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CO-01 | BLEEDING EVENTS IN MILD HEMOPHILIA A AND VON WILLEBRAND DISEASE TYPE 1 YEARLY TREATABLE WITH DESMOPRESSIN IN ITALY A. Gringeri1|2, P.M. Mannucci3, G.C. Castaman4, F. Peyvandi3|5 | 1Fondazione Charta, Milano, Italy; 2Centro di Sanità Solidale, Lucca, Italy; 3Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milano, Italy; 4Centre for Bleeding Disorders and Coagulation, Careggi University Hospital, Firenze, Italy; 5Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Italy
81PDF: 14 -
PO45 | Routine prophylaxis with PD-FX concentrate in severe inherited FX deficiency: a case report S. Linari, L. Pieri, M. Attanasio, F. Salvianti, M. Coppo, E. Grisanti, F. Demartis, R. Fjerza, G. Castaman | Centre for Bleeding Disorders and Coagulation, Azienda Ospedaliera Universitaria Careggi, Firenze, Italy
625PDF: 20 -
PO39 | Bleeding events in Von Willebrand disease type 1 yearly treatable with desmopressin A. Gringeri1,2, P.M. Mannucci3, M. Gringeri4, G. Castaman5, F. Peyvandi3,6 | 1Fondazione Charta, Milan; 2Centro di Sanità Solidale, Lucca; 3Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan; 4Department of Medical Biotechnologies and Translational Medicine, Postgraduate School of Clinical Pharmacology and Toxicology, University of Milan; 5Centre for Bleeding Disorders and Coagulation, Careggi University Hospital, Firenze; 6Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Italy
645PDF: 23 -
CO38 | Management of pregnancy, delivery, and postpartum in Italian carriers and women with hemophilia A and B S. Linari1, B. Pollio2, A. Ferretti3, M. Napolitano4, E. Grandone5, S. Siboni6, Ab. Aru7, E. Bertoni8, C. Biasoli9, I. Cantori10, P. Di Gregorio11, G. Lassandro12, G. Sottilotta13, A. Strangio14, Mr. Villa15, A. Rocino16, G. Castaman1 | 1Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Firenze; 2Regional Reference Center for Inherited Bleeding and Thrombotic Disorders, Transfusion Medicine, Regina Margherita Children Hospital, Turin; 3Center for Hemorrhagic and Thrombotic Diseases, IRCCS A. Gemelli University Hospital Foundation, Roma; 4Department of Health Promotion, Maternal-Childhood, Internal Medicine of Excellence G. D’Alessandro, University of Palermo; 5Thrombosis and Hemostasis Unit, Fondazione IRCCS Casa Sollievo della Sofferenza, San Giovanni Rotondo; 6Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan; 7Pediatric Onco-Hematology Center, Microcitemico Hospital Cagliari; 8Pediatric Oncohematology and Bone Marrow Transplant “BMT” Unit, Children’s Hospital, Spedali Civili, Brescia; 9Hemophilia and Transfusion Center, Bufalini Hospital, Cesena; 10Hemophilia Center, Department of Transfusion Medicine, Hospital of Macerata; 11Transfusion Medicine Department, SS. Annunziata Hospital, Chieti; 12B. Trambusti General and Specialised Pediatrics Unit, Giovanni XXIII Hospital, University of Bari; 13Hemophilia Center, Grande Ospedale Metropolitano, Reggio Calabria; 14Center for Hemorrhagic and Thrombotic Disorders, Pugliese Ciaccio Hospital, Catanzaro; 15Hemophilia and Thrombosis Center, Haematology Unit, Ospedale del Mare Hospital, Naples; 16Past President of the Italian Association of Hemophilia Centers, Italy
586PDF: 15 -
CO-64 | REAPPRAISAL OF DESMOPRESSIN FOR MANAGEMENT OF MILD/MODERATE VON WILLEBRAND DISEASE: RESULTS OF A RETROSPECTIVE ANALYSIS IN 127 ITALIAN PATIENTS SUGGEST FURTHER ORGANIZATION OF PROSPECTIVE OBSERVATIONAL STUDIES TO PREDICT DESMOPRESSIN FAILURE WITH IMMEDIATE VWF CONCENTRATES REQUIREMENT A.B. Federici1, G. Castaman2 | 1Ematologia e Medicina Trasfusionale, Ospedale Luigi Sacco, Polo Didattico Università di Milano, Italy; 2Centro Emofilia e Malattie Emorragiche Regione Toscana, Ospedale Careggi, Firenze, Italy
44PDF: 15 -
CO-07 | ITALIAN RESOURCE USE IN MILD HEMOPHILIA A AND TYPE 1 VON WILLEBRAND DISEASE A. Gringeri1|2, G.C. Castaman3 | 1Fondazione Charta, Milano, Italy; 2Centro di Sanità Solidale, Lucca, Italy; 3Centre for Bleeding Disorders and Coagulation, Careggi University Hospital, Firenze, Italy
94PDF: 17 -
The evolving landscape of gene therapy for congenital severe hemophilia: a 2024 state of the art
2089PDF: 359SUPPLEMENTARY MATERIAL: 218 -
PO043 | MULTICENTRE REAL‑WORLD EXPERIENCE OF EMICIZUMAB PROPHYLAXIS IN AN ITALIAN COHORT OF 49 CHILDREN WITH SEVERE HEMOPHILIA A - EMIPED STUDY L. Pieri1, B. Pollio2, P. Giordano3, G. Lassandro4, S. Linari1, R. Pasquino1, E. Piellucci1, MC. Di Stefano1, F. Demartis1, R. Fjerza1, G. Castaman1 | 1Center for Bleeding Disorders, Careggi University Hospital, Firenze, Italy; 2Center for Inherited Bleeding and Thrombotic Disorders, Transfusion Medicine, "Regina Margherita" Children Hospital, Torino, Italy; 3Department of Interdisciplinary of Medicine, University of Bari 'Aldo Moro', Bari, Italy; 4General and Specialised Pediatrics Unit, "Giovanni XXIII" Hospital, University of Bari, Bari, Italy
27PDF: 8 -
CO029 | REAL WORLD EVALUATION OF A NOVEL PATIENT CENTERED OUTCOME INDICATOR TO SUPPORT VALUE BASED HEALTHCARE DECISION-MAKING IN HEMOPHILIA A IN ITALY: THE HEMPROVE NON-INTERVENTIONAL STUDY G. Castaman1, C. Santoro2, E. Ferri Grazzi3, S. Bendinelli4, R. Perrone4, C. Lainez Diaz4, P.A. Cortesi5, M.N.D. Di Minno6 | 1Dep. of Oncology, Centre for Bleeding Disorders and Coagulation, Careggi Univ. Hosp., Firenze, Italy; 2Hematology, Dep. of Translational and Precision Medicine, Sapienza University of Roma, Italy; 3FedEmo, Milano, Italy; 4Roche S.p.A. Italy, Monza, Italy; 5CESP, Univ. of Milano-Bicocca, Monza, Italy; 6Dep. of Clinical Medicine and Surgery, Regional Reference Center for Coagulation Disorders, Federico II Univ., Napoli, Italy
34PDF: 12 -
BEST-04 | MAJOR ORTHOPEDIC SURGERY WITH EMICIZUMAB: A SERIES OF 22 PATIENTS (40 PROCEDURES) WITH A MID-TERM FOLLOW-UP G. Daniele1, C. Carulli1, S. Linari2, L. Pieri2, G. Castaman2 | 1Clinica Ortopedica, Azienda Ospedaliero-Universitaria Careggi, Università degli Studi di Firenze, Italy; 2Centro Malattie Emorragiche e della Coagulazione, Azienda Ospedaliero-Universitaria Careggi, Firenze, Italy
86PDF: 13 -
CO006 | PREVALENCE OF GASTROINTESTINAL BLEEDING IN GLANZMANN THROMBASTHENIA PATIENTS: RESULTS OF A WORLDWIDE SURVEY E. Falcinelli1, L. Bury1, S. Morais2, M. de Oliveira2, M. Kannan3, M. Faranoush4, D. Desprez5, V. Jean-Baptiste6, C. Santoro7, C. Falaise8, P. Saultier8, M. Fiore9, C. Albayrak10, A. Sánchez11, J.M. Bastida12, A. Artoni13, G. Castaman14, K. Gomez15, R. Schutgens16, L. Spiezia17, M. Sridharan18, N. Butta19, P. Buur van Kooten Niekerk20, E. Leinøe21, B. Guillet22, C. Panagiotis23, S. Crivelli24, E. De Candia25, M. Lewandowska26, P. De Bonis27, G. Kenet28, F. Heubel-Moenen29, J. Payne30, L. Banov31, L. Puccetti32, M. Olivieri33, M. Chitlur34, M. Lambert35, A. Pecci36, D. Martínez-Carballeira37, E. Zetterberg38, J. Curnow39, M. Napolitano40, A. Morotti41, R.C. Santoro42, R. Johann43, S. Saulyte Trakymiene44, A. Falanga45, C. Casas46, E. Zanon47, J. Moppet48, N.M. Popa-Fotea49, P. Gresele1 | 1Department of Medicine and Surgery, Section of Internal and Cardiovascular Medicine, University of Perugia, Italy; 2Centro Hospitalar Universitário de Santo António, Portugal; 3Blood and Vascular Biology Research Lab, Department of Biotechnology, Central University of Tamil Nadu, India; 4Iranian Hemophilia and Thrombophilia Association, MAHTA, Iran; 5CRC MHC Hopital Universaitire de Strasbourg, France; 6CHRU de Tours, University Hospital of Tours, France; 7UOC Ematologia, Azienda Ospedaliero Universitaria Policlinico Umberto I, Roma, Italy; 8AP-HM Timone, Marseille, France; 9CHU Bordeaux, France; 10Mayıs University Medical Faculty Pediatric Hematology and Oncology Dept, Türkiye; 11Hospital Universitario Morales Meseguer – Centro Regional de Hemodonación, Murcia, Spain; 12Complejo Asistencial Universitario De Salamanca, Spain; 13Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milano, Italy; 14Azienda Ospedaliera Universitaria Careggi Florence, Italy; 15Royal Free Hospital, London, UK; 16Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, UMC Utrecht, The Netherlands; 17University of Padova, Italy; 18Mayo Clinic, Rochester, USA; 19Hospital Universitario La Paz-IdiPAZ, Madrid, Spain; 20Aarhus University Hospital, Denmark; 21Department of Hematology, Rigshospitalet, Copenhagen University Hospital, Copenhagen, Denmark; 22University Hospital of Rennes, France; 23Blood unit and National Reference Center for Congenital Bleeding Disorders, ''Laiko'' General Hospital, Greece; 24Ospedale L. Sacco e Polo Universitario, Milano, Italy; 25Università Cattolica del Sacro Cuore-Roma, Fondazione Policlinico Universitario A. Gemelli IRCCS, Italy; 26Indiana Hemophilia and Thrombosis Center Innovative Hematology, USA; 27IRCCS Casa Sollievo della Sofferenza, San Giovanni Rotondo, Italy; 28Sheba Medical Center, Israel; 29Department of Hematology Internal Medicine, Maastricht University Medical Center and Hemophilia Treatment Center Nijmegen Eindhoven Maastricht and CARIM, Maastricht University, Maastricht, Netherlands; 30Sheffield Children’s NHS foundation Trust, UK; 31Hemostasis and Thrombosis Unit, Gaslini Children’s Hospital, Italy; 32University Teaching Hospital of Siena, Italy; 33Pediatric Thrombosis and Hemostasis Unit, Dr. von Hauner Childreńs Hospital, LMU Munich, Munich, Germany; 34Children's Hospital of Michigan, USA; 35Children’s Hospital of Philadelphia, USA; 36IRCCS Policlinico San Matteo Foundation and University of Pavia, Italy; 37Instituto de Investigación Sanitaria del Principado de Asturias (ISPA), Spain; 38Department of Hematology, Coagulation Unit, Skane University hospital, Sweden; 39Westmead Hospital, Sydney, Australia; 40PROMISE Department, University of Palermo, Italy; 41AOU San Luigi Gonzaga Hospital, University of Turin, Italy; 42Hemostasis and Thrombosis, Az. Osp. Univ. Dulbecco, Catanzaro, Italy; 43Hospital Center of Le Mans, France; 44Vilnius University Hospital Santaros Klinikos, Lithuania; 45ASST Papa Giovanni XXIII, Bergamo, Italy; 46Hospital San José, Bogotà, Colombia; 47Haemophilia Center-General Medicine, Padova University Hospital, Padova, Italy; 48University Hospitals Bristol NHS Foundation Trust, UK; 49University of Medicine and Pharmacy “Carol Davila”, Bucharest, Romania
39PDF: 11 -
Development of a shared decision-making tool for gene therapy in hemophilia A in Italy
933PDF: 139SUPPLEMENTARY MATERIAL: 78 -
CO-70 | CONGENITAL AFIBRINOGENEMIA: BLEEDING PHENOTYPE, THROMBOTIC COMPLICATIONS, AND LONG-TERM MANAGEMENT IN A MULTICENTER COHORT M. Biglietto1|2, M. Presti1, R.C. Santoro3, I. Nichele4, M. Napolitano5, S. Pasca6, A. Borchiellini7, B. Pollio8, E. Zanon9, G. Barillari10, A. Poz10, S. Sorella1, S. Ligia1, R. Mormile1, E. Baldacci1, G. Castaman11, F. Peyvandi12|13, C. Santoro1 | 1Hematology, Department of Translational and Precision Medicine, Sapienza University of Rome, Italy; 2Department of Molecular Biology, Sapienza University of Rome, Italy; 3Hemostasis and Thrombosis Unit, Azienda Ospedaliera Pugliese-Ciaccio, Catanzaro, Italy; 4Hematology Unit, Azienda ULSS 8 Berica - San Bortolo Hospital, Vicenza, Italy; 5Department of Health Promotion, Mother and Child Care, Internal Medicine and Medical Specialties, University of Palermo, Italy; 6Coagulation Diseases Center, Transfusion Medicine Department - Azienda Sanitaria Universitaria Integrata del Trentino, Trento, Italy; 7Centro di Riferimento Regionale Malattie Emorragiche e Trombotiche dell'Adulto, Divisione di Ematologia, Università Città della Salute e della Scienza di Torino, Italy; 8SIMT Immunohematology and Transfusion Medicine Service, Diagnostic Department, A.O. Città della Salute e della Scienza, Torino, Italy; 9Department of Medicine, Haemophilia Centre, University Hospital of Padua, Italy; 10Center for Hemorrhagic and Thrombotic Diseases, General and University Hospital S. Maria della Misericordia, Udine, Italy; 11Centre for Bleeding Disorders and Coagulation, Careggi University Hospital, Firenze, Italy; 12IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milano, Italy; 13Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milano, Italy
43PDF: 16 -
CO-67 | CONGENITAL DYSFIBRINOGENEMIA: BLEEDING PHENOTYPE, THROMBOTIC COMPLICATIONS, AND LONG-TERM MANAGEMENT IN A MULTICENTER COHORT M. Biglietto1|2, M. Presti1, R.C. Santoro3, I. Nichele4, M. Napolitano5, S. Pasca6, A. Borchiellini7, B. Pollio8, E. Zanon9, G. Barillari10, A. Poz10, S. Sorella1, S. Ligia1, R. Mormile1, E. Baldacci1, G. Castaman11, F. Peyvandi12|13, C. Santoro1 | 1Hematology, Department of Translational and Precision Medicine, Sapienza University of Rome, Italy; 2Department of Molecular Biology, Sapienza University of Rome, Italy; 3Hemostasis and Thrombosis Unit, Azienda Ospedaliera Pugliese-Ciaccio, Catanzaro, Italy; 4Hematology Unit, Azienda ULSS 8 Berica - San Bortolo Hospital, Vicenza, Italy; 5Department of Health Promotion, Mother and Child Care, Internal Medicine and Medical Specialties, University of Palermo, Italy; 6Coagulation Diseases Center, Transfusion Medicine Department - Azienda Sanitaria Universitaria Integrata del Trentino, Trento, Italy; 7Centro di Riferimento Regionale Malattie Emorragiche e Trombotiche dell'Adulto, Divisione di Ematologia, Università Città della Salute e della Scienza di Torino, Italy; 8SIMT Immunohematology and Transfusion Medicine Service, Diagnostic Department, A.O. Città della Salute e della Scienza, Torino, Italy; 9Department of Medicine, Haemophilia Centre, University Hospital of Padua, Italy; 10Center for Hemorrhagic and Thrombotic Diseases, General and University Hospital S. Maria della Misericordia, Udine, Italy; 11Centre for Bleeding Disorders and Coagulation, Careggi University Hospital, Firenze, Italy; 12IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milano, Italy; 13Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milano, Italy
46PDF: 13
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