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Meta-analysis on incidence of inhibitors in 1,945 previously untreated patients treated with recombinant factor VIII products: is there a difference?
Mantovani LG, Rota M, Cortesi P, Steinitz K, Reininger A, Gringeri A
Blood. 2015;126((23)): Abstract No. 289.
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Direct costs of children with haemophilia A undergoing prophylaxis or episodic treatment: Results from the ESPRIT study
Gringeri A, Fusco F, Riva S, Von MacKensen S, Mantovani LG
Journal of Thrombosis and Haemostasis. 2011;9((Suppl 2):):927. Abstract No. P-TH-510.
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3.
Cost-effectiveness of prophylaxis with anti-inhibitor complex concentrate in patients with hemophilia A and inhibitors: results from the Pro-FEIBA study
Gringeri A, Leissinger CA, Cortesi P, Jo H, Mantovani L
Blood. 2011;118((21):): Abstract No. 4187.
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4.
A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study)
Gringeri A, Lundin B, Von Mackensen S, Mantovani L, Mannucci PM
Journal of Thrombosis and Haemostasis. 2011;9((4):):700-10.
Abstract
Background: Prevention of arthropathy is a major goal of hemophilia treatment. While studies in adults have demonstrated an impact of prophylaxis on the incidence of joint bleeds and patients' well-being in terms of improved quality of life (QoL), it is unclear whether or not prophylaxis influences the outcome and perception of well- of children with hemophilia. Objective:This randomized controlled study compared the efficacy of prophylaxis with episodic therapy in preventing hemarthroses and image-proven joint damage in children with severe hemophilia A (factor VIII <1%) over a 10-year time period. Methods: Forty-five children with severe hemophilia A, aged 1-7years (median 4), with negative clinical-radiologic joint score at entry and at least one bleed during the previous 6 months, were consecutively randomized to prophylaxis with recombinant factor VIII (25IUkg-1 3x week) or episodic therapy with >=25IUkg-1 every 12-24h until complete clinical bleeding resolution. Safety, feasibility, direct costs and QoL were also evaluated. Results:Twenty-one children were assigned to prophylaxis, 19 to episodic treatment. Children on prophylaxis had fewer hemarthroses than children on episodic therapy: 0.20 vs. 0.52 events per patient per month (P<0.02). Plain-film radiology showed signs of arthropathy in six patients on prophylaxis (29%) vs. 14 on episodic treatment (74%) (P< 0.05). Prophylaxis was more effective when started early (<=36 months), with patients having fewer joint bleeds (0.12 joint bleeds per patient per month) and no radiologic signs of arthropathy. Conclusion:This randomized trial confirms the efficacy of prophylaxis in preventing bleeds and arthropathy in children with hemophilia, particularly when it is initiated early in life. 2011 International Society on Thrombosis and Haemostasis.
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5.
Quality of life of children with hemophilia A undergoing prophylaxis or episodic treatment: Results from the ESPRIT study
von MacKensen S, Riva S, Fusco F, Mantovani L, Gringeri A
Journal of Thrombosis and Haemostasis. 2011;9((Suppl 2):):745. Abstract No. O-TH-075.
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6.
Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review
Iorio A, Halimeh S, Holzhauer S, Goldenberg N, Marchesini E, Marcucci M, Young G, Bidlingmaier C, Brandao LR, Ettingshausen CE, et al
Journal of Thrombosis and Haemostasis. 2010;8((6):):1256-65.
Abstract
Background: Different rates of inhibitor development after either plasma-derived (pdFVIII) or recombinant (rFVIII) FVIII have been suggested. However, conflicting results are reported in the literature. Objectives: To systematically review the incidence rates of inhibitor development in previously untreated patients (PUPs) with hemophilia A treated with either pdFVIII or rFVIII and to explore the influence of both study and patient characteristics. Methods: Summary incidence rates (95% confidence interval) from all included studies for both pdFVIII and rFVIII results were recalculated and pooled. Sensitivity analysis was used to investigate the effect of study design, severity of disease and inhibitor characteristics. Meta-regression and analysis-of-variance were used to investigate the effect of covariates (testing frequency, follow-up duration and intensity of treatment). Results: Two thousand and ninety-four patients (1167 treated with pdFVIII, 927 with rFVIII; median age, 9.6 months) from 24 studies were investigated and 420 patients were observed to develop inhibitors. Pooled incidence rate was 14.3% (10.4-19.4) for pdFVIII and 27.4% (23.6-31.5) for rFVIII; high responding inhibitor incidence rate was 9.3% (6.2-13.7) for pdFVIII and 17.4% (14.2-21.2) for rFVIII. In the multi-way anova study design, study period, testing frequency and median follow-up explained most of the variability, while the source of concentrate lost statistical significance. It was not possible to analyse the effect of intensity of treatment or trigger events such as surgery, and to completely exclude multiple reports of the same patient or changes of concentrate. Conclusions: These findings underscore the need for randomized controlled trials to address whether or not the risk of inhibitor in PUPs with hemophilia A differs between rFVIII and pdFVIII. 2010 International Society on Thrombosis and Haemostasis.
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7.
Immunotolerance induction with von Willebrand factor/factor VIII concentrates in patients at high risk of failure
Gringeri A, Ewing NP, Heisel Kurth M, Hoots WK, Negrier C
Haematologica. 2009;94((6, Suppl 2):):677. Abstract No. 1782.
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8.
VWF/FVIII concentrates in high-risk immunotolerance: the RESIST studies
Gringeri A, Ewing N, Heisel-Kurth MA, Hoots K, Negrier C, Bianchi Bonomi A
ISTH Congress. 2009;: Abstract No. PP-MO-586.
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9.
Primary and secondary prophylaxis in children with haemophilia A reduces bleeding frequency and arthropathy development compared to on-demand treatment: a 10-year, randomized, clinical trial
Gringeri A, Lundin B, von Mackensen S, Mantovani LG, Mannucci PM, Bianchi Bonomi A
ISTH Congress. 2009;: Abstract No. OC-MO-034.
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10.
Rate of inhibitor development in hemophilia A patients treated with plasma derived or recombinant factor VIII concentrates. A systematic review of the literature
Iorio A, Halimeh S, Bidlingmaier C, Brandao LR, Escuriola-Ettingshausen C, Goldenberg NA, Gringeri A, Holzhauer S, Kenet G, Knoefler R, et al
Blood. 2009;114((22):): Abstract No. 3154.