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1. Demographic and clinical data in acquired hemophilia A

Author

Knoebl, P, Marco, P, Baudo, F, Collins, P, Huth Kühne, A, Nemes, L, Pellegrini, F, Tengborn, L, Lévesque, H, Aspoeck G, Heistinger M, Knöbl P, Makipernaa A, André H, Aouba A, Bellucci S, Beurrier P, Borg JY, Darnige L, Devignes J, D'Oiron R, Gautier P, Gay V, Girault S, Gruel Y, Guerin V, Hézard N, Khellaf M, Koenig M, Lévesque H, Lifermann F, Marlu R, Peynet J, Quéméneur T, Rothschild C, Schleinitz N, Sigaud M, Trouillier S, Voisin S, Giebl A, Holstein K, Huth Kühne A, Loreth RM, Steigerwald U, Tiede A, Theodossiades G, Nemes L, Radvanyi G, Schlammadinger A, Barillari G, Pasca S, Baudo F, Caimi T, Contino L, Di Minno G, Cerbone AM, Di Minno D, D'incà M, Falanga A, Maggioni A, Lerede T, Franchini M, Gaidano G, De Paoli L, Gamba G, Ghirardi R, Girotto M, Tasca D, Grandone E, Tiscia G, Imberti D, Iorio A, Landolfi R, Di Gennaro L, Novarese L, Mariani G, Lapecorella M, Marietta M, Pedrazzi P, Mazzucconi MG, Santoro C, Morfini M, Linari S, Moratelli S, Paolini R, Piseddu G, Poggio R, POGLIANI, ENRICO MARIA, Carpenedo M, Remiddi C, Santagostino E, Santoro R, Papaleo G, Schinco P, Borchiellini A, Scortechini AR, Siragusa S, Sottilotta G, Squizzato A, Sartori R, Tagariello G, Tagliaferri AR, Di Perna C, Rivolta GF, Testa S, Paoletti O, Toschi V, Zanon E, Brandolini B, Hamulyák K, Kamphuisen P, Laros van Gorkom B, Leebeek FW, Marten N, Novakova I, Schutgens R, van der Linden PW, van Esser J, van der Meer J, Ypma P, Campos M, Aguilar C, Altisent C, Bermejo N, Del Campo R, Ferreiro Argüelles M, González Boullosa R, Gutiérrez Pimentel MJ, Jiménez Yuste V, Jose Felix L, Pascual M, Mingot ME, Perez Garrido R, Perez Gonzale Nz, Prieto Garcia M, Rodriguez Huerta AM, Sedano C, Tolosa Munoz A, Baghaei F, Tengborn L, Boehlen F, Korte W, Chowdary P, Collins P, Evans G, Pavord S, Rangarajan S, Wilde J., Knoebl, P, Marco, P, Baudo, F, Collins, P, Huth Kühne, A, Nemes, L, Pellegrini, F, Tengborn, L, Lévesque, H, Group Author: Aspoeck, G, Heistinger, M, Knöbl, P, Makipernaa, A, André, H, Aouba, A, Bellucci, S, Beurrier, P, Borg, Jy, Darnige, L, Devignes, J, D'Oiron, R, Gautier, P, Gay, V, Girault, S, Gruel, Y, Guerin, V, Hézard, N, Khellaf, M, Koenig, M, Lifermann, F, Marlu, R, Peynet, J, Quéméneur, T, Rothschild, C, Schleinitz, N, Sigaud, M, Trouillier, S, Voisin, S, Giebl, A, Holstein, K, Loreth, Rm, Steigerwald, U, Tiede, A, Theodossiades, G, Radvanyi, G, Schlammadinger, A, Barillari, G, Pasca, S, Caimi, T, Contino, L, DI MINNO, Giovanni, Cerbone, Am, DI MINNO, matteo nicola dario, D'Incà, M, Falanga, A, Maggioni, A, Lerede, T, Franchini, M, Gaidano, G, De Paoli, L, Gamba, G, Ghirardi, R, Girotto, M, Tasca, D, Grandone, E, Tiscia, G, Imberti, D, Iorio, A, Landolfi, R, Di Gennaro, L, Novarese, L, Mariani, G, Lapecorella, M, Marietta, M, Pedrazzi, P, Mazzucconi, Mg, Santoro, C, Morfini, M, Linari, S, Moratelli, S, Paolini, R, Piseddu, G, Poggio, R, Pogliani, E, Carpenedo, M, Remiddi, C, Santagostino, E, Santoro, R, Papaleo, G, Schinco, P, Borchiellini, A, Scortechini, Ar, Siragusa, S, Sottilotta, G, Squizzato, A, Sartori, R, Tagariello, G, Tagliaferri, Ar, Di Perna, C, Rivolta, Gf, Testa, S, Paoletti, O, Toschi, V, Zanon, E, Hamulyák, K, Kamphuisen, P, Laros van Gorkom, B, Leebeek, Fw, Marten, N, Novakova, I, Schutgens, R, van der Linden, Pw, van Esser, J, van der Meer, J, Ypma, P, Campos, M, Aguilar, C, Altisent, C, Bermejo, N, Del Campo, R, Ferreiro Argüelles, M, González Boullosa, R, Gutiérrez Pimentel, Mj, Jiménez Yuste, V, Jose Felix, L, Pascual, M, Mingot, Me, Perez Garrido, R, Perez Gonzale, Nz, Prieto Garcia, M, Rodriguez Huerta, Am, Sedano, C, Tolosa Munoz, A, Baghaei, F, Boehlen, F, Korte, W, Chowdary, P, Evans, G, Pavord, S, Rangarajan, S, Wilde, J., Aspoeck, G, Borg, JY, Huth-Kühne, A, Loreth, RM, Di Minno, G, Cerbone, AM, Di Minno, D, D'incà, M, Mazzucconi, MG, Scortechini, AR, Tagliaferri, AR, Rivolta, GF, Laros-van Gorkom, B, Leebeek, FW, van der Linden, PW, Gutiérrez Pimentel, MJ Jiménez-Yuste, V, Jose-Felix, L, Pascual, M, Mingot, ME, Rodriguez-Huerta, AM, Wilde, J, Faculteit Medische Wetenschappen/UMCG, Cardiovascular Centre (CVC), Vascular Ageing Programme (VAP), Huth Kühne, A, Lévesque, H, Borg, J, Loreth, R, Cerbone, A, Mazzucconi, M, Scortechini, A, Tagliaferri, A, Rivolta, G, Brandolini, B, Leebeek, F, van der Linden, P, Gutiérrez Pimentel, M, Mingot, M, Perez Gonzale, N, Rodriguez Huerta, A, and Other departments

Subjects
Registrie, Male, Pediatrics, diagnosis, medicine.medical_treatment, Hemostatic Technique, Kaplan-Meier Estimate, registry, THERAPY, Settore MED/15 - Malattie Del Sangue, Immunosuppressive Agent, IMMUNOADSORPTION, Risk Factors, Pregnancy, 80 and over, Prospective Studies, Registries, Prospective cohort study, health care economics and organizations, Aged, 80 and over, treatment, Immunosuppression, Hematology, Middle Aged, FACTOR-VIII INHIBITOR, Acquired hemophilia, Demographics, Diagnosis, Outcome, Registry, Treatment, Aged, Autoantibodies, Chi-Square Distribution, Europe, Factor VIII, Female, Hemostatic Techniques, Humans, Immunosuppressive Agents, Risk Assessment, Treatment Outcome, Hemophilia A, Hemorrhage, Autoantibodie, acquired hemophilia, demographics, outcome, INTRAVENOUS GAMMA-GLOBULIN, Human, medicine.medical_specialty, Malignancy, hemophilia, registry, medicine, METAANALYSIS, Autoimmune disease, business.industry, Risk Factor, Autoantibody, medicine.disease, Surgery, Prospective Studie, Hemostasis, business, Chi-squared distribution
Abstract

Summary. Background: Acquired hemophilia A (AHA) is a rare autoimmune disease caused by autoantibodies against coagulation factor VIII and characterized by spontaneous hemorrhage in patients with no previous family or personal history of bleeding. Although data on several AHA cohorts have been collected, limited information is available on the optimal management of AHA. Objectives: The European Acquired Hemophilia Registry (EACH2) was established to generate a prospective, large-scale, pan-European database on demographics, diagnosis, underlying disorders, bleeding characteristics, treatment and outcome of AHA patients. Results: Five hundred and one (266 male, 235 female) patients from 117 centers and 13 European countries were included in the registry between 2003 and 2008. In 467 cases, hemostasis investigations and AHA diagnosis were triggered by a bleeding event. At diagnosis, patients were a median of 73.9 years. AHA was idiopathic in 51.9%; malignancy or autoimmune diseases were associated with 11.8% and 11.6% of cases. Fifty-seven per cent of the non-pregnancy-related cases were male. Four hundred and seventy-four bleeding episodes were reported at presentation, and hemostatic therapy initiated in 70.5% of patients. Delayed diagnosis significantly impacted treatment initiation in 33.5%. Four hundred and seventy-seven patients underwent immunosuppression, and 72.6% achieved complete remission. Conclusions: Representing the largest collection of consecutive AHA cases to date, EACH2 facilitates the analysis of a variety of open questions in AHA.

Published
2012

3. Sopravvivenza libera da trombosi in 734 pazienti con trombocitemia essenziale (WHO 2016) stratificati secondo lo score 'Revised International Prognostic Score for Thrombosis in Essential Thrombocythemia (R-IPSET-Th)'. Studio del Regostro ItalianoTrombocitemie (RIT)

Author

Gugliotta, L, Iurlo, A, Gugliotta, G, Tieghi, A, Gaidano, G, Dragani, A, Scalzulli, P, Martinelli, V, Martino, B, Randi, Ml, Cattaneo, D, Appolloni, V, Maschio, N, Langella, M, Santoro, C, Rago, A, Cacciola, Emma, Cacciola, Rossella Rosaria, Caocci, G, Plebani, S, Santoro, U, Vianelli, N, Mazzucconi, Mg, Specchia, G, Passamonti, F, and Vannucchi, Am

Published
2017

5. SEQUENTIAL USE OF THROMBOPOIETIN RECEPTOR AGONISTS IN ADULT PRIMARY IMMUNE THROMBOCYTOPENIA PATIENTS: A RETROSPECTIVE COLLABORATIVE SURVEY FROM ITALIAN HEMATOLOGY CENTERS

Author

Carpenedo, M, Cantoni, S, Mazzucconi, MG, De Stefano, V, Carrai, V, Ruggeri, M, Specchia, G, Vianelli, N, Pane, F, Consoli, U, Zaja, F, Artoni, A, Trentin, L, Ferrara, F, Barcellini, W, Caramazza, D, Rossi, E, Baldacci, E, Ciminello, A, Carbone, C, Cairoli, R, Carpenedo, M, Cantoni, S, Mazzucconi, M, De Stefano, V, Carrai, V, Ruggeri, M, Specchia, G, Vianelli, N, Pane, F, Consoli, U, Zaja, F, Artoni, A, Trentin, L, Ferrara, F, Barcellini, W, Caramazza, D, Rossi, E, Baldacci, E, Ciminello, A, Carbone, C, and Cairoli, R

Subjects
Hematology
Published
2017

8. Comparison of the rates of joint arthroplasty in patients with severe factor VIII and IX deficiency: an index of different clinical severity of the 2 coagulation disorders

Author

Tagariello G, Iorio A, Santagostino E, Morfini M, Bisson R, Innocenti M, Mancuso ME, Mazzucconi MG, Pasta GL, Radossi P, Rodorigo G, Santoro C, Sartori R, Scaraggi A, Solimeno LP, Mannucci PM, Ciavarella N, Valdrè L, Targhetta R, Biasioli C, Vincenzi D, Molinari C, Boeri E, Mariani G, Lapecorella M, Coppola A, Schiavulli M, Rocino A, Mancuso G, Siragusa S, Malato A, Tagliaferri A, Rivolta F, Oliovecchio E, Marcucci M, Dragani A, Mancino A, D'Incà M, De Rossi G, Luciani M, Landolfi R, Piseddu G, Schinco PC, Rossetti G, Barillari G, Feola G, Gandini G, Giuffrida A, Castaman G., DI MINNO, GIOVANNI, Tagariello, G, Iorio, A, Santagostino, E, Morfini, M, Bisson, R, Innocenti, M, Mancuso, ME, Mazzucconi, MG, Pasta, GL, Radossi, P, Rodorigo, G, Santoro, C, Sartori, R, Scaraggi, A, Solimeno, LP, Mannucci, PM, Ciavarella, N, Valdrè, L, Targhetta, R, Biasioli, C, Vincenzi, D, Molinari, C, Boeri, E, Mariani, G, Lapecorella, M, di Minno, G, Coppola, A, Schiavulli, M, Rocino, A, Mancuso, G, Siragusa, S, Malato, A, Tagliaferri, A, Rivolta, F, Oliovecchio, E, Marcucci, M, Dragani, A, Mancino, A, D'Incà, M, De Rossi, G, Luciani, M, Landolfi, R, Piseddu, G, Schinco, PC, Rossetti, G, Barillari, G, Feola, G, Gandini, G, Giuffrida, A, Castaman, G, Mancuso, Me, Mazzucconi, Mg, Pasta, Gl, Solimeno, Lp, Mannucci, Pm, DI MINNO, Giovanni, Schinco, Pc, and Castaman, G.

Subjects
Adult, medicine.medical_specialty, Adolescent, medicine.medical_treatment, Immunology, Population, macromolecular substances, Gene mutation, Hemophilia A, Hemophilia B, Severity of Illness Index, Biochemistry, Arthroplasty, Settore MED/15 - Malattie Del Sangue, Cohort Studies, Young Adult, Risk Factors, hemic and lymphatic diseases, Internal medicine, Severity of illness, medicine, Coagulopathy, Humans, Child, education, Retrospective Studies, education.field_of_study, Hematology, hamophilia, arthroplasty, business.industry, Retrospective cohort study, Cell Biology, Odds ratio, Blood Coagulation Disorders, Middle Aged, medicine.disease, Surgery, Italy, Joint Diseases, business, Algorithms
Abstract

Data from the Italian Hemophilia Centres were collected to perform a retrospective survey of joint arthroplasty in patients with severe hemophilia. Twenty-nine of 49 hemophilia centers reported that 328 of the 347 operations were carried out in 253 patients with severe hemophilia A (HA) and 19 in 15 patients with severe hemophilia B (HB). When results were normalized to the whole Italian hemophilia population (1770 severe HA and 319 severe HB), patients with HA had a 3-fold higher risk of undergoing joint arthroplasty (odds ratio [OR], 3.38; 95% confidence interval [CI], 1.97-5.77; P < .001). These results were confirmed after adjustment for age, HIV, hepatitis C virus (HCV), and inhibitor in a Cox regression model (HR, 2.65; 95% CI, 1.62-4.33; P < .001). The survival analysis of time to joint arthroplasty in the subset of patients with severe HA was not affected by the severity of factor VIII (FVIII) gene mutations. A systematic review of literature articles reporting joint arthroplasties in HA and HB showed that the proportion of HA patients who had undergone arthroplasties was higher than that of HB patients, in agreement with the findings in our Italian cohort. These data suggest that the 2 inherited coagulation disorders have a different severity of clinical phenotype.

Published
2009

9. Clinical and biological features in patients with Ph-negative chronic myeloproliferative neoplasms showing different molecular pattern comparative study in 596 patients of the Registro Italiano Trombocitemie (RIT)

Author

Gugliotta, L, Iurlo, A, Gugliotta, G, Tieghi, A, Specchia, G, Gaidano, G, Candoni, A, Randi, Ml, Scalzulli, Pr, Dragani, A, Martinelli, V, Tagariello, G, Antonioli, E, Liberati, Am, Palmieri, R, Langella, M, Rago, A, Cacciola, Rossella Rosaria, Pierri, I, Ricco, A, Cattaneo, D, Santoro, U, Rupoli, S, Santoro, C, Mastrullo, L, Mazzucconi, Mg, Vianelli, N, De Stefano, V, Passamonti, F, and Vannucchi, Am

Published
2015

10. THROMBOPOIETIN RECEPTOR AGONIST SWITCHING IN ADULT IMMUNE THROMBOCYTOPENIA PATIENTS: A RETROSPECTIVE COLLABORATIVE SURVEY FROM 8 ITALIAN CENTERS

Author

Carpenedo M, Cantoni S, Mazzucconi MG, De Stefano V, Ruggeri M, Vianelli N, Zaja F, Barcellini W, Nichelatti M, Coccini V, Baldacci E, Rossi E, Ciminello A, CAIROLI R, Carpenedo, M, Cantoni, S, Mazzucconi, M, De Stefano, V, Ruggeri, M, Vianelli, N, Zaja, F, Barcellini, W, Nichelatti, M, Coccini, V, Baldacci, E, Rossi, E, Ciminello, A, and Cairoli, R

Subjects
hematology
Published
2015

11. Immunosuppression for acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2)

Author

Collins, Peter, Baudo, Francesco, Knoebl, Paul, Lévesque, Hervé, Nemes, László, Pellegrini, Fabio, Marco, Pascual, Tengborn, Lilian, Huth Kühne, Angela, Group Author: Aspoeck G, Heistinger M, Knöbl P, Makipernaa A, André H, Aouba A, Bellucci S, Beurrier P, Borg JY, Darnige L, Devignes J, d'Oiron R, Gautier P, Gay V, Girault S, Gruel Y, Guerin V, Hézard N, Khellaf M, Koenig M, Lévesque H, Lifermann F, Marlu R, Ninet J, Peynet J, Quéméneur T, Rothschild C, Schleinitz N, Sigaud M, Trouillier S, Voisin S, Giebl A, Holstein K, Huth Kühne A, Loreth RM, Steigerwald U, Tiede A, Theodossiades G, Nemes L, Radvanyi G, Schlammadinger A, Barillari G, Pasca S, Baudo F, Caimi T, Contino L, D'Angelo Armando CL, Fattorini A, Cerbone AM, D'incà M, Falanga A, Maggioni A, Lerede T, Franchini M, Gaidano G, De Paoli L, Gamba G, Ghirardi R, Girotto M, Tasca D, Grandone E, Tiscia G, Imberti D, Iorio A, Landolfi R, Di Gennaro L, Novarese L, Mariani G, Lapecorella M, Marietta M, Pedrazzi P, Mazzucconi MG, Santoro C, Morfini M, Linari S, Moratelli S, Paolini R, Piseddu G, Poggio R, Pogliani E, Carpenedo M, Remiddi C, Santagostino E, Mancuso ME, Santoro R, Papaleo G, Schinco P, Borchiellini A, Valeri F, Scortechini AR, Siragusa S, Sottilotta G, Squizzato A, Tagariello G, Sartori R, Tagliaferri AR, Di Perna C, Rivolta GF, Testa S, Paoletti O, Toschi V, Zanon E, Brandolin B, Hamulyák K, Kamphuisen P, Laros van Gorkom B, Leebeek FW, Marten N, Novakova I, Schutgens R, van der Linden PW, van Esser J, van der Meer J, Ypma P, Campos M, Aguilar C, Altisent C, Bermejo N, Del Campo R, Ferreiro Argüelles M, González Boullosa R, Gutiérrez Pimentel MJ, Jiménez Yuste V, Jose Felix L, Marco P, Mingot ME, Perez Garrido R, Perez Gonzale NZ, Prieto Garcia M, Rodriguez Huerta AM, Sedano C, Tolosa Munoz A, Baghaei F, Tengborn L, Boehlen F, Korte W, Chowdary P, Collins P, Evans G, Pavord S, Rangarajan S, Wilde J., DI MINNO, GIOVANNI, DI MINNO, MATTEO, Interne Geneeskunde, Foundations and methods of Law, RS: CARIM School for Cardiovascular Diseases, Faculteit Medische Wetenschappen/UMCG, Cardiovascular Centre (CVC), Vascular Ageing Programme (VAP), Collins, Peter, Baudo, Francesco, Knoebl, Paul, Lévesque, Hervé, Nemes, László, Pellegrini, Fabio, Marco, Pascual, Tengborn, Lilian, Huth Kühne, Angela, Group Author: Aspoeck, G, Heistinger, M, Knöbl, P, Makipernaa, A, André, H, Aouba, A, Bellucci, S, Beurrier, P, Borg, Jy, Darnige, L, Devignes, J, D'Oiron, R, Gautier, P, Gay, V, Girault, S, Gruel, Y, Guerin, V, Hézard, N, Khellaf, M, Koenig, M, Lévesque, H, Lifermann, F, Marlu, R, Ninet, J, Peynet, J, Quéméneur, T, Rothschild, C, Schleinitz, N, Sigaud, M, Trouillier, S, Voisin, S, Giebl, A, Holstein, K, Huth Kühne, A, Loreth, Rm, Steigerwald, U, Tiede, A, Theodossiades, G, Nemes, L, Radvanyi, G, Schlammadinger, A, Barillari, G, Pasca, S, Baudo, F, Caimi, T, Contino, L, D'Angelo Armando, Cl, Fattorini, A, DI MINNO, Giovanni, Cerbone, Am, DI MINNO, Matteo, D'Incà, M, Falanga, A, Maggioni, A, Lerede, T, Franchini, M, Gaidano, G, De Paoli, L, Gamba, G, Ghirardi, R, Girotto, M, Tasca, D, Grandone, E, Tiscia, G, Imberti, D, Iorio, A, Landolfi, R, Di Gennaro, L, Novarese, L, Mariani, G, Lapecorella, M, Marietta, M, Pedrazzi, P, Mazzucconi, Mg, Santoro, C, Morfini, M, Linari, S, Moratelli, S, Paolini, R, Piseddu, G, Poggio, R, Pogliani, E, Carpenedo, M, Remiddi, C, Santagostino, E, Mancuso, Me, Santoro, R, Papaleo, G, Schinco, P, Borchiellini, A, Valeri, F, Scortechini, Ar, Siragusa, S, Sottilotta, G, Squizzato, A, Tagariello, G, Sartori, R, Tagliaferri, Ar, Di Perna, C, Rivolta, Gf, Testa, S, Paoletti, O, Toschi, V, Zanon, E, Brandolin, B, Hamulyák, K, Kamphuisen, P, Laros van Gorkom, B, Leebeek, Fw, Marten, N, Novakova, I, Schutgens, R, van der Linden, Pw, van Esser, J, van der Meer, J, Ypma, P, Campos, M, Aguilar, C, Altisent, C, Bermejo, N, Del Campo, R, Ferreiro Argüelles, M, González Boullosa, R, Gutiérrez Pimentel, Mj, Jiménez Yuste, V, Jose Felix, L, Marco, P, Mingot, Me, Perez Garrido, R, Perez Gonzale, Nz, Prieto Garcia, M, Rodriguez Huerta, Am, Sedano, C, Tolosa Munoz, A, Baghaei, F, Tengborn, L, Boehlen, F, Korte, W, Chowdary, P, Collins, P, Evans, G, Pavord, S, Rangarajan, S, Wilde, J., and Other departments

Subjects
Registrie, Male, Clinical Trials and Observations, medicine.medical_treatment, THERAPY, Biochemistry, Gastroenterology, Immunosuppressive Agent, Antibodies, Monoclonal, Murine-Derived, Adult, Aged, Aged, 80 and over, Autoantibodies, Cyclophosphamide, Cyclosporine, Europe, Factor VIII, Female, Follow-Up Studies, Hemophilia A, Humans, Immunosuppressive Agents, Middle Aged, Registries, Rituximab, Secondary Prevention, Steroids, Treatment Outcome, FACTOR-VIII INHIBITORS, Monoclonal, 80 and over, UNITED-KINGDOM, Hematology, RECLASSIFICATION, Immunosuppression, Autoantibodie, Human, medicine.drug, Murine-Derived, medicine.medical_specialty, Immunology, DIAGNOSIS, Antibodies, Follow-Up Studie, Internal medicine, MANAGEMENT, medicine, Steroid, Autoimmune disease, business.industry, Settore MED/09 - MEDICINA INTERNA, Autoantibody, Cell Biology, Odds ratio, CENTER DOCTORS ORGANIZATION, medicine.disease, Surgery, DISCRIMINATION, Etiology, business
Abstract

Acquired hemophilia A (AHA) is an autoimmune disease caused by an autoantibody to factor VIII. Patients are at risk of severe and fatal hemorrhage until the inhibitor is eradicated, and guidelines recommend immunosuppression as soon as the diagnosis has been made. The optimal immunosuppressive regimen is unclear; therefore, data from 331 patients entered into the prospective EACH2 registry were analyzed. Steroids combined with cyclophosphamide resulted in more stable complete remission (70%), defined as inhibitor undetectable, factor VIII more than 70 IU/dL and immunosuppression stopped, than steroids alone (48%) or rituximab-based regimens (59%). Propensity score-matched analysis controlling for age, sex, factor VIII level, inhibitor titer, and underlying etiology confirmed that stable remission was more likely with steroids and cyclophosphamide than steroids alone (odds ratio = 3.25; 95% CI, 1.51-6.96; P < .003). The median time to complete remission was approximately 5 weeks for steroids with or without cyclophosphamide; rituximab-based regimens required approximately twice as long. Immunoglobulin administration did not improve outcome. Second-line therapy was successful in approximately 60% of cases that failed first-line therapy. Outcome was not affected by the choice of first-line therapy. The likelihood of achieving stable remission was not affected by underlying etiology but was influenced by the presenting inhibitor titer and FVIII level.

Published
2012

12. Management of bleeding in acquired hemophilia A: results from the European Acquired Haemophilia (EACH2) Registry

Author

Baudo, Francesco, Collins, Peter, Huth Kühne, Angela, Lévesque, Hervé, Marco, Pascual, Nemes, László, Pellegrini, Fabio, Tengborn, Lilian, Knoebl, Paul, Group Author: Aspoeck, G, Heistinger, M, Knöbl, P, Makipernaa, A, André, H, Aouba, A, Bellucci, S, Beurrier, P, Borg, Jy, Darnige, L, Devignes, J, D'Oiron, R, Gautier, P, Gay, V, Girault, S, Gruel, Y, Guerin, V, Hézard, N, Khellaf, M, Koenig, M, Lévesque, H, Lifermann, F, Marlu, R, Ninet, J, Peynet, J, Quéméneur, T, Rothschild, C, Schleinitz, N, Sigaud, M, Trouillier, S, Voisin, S, Giebl, A, Holstein, K, Huth Kühne, A, Loreth, Rm, Steigerwald, U, Tiede, A, Theodossiades, G, Nemes, L, Radvanyi, G, Schlammadinger, A, Barillari, G, Pasca, S, Baudo, F, Caimi, T, Contino, L, D'Angelo Armando, Cl, Fattorini, A, Cerbone, Am, D'Incà, M, Falanga, A, Maggioni, A, Lerede, T, Franchini, M, Gaidano, G, De Paoli, L, Gamba, G, Ghirardi, R, Girotto, M, Tasca, D, Grandone, E, Tiscia, G, Imberti, D, Iorio, A, Landolfi, R, Di Gennaro, L, Novarese, L, Mariani, G, Lapecorella, M, Marietta, M, Pedrazzi, P, Mazzucconi, Mg, Santoro, C, Morfini, M, Linari, S, Moratelli, S, Paolini, R, Piseddu, G, Poggio, R, Pogliani, E, Carpenedo, M, Remiddi, C, Santagostino, E, Mancuso, Me, Santoro, R, Papaleo, G, Schinco, P, Borchiellini, A, Valeri, F, Scortechini, Ar, Siragusa, S, Sottilotta, G, Squizzato, A, Tagariello, G, Sartori, R, Tagliaferri, Ar, Di Perna, C, Rivolta, Gf, Testa, S, Paoletti, O, Toschi, V, Zanon, E, Brandolin, B, Hamulyák, K, Kamphuisen, P, Laros van Gorkom, B, Leebeek, Fw, Marten, N, Novakova, I, Schutgens, R, van der Linden, Pw, van Esser, J, van der Meer, J, Ypma, P, Campos, M, Aguilar, C, Altisent, C, Bermejo, N, Del Campo, R, Ferreiro Argüelles, M, González Boullosa, R, Gutiérrez Pimentel, Mj, Jiménez Yuste, V, Jose Felix, L, Marco, P, Mingot, Me, Perez Garrido, R, Perez Gonzale, Nz, Prieto Garcia, M, Rodriguez Huerta, Am, Sedano, C, Tolosa Munoz, A, Baghaei, F, Tengborn, L, Boehlen, F, Korte, W, Chowdary, P, Collins, P, Evans, G, Pavord, S, Rangarajan, S, Wilde, J., DI MINNO, GIOVANNI, DI MINNO, MATTEO, Other departments, Baudo, Francesco, Collins, Peter, Huth Kühne, Angela, Lévesque, Hervé, Marco, Pascual, Nemes, László, Pellegrini, Fabio, Tengborn, Lilian, Knoebl, Paul, Group Author: Aspoeck, G, Heistinger, M, Knöbl, P, Makipernaa, A, André, H, Aouba, A, Bellucci, S, Beurrier, P, Borg, Jy, Darnige, L, Devignes, J, D'Oiron, R, Gautier, P, Gay, V, Girault, S, Gruel, Y, Guerin, V, Hézard, N, Khellaf, M, Koenig, M, Lévesque, H, Lifermann, F, Marlu, R, Ninet, J, Peynet, J, Quéméneur, T, Rothschild, C, Schleinitz, N, Sigaud, M, Trouillier, S, Voisin, S, Giebl, A, Holstein, K, Huth Kühne, A, Loreth, Rm, Steigerwald, U, Tiede, A, Theodossiades, G, Nemes, L, Radvanyi, G, Schlammadinger, A, Barillari, G, Pasca, S, Baudo, F, Caimi, T, Contino, L, D'Angelo Armando, Cl, Fattorini, A, DI MINNO, Giovanni, Cerbone, Am, DI MINNO, Matteo, D'Incà, M, Falanga, A, Maggioni, A, Lerede, T, Franchini, M, Gaidano, G, De Paoli, L, Gamba, G, Ghirardi, R, Girotto, M, Tasca, D, Grandone, E, Tiscia, G, Imberti, D, Iorio, A, Landolfi, R, Di Gennaro, L, Novarese, L, Mariani, G, Lapecorella, M, Marietta, M, Pedrazzi, P, Mazzucconi, Mg, Santoro, C, Morfini, M, Linari, S, Moratelli, S, Paolini, R, Piseddu, G, Poggio, R, Pogliani, E, Carpenedo, M, Remiddi, C, Santagostino, E, Mancuso, Me, Santoro, R, Papaleo, G, Schinco, P, Borchiellini, A, Valeri, F, Scortechini, Ar, Siragusa, S, Sottilotta, G, Squizzato, A, Tagariello, G, Sartori, R, Tagliaferri, Ar, Di Perna, C, Rivolta, Gf, Testa, S, Paoletti, O, Toschi, V, Zanon, E, Brandolin, B, Hamulyák, K, Kamphuisen, P, Laros van Gorkom, B, Leebeek, Fw, Marten, N, Novakova, I, Schutgens, R, van der Linden, Pw, van Esser, J, van der Meer, J, Ypma, P, Campos, M, Aguilar, C, Altisent, C, Bermejo, N, Del Campo, R, Ferreiro Argüelles, M, González Boullosa, R, Gutiérrez Pimentel, Mj, Jiménez Yuste, V, Jose Felix, L, Marco, P, Mingot, Me, Perez Garrido, R, Perez Gonzale, Nz, Prieto Garcia, M, Rodriguez Huerta, Am, Sedano, C, Tolosa Munoz, A, Baghaei, F, Tengborn, L, Boehlen, F, Korte, W, Chowdary, P, Collins, P, Evans, G, Pavord, S, Rangarajan, S, Wilde, J., Faculteit Medische Wetenschappen/UMCG, Cardiovascular Centre (CVC), Vascular Ageing Programme (VAP), Huth Kühne, A, Lévesque, H, Pellegrini, F, Knoebl, P, Aspoeck, G, Borg, J, Loreth, R, D'Angelo Armando, C, Di Minno, G, Cerbone, A, Di Minno, D, Mazzucconi, M, Mancuso, M, Scortechini, A, Tagliaferri, A, Rivolta, G, Leebeek, F, van der Linden, P, Gutiérrez Pimentel, M, Mingot, M, Perez Gonzale, N, Rodriguez Huerta, A, and Wilde, J

Subjects
Registrie, Male, SURGERY, Biochemistry, THERAPY, Hemostatics, Hemostatic, FACTOR-VIII INHIBITORS, 80 and over, Deamino Arginine Vasopressin, Registries, Desmopressin, UNITED-KINGDOM, Factor IX, Aged, 80 and over, Hematology, biology, Incidence, FEIBA, Recombinant Protein, Middle Aged, Blood Coagulation Factors, Recombinant Proteins, Europe, Treatment Outcome, Coagulation, Female, medicine.drug, Blood Coagulation Factor, Human, Adult, medicine.medical_specialty, Adolescent, Immunology, Aged, Factor VIII, Factor VIIa, Hemophilia A, Hemorrhage, Humans, Young Adult, DIAGNOSIS, Internal medicine, BYPASSING ACTIVITY, medicine, business.industry, Settore MED/09 - MEDICINA INTERNA, RECOMBINANT FACTOR VIIA, Retrospective cohort study, Cell Biology, FACTOR-IX, CENTER DOCTORS ORGANIZATION, Surgery, Recombinant factor VIIa, Propensity score matching, biology.protein, business
Abstract

Acquired hemophilia A is a rare bleeding disorder caused by autoantibodies to coagulation FVIII. Bleeding episodes at presentation are spontaneous and severe in most cases. Optimal hemostatic therapy is controversial, and available data are from observational and retrospective studies only. The EACH2 registry, a multicenter, pan-European, Web-based database, reports current patient management. The aim was to assess the control of first bleeding episodes treated with a bypassing agent (rFVIIa or aPCC), FVIII, or DDAVP among 501 registered patients. Of 482 patients with one or more bleeding episodes, 144 (30%) received no treatment for bleeding; 31 were treated with symptomatic therapy only. Among 307 patients treated with a first-line hemostatic agent, 174 (56.7%) received rFVIIa, 63 (20.5%) aPCC, 56 (18.2%) FVIII, and 14 (4.6%) DDAVP. Bleeding was controlled in 269 of 338 (79.6%) patients treated with a first-line hemostatic agent or ancillary therapy alone. Propensity score matching was applied to allow unbiased comparison between treatment groups. Bleeding control was significantly higher in patients treated with bypassing agents versus FVIII/DDAVP (93.3% vs 68.3%; P = .003). Bleeding control was similar between rFVIIa and aPCC (93.0%; P = 1). Thrombotic events were reported in 3.6% of treated patients with a similar incidence between rFVIIa (2.9%) and aPCC (4.8%).

Published
2012

13. Pregnancy-associated acquired haemophilia A: Results from the European Acquired Haemophilia (EACH2) registry

Author

Tengborn, L, Baudo, F., Huth Kühne, A., Knoebl, P., Lévesque, H., Marco, P., Pellegrini, F., Nemes, L., Collins, P, Group Author: Aspoeck G, Heistinger M, Knöbl P, Makipernaa A, André H, Aouba A, Bellucci S, Beurrier P, Borg JY, Darnige L, Devignes J, d'Oiron R, Gautier P, Gay V, Girault S, Gruel Y, Guerin V, Hézard N, Khellaf M, Koenig M, Lévesque H, Lifermann F, Marlu R, Ninet J, Peynet J, Quéméneur T, Rothschild C, Schleinitz N, Sigaud M, Trouillier S, Voisin S, Giebl A, Holstein K, Huth Kühne A, Loreth RM, Steigerwald U, Tiede A, Theodossiades G, Nemes L, Radvanyi G, Schlammadinger A, Barillari G, Pasca S, Baudo F, Caimi T, Contino L, D'Angelo Armando CL, Fattorini A, Cerbone AM, D'incà M, Falanga A, Maggioni A, Lerede T, Franchini M, Gaidano G, De Paoli L, Gamba G, Ghirardi R, Girotto M, Tasca D, Grandone E, Tiscia G, Imberti D, Iorio A, Landolfi R, Di Gennaro L, Novarese L, Mariani G, Lapecorella M, Marietta M, Pedrazzi P, Mazzucconi MG, Santoro C, Morfini M, Linari S, Moratelli S, Paolini R, Piseddu G, Poggio R, Pogliani E, Carpenedo M, Remiddi C, Santagostino E, Mancuso ME, Santoro R, Papaleo G, Schinco P, Borchiellini A, Valeri F, Scortechini AR, Siragusa S, Sottilotta G, Squizzato A, Tagariello G, Sartori R, Tagliaferri AR, Di Perna C, Rivolta GF, Testa S, Paoletti O, Toschi V, Zanon E, Hamulyák K, Kamphuisen P, Laros van Gorkom B, Leebeek FW, Marten N, Novakova I, Schutgens R, van der Linden PW, van Esser J, van der Meer J, Ypma P, Campos M, Aguilar C, Altisent C, Bermejo N, Del Campo R, Ferreiro Argüelles M, González Boullosa R, Gutiérrez Pimentel MJ, Jiménez Yuste V, Jose Felix L, Marco P, Mingot ME, Perez Garrido R, Perez Gonzale NZ, Prieto Garcia M, Rodriguez Huerta AM, Sedano C, Tolosa Munoz A, Baghaei F, Tengborn L, Boehlen F, Korte W, Chowdary P, Collins P, Evans G, Pavord S, Rangarajan S, Wilde J., DI MINNO, GIOVANNI, DI MINNO, MATTEO, Tengborn, L, Baudo, F., Huth Kühne, A., Knoebl, P., Lévesque, H., Marco, P., Pellegrini, F., Nemes, L., Collins, P, Group Author: Aspoeck, G, Heistinger, M, Knöbl, P, Makipernaa, A, André, H, Aouba, A, Bellucci, S, Beurrier, P, Borg, Jy, Darnige, L, Devignes, J, D'Oiron, R, Gautier, P, Gay, V, Girault, S, Gruel, Y, Guerin, V, Hézard, N, Khellaf, M, Koenig, M, Lévesque, H, Lifermann, F, Marlu, R, Ninet, J, Peynet, J, Quéméneur, T, Rothschild, C, Schleinitz, N, Sigaud, M, Trouillier, S, Voisin, S, Giebl, A, Holstein, K, Huth Kühne, A, Loreth, Rm, Steigerwald, U, Tiede, A, Theodossiades, G, Nemes, L, Radvanyi, G, Schlammadinger, A, Barillari, G, Pasca, S, Baudo, F, Caimi, T, Contino, L, D'Angelo Armando, Cl, Fattorini, A, DI MINNO, Giovanni, Cerbone, Am, DI MINNO, Matteo, D'Incà, M, Falanga, A, Maggioni, A, Lerede, T, Franchini, M, Gaidano, G, De Paoli, L, Gamba, G, Ghirardi, R, Girotto, M, Tasca, D, Grandone, E, Tiscia, G, Imberti, D, Iorio, A, Landolfi, R, Di Gennaro, L, Novarese, L, Mariani, G, Lapecorella, M, Marietta, M, Pedrazzi, P, Mazzucconi, Mg, Santoro, C, Morfini, M, Linari, S, Moratelli, S, Paolini, R, Piseddu, G, Poggio, R, Pogliani, E, Carpenedo, M, Remiddi, C, Santagostino, E, Mancuso, Me, Santoro, R, Papaleo, G, Schinco, P, Borchiellini, A, Valeri, F, Scortechini, Ar, Siragusa, S, Sottilotta, G, Squizzato, A, Tagariello, G, Sartori, R, Tagliaferri, Ar, Di Perna, C, Rivolta, Gf, Testa, S, Paoletti, O, Toschi, V, Zanon, E, Hamulyák, K, Kamphuisen, P, Laros van Gorkom, B, Leebeek, Fw, Marten, N, Novakova, I, Schutgens, R, van der Linden, Pw, van Esser, J, van der Meer, J, Ypma, P, Campos, M, Aguilar, C, Altisent, C, Bermejo, N, Del Campo, R, Ferreiro Argüelles, M, González Boullosa, R, Gutiérrez Pimentel, Mj, Jiménez Yuste, V, Jose Felix, L, Marco, P, Mingot, Me, Perez Garrido, R, Perez Gonzale, Nz, Prieto Garcia, M, Rodriguez Huerta, Am, Sedano, C, Tolosa Munoz, A, Baghaei, F, Boehlen, F, Korte, W, Chowdary, P, Evans, G, Pavord, S, Rangarajan, S, and Wilde, J.

Subjects
Adult, Registrie, treatment, Pregnancy Complications, Hematologic, Obstetrics and Gynecology, Factor VIIa, Kaplan-Meier Estimate, Recombinant Protein, Antifibrinolytic Agent, Hemophilia A, Follow-Up Studie, Europe, diagnosi, Immunosuppressive Agent, Prospective Studie, Hemostatic, Treatment Outcome, Pregnancy, Drug Therapy, Combination, Female, Acquired haemophilia A, Blood Coagulation Factor, Human
Published
2012

14. Consumption of clotting factors in severe haemophilia patients undergoing prophylaxis and on-demand treatment in Italy

Author

Giampaolo A, Abbonizio F, Puopolo M, Arcieri R, Mannucci PM, Hassan HJ, Accorsi A, Ettorre PC, Schiavoni M, Palareti G, Rodorigo G, Valdré L, Amaddii G, Billio A, Notarangelo L, Iannacaro P, Muleo P, Biasioli C, Rossi V, Alatri A, Testa S, Vincenzi D, Scapoli G, Morfini M, Molinari AC, Lapecorella M, Mariani G, Baudo F, Caimi MT, Federici AB, Gringeri A, Santagostino E, Marietta M, Coppola A, Perricone C, Schiavulli M, Miraglia E, Rocino A, Zanon E, Gagliano F, Mancuso G, Siragusa S, Rivolta F, Tagliaferri A, Gamba G, Iorio A, Oliovecchio E, Dragani A, Arbasi MC, Albertini P, Mancino A, Lombardo VT, Latella C, D'Incà M, Landolfi Raffaele, Biondo Francesca, Mazzucconi MG, Santoro Cristina, Mameli AL, Piseddu G, Schinco PC, Messina M, Rossetti G, Barillari G, Feola G, Franchini M, Gandini G, Castaman G, Rodeghiero F, Italian Association of Haemophilia Centres, DI MINNO, GIOVANNI, Giampaolo, A, Abbonizio, F, Puopolo, M, Arcieri, R, Mannucci, Pm, Hassan, Hj, Accorsi, A, Ettorre, Pc, Schiavoni, M, Palareti, G, Rodorigo, G, Valdré, L, Amaddii, G, Billio, A, Notarangelo, L, Iannacaro, P, Muleo, P, Biasioli, C, Rossi, V, Alatri, A, Testa, S, Vincenzi, D, Scapoli, G, Morfini, M, Molinari, Ac, Lapecorella, M, Mariani, G, Baudo, F, Caimi, Mt, Federici, Ab, Gringeri, A, Santagostino, E, Marietta, M, Coppola, A, DI MINNO, Giovanni, Perricone, C, Schiavulli, M, Miraglia, E, Rocino, A, Zanon, E, Gagliano, F, Mancuso, G, Siragusa, S, Rivolta, F, Tagliaferri, A, Gamba, G, Iorio, A, Oliovecchio, E, Dragani, A, Arbasi, Mc, Albertini, P, Mancino, A, Lombardo, Vt, Latella, C, D'Incà, M, Landolfi, Raffaele, Biondo, Francesca, Mazzucconi, Mg, Santoro, Cristina, Mameli, Al, Piseddu, G, Schinco, Pc, Messina, M, Rossetti, G, Barillari, G, Feola, G, Franchini, M, Gandini, G, Castaman, G, Rodeghiero, F, and Italian Association of Haemophilia, Centres

Subjects
Clotting factor, Male, Pediatrics, medicine.medical_specialty, Factor VIII, business.industry, Haemophilia A, Hematology, Disease, medicine.disease, Haemophilia, Hemophilia A, Hemophilia B, Factor IX, On demand treatment, Coagulation, Italy, Surveys and Questionnaires, medicine, Humans, Female, National registry, business, Coagulation Disorder
Abstract

Dear Sir, Treatment of haemophilia is costly, and it must be carefully monitored by physicians and health authorities, so as to improve the planning of the supply of factor concentrates obtained from the fractionation of human plasma or by recombinant technology. Therapy with coagulation factors can be used either prophylactically to prevent bleeding, which entails multiple weekly infusions, or on-demand, through infusion of the amount of factors determined by the severity of the disease and patient’s weight, to stop ongoing bleeding. According to a recent study, factor VIII (FVIII) consumption among persons with haemophilia A (HA) has increased in countries with high-income economies (Stonebraker et al., 2010). In Italy, haemophiliacs and other persons with congenital coagulation disorders are monitored by the National Registry of Congenital Coagulopathies (NRCC) established at the Istituto Superi=

Published
2011

15. Replacement therapy with recombinant factor IX. A multicentre evaluation of current dosing practices in Italy

Author

Rocca A, Pizzinelli S, Oliovecchio E, Santagostino E, Rocino A, Iorio A, Contino L, Accorsi A, Ettorre C, Scaraggi FA, Rodorigo G, Valdrè L, Targhetta R, Tagariello G, Radossi P, Muleo G, Biasioli C, Morfini M, Molinari AC, Mariani G, Carloni MT, Cantori I, Mannucci PM, Coppola A, Schiavulli M, Zanon E, Tagliaferri A, Rivolta F, Marchesini E, Marcucci M, Dragani A, D'Inca M, Luciani M, Mazzucconi MG, Santoro C, Schinco P, Rossetti G, Barillari G, Gandini G, Castaman G., DI MINNO, GIOVANNI, Rocca, A, Pizzinelli, S, Oliovecchio, E, Santagostino, E, Rocino, A, Iorio, A, Contino, L, Accorsi, A, Ettorre, C, Scaraggi, Fa, Rodorigo, G, Valdrè, L, Targhetta, R, Tagariello, G, Radossi, P, Muleo, G, Biasioli, C, Morfini, M, Molinari, Ac, Mariani, G, Carloni, Mt, Cantori, I, Mannucci, Pm, DI MINNO, Giovanni, Coppola, A, Schiavulli, M, Zanon, E, Tagliaferri, A, Rivolta, F, Marchesini, E, Marcucci, M, Dragani, A, D'Inca, M, Luciani, M, Mazzucconi, Mg, Santoro, C, Schinco, P, Rossetti, G, Barillari, G, Gandini, G, and Castaman, G.

Abstract

Background: The in vivo recovery of recombinant factor IX (rFIX) is reported to be lower than that of plasma-derived products, with potential clinical implications for dosing. In clinical practice, a conversion (augmentation) factor is suggested to calculate the necessary doses of rFIX. The aim of this study was to assess the range of values for the conversion factor in usual clinical practice in Italy. Materials and methods: The study was questionnaire-based and proposed to all Italian Haemophilia centres treating patients with haemophilia B. Age, weight, dosage used in the last effective infusion, treatment regimen (prophylaxis versus on-demand), human immunodeficiency virus (HIV) and hepatitis C virus (HCV) status, and years of previous therapy with rFIX were recorded for patients with severe haemophilia B treated with rFIX. Mean, standard deviation, median and range were calculated for demographic and treatment data for the overall population and for subgroups. The conversion factor for the theoretical dosage of 40 IU/Kg was calculated. Results: Among 207 patients with severe haemophilia B being followed in 24 centres, 138 (66.7%) were being treated with rFIX. The sample of 207 patients represents 83.1% of the population of Italian patients with severe haemophilia B. The age range of the studied patients was 0-72 years (mean, 24 years) and the weight range was 3-108 kg (mean, 60 kg). Nineteen patients (14.4%) were positive for HIV and 51 (42.9%) were positive for HCV. The mean dosage of rFIX was 44 IU/Kg, with no significant difference between those receiving the product as prophylaxis or on-demand. A reduction in dosage was observed with increasing age (0.23 IU/kg/year). The mean value for the conversion factor was 1.10 ± 0.36 (median 1.00, range 0.51- 2.08), when estimated for the whole population. No effect of HIV and HCV status was found on the dose prescribed. No evident correlation was found with the underlying genetic mutation. Discussion: We found that dosing of rFIX in clinical practice is very close to that of plasmaderived FIX concentrates. As a consequence, dosing in the non-surgical setting should be started using the same criteria as those for plasma-derived FIX and treatment effectiveness verified on a clinical basis rather than relying on in vivo recovery assessments.

Published
2011

16. Mortality and causes of death in Italian persons with haemophilia, 1990-2007

Author

Tagliaferri, A, Rivolta, Gf, Iorio, A, Oliovecchio, E, Mancuso, Me, Morfini, M, Rocino, A, Mazzucconi, Mg, Franchini, M, Italian Association of Hemophilia Centers, Ciavarella, N, Scaraggi, A, Valdrè, L, Tagariello, G, Radossi, P, Muleo, G, Iannaccaro, Pg, Biasoli, C, Vincenzi, D, Serino, Ml, Linari, S, Molinari, C, Boeri, E, La Pecorella, M, Carloni, Mt, Santagostino, E, Di Minno, G, Coppola, A, Zanon, E, Spiezia, L, Di Perna, C, Marchesini, M, Marcucci, M, Dragani, A, Macchi, S, Albertini, P, D'Incà, M, Santoro, C, Biondo, F, Piseddu, G, Rossetti, G, Barillari, G, Gandini, Giorgio, Giuffrida, Anna Chiara, Castaman, G., Tagliaferri, A, Rivolta, Gf, Iorio, A, Oliovecchio, E, Mancuso, Me, Morfini, M, Rocino, A, Mazzucconi, Mg, Franchini, M, Ciavarella, N, Scaraggi, A, Valdrè, L, Tagariello, G, Radossi, P, Muleo, G, Iannaccaro, Pg, Biasoli, C, Vincenzi, D, Serino, Ml, Linari, S, Molinari, C, Boeri, E, La Pecorella, M, Carloni, Mt, Santagostino, E, DI MINNO, Giovanni, Coppola, A, Zanon, E, Spiezia, L, Di Perna, C, Marchesini, M, Marcucci, M, Dragani, A, Macchi, S, Albertini, P, D'Incà, M, Santoro, C, Biondo, F, Piseddu, G, Rossetti, G, Barillari, G, Gandini, G, Giuffrida, Ac, Castaman, G, and Italian Association of Hemophilia, Centers

Subjects
Pediatrics, retrospective study, HIV Infections, cause of death, Hepatitis C: complications, Hemophilia A: complications, hemophilia, Medicine, Young adult, Child, Genetics (clinical), Cause of death, education.field_of_study, Mortality rate, adult, article, Hematology, General Medicine, Hepatitis C, Middle Aged, Italy: epidemiology, aged, comorbidity, disease association, hepatitis C, human, Italy, life expectancy, major clinical study, male, mortality, priority journal, sex difference, HIV Infections: mortality, Child, Preschool, Female, medicine.medical_specialty, Adolescent, Population, Hepatitis C: mortality, Hemophilia A: mortality, Haemophilia, Hemophilia A, Hemophilia B, Young Adult, cancer, haemophilia, hcv, hiv, Humans, Cancer, HCV, HIV, Mortality, Preschool, education, Retrospective Studies, Hemophilia B: complications, HIV Infections: complications, business.industry, Retrospective cohort study, medicine.disease, Adult, Aged, Cause of Death, Hemophilia B: mortality, Life Expectancy, Male, Life expectancy, business
Abstract

Although a number of studies have analysed so far the causes of death and the life expectancy in haemophilic populations, no investigations have been conducted among Italian haemophilia centres. Thus, the aim of this study was to investigate mortality, causes of deaths, life expectancy and co-morbidities in Italian persons with haemophilia (PWH). Data pertaining to a total of 443 PWH who died between 1980 and 2007 were retrospectively collected in the 30 centres who are members of the Italian Association of Haemophilia Centres that chose to participate. The mortality rate ratio standardized to the male Italian population (SMR) was reduced during the periods 1990-1999 and 2000-2007 such that during the latter, death rate overlapped that of the general population (SMR 1990-1999: 1.98 95% CI 1.54-2.51; SMR 2000-2007: 1.08 95% CI 0.83-1.40). Similarly, life expectancy in the whole haemophilic population increased in the same period (71.2 years in 2000-2007 vs. 64.0 in 1990-1999), approaching that of the general male population. While human immunodeficiency virus infection was the main cause of death (45%), 13% of deaths were caused by hepatitis C-associated complications. The results of this retrospective study show that in Italian PWH improvements in the quality of treatment and global medical care provided by specialized haemophilia centres resulted in a significantly increased life expectancy.

Published
2010

17. Italian Registry of Haemophilia and Allied Disorders. Objectives, methodology and data analysis

Author

IORIO A, OLIOVECCHIO E, MORFINI M, MANNUCCI PM, CONTINO L, ACCORSI A, CIAVARELLA N, SCHIAVONI M, SCARAGGI FA, RODORIGO G, VALDRÉ L, TARGHETTA R, TAGARIELLO G, RADOSSI P, MUSSO R, CULTRERA D, MULEO G, IANNACARO P, BIASIOLI C, TESTA S, ALATRI A, VINCENZI D, SCAPOLI G, MOLINARI AC, BOERI E, CAPRINO D, MARIANI G, LAPECORELLA M, CARLONI MT, CANTORI I, SANTAGOSTINO E, GRINGERI A, FEDERICI AB, MARIETTA M, PEDRAZZOLI P, DI MINNO G, PERRICONE C, SCHIAVULLI M, ROCINO A, BERRETTINI M, ZANON E, MANCUSO G, TAGLIAFERRI A, RIVOLTA F, FERRANTE F, DRAGANI A, ROSSI A, ALBERTINI P, MACCHI S, D'INCA M, DE ROSSI G, LUCIANI M, LANDOLFI R, MAZZUCCONI MG, SANTORO C, PISEDDU G, CARLA SCHINCO P, ROSSETTI G, BARILLARI G, FEOLA G, GANDINI G, FRANCHINI M, CASTAMAN G., COPPOLA, Antonino, SIRAGUSA, Sergio, MALATO, Alessandra, SACCULLO, Giorgia, MANCINO, Antonio, IORIO A, OLIOVECCHIO E, MORFINI M, MANNUCCI PM, CONTINO L, ACCORSI A, CIAVARELLA N, SCHIAVONI M, SCARAGGI FA, RODORIGO G, VALDRÉ L, TARGHETTA R, TAGARIELLO G, RADOSSI P, MUSSO R, CULTRERA D, MULEO G, IANNACARO P, BIASIOLI C, TESTA S, ALATRI A, VINCENZI D, SCAPOLI G, MOLINARI AC, BOERI E, CAPRINO D, MARIANI G, LAPECORELLA M, CARLONI MT, CANTORI I, SANTAGOSTINO E, GRINGERI A, FEDERICI AB, MARIETTA M, PEDRAZZOLI P, DI MINNO G, COPPOLA A, PERRICONE C, SCHIAVULLI M, ROCINO A, BERRETTINI M, ZANON E, MANCUSO G, SIRAGUSA S, MALATO A, SACCULLO G, TAGLIAFERRI A, RIVOLTA F, FERRANTE F, DRAGANI A, ROSSI A, MANCINO A, ALBERTINI P, MACCHI S, D'INCA M, DE ROSSI G, LUCIANI M, LANDOLFI R, MAZZUCCONI MG, SANTORO C, PISEDDU G, CARLA SCHINCO P, ROSSETTI G, BARILLARI G, FEOLA G, GANDINI G, FRANCHINI M, and CASTAMAN G

Subjects
methodology, haemophilia, Settore MED/15 - Malattie Del Sangue
Published
2008

19. Pregnancy-associated acquired haemophilia A: results from the European Acquired Haemophilia (EACH2) registry

Author

Tengborn L, Baudo F, Huth-Kühne A, Knoebl P, Lévesque H, Marco P, Pellegrini F, Nemes L, Collins P, EACH2 registry contributors, Aspoeck G, Heistinger M, Knöbl P, Makipernaa A, André H, Aouba A, Bellucci S, Beurrier P, Borg JY, Darnige L, Devignes J, d'Oiron R, Gautier P, Gay V, Girault S, Gruel Y, Guerin V, Hézard N, Khellaf M, Koenig M, Lifermann F, Marlu R, Ninet J, Peynet J, Quéméneur T, Rothschild C, Schleinitz N, Sigaud M, Trouillier S, Voisin S, Giebl A, Holstein K, Loreth RM, Steigerwald U, Tiede A, Theodossiades G, Radvanyi G, Schlammadinger A, Barillari G, Pasca S, Caimi T, Contino L, D'Angelo Armando CL, Fattorini A, Di Minno G, Cerbone AM, Di Minno D, D'incà M, Falanga A, Maggioni A, Lerede T, Franchini M, Gaidano G, De Paoli L, Gamba G, Ghirardi R, Girotto M, Tasca D, Grandone E, Tiscia G, Imberti D, Iorio A, Landolfi R, Di Gennaro L, Novarese L, Mariani G, Lapecorella M, Marietta M, Pedrazzi P, Mazzucconi MG, Santoro C, Morfini M, Linari S, Moratelli S, Paolini R, Piseddu G, Poggio R, Pogliani E, Carpenedo M, Remiddi C, Santagostino E, Mancuso ME, Santoro R, Papaleo G, Schinco P, Borchiellini A, Valeri F, Scortechini AR, Siragusa S, Sottilotta G, Squizzato A, Tagariello G, Sartori R, Tagliaferri AR, Di Perna C, Rivolta GF, Testa S, Paoletti O, Toschi V, Zanon E, Hamulyák K, Kamphuisen P, Laros-van Gorkom B, Leebeek FW, Marten N, Novakova I, Schutgens R, van der Linden PW, van Esser J, van der Meer J, Ypma P, Campos M, Aguilar C, Altisent C, Bermejo N, Del Campo R, Ferreiro Argüelles M, González Boullosa R, Gutiérrez Pimentel MJ, Jiménez-Yuste V, Jose-Felix L, Mingot ME, Perez Garrido R, Perez Gonzale NZ, Prieto Garcia M, Rodriguez-Huerta AM, Sedano C, Tolosa Munoz A, Baghaei F, Boehlen F, Korte W, Chowdary P, Evans G, Pavord S, Rangarajan S, Wilde J, Tengborn, L, Baudo, F, Huth-Kühne, A, Knoebl, P, Lévesque, H, Marco, P, Pellegrini, F, Nemes, L, Collins, P, EACH2 registry, C, Aspoeck, G, Heistinger, M, Knöbl, P, Makipernaa, A, André, H, Aouba, A, Bellucci, S, Beurrier, P, Borg, J, Darnige, L, Devignes, J, D'Oiron, R, Gautier, P, Gay, V, Girault, S, Gruel, Y, Guerin, V, Hézard, N, Khellaf, M, Koenig, M, Lifermann, F, Marlu, R, Ninet, J, Peynet, J, Quéméneur, T, Rothschild, C, Schleinitz, N, Sigaud, M, Trouillier, S, Voisin, S, Giebl, A, Holstein, K, Loreth, R, Steigerwald, U, Tiede, A, Theodossiades, G, Radvanyi, G, Schlammadinger, A, Barillari, G, Pasca, S, Caimi, T, Contino, L, D'Angelo Armando, C, Fattorini, A, Di Minno, G, Cerbone, A, Di Minno, D, D'Incà, M, Falanga, A, Maggioni, A, Lerede, T, Franchini, M, Gaidano, G, De Paoli, L, Gamba, G, Ghirardi, R, Girotto, M, Tasca, D, Grandone, E, Tiscia, G, Imberti, D, Iorio, A, Landolfi, R, Di Gennaro, L, Novarese, L, Mariani, G, Lapecorella, M, Marietta, M, Pedrazzi, P, Mazzucconi, M, Santoro, C, Morfini, M, Linari, S, Moratelli, S, Paolini, R, Piseddu, G, Poggio, R, Pogliani, E, Carpenedo, M, Remiddi, C, Santagostino, E, Mancuso, M, Santoro, R, Papaleo, G, Schinco, P, Borchiellini, A, Valeri, F, Scortechini, A, Siragusa, S, Sottilotta, G, Squizzato, A, Tagariello, G, Sartori, R, Tagliaferri, A, Di Perna, C, Rivolta, G, Testa, S, Paoletti, O, Toschi, V, Zanon, E, Hamulyák, K, Kamphuisen, P, Laros-van Gorkom, B, Leebeek, F, Marten, N, Novakova, I, Schutgens, R, van der Linden, P, van Esser, J, van der Meer, J, Ypma, P, Campos, M, Aguilar, C, Altisent, C, Bermejo, N, Del Campo, R, Ferreiro Argüelles, M, González Boullosa, R, Gutiérrez Pimentel, M, Jiménez-Yuste, V, Jose-Felix, L, Mingot, M, Perez Garrido, R, Perez Gonzale, N, Prieto Garcia, M, Rodriguez-Huerta, A, Sedano, C, Tolosa Munoz, A, Baghaei, F, Boehlen, F, Korte, W, Chowdary, P, Evans, G, Pavord, S, Rangarajan, S, and Wilde, J

Subjects
Adult, Acquired haemophilia A, diagnosis, pregnancy, treatment, Antifibrinolytic Agents, Blood Coagulation Factors, Drug Therapy, Combination, Europe, Factor VIIa, Female, Follow-Up Studies, Hemostatics, Humans, Immunosuppressive Agents, Kaplan-Meier Estimate, Pregnancy, Prospective Studies, Recombinant Proteins, Registries, Treatment Outcome, Hemophilia A, Pregnancy Complications, Hematologic, Drug Therapy, Hematologic, Acquired Haemophilia, Pregnancy, Pregnancy Complications, Combination
Abstract

Objective The European Acquired Haemophilia registry (EACH2) collected data on the demographics, diagnosis, underlying disorders, bleeding characteristics, treatment, and outcome of women with acquired haemophilia A (AHA), a rare and often severe bleeding disorder caused by autoantibodies directed against coagulation factor VIII. Design Prospective, multi-centre, large-scale, pan-European registry. Setting A total of 117 haemophilia centres in 13 European countries. Population Pregnancy-associated AHA. Methods Data were reported using a web-based electronic case report form. Diagnosis was based on the presence of a prolonged activated partial thromboplastin time, reduced coagulation Factor VIII level and positive inhibitor assay. Main outcome measures Presenting characteristics, time to diagnosis, haemostatic treatment and outcome, immunosuppressive treatment and outcome. Results The EACH2 registry (n = 501) documented 42 (8.4%) cases of AHA associated with the peripartum period, a median Factor VIII level at diagnosis of 2.5 (range 0-25) IU/dl and inhibitor titre of 7.8 (range 0.7-348) BU/ml. Antepartum inhibitors were evident in eight women. Time to diagnosis of AHA after delivery was 89 (range 21-120) days. First-line haemostatic treatment was successful in 20/23 (87%) women treated. Bleeding episodes resolved in 17/18 (94%) women treated with a bypassing agent and 29/39 (74%) women achieved complete remission with first-line immunosuppressive treatment. Two babies experienced postnatal bleeding, suggesting transplacental transfer of the antibody. All women were alive at last follow-up. Conclusions Although rare, pregnancy-associated AHA may cause severe bleeding-related morbidity. Once diagnosed, women respond well to haemostatic treatment with bypassing agents and immunosuppression. Awareness of peripartum AHA requires improvement to facilitate rapid and appropriate management.

Published
2012

20. Cytoreductive combination therapy in essential trhombocythemia: preliminary report of the Registro Italiano trombocitemia (RIT)

Author

Gugliotta, L, Scalzulli, P, Tighi, A, Codeluppi, K, Valvano, R, Candoni, A, Chiozzotto, E, Liberati, M, Apolloni, V, Specchia, G, Cacciola, Emma, Cacciola, Rossella Rosaria, Mastrullo, R, Villa, M, Prugnola, M, Martinelli, V, Ciancia, R, Mazzucconi, Mg, Santoro, C, Campana, P, Randi, Ml, De Biasi, E, Maschio, N, Lanza, F, Antonioli, E, Pierri, I, Di Ianni, M, Iurlo, A, Piccin, A, and Cascavilla, N.

Published
2012

21. Intracranial haemorrhage in the Italian population of haemophilia patients with and without inhibitors

Author

Zanon, E, Iorio, A, Rocino, A, Artoni, A, Santoro, R, Tagliaferri, A, Coppola, A, Castaman, G, Mannucci, Pm, Italian Association of Hemophilia Centers, Barillari, G, Dragani, A, Gamba, G, Giuffrida, A, Lapecorella, M, Mancuso, G, Lucia, L, Mazzucconi, Mg, Messina, M, Musso, R, De Martis, F, Rossetti, G, Schinco, P, Spiezia, L, and Valdrè, L

Subjects
Questionnaires, Adult, Male, complications/immunology, Adolescent, Hemophilia A, Hemophilia B, fviii inhibitor, haemophilia a, haemophilia b, intracranial haemorrhage, Cohort Studies, Young Adult, Age Distribution, blood, Risk Factors, Surveys and Questionnaires, Prevalence, Humans, Preschool, Child, Aged, Autoantibodies, Retrospective Studies, Incidence, Infant, Newborn, Infant, Middle Aged, Newborn, epidemiology/prevention /&/ control, Blood Coagulation Factors, Adolescent, Adult, Age Distribution, Aged, Autoantibodies, blood, Blood Coagulation Factors, therapeutic use, Child, Child, Preschool, Cohort Studies, Hemophilia A, complications/immunology, Hemophilia B, complications/immunology, Humans, Incidence, Infant, Infant, Newborn, Intracranial Hemorrhages, epidemiology/prevention /&/ control, Italy, epidemiology, Male, Middle Aged, Prevalence, Questionnaires, Retrospective Studies, Risk Factors, Young Adult, Italy, therapeutic use, Child, Preschool, epidemiology, Intracranial Hemorrhages
Abstract

Intracranial haemorrhage (ICH) is the most serious bleeding symptom in haemophiliacs, resulting in high rates of mortality and disabling sequelae. The Association of Italian Haemophilia Centres carried out a retrospective survey (1987-2008) of ICH occurring in haemophiliacs with the goals to establish: (i) incidence, location of bleeding, death rate and disabling sequels; (ii) risk factors for ICH; and (iii) treatment used during the acute phase of ICH and for recurrence prevention. A total of 112 ICH episodes had occurred in 88 patients (78 haemophilia A, 10 haemophilia B), 24 of whom experienced recurrences. The cumulative hazard of ICH for the whole cohort over the entire follow-up period was 26.7 per 1000 patients, and the annualized rate of ICH was 2.50 events per 1000 patients (95% CI 1.90-3.31). The risk of ICH was higher in the youngest children (24.4 per 1000, 95% CI 12.7-47.0 in the first year of age and 14.9, 95% CI 7.1-31.4 in the second year of age) and then progressively rose again after the age of 40. Univariate, bivariate (age-adjusted) and multivariate analysis investigating the effects of patient characteristics on ICH occurrence showed that haemophilia severity and inhibitor status were strongly associated with ICH [severe vs. mild, HR 3.96 (2.39-6.57); inhibitor vs. non-inhibitor 2.52 (1.46-4.35)]. HCV infection was also associated with the risk of ICH [HR 1.83 (1.25-2.69)]. Therapeutic suggestions based upon our experience to control ICH recurrence are provided.

Published
2011

22. Low impact of cardiovascular adverse events on anagrelide treatment discontinuation in a cohort of 232 patients with essential thrombocythemia

Author

Gugliotta, L, Tieghi, A, Tortorella, G, Scalzulli, Pr, Ciancia, R, Lunghi, M, Cacciola, Emma, Cacciola, Rossella Rosaria, Candoni, A, Crugnola, M, Codeluppi, K, Usala, E, Specchia, G, Martinelli, V, Palmieri, F, Pierri, I, Liberati, M, Iurlo, A, Grossi, A, Vannucchi, Am, Vianelli, N, Mazzucconi, Mg, on behalf of the Registro Italiano Trombocitemai, Gugliotta, L, Tieghi, A, Tortorella, G, Scalzulli, Pr, Ciancia, R, Lunghi, M, Cacciola, E, Cacciola, R, Candoni, A, Crugnola, M, Codeluppi, K, Usala, E, Specchia, G, Martinelli, Vincenzo, Palmieri, F, Pierri, I, Liberati, Am, Iurlo, A, Grossi, A, Vannucchi, Am, Vianelli, N, and Mazzucconi, M. G.

Subjects
Male, Cancer Research, Adolescent, Adult, Aged, Aged, 80 and over, Cardiovascular Diseases, Child, Cohort Studies, Drug-Related Side Effects and Adverse Reactions, Female, Follow-Up Studies, Humans, Middle Aged, Platelet Aggregation Inhibitors, Quinazolines, Retrospective Studies, Thrombocythemia, Essential, Withholding Treatment, Young Adult, Echocardiogram, Essential thrombocythemia, Essential, 80 and over, Palpitations, Medicine, Thrombocythemia, Hematology, Oncology, Anagrelide, Safety, Toxicity, Cardiovascular adverse events, Palpitation, Cohort, Cardiology, medicine.symptom, Cohort study, medicine.drug, medicine.medical_specialty, Internal medicine, Adverse effect, business.industry, Retrospective cohort study, medicine.disease, Discontinuation, business
Abstract

This retrospective study of the thrombocythemia Italian registry (RIT) documented that 71 (30.6%) out of 232 ET patients experienced 88 cardiovascular adverse events (CV-AEs) during anagrelide treatment (522 pt-y). The rate of CV-AEs was: 24.1% for palpitations, 4.3% for angina, 3.5% for arterial hypertension, 3.0% for congestive heart failure, 1.8% for arrhythmia, 0.9% for AMI, 0.4% for pericardial effusion. CV-AEs led to treatment discontinuation in nine (3.9%) patients, while in the remaining cases they were managed by pharmacological intervention and/or patient life style improvement. CV-AEs had no relationship with patient characteristics (including older age). A significant relationship was found only with a higher anagrelide induction dose. In the absence of any agreed protocol, a cardiovascular instrumental evaluation (CV-IE) was performed in 102 (44%) patients before commencement of anagrelide (with higher rate after the anagrelide/Xagrid EMA approval of 2004), and in 84 (36%) patients during treatment. Patients with and without CV-IEs, who resulted completely balanced for all their characteristics, did not significantly differ in the occurrence of CV-AEs. In conclusion, this study on ET patients treated with anagrelide shows that CV-AEs, equally distributed in younger and older subjects, were mostly mild and easily manageable, allowing safe treatment continuation in the majority of cases. Moreover, routinely performing a CV-IE did not appear to anticipate the occurrence of CV-AEs.

Published
2011

26. Extra-haematological adverse events in 1075 essential thrombocythemia patients treated with hydroxyurea: a preliminary report of the Registro Italiano Trombocitemia (RIT)

Author

Gugliotta, L, Tieghi, A, Piccin, A, Papayannidis, C, Candoni, A, Specchia, G, Passamonti, F, Scalzulli, Pr, Lunghi, M, Radaelli, F, Iurlo, A, Vannucchi, Am, Ciancia, R, Randi, Ml, Palmieri, F, Liberati, M, Mazzucconi, Mg, Cacciola, Rossella Rosaria, Cacciola, Emma, Dragani, A, Cimino, G, Mastrullo, L, Fecerici, Ab, Porretto, F, Lucchesi, A, Vinelli, N, FOR THE, MARTINELLI G., and Rit

Published
2010

27. Hydroxyurea treatment in 1075 patients with essential thrombocythemia and occirrence of extra- hematological adverse events: a preliminary report of the Registro Italiano Trrombocitemia (RIT)

Author

Gugliotta, L, Papayannidis, C, Piccin, A, Pieghi, A, Vinelli, M, Scalzulli, Pr, Candoni, A, Dragani, A, Patriarca, A, Lunghi, M, Ciancia, R, Martionelli, V, Liberati, Am, Specchia, G, Imovilli, A, Vannucchi, Am, Maschio, A, Iurlo, A, Randi, Ml, Passamonti, F, Calmieri, Mazzucconi, Mg, LA TAGLIATA, R, Mastrullo, R, Cimino, G, Pierri, I, Cacciola, Emma, Lucchesi, A, Luraschi, A, Cozzani, E, Parodi, A, Martinelli, G, and Baccarani, M.

Published
2010

30. Anagrelide in assential thronbocythemia: a retrospective study

Author

Gugliotta, L, Grossi, A, Mazzucconi, Mg, Bulgarelli, S, Beggi, C, Liso, V, Specchia, G, Coser, P, Amato, B, Angelucci, E, Di Tucci, A, Sciorino, A, Giustolisi, R, Cacciola, Emma, Cacciola, Rossella Rosaria, Iuliano, F, Giordano, M, Bosi, A, Balestri, F, Ghio, R, Balleari, E, Spriano, M, Sacchi, S, Marcheselli, R, Rotoli, B, Martinelli, V, Ciancia, R, Gaidano, G, Conconi, A, Rizzoli, V, Prugnola, M, Trincali, S, Balduini, C, Noris, P, Martelli, M, Tabilio, A, Liberati, Am, Germani, A, Andriani, A, Lauria, F, Gentili, S, Carella, Am, Scalzulli, Pr, Boccadoro, M, Ciocca Vasino, A, Fanin, R, Candoni, A, Pizzolo, G, Ambrosetti, A, and Canotti, R.

Published
2003

32. Peg-Interferon -2b in essential thrombocythemia: phase II study for de- termination of the minimum effective, safe and tolerated dose. Prelimina- ry data

Author

Gugliotta, L, Russo, D, Bulgarelli, S, Vianelli, N, Caglio, S, Martinelli, V, Sacchi, S, Rupoli, S, Passamonti, F, Bucalossi, A, De Biasi, E, Cacciola, Rossella Rosaria, Cacciola, Emma, Candoni, A, Valdrè, L, Ciancia, R, Barulli, L, Agostini, V, Finazzi, G, Latagliata, R, Tabilio, A, Marcomigni, L, Miglino, M, Palazzo, G, Molinari, A, Grossi, A, Mazzucconi, Mg, Gobbi, M, Martelli, M, Zaccaria, A, Mazza, P, Boccadoro, M, Giustolisi, R, Lauria, F, Lazzarino, M, Leoni, P, Rotoli, B, Fanin, R, Rossi Ferrini PL, Barbui, T, Mandelli, F, Fincato, G, and Baccarani, M.

Published
2001

33. HIV-HCV RNA loads and liver failure in coinfected patients with coagulopathy

Author

Dragoni, F., Cafolla, A., Gentile, G., Mazzucconi, Mg, Vella, S., Di Corpo, U., Tosti, Me, Giulio Pisani, Donato, V., and Martino, P.

Subjects
Adult, CD4-Positive T-Lymphocytes, Male, HIV-RNA load, HCV-RNA load, liver failure, coinfection, coagulopathic patients, HIV Infections, Hepacivirus, Humans, Lymphocyte Count, Ultrasonography, Portal Vein, HIV, Alanine Transaminase, Organ Size, Blood Coagulation Disorders, Middle Aged, Hepatitis C, Cross-Sectional Studies, Liver, RNA, Viral, Female, Spleen, Follow-Up Studies
Abstract

The aim of this study was to measure contemporaneously HCV-RNA load, HIV-RNA load and CD4+ lymphocyte count in HCV/HIV coinfected patients with coagulopathy and to examine the relationship between these parameters and the liver failure.A cross-sectional study was performed on 54 patients with severe coagulopathy: 39 HCV/HIV coinfected and 15 HCV+/HIV- comparable for age and HCV exposure time. HCV-RNA and HIV-RNA load, CD4+ lymphocyte count, biochemical and ultrasonographic parameters were evaluated at the time of entry to the study.Mean HCV-RNA load was significantly higher in coinfected patients (643,872 717,687 copies/mL) than in HCV+/HIV- (mean 161,573 276,896 copies/mL) (p = 0.01). The 39 HCV/HIV coinfected patients had a mean HIV-RNA load of 205,913 456,311 copies/mL (range 4,000-2,500,000) and a mean CD4+ lymphocyte count of 206.5171/microL (range 5-693). Five of the 39 (12.8%) coinfected patients had liver failure. In these five patients the mean HCV-RNA load (770,200 996,426 copies/mL) was high but not significantly different from that in the 34 HCV+/HIV+ patients (623,496 682,239 copies/mL) without liver failure (p = 1.0). Coinfected patients with liver failure had a significantly higher HIV-RNA load (mean 764, 599 978,542 copies/mL) and lower CD4+ lymphocyte count (mean 52.655. 6/microL) than those observed in coinfected patients without liver failure (p = 0.007 and p = 0.03, respectively). A significant inverse correlation was found between CD4+ lymphocyte count and HIV-RNA load (r = -0.37, p = 0.01).HCV-RNA load is significantly higher in HIV+ than in HIV- patients with coagulopathy. Liver failure was found only in the HCV/HIV coinfected patients with severe immunodepression, expressed either by low CD4+ lymphocyte count or by high HIV-RNA load.

Published
1999

34. Essential Thrombocythaemia: prognostic factors in the italian series of two thousand patients

Author

Gugliotta, L, Lazzarino, M, Marchioli R, R., Ambrosetti, A, Baravelli, S, Bazzan, M, Cacciola, Emma, Calori, R, Ciocca Vasino, A, De Vivo, A, Fiacchini, M, Finazzi, G, Gargantini, L, Grossi, A, Iannacaro, Pg, Leva, T, Magrini, U, Marfisi, Mr, Martinelli, V, Mazzucconi, Mg, Morelli, A, Novarino, A, Palmieri, F, Pogliani, E, Radaelli, F, Randi, L, Rege Cambrin, G, Ronco, F, Ruggeri, M, Rupoli, S, Russo, D, Sacchi, S, Valdrè, L, Vianelli, N, Rodeghiero, F, Barbui, T, and for the GIMMC, Tura S.

Published
1999

35. Trombocitemia essenziale: aspetti diagnostici e terapeutici nella casistica ita- liana

Author

Gugliotta, L, Vianelli, N, Baravelli, S, Fiacchini, M, Marchioli, R, Marfisi, Rm, Valdrè, L, Radaelli, F, Gargantini, L, Finazzi, G, Grossi, A, Zaccaria, A, Ambrosetti, A, Mazzucconi, Mg, Ruggeri, M, Cacciola, Emma, Randi, L, Bazzan, M, Morelli, A, Volpe, E, Martinelli, V, Fasano, A, Specchia, G, Pardini, S, Iannaccaro, P, Ronco, F, Russo, D, and per il GIMMC, Rupoli S. e. Lazzarino M.

Published
1997

36. Epidermiological, diagnostic, therapeutic and prognostic aspects of essential thrombocithemia in a retrospective study of the GIMMC group in two thousand patients

Author

Gugliotta, L, Marchioli, R, Fiacchini, M, Vianelli, N, Baravelli, S, Valdrè, L, De vivo, A, Marfisi, R, Radaelli, F, Garantini, L, Ambrosetto, A, Mazzucconi, Mg, Russo, D, Bazzan, M, Zaccaria, A, Finazzi, G, Grossi, A, Ruggeri, M, Pogliani, E, Sacchi, Stefano, Martelli, V, Barbui, T, and Lazzarino, M.

Subjects
Thrombocythemia
Published
1997

44. Heterogeneity of type I von Willebrand disease: evidence for a subgroup with an abnormal von Willebrand factor

Author

Mannucci, PM, Lombardi, R, Bader, R, Vianello, L, Federici, AB, Solinas, S, Mazzucconi, MG, and Mariani, G

Abstract

Type I von Willebrand disease (vWD) is characterized by equally low plasma concentrations of von Willebrand factor antigen (vWF:Ag) and ristocetin cofactor (RiCof) and by the presence of all vWF multimers in sodium dodecyl sulfate (SDS)-agarose gel electrophoresis. For 17 patients (13 kindreds) diagnosed with these criteria, we have studied the platelet contents of vWF:Ag and RiCof and the changes of these in plasma after DDAVP infusion. Platelet vWF:Ag and RiCof were normal in four kindreds (called “platelet normal” subgroup); following 1-deamino- 8-D-arginine vasopressin; plasma vWF:Ag, RiCof and the bleeding time (BT) became normal. In six kindreds, platelet vWF:Ag and RiCof were equally low (platelet low); after DDAVP, plasma vWF:Ag and RiCof remained low, and the BT was prolonged. In three additional kindreds, platelets contained normal concentrations of vWF:Ag, but RiCof was very low (platelet discordant); even though a complete set of multimers was found in plasma and platelets, there was a relatively small amount of large multimers. After DDAVP, plasma vWF:Ag became normal, but RiCof remained low and the BT was very prolonged. These findings demonstrated that there can be an abnormal vWF (RiCof less than vWF:Ag) even in type I vWD, coexisting with a complete set of vWF multimers (platelet discordant); that the abnormal vWF can be shown more clearly in platelets than in plasma or else in plasma after DDAVP infusion; and that DDAVP normalizes the BT only in those patients with normal platelet levels of both vWF:Ag and RiCof (platelet normal).

Published
1985
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45. A variant of von Willebrand's disease characterized by recessive inheritance and missing triplet structure of von Willebrand factor multimers

Author

Mannucci, PM, Lombardi, R, Pareti, FI, Solinas, S, Mazzucconi, MG, and Mariani, G

Abstract

A 10-yr-old girl had bleeding symptoms of moderate severity; her mother and maternal aunt had milder bleeding symptoms, and other members of the kindred were asymptomatic. In the child, factor VIII coagulant activity (VIII:C) and von Willebrand factor antigen (vWF:Ag) were normal, ristocetin cofactor very low, and the bleeding time (BT) markedly prolonged. These values were normal in the rest of the kindred, but the mother and maternal aunt had prolonged BT and a high VIII:C/vWF:Ag ratio. Crossed immunoelectrophoresis (CIE) showed a vWF:Ag peak migrating more anodally in the propositus, two distinct peaks, one migrating anodally, in the father, paternal uncle, and grandmother, and normal peaks in the rest of the kindred. In the propositus, analysis of vWF multimers in plasma on 1.6% sodium dodecyl sulfate (SDS) agarose revealed that there were no larger multimers and there was a relative increase of the smallest multimer. This relative increase was also seen in her relatives with a double peak on CIE. Using gels of smaller porosity, each multimer of the propositus's plasma consisted of a single band, instead of the repeating triplet seen in normal and von Willebrand's disease varients types IIA and IIB. The abnormalities found in the propositus are tentatively interpreted as being due to double heterozygosity for two different genes. The defective gene carried by the father affects the triplet structure of vWF multimers, whereas a prolonged BT and a high VII:C/vWF:Ag ratio are the only phenotypic expressions of the defective gene of the mother. The findings of aberrant triplet structure in congenital vWD strengthen the view that this structure is an intrinsic feature of the normal vWF molecule.

Published
1983
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46. Anagrelide in essential thrombocythemia: A retrospective analysis of 220 patients

Author

Gugliotta, L., Grossi, A., Mazzucconi, Mg, Bulgarelli, S., Gamberi, B., Annalisa Imovilli, Balestri, F., Liso, V., Specchia, G., Scalzulli, Pr, Liberati, Am, Bassetti, A., Angelucci, E., Di Tucci, A., Iuliano, F., Gaidano, G., Conconi, Ar, Cacciola, E., Cacciola, R., Spriano, M., Crugnola, M., Tabilio, A., Balduini, C., Noris, P., Amato, B., Candoni, A., Balleari, E., Sacchi, S., Ambrosetti, A., Zanotti, R., Giordano, M., Andriani, A., Sciorio, A., Vasino, Ac, Martinelli, V., Ciancia, R., Tringali, S., and Lauria, F.

47. THE IMPACT OF ANTIVIRAL THERAPY WITH ZIDOVUDINE - A RETROSPECTIVE STUDY ON HIV-POSITIVE HEMOPHILIACS IN ITALY

Author

Arcieri, R., maria Puopolo, Baudo, F., Chiarotti, F., Derosa, V., Schinaia, N., Mori, Pg, Ghirardini, A., Greco, D., Bellocco, R., Palombi, M., Zanetti, L., Mannucci, Pm, Debiasi, R., Mariani, G., Morfini, M., Ciavarella, N., Perugini, L., Tamponi, G., Schinco, Pc, Quartino, Vr, Galletti, A., Barone, E., Gamba, G., Caimi, T., Saladini, A., Carnelli, V., Gringeri, A., Santagostino, E., Tradati, F., Duse, M., Soresina, Ar, Rubertelli, M., Rossetti, G., Coser, P., Traldi, A., Tagariello, G., Rodeghiero, F., Castaman, G., Girolami, A., Mares, M., Sartori, Mt, Gandini, G., Biffoni, F., Dettori, Ag, Tagliaferri, Ar, Bencivelli, V., Rodorigo, G., Coccheri, S., Ballerini, G., Berrettini, M., Longo, G., Rafanelli, D., Panicucci, F., Pula, Ep, Ribichini, G., Mazzucconi, Mg, Chistolini, A., Desanctis, V., Dantonio, A., Mastrullo, L., Rocino, A., Carola, A., Schiavoni, M., Fasano, A., Scaraggi, Fa, Perricci, A., Marino, R., Lombardo, Vt, Muleo, G., Santoro, R., Musso, R., Cabibbo, S., Mancuso, G., Alba, B., Biddau, Pf, Pitturro, C., Cera, Mmg, and Piseddu, G.

48. Real-world use of thrombopoietin receptor agonists in elderly patients with primary immune thrombocytopenia

Author

Wilma Barcellini, Michele Cavo, Emanuele Sutto, Francesca Palandri, Andrea Patriarca, Daniela Bartoletti, Erminia Baldacci, Gaetano Giuffrida, Francesco Zaja, Monica Carpenedo, Ugo Consoli, Antonietta Ferretti, Federico Chiurazzi, Daniela Nicolosi, Elena Rivolti, Giuseppe Auteri, Esther Oliva, Maria Gabriella Mazzucconi, Elisa Lucchini, Silvia Cantoni, Giuseppe Carli, Valerio De Stefano, Nicola Vianelli, Giovanni Caocci, Francesco Rodeghiero, Elena Rossi, Valentina Carrai, Alessandra Borchiellini, Marco Ruggeri, Palandri, F, Rossi, E, Bartoletti, D, Ferretti, A, Ruggeri, M, Lucchini, E, Carrai, V, Barcellini, W, Patriarca, A, Rivolti, E, Consoli, U, Cantoni, S, Oliva, En, Chiurazzi, F, Caocci, G, Giuffrida, G, Borchiellini, A, Auteri, G, Baldacci, E, Carli, G, Nicolosi, D, Sutto, E, Carpenedo, M, Cavo, M, Mazzucconi, Mg, Zaja, F, De Stefano, V, Rodeghiero, F, Vianelli, N., Palandri F., Rossi E., Bartoletti D., Ferretti A., Ruggeri M., Lucchini E., Carrai V., Barcellini W., Patriarca A., Rivolti E., Consoli U., Cantoni S., Oliva E.N., Chiurazzi F., Caocci G., Giuffrida G., Borchiellini A., Auteri G., Baldacci E., Carli G., Nicolosi D., Sutto E., Carpenedo M., Cavo M., Mazzucconi M.G., Zaja F., De Stefano V., Rodeghiero F., and Vianelli N.

Subjects
Male, Receptors, Fc, Biochemistry, Gastroenterology, Benzoates, chemistry.chemical_compound, Older patients, Romiplostim, Retrospective Studie, Hydrazine, Medicine, Platelet, Aged, 80 and over, Hematology, Middle Aged, Thrombosis, Hydrazines, Thrombopoietin, Idiopathic Thrombocytopenic Purpura, Eltrombopag, Female, Receptors, Thrombopoietin, medicine.drug, Human, Thrombopoietin Receptor Agonists, medicine.medical_specialty, Immune Thrombocytopenia, Recombinant Fusion Proteins, Immunology, Hemorrhage, Follow-Up Studie, Benzoate, Internal medicine, Humans, Retrospective Studies, Aged, Purpura, Thrombocytopenic, Idiopathic, business.industry, Cell Biology, medicine.disease, Immune thrombocytopenia, Settore MED/15 - MALATTIE DEL SANGUE, chemistry, Pyrazole, Pyrazoles, business, Fibrinolytic agent, Follow-Up Studies, Recombinant Fusion Protein
Abstract

The efficacy and safety of thrombopoietin receptor agonists (TRAs) in older patients with primary immune thrombocytopenia (ITP) are unknown. We investigated TRA response and switch, thrombotic/hemorrhagic risk, and sustained responses off-treatment (SROTs) in 384 patients with ITP aged ≥60 years. After 3 months, 82.5% and 74.3% of eltrombopag- and romiplostim-treated patients, respectively, achieved a response; 66.7% maintained the response (median follow-up, 2.7 years). Eighty-five (22.2%) patients switched to the alternative TRA; although no cross-toxicity was observed, 83.3% of resistant patients had a response after the switch. Thirty-four major thromboses (3 fatal) and 14 major hemorrhages (none fatal) occurred in 18 and 10 patients, respectively, while on TRAs and were associated with thrombosis history (subdistribution hazard ratio, 2.04, P = .05) and platelet count

Published
2021

49. Activated prothrombin complex concentrate (FEIBA® ) in acquired haemophilia A: a large multicentre Italian study - the FAIR Registry

Author

Gabriella Gamba, Anna Falanga, Angiola Rocino, Corrado Lodigiani, Gaetano Giuffrida, Ezio Zanon, Rita Santoro, Mariasanta Napolitano, Cristina Santoro, Sergio Siragusa, Maria Gabriella Mazzucconi, Marta Milan, Luciana Mameli, Augusto B. Federici, Samantha Pasca, Isabella Cantori, Chiara Ambaglio, Zanon E, Pasca S, Santoro C, Gamba G, Siragusa S, Rocino A, Cantori I, Federici AB, Mameli L, Giuffrida G, Falanga A, Lodigiani C, Santoro RC, Milan M, Ambaglio C, Napolitano M, Mazzucconi MG, Zanon, E, Pasca, S, Santoro, C, Gamba, G, Siragusa, S, Rocino, A, Cantori, I, Federici, A, Mameli, L, Giuffrida, G, Falanga, A, Lodigiani, C, Santoro, R, Milan, M, Ambaglio, C, Napolitano, M, and Mazzucconi, M

Subjects
Male, Pediatrics, medicine.medical_specialty, haemophilia, coagulation factors, 030204 cardiovascular system & hematology, Hemophilia A, Haemophilia, 03 medical and health sciences, 0302 clinical medicine, Acquired haemophilia, Humans, Medicine, coagulation factor, Prospective Studies, Registries, Prospective cohort study, Activated prothrombin complex concentrate, Retrospective Studies, bleeding disorders, factor VIII, Blood Coagulation Factors, Female, Italy, bleeding disorder, business.industry, Retrospective cohort study, Hematology, medicine.disease, business, 030215 immunology
Published
2018

50. A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A

Author

Kaan Kavakli, Mamta Manglani, Ezio Zanon, Christoph Male, Tarek Owaidah, Johnny Mahlangu, Ramabadran Varadarajan, Veronica Soto Arellano, Flora Peyvandi, Alessandra Nunes Loureiro Prezotti, Monica Martinez, Frits R. Rosendaal, Cecil Ross, Suresh Hanagavadi, Suvankar Majumdar, Bulent Zulfikar, Anupam Sachdeva, Brian M. Wicklund, Amal El-Beshlawy, Marilyn J. Manco-Johnson, M. Cerqueira, Dinesh M Nayak, Pier Mannuccio Mannucci, Santiago Bonanad Boix, Nadia P. Ewing, Klaus Schmitt, Angeles Palomo Bravo, Nathan L Kobrinsky, Maria Elisa Mancuso, Shashikant Apte, Mathew Thomas, Isabella Garagiola, Rogelio Paredes Aguilera, Guy Young, Maria Gabriella Mazzucconi, Esperanza Marzouka, Rosario Perez Garrido, Tulika Seth, Bülent Antmen, Mohsen Saleh Elalfy, Peyman Eshghi, E. Santagostino, Mindy L. Simpson, Vijay Ramanan, Daniela Neme, Mehran Karimi, Adriana C Sandoval Gonzalez, Çukurova Üniversitesi, Ege Üniversitesi, [Peyvandi,F, Mannucci,PM, Santagostino,E, Mancuso,ME] the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda Ospedale Maggiore Policlinico, Italy. [Peyvandi,F, and Garagiola,I] Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Italy. [Zanon,E] Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua. Italy. [Mazzucconi,MG] Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell’Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome, Italy. [El-Beshlawy,A] The Pediatric Hematology Department, Cairo University Pediatric Hospital, Cairo. [Elalfy,M] Department of Pediatrics, Faculty of Medicine, Ain Shams University, Cairo. [Ramanan,V] Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises, India. , [Apte,S] Sahyadri Speciality Hospita, l India. [Hanagavadi,S] Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere, India. [Baradajaran,P] Center for Blood Disorders, Chennai, India. [Manglani,MV] Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai, India. [Ross,C] St. John’s Medical College Hospital, Bangalore India. [Seth,T] India Institute of Medical Sciences, Department of Hematology, India. [Sachdeva,A] Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital, India. [Nayak,DM] New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal, India. [Thomas,M] Kerala Institute of Medical Science, Trivandrum, India. [Eshghi,P] The Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran, Iran. [Karimi,M] Hematology Research Center, Shiraz University of Medical Sciences, Shiraz, Iran. [Young,G] Children’s Hospital Los Angeles, Los Angeles,California. [Ewing,NP] City of Hope National Medical Center, Duarte, California. [Sandoval Gonzalez, AC] Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey, Mexico. [Paredes Aguilera,R] Instituto Nacional de Pediatria, Mexico City, Mexico. [Mahlangu,JN] Faculty of Health Sciences, School of Pathology, University of the Witwatersrand, National Health Laboratory Service and Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg.[Bonanad Boix,S] Hospital Universitario La Fe, Unidad Coagulopatias Congenitas, Valencia, Spain. [Perez Garrido, R] Hospital Universitario Virgen del Rocío, Unidad de Hemofilia, Seville, Spain. [Palomo Bravo,A] Hospital Regional Universitario Carlos Haya, Malaga, Spain. [Cerqueira,M] Centro de Pesquisa Clinica Hemorio–Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti, Rio de Janeiro, Brazil. [Prezotti,A] Centro de Hematologia e Hemoterapia do Espírito Santo, Vitoria, Brazil. [Male,C] Medizinische Universität Wien, Department of Pediatrics, Vienna, Austria. [Schmitt,K] Department of Pediatric and Adolescent Medicine, Kepler University Clinic, Linz, Austria. [Owaidah,T] King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. [Soto Arellano,V] Centro de Hemofílicos del Hospital de Niños Dr. Roberto del Río, Chile. [Marzouka,E] Hospital de Niños Dr. Luis Calvo Mackenna, Centro Hemofílico, Santiago, Chile. [Kobrinsky, NL] Sanford Roger Maris Cancer Center, Fargo, ND. [Majundar,S] University of Mississippi Medical Center, Division of Pediatric Hematology–Oncology, Jackson. [Simpson,M] Rush Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago. [Antmen,B] Cukurova Universitesi, Tip Fakultesi Pediatrik Hematoloji Bilim Dali, Adana, Turkey. [Kabakli,K] Ege Universitesi Tip Fakultesi Cocuk Sagligi ve Hastalikari Anabilim Dali, Pediatrik Hematoloji Bilim Dali, Turkey. [Zulfikar,B] Istanbul Universitesi Cerrahpasa Tip Fakultesi, Pediatrik Hematoloji Bilim Dali, Istanbul,Turkey. [Manco-Johnson,MJ] Hemophilia and Thrombosis Center, University of Colorado Denver, Aurora. [Martinez,M] Hospital de Niños Sor María Ludovica La Plata, Servicio de Hematología, Buenos Aires. [Neme,D] Fundación de la Hemofilia, Buenos Aires.[Wicklund,BM] Children’s Mercy Hospital, Kansas City, MO, The Netherlands. [Rosendaal,FR] he Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, The Netherlands.

Subjects
Male, Analytical, Diagnostic and Therapeutic Techniques and Equipment::Investigative Techniques::Epidemiologic Methods::Statistics as Topic::Models, Statistical::Proportional Hazards Models [Medical Subject Headings], Named Groups::Persons::Age Groups::Adult::Middle Aged [Medical Subject Headings], Adulto joven, 030204 cardiovascular system & hematology, Gastroenterology, law.invention, Organisms::Eukaryota::Animals::Chordata::Vertebrates::Mammals::Primates::Haplorhini::Catarrhini::Hominidae::Humans [Medical Subject Headings], 0302 clinical medicine, Randomized controlled trial, Isoantibodies, law, Hemorragia, hemic and lymphatic diseases, Medicine, Child, Masculino, Adolescente, Named Groups::Persons::Age Groups::Child::Child, Preschool [Medical Subject Headings], biology, Adulto, Incidence, Hemofilia A, General Medicine, Middle Aged, Modelos de riesgos proporcionales, Humanos, Diseases::Hemic and Lymphatic Diseases::Hematologic Diseases::Hemorrhagic Disorders::Hemophilia A [Medical Subject Headings], Analytical, Diagnostic and Therapeutic Techniques and Equipment::Therapeutics::Drug Therapy::Drug Administration Routes::Injections::Injections, Subcutaneous [Medical Subject Headings], Child, Preschool, Niño, Named Groups::Persons::Age Groups::Adolescent [Medical Subject Headings], Drug Therapy, Combination, Named Groups::Persons::Age Groups::Infant [Medical Subject Headings], Antibody, Incidencia, Niño preescolar, Adult, medicine.medical_specialty, Randomization, Adolescent, Named Groups::Persons::Age Groups::Adult::Young Adult [Medical Subject Headings], Injections, Subcutaneous, Anciano, Analytical, Diagnostic and Therapeutic Techniques and Equipment::Therapeutics::Drug Therapy::Drug Therapy, Combination [Medical Subject Headings], Check Tags::Male [Medical Subject Headings], Hemorrhage, Hemophilia A, Isoanticuerpos, Bethesda unit, Anticuerpos neutralizantes, Diseases::Pathological Conditions, Signs and Symptoms::Pathologic Processes::Hemorrhage [Medical Subject Headings], Young Adult, 03 medical and health sciences, Pharmacotherapy, Analytical, Diagnostic and Therapeutic Techniques and Equipment::Investigative Techniques::Epidemiologic Methods::Data Collection::Vital Statistics::Morbidity::Incidence [Medical Subject Headings], Von Willebrand factor, Internal medicine, von Willebrand Factor, Named Groups::Persons::Age Groups::Adult [Medical Subject Headings], Humans, Factor de von Willebrand, Named Groups::Persons::Age Groups::Adult::Aged [Medical Subject Headings], Named Groups::Persons::Age Groups::Child [Medical Subject Headings], Aged, Proportional Hazards Models, Emicizumab, Mediana edad, Factor VIII, Dose-Response Relationship, Drug, business.industry, Phenomena and Processes::Physiological Phenomena::Pharmacological Phenomena::Dose-Response Relationship, Drug [Medical Subject Headings], Chemicals and Drugs::Amino Acids, Peptides, and Proteins::Proteins::Globulins::Serum Globulins::Immunoglobulins::Antibodies::Isoantibodies [Medical Subject Headings], Infant, Inyecciones subcutáneas, Lactante, Antibodies, Neutralizing, Relación dosis-respuesta de medicamentos, Chemicals and Drugs::Biological Factors::Blood Coagulation Factors::von Willebrand Factor [Medical Subject Headings], Surgery, Farmacoterapia combinada, Chemicals and Drugs::Biological Factors::Blood Coagulation Factors::Factor VIII [Medical Subject Headings], biology.protein, Chemicals and Drugs::Amino Acids, Peptides, and Proteins::Proteins::Globulins::Serum Globulins::Immunoglobulins::Antibodies::Antibodies, Neutralizing [Medical Subject Headings], business, 030215 immunology
Abstract

WOS: 000376443500008, PubMed ID: 27223147, BACKGROUND The development of neutralizing anti-factor VIII alloantibodies (inhibitors) in patients with severe hemophilia A may depend on the concentrate used for replacement therapy. METHODS We conducted a randomized trial to assess the incidence of factor VIII inhibitors among patients treated with plasma-derived factor VIII containing von Willebrand factor or recombinant factor VIII. Patients who met the eligibility criteria (male sex, age = 5 Bethesda units). Inhibitors developed in 29 of the 125 patients treated with plasma-derived factor VIII (20 patients had high-titer inhibitors) and in 47 of the 126 patients treated with recombinant factor VIII (30 patients had high-titer inhibitors). The cumulative incidence of all inhibitors was 26.8% (95% confidence interval [CI], 18.4 to 35.2) with plasma-derived factor VIII and 44.5% (95% CI, 34.7 to 54.3) with recombinant factor VIII; the cumulative incidence of high-titer inhibitors was 18.6% (95% CI, 11.2 to 26.0) and 28.4% (95% CI, 19.6 to 37.2), respectively. In Cox regression models for the primary end point of all inhibitors, recombinant factor VIII was associated with an 87% higher incidence than plasma-derived factor VIII (hazard ratio, 1.87; 95% CI, 1.17 to 2.96). This association did not change in multivariable analysis. For high-titer inhibitors, the hazard ratio was 1.69 (95% CI, 0.96 to 2.98). When the analysis was restricted to recombinant factor VIII products other than second-generation full-length recombinant factor VIII, effect estimates remained similar for all inhibitors (hazard ratio, 1.98; 95% CI, 0.99 to 3.97) and high-titer inhibitors (hazard ratio, 2.59; 95% CI, 1.11 to 6.00). CONCLUSIONS Patients treated with plasma-derived factor VIII containing von Willebrand factor had a lower incidence of inhibitors than those treated with recombinant factor VIII. (Funded by the Angelo Bianchi Bonomi Foundation and others; ClinicalTrials.gov number, NCT01064284; EudraCT number, 2009-011186-88.), Angelo Bianchi Bonomi Foundation, Funded by the Angelo Bianchi Bonomi Foundation and others

Published
2016

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