Economic Evaluation of Immune Tolerance Induction in Children With Severe Hemophilia A and High-Responding Inhibitors: A Cost-Effectiveness Analysis of Prophylaxis With Emicizumab

Camelo, Ricardo Mesquita; Barbosa, Mariana Michel; Araújo, Maiara Silva; Muniz, Roberto Lúcio; Guerra, Augusto Afonso; Godman, Brian; Rezende, Suely Meireles; Acúrcio, Francisco de Assis; Martin, Antony; Álvares-Teodoro, Juliana

doi:10.1016/j.vhri.2022.10.007

Cited by 3 publications

(2 citation statements)

References 43 publications

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“…Though, economic evaluation of prophylactic emicizumab is not conducted in Indian setting, the model findings were in line with previous models developed for other countries like Brazil, France, Italy, Korea, Iran, and Peru [ 4 , 6 , [25] , [26] , [27] , [28] ]. Though, there is difference across these studies in terms of the time horizon, all these studies included Markov models reporting results in terms of health system perspective.…”

Section: Discussionsupporting

confidence: 84%

Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India

Krishnamoorthy,

Govindan,

Kannan

et al. 2024

Heliyon

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Section: Discussionsupporting

confidence: 84%

Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India

Krishnamoorthy,

Govindan,

Kannan

et al. 2024

Heliyon

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“…A recent European survey indicated that ITI is still the mainstay of treatment for patients with HA and inhibitors. Emicizumab prophylaxis can be considered a cost-saving treatment for patients with HA and inhibitors during ITI [ [21] , [22] , [23] ].…”

Section: Discussionmentioning

confidence: 99%

Factor VIII inhibitors in hemophilia A treated with emicizumab: longitudinal follow-up of outcomes

Levy-Mendelovich,

Atia,

Budnik

et al. 2023

Research and Practice in Thrombosis and Haemostasis

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Determinants of bleeding before and during immune tolerance in 222 boys with severe hemophilia A and inhibitors >5 BU

Fischer,

Kenet,

Kurnik

et al. 2024

Blood Advances

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Prevention of bleeding and its consequences is the main goal of hemophilia treatment and determines treatment choices for patients who develop inhibitors. To assess bleeding before and during immune tolerance induction (ITI) and its association with ITI regimen and inhibitor titer, we selected and analyzed data on patients receiving high-titer inhibitors from the international prospective PedNet cohort study. In total, 222 patients with severe hemophilia A and inhibitor titers of >5 Bethesda units (BU) were followed from the first positive to the first negative inhibitor result (median overall follow-up, 1.7 years). Mean annual (joint) bleeding rates (AJBR) and 95% confidence intervals (CIs) were compared according to treatment and inhibitor titer using multivariable negative binomial regression. Before ITI, 115 patients showed an ABR of 6.1 (5.0-7.4) and an AJBR 2.6 (2.1-3.2). Bleeding was independent of inhibitor titer. During ITI, 202 patients had an ABR of 4.4 (3.9-5.1) and an AJBR of 1.7 (1.5-2.0). AJBR during ITI increased with inhibitor titer (hazard ratio [HR] for ≥200 BU vs 5 to 39 BU [4.9; CI, 3.2-7.4]) and decreased with daily ITI infusions (HR, 0.4; CI, 0.3-0.6) or activated prothrombin complex concentrate prophylaxis (HR, 0.4; CI, 0.2-0.8), whereas ITI dose and recombinant activated factor VII prophylaxis did not independently affect bleeding. These data provide evidence for a protective effect of repeated FVIII infusions (ITI) on bleeding in patients who have developed inhibitors; these data should be used to plan ITI and/or serve as a comparator for prophylaxis with nonreplacement therapy.

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Economic Evaluation of Immune Tolerance Induction in Children With Severe Hemophilia A and High-Responding Inhibitors: A Cost-Effectiveness Analysis of Prophylaxis With Emicizumab

Cited by 3 publications

References 43 publications

Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India

Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India

Factor VIII inhibitors in hemophilia A treated with emicizumab: longitudinal follow-up of outcomes

Determinants of bleeding before and during immune tolerance in 222 boys with severe hemophilia A and inhibitors >5 BU

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Economic Evaluation of Immune Tolerance Induction in Children With Severe Hemophilia A and High-Responding Inhibitors: A Cost-Effectiveness Analysis of Prophylaxis With Emicizumab

Cited by 3 publications

References 43 publications

Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India

Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India

Factor VIII inhibitors in hemophilia A treated with emicizumab: longitudinal follow-up of outcomes

Determinants of bleeding before and during immune tolerance in 222 boys with severe hemophilia A and inhibitors &gt;5 BU

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Product

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Determinants of bleeding before and during immune tolerance in 222 boys with severe hemophilia A and inhibitors >5 BU