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1.
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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2.
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.
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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4.
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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5.
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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6.
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.
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7.
A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study
Astermark J, Donfield SM, DiMichele DM, Gringeri A, Gilbert SA, Waters J, Berntorp E, FENOC Study Group
Blood. 2007;109((2):):546-51.
Abstract
The development of inhibitory antibodies to factor VIII is a serious complication of hemophilia. FEIBA (factor VIII inhibitor-bypassing activity), an activated prothrombin complex concentrate (aPCC), and NovoSeven, recombinant factor VIIa (rFVIIa), are used as hemostatic bypassing agents in treating patients with inhibitors. The FENOC study was designed to test equivalence of the products in the treatment of ankle, knee, and elbow joint bleeding. A prospective, open-label, randomized, crossover, equivalency design was used. The parameters of interest were the percentage of patients who reported efficacy in response to FEIBA and the percentage that reported efficacy in response to NovoSeven. A difference in these percentages of no more than 15% was determined to be a clinically acceptable magnitude for equivalence of the 2 products. The primary outcome was evaluation 6 hours after treatment. Data for 96 bleeding episodes contributed by 48 participants were analyzed. The criterion for declaring the 2 products equivalent at 6 hours was not met; however, the confidence interval of the difference in percentages of efficacy reported for each product only slightly exceeded the 15% boundary (-11. 4%-15. 7%), P=. 059. FEIBA and NovoSeven appear to exhibit a similar effect on joint bleeds, although the efficacy between products is rated differently by a substantial proportion of patients. This trial was registered at www. clinicaltrials. gov as #NCT00166309.
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8.
Italian guidelines for the diagnosis and treatment of patients with haemophilia and inhibitors
Gringeri A, Mannucci PM, Italian Association of Haemophilia Centres
Haemophilia. 2005;11((6):):611-9.
Abstract
The Italian Association of Haemophilia Centres reviewed and finally approved in November 2004 the new Italian Guidelines for the diagnosis and treatment of patients with clotting factor inhibitors. The recommendations have been based on the identification of levels of clinical evidence derived from the systematic review carried out in 2003 by the School of Health and Related Research, the University of Sheffield, UK, and further integrated by clinical studies published from 2003 to 2004. The Italian guidelines consist of six major domains concerning inhibitor definition, epidemiology, risk factors, diagnosis, inhibitor eradication, management of bleeding episodes, in patients with congenital and acquired coagulation disorders, with 121 statements, 59 synthesis and 54 recommendations. We report here recommendations and open issues concerning the diagnosis and monitoring of inhibitors, inhibitor eradication and the management of bleeding in patients with haemophilia A and B.
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9.
The FEIBA® NovoSeven® comparative study (FENOC) a randomized evaluation of by-passing agents in hemophilia complicated by inhibitors
Berntorp E, Donfield S, Waters J, Mattson E, DiMichele D, Gringeri A, Astermark J
Blood. 2005;106((11):): Abstract No. 324.
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10.
Pharmacokinetic comparison of three FVIII preparations: full-length rFVIII, full-length sucrose-formulated rFVIII, and b-domain deleted rFVII
Morfini M, Gringeri A, Cinotti S, Paladino E, Bizzoni L, Musso R, Piseddu G, Mannucci PM
Blood. 2002;100((11, Pt 1):):710a.. Abstract No. 2798.