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  The evolving landscape of gene therapy for congenital severe hemophilia: a 2024 state of the art

  Giovanni Di Minno, Gaia Spadarella, Ilenia Lorenza Calcaterra, Giancarlo Castaman, Paolo Simioni, Raimondo De Cristofaro, Cristina Santoro, Flora Peyvandi, Matteo Di Minno

  04-09-2024

  https://doi.org/10.4081/btvb.2024.144

  1048

  PDF: 226

  SUPPLEMENTARY MATERIAL: 105

• 29th National Conference of the Italian Society for the Study of Hemostasis and Thrombosis, 2025

  Guest Editor: Valerio De Stefano, Course President

  22-10-2025

  https://doi.org/10.4081/btvb.2025.408

  47

• CO35 | Bleeding phenotype characterization in patients with congenital bleeding disorders: a multigenic approach using next generation sequencing to identify coagulation-modulating variants C. Simion, L. Gianesello, C. Bulato, A. Benetti, E. Campello, A. Giannella, G. Ceolotto, E. Zanon, L. Spiezia, P. Simioni | Department of Medicine – DIMED, University of Padua, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.242

  36

• 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

  22-10-2025

  https://doi.org/10.4081/btvb.2025.245

  39

• PO02 | Pregnancy and delivery in women with hemophilia and carriers are still challenging: a ten years analysis from The Italian Association of Hemophilia Centers (AICE) M. Napolitano¹, M. Mattana², C. Cammarata¹, M. Leotta³, S. Linari⁴, M.E. Mancuso⁵, G. F. Rivolta⁶, C. Santoro⁷, L. Valdrè⁸, R.C. Santoro³, S. Siragusa¹ | ¹Department of Health Promotion, Mother and Child Care, Internal Medicine and Med, Palermo; University of Palermo, Italy; ²Department of Precision Medicine in Medical, Surgical and Critical Care “Me. Pre. Cc”, University of Palermo; ³Hemostasis and Thrombosis Unit, Azienda Ospedaliera Universitaria Dulbecco, Catanzaro; ⁴Center for Bleeding Disorders, Careggi University Hospital, Firenze; ⁵Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Rozzano, Milan; ⁶Regional Reference Center for the Treatment of Congenital Hemorrhagic Diseases, Parma University Hospital, Parma; ⁷Hematology, University Hospital Policlinico Umberto I, Roma; ⁸Inherited Bleeding Disorders Unit, IRCCS AOUBO, Bologna, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.312

  39

• CO37 | Assessment of clotting factor activity and sterility in thawed fresh frozen plasma supplied to helicopter emergency medical services for prehospital transfusion D. Romeo¹, M. Marchetti^1,2, M. Testa¹, A. Motta¹, P. Gomez-Rosas¹, G. Ravasio¹, F. Daminelli³, L. Barcella¹, A. Falanga^1,2 | ¹Immunohematology and Transfusion Medicine Department, Hospital Papa Giovanni XXIII, Bergamo; ²School of Medicine and Surgery, University Milano Bicocca, Milan; ³Anesthesia and Resuscitation Department, Hospital Papa Giovanni XXIII, Bergamo, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.244

  42

• CO34 | Evaluation of emicizumab concentration and thrombin generation in a cohort of severe hemophilia A patients with and without inhibitor A. Taglietti¹, S. Ligia¹, F. Paoletti¹, R. Mormile¹, E. Baldacci², A. Chistolini¹, C. Santoro^1,2 | ¹Hematology, Department of Translational and Precision Medicine, Sapienza University of Roma; ²Hematology, Azienda Ospedaliera Universitaria Policlinico Umberto I, Roma, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.241

  37

• CO27 | Intracerebral hemorrhage in hemophilia A and B: a 10-year prospective multicenter study in Italy (2012–2022) E. Zanon¹, A. Napolitano¹, A. Porreca², F. Demartis³, A. Coppola⁴, C. Santoro⁵, I. Cantori⁶, A. C. Molinari⁷, C. Biasioli⁸, M. Luciani⁹, G. Sottilotta¹⁰, B. Pollio¹¹, A. Borchiellini¹², A. Tosetto¹³, P. Flora¹⁴, P. Simioni¹ | ¹Haemophilia Center, First Chair of Internal Medicine, Department of Medicine, University-Hospital of Padua Medical School, Padua; ²Department of Human Sciences and Promotion of the Quality of Life, Unit of Clinical and Molecular Epidemiology IRCCS San Raffaele Roma Open University, Roma; Unit of Clinical and Molecular Epidemiology IRCCS San Raffaele, Roma; ³Centre for Bleeding Disorders, Careggi University Hospital of Firenze; ⁴Azienda Ospedaliero Universitaria di Parma; ⁵Hematology Division, Umberto I University Hospital of Roma; ⁶Haemophilia Center, Department of Transfusion Medicine, Hospital of Macerata; ⁷IRCCS Istituto Giannina Gaslini, Genova; ⁸Haemophilia and Transfusion Centre, Bufalini Hospital, Cesena; ⁹Children’s Hospital Bambino Gesù, Roma; ¹⁰Haemophilia Center, Department of Onco-Hematology and Radioterapy, Hospital of Reggio Calabria; ¹¹Haemophilia Pediatric Center, Transfusion Medicine, City of Health and Science of Turin; ¹²City of Health and Science of Turin; ¹³Hemorrhagic and Thrombotic Diseases Unit, S. Bortolo Hospital of Vicenza; ¹⁴RCCS Foundation Ca’ Granda Maggiore Policlinico Hospital, Milan, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.233

  42

• CO33 | Acquired hemophilia A in Liguria, Italy: what six years of data reveal C. Vernarecci¹, A. C. Molinari¹, P. Ballarino², M. Caiti², E. Cenni², P. Moscatelli², L. Banov¹ | ¹IRCCS Istituto Giannina Gaslini, Genova; ²IRCCS Policlinico San Martino, Genova, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.240

  47

• CS06 | Patient specific iPSCs-derived sinusoidal endothelial cells for phenotypic correction of haemophilia A A. Benetti¹, C.M. Radu¹, C. Samà¹, M. Trevisan², C. Simion¹, E. Campello¹, P. Simioni¹ | ¹Department of Medicine - DIMED, Padua University Hospital; ²Department of Molecular Medicine, University of Padua, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.203

  44

• CO30 | Joint prosthetic surgery in patients with hemophilia A: clinical-functional outcomes and quality of life E. Zanon¹, A. Napolitano¹, A. Berizzi², P. Simioni¹ | ¹Haemophilia Center, First Chair of Internal Medicine, Department of Medicine, University-Hospital of Padua Medical School, Padua; ²Orthopedics and Traumatology Unit, University-Hospital of Padua Medical School, Padua, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.236

  49
• 

  Thrombosis and hemostasis at the University of Padua: a reappraisal on the occasion of its 800th year of history

  Paolo Simioni, Vittorio Pengo, Paolo Prandoni

  07-11-2022

  https://doi.org/10.4081/btvb.2022.52

  2128

  PDF: 253

• CO36 | Inhibitor titer at diagnosis and clinical course in acquired hemophilia A: a single-center experience C. Simion, B. Stocco, E. Dalla Porta, E. Campello, G. Furlan, L. Spiezia, G. Saggiorato, F. Sartorello, E. Zanon, P. Simioni | Department of Medicine – DIMED, University of Padua, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.243

  38

• CS05 | Joint ultrasound follow-up in pediatric patients with haemophilia: a 12-year prospective single-center experience B. Borsellino^1,2, B. Pollio¹, C. Martinoli³, T. Martini¹, R. Albiani¹, I. Ricca¹ | ¹Haemophilia Centre, Immune-Haematology and Transfusion Medicine, University Hospital “Città della Salute e della Scienza”, Turin; ²Department of Biomedicine and Prevention, PhD in Immunology, Molecular Medicine and Applied Biotechnology, University of Roma Tor Vergata, Roma; ³Department of Health Science DISSAL, University of Genoa, Unit of Radiology and IRCCS San Martino Hospital, Genoa, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.202

  48

• PO07 | Liver transplant in a case of congenital afibrinogenemia G. Murante^1,2,3, S. Bergonzi^1,2,3, C. Sella^1,2,3, F. Valeri^1,2, C. Dainese^1,2, A. Valpreda⁴, R. Pastorino^1,3, R. Romagnoli⁵, D. Patrono⁵, D. Cussa⁵, S. Martini⁶, B. Bruno^2,3, A. Borchiellini^1,2 | ¹Regional Centre for Hemorrhagic and Thrombotic Diseases, AOU Città della Salute e della Scienza, Torino; ²Division of Hematology, AOU Città della Salute e della Scienza, University of Turin; ³Department of Molecular Biotechnology and Health Sciences, University of Turin; ⁴Central Laboratory Baldi and Riberi, AOU Città della Salute e della Scienza, Torino; ⁵General Surgery 2U - Liver Transplant Center, Molinette Hospital, AOU Città della Salute e della Scienza, Torino; ⁶SSD Hepatic insufficiency and Liver Transplant, AOU Città della Salute e della Scienza, Torino, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.269

  39

• CO29 | Emergency room physicians and oral anticoagulants related bleeding D. Barcellona^1,2, G. Melis¹, A. Bussu¹, M. Orrù¹, Ml. Caddeo¹, E. Antonucci², A. Mameli¹, F. Marongiu^1,2 | ¹SSD Emostasi e Trombosi, AOU Cagliari; ²Fondazione Arianna, Bologna, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.235

  41

• CO28 | Cognitive, adaptive abilities and behavioral symptoms in children and adolescents with hemophilia: a pilot study R. Vallone¹, M. Luciani², A. Smigliani², M. F. Mansueto³, S. Vicari^1,4, D. Menghini¹ | ¹Child and Adolescent Neuropsychiatry Unit, Bambino Gesù Children’s Hospital, IRCCS, Roma; ²OncoHematology Unit, Bambino Gesù Children’s Hospital, IRCCS, Roma; ³Department of Oncology U.O. Hematology AUO Policlinico, Paolo Giaccone, Palermo; ⁴Department of Life Science and Public Health, Università Cattolica del Sacro Cuore, Roma, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.234

  48

• CO39 | Artificial intelligence in hemophilia management: challenging the need for experts G. Sottilotta¹, D. Megalizzi¹, F. Luise² | ¹UOSD Microcitemie - Emostasi e Trombosi. Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli, Reggio Calabria; ²Laboratorio Analisi, Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli, Reggio Calabria, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.246

  40

• CO32 | Major surgery in patients with severe hemophilia A without inhibitors receiving prophylaxis with fitusiran E. Zanon, C. Simion, A. Napolitano, L. Spiezia, P. Simioni | Haemophilia Center, First Chair of Internal Medicine, Department of Medicine, University-Hospital of Padua Medical School, Padua, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.239

  38

• CO11 | Oral anticoagulation with vitamin K antagonists in early childhood: a single centre-experience B. Borsellino^1,2, B. Pollio¹, I. Ricca¹, R. Albiani¹, T. Martini¹ | ¹Haemophilia Centre, Immune-Haematology and Transfusion Medicine, University Hospital “Città della Salute e della Scienza”, Torino; ²Department of Biomedicine and Prevention, PhD in Immunology, Molecular Medicine and Applied Biotechnology, University of Roma Tor Vergata, Roma, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.215

  52

• CO31 | Immune system profiling in FVIII-treated and emicizumab-treated pediatric HA patients A. Cottonaro¹, S. Akula¹, B. Pollio², I. Ricca², T. Martini², J. Agnelli Giacchello³, P. Sciancalepore³, R. Santi³, A. Follenzi^1,3, S. Merlin¹ | ¹Università del Piemonte Orientale, Health Sciences, Novara; ²A.O. Città della Salute e della Scienza, SIMT Immunohematology and Transfusion Medicine Service, Diagnostic Department, Torino; ³Azienda Ospedaliera SS. Antonio e Biagio e Cesare Arrigo, Alessandria, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.238

  40