;Knight et al, 2003;Jiménez-Yuste et al, 2013).…”

Section: Estrutura Do Modelounclassified

“…Uma busca na literatura sobre estudos econômicos avaliando a relação de custo-efetividade do uso de rFVIIa em comparação ao CCPa no mundo mostrou resultados favorá-veis para rFVIIa, motivando a elaboração de uma análise de custo-efetividade para a realidade brasileira (You et al, 2009;Dundar et al, 2005;Ozelo et al, 2007;Putnam et al, 2005;Seremetis et al, 2006;Lyseng & Plosker, 2007;Treur et al, 2009;Knight et al, 2003;Jiménez-Yuste et al, 2013).…”

Section: Introductionunclassified

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Custo-efetividade do fator VII ativado recombinante (rFVIIa) e complexo protrombínico parcialmente ativado (CCPa) no tratamento sob demanda do sangramento leve/moderado de pacientes com hemofilia congênita complicada por inibidores sob a perspectiva

Pepe¹,

Fagundes²,

Brandon³

et al. 2017

JBES

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Section: Discussionunclassified

Section: Estrutura Do Modelounclassified

Section: Introductionunclassified

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Custo-efetividade do fator VII ativado recombinante (rFVIIa) e complexo protrombínico parcialmente ativado (CCPa) no tratamento sob demanda do sangramento leve/moderado de pacientes com hemofilia congênita complicada por inibidores sob a perspectiva

Pepe¹,

Fagundes²,

Brandon³

et al. 2017

JBES

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“…There was no requirement that FEIBA be used exclusively in inhibitor patients during the study period. Should cost, lack of immediate availability or clinical considerations preclude treatment with FEIBA, the physicians participating in the registry could freely utilize other treatment options available in Turkey during the study period, namely, rFVIIa, high‐dose FVIII or prothrombin complex concentrate [11].…”

Section: Methodsmentioning

confidence: 99%

Efficacy of FEIBA for acute bleeding and surgical haemostasis in haemophilia A patients with inhibitors: a multicentre registry in Turkey

Zülfikar

Aydoğan²,

Salcioglu

et al. 2011

Haemophilia

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Long used in established industrialized nations to treat patients with haemophilia and inhibitors, factor eight inhibitor bypassing activity (FEIBA) has, in recent years, been introduced into more geographically diverse settings. Data are needed on how successfully FEIBA therapy has been implemented in new regions. To determine the efficacy and safety of FEIBA for the treatment of acute bleeding and surgical haemostasis in a newly industrialized country. A multicentre registry of haemophilia A patients with inhibitors receiving FEIBA treatment was established in Turkey. With a standardized case report form, data were collected retrospectively on: patient demographics; characteristics of acute bleeding episodes and surgical interventions; FEIBA regimen; and treatment outcomes. Thirty-seven patients received a total of 112 FEIBA treatment courses, 90 for acute bleeding and 22 for surgical haemostasis. The median FEIBA dose per infusion for acute bleeding was 50 IU kg(-1), and for surgery was 100 IU kg(-1). For both acute joint and muscle/soft tissue bleeding and in surgery, haemostasis was attained in a median of two FEIBA infusions. FEIBA was judged effective in 92% of treatment courses for acute bleeding, with a 95% confidence interval (CI) of 85-97%. Rates of haemostatic efficacy did not differ significantly between anatomical sites of acute bleeding. The haemostatic efficacy rate of FEIBA in surgery was 86% (CI, 65-97%). No thromboembolic complications or other adverse events occurred during any treatment course. FEIBA has been successfully integrated into clinical practice in Turkey, with rates of haemostatic efficacy comparable to those reported in countries with a longer history of FEIBA usage.

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“…A study by Dundar et al. evaluated the economic impact of four different treatment regimens (high‐dose factor VIII or IX, PCC, APCC and rFVIIa) for mild‐to‐moderate bleeds in patients with haemophilia and inhibitors [24]. They constructed a decision‐analysis model, similar to Fig.…”

Section: Cost‐effectiveness Models Based On Observational Datamentioning

confidence: 99%

A systematic review of the cost‐effectiveness of rFVIIa and APCC in the treatment of minor/moderate bleeding episodes for haemophilia patients with inhibitors

Knight¹,

Dano

Kennedy-Martin

2009

Haemophilia

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and àKMHO, Brighton, UK Summary. The clinical, humanistic and economic consequences associated with haemophilia and inhibitors are considerable. Primary treatment for mild-to-moderate bleeding disorders in such patients is recombinant factor VIIa (rFVIIa) or activated prothrombin complex concentrate (APCC). The aims of this study were to identify, review and evaluate the quality of the published literature on the relative cost-effectiveness of rFVIIa and APCC in treating haemophilia patients with inhibitors. The review concentrates on model type, design and assumptions, and results. The results of this study suggest that rFVIIa may be the cost-effective alternative to treatment with APCC. In seven out of the nine studies, rFVIIa had the lower average treatment cost. The difference in average treatment cost to resolve a bleed, between rFVIIa and APCC in these seven studies, ranged from $3000 to $17 000. The adapted modelling framework is similar in all the economic models reviewed, suggesting clinical acceptability of the approach used. The estimates of efficacy varied between the models, especially for APCC. The efficacy for APCC derived from retrospective studies was lower than reported in the literature. Sensitivity analysis was undertaken in the majority of the economic analyses and the results were found to be robust to realistic parameter variations. Only one of the studies was a cost-utility study, showing the lack of measuring health status within this area. This systematic review showed that models based on different sources of data produced fairly similar robust results despite differences in the estimates of efficacy, average dosage required, and unit costs. However, ideally there should be a systematic approach to identifying the relevant data.

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A cost evaluation of treatment alternatives in mild-to-moderate bleeding episodes in haemophilia patients with inhibitors in Turkey

Cited by 22 publications

References 13 publications

Custo-efetividade do fator VII ativado recombinante (rFVIIa) e complexo protrombínico parcialmente ativado (CCPa) no tratamento sob demanda do sangramento leve/moderado de pacientes com hemofilia congênita complicada por inibidores sob a perspectiva

Custo-efetividade do fator VII ativado recombinante (rFVIIa) e complexo protrombínico parcialmente ativado (CCPa) no tratamento sob demanda do sangramento leve/moderado de pacientes com hemofilia congênita complicada por inibidores sob a perspectiva

Efficacy of FEIBA for acute bleeding and surgical haemostasis in haemophilia A patients with inhibitors: a multicentre registry in Turkey

A systematic review of the cost‐effectiveness of rFVIIa and APCC in the treatment of minor/moderate bleeding episodes for haemophilia patients with inhibitors

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