Università degli Studi di Cagliari
Olimpia Giuliana Loddo
The Emerging Role of Liver Stiffness Measurement in Transfusion Dependent Thalassemia.
2026-01-01 Gianesin, B; Ferrero, Gb; Longo, F; Barella, S; Origa, R; Lisi, R; Pasanisi, A; Bacigalupo, L; De Franceschi, L; Forni, Gl.
Real-world evidence supports the safety and efficacy profile of luspatercept in clinically complex and heavily iron-overloaded patients
2026-01-01 Gianesin, B; Zappu, A; Ferrero, Gb; Gaglioti, Cm; Santodirocco, M; Fania, G; Graziadei, G; Panzieri, Dl; Massa, A; Mina, T; Polese, F; Vitucci, A; Arcioni, F; Putti, Mc; Cecinati, V; Fotzi, I; Sanna, Pmg; Cappello, A; Longo, F; Lisi, R; Pasanisi, A; Pietrasanta, D; Bocchia, M; Bertoni, E; Cossu, A; Serra, M; Barella, S; Gigante, A; Forni, Gl; Origa, R.
A Combination of Plerixafor and Filgrasting Promotes Successful CD34+ Cell Collection for CRISPR/Cas9 Therapy in Sickle Cell Disease Patients With Insufficient Response to Plerixafor Alone.
2026-01-01 Arcioni, F; Bonetti, M; Sau, A; Zecca, M; Alzahrani, M; Alahmari, B; Alsadi, H; Almozaini, A; Ahmed, M; Essa, M; Perotti, C; Del Fante, C; Passeri, C; Iuliani, O; Russo, Ac; Di Ianni, M; Marchesi, M; Pasqua, Bl; Onorato, M; Berchicci, L; Pierini, A; Zei, T; Iacucci, R; Cerri, C; Gurdo, G; Innocente, A; Capolsini, I; Gorello, P; Colombatti, R; Origa, R; Caniglia, M.
Atrial fibrillation prevalence and its management in aging, transfusion-dependent patients with thalassemia: the FATHAL study
2026-01-01 Di Stefano, V; Gianesin, B; Orecchia, V; Longo, F; Barella, S; Barone, A; Culcasi, M; Denotti, Ar; Costantini, S; Ferrara, F; Foggetti, I; Gigante, A; Manca, Mr; Mazzi, F; Pasanisi, A; Pisaniello, M; Ricchi, P; Serra, M; Origa, R; Bertini, M; Motta, I.
Atrial fibrillation prevalence and its management in aging transfusion-dependent thalassemia patients: the FATHAL study
2026-01-01 Di Stefano, V; Gianesin, B; Orecchia, V; Longo, F; Susanna, B; Barone, A; Culcasi, M; Denotti, Ar; Costantini, S; Ferrara, F; Foggetti, I; Gigante, A; Manca, Mr; Mazzi, F; Pasanisi, A; Pisaniello, M; Ricchi, P; Serra, M; Origa, R; Bertini, M; Motta, I.
Pregnancy and childbirth in women with thalassemia: past and present
2026-01-01 Demurtas, A; Gianesin, B; Zappu, A; Cassinerio, E; Leoni, S; Ricchi, P; Fatigati, C; Ruffo, G; Miciotto, F; Roberti, D; Tartaglione, I; Pasanisi, A; Longo, F; Sorrentino, F; Salvo, A; De Franceschi, L; Lisi, R; Murgia, M; Pisano, V; Sanna, P; Testa, E; Atzeni, I; Mariani, R; Valentini, C; Arcioni, F; Barcella, L; Culcasi, M; Vitucci, A; Miulli, E; Pugliese, P; Fotzi, I; Paci, C; Barella, S; Gigante, A; Forni, G; Origa, R
Transfusion strategies in thalassemia and sickle cell disease SITE-SIMTI-SIdEM Good Practice
2025-01-01 Forni Gian, Luca; Vassanelli, Aurora; De Franceschi, Lucia; Marson, Piero; Lisi, Roberto; Ostuni, Angelo; Gigante, Antonia; Origa, Raffaella; Fiorin, Francesco
Thyroid Nodule Surveillance in Transfusion-Dependent Thalassemia: A Comparative Ultrasonographic Study
2025-01-01 Casale, M; Errico, M; Origa, R; Mureddu, P; Allosso, F; Digitale Selvaggio, L; Grande, G; Di Ludovico, C; Navarra, R; Roberti, D; Capellupo, Mc; Perrotta, S; Pasquali, D.
Long-term efficacy and safety of luspatercept for the treatment of anaemia in patients with transfusion-dependent β-thalassaemia (BELIEVE): final results from a phase 3 randomised trial
2025-01-01 Domenica Cappellini, Maria; Viprakasit, Vip; Georgiev, Pencho; D Coates, Thomas; Origa, Raffaella; Khelif, Abderrahim; Liew, Hong-Keng; Tantiworawit, Adisak; Chew, Lee-Ping; Khalil, Abdalla; Joy Ho, P; M Kuo, Kevin H; Holot, Natalia; Perin, Martina; Carolina Giuseppi, Ana; Kuo, Wen-Ling; Lai, Yinzhi; Felber Medlin, Loyse; Moro Bueno, Luciana; Kattamis, Antonis; T Taher, Ali
The novel SCD MED ALERT mHealth app: empowering patients with sickle cell disease
2025-01-01 Villaboni, S; Pinto, Vm; Gianesin, B; Casale, M; Perrotta, S; Mazzi, F; Ceolan, J; Pirrone, R; Cavellini, P; Gigante, A; Calvillo, M; Origa, R; Forni, Gl; De Franceschi, L
Luspatercept for Transfusion-Dependent Beta-Thalassemia: Real-World Experience in a Large Cohort of Patients From Italy
2025-01-01 Origa, Raffaella; Gianesin, Barbara; Zappu, Antonietta; Denotti Anna, Rita; Di Giorgio Mary, Ann; Sciortino, Roberta; Motta, Irene; Panzieri Daniele, Lello; Rosso, Rosamaria; Bulla, Anna; Culcasi, Martina; Pasanisi Anna, Maria; De Franceschi, Lucia; Di Maggio, Rosario; Pinto Valeria, Maria; Sanna Paola Maria, Grazia; Ricchi, Paolo; Ruffo Giovan, Battista; Schieppati, Francesca; Roberti, Domenico; Ferrero Giovanni, Battista; De Michele, Elisa; Arcioni, Francesco; Fotzi, Ilaria; Marktel, Sarah; Poloni, Antonella; Soverini, Giulia; Testa Epifania, Rita; Cabiddu, Giusy; Fortugno, Carmelo; Gigante, Antonia; Polese, Francesca; Rapezzi, Davide; Sau, Antonella; Forni Gian, Luca; Cappellini Maria, Domenica; Longo, Filomena
Thalassemias and Sickle Cell Diseases in Pregnancy: SITE Good Practice.
2025-01-01 Maria Pinto, Valeria; Cima, Rosanna; Di Maggio, Rosario; Livia Alga, Maria; Gigante, Antonia; Longo, Filomena; Maria Pasanisi, Anna; Venturelli, Donatella; Cassinerio, Elena; Casale, Maddalena; Origa, Raffaella; Zanconato, Giovanni; Luca Forni, Gian; De Franceschi, Lucia
Prevalence and mortality trends of hemoglobinopathies in Italy: a nationwide study
2025-01-01 Gianesin, B; Piel, Fb; Musallam, Km; Barella, S; Casale, M; Cassinerio, E; Di Maggio, R; Gigante, A; Gamberini, Mr; Graziadei, G; Lisi, R; Longo, F; Maggio, A; Origa, R; Pasanisi, A; Perrotta, S; Piga, Ag; Pinto, Vm; Rosso, R; Robello, G; Russo, G; Zecca, M; De Franceschi, L; Forni, Gl
Beta Thalassemia in Children: Established Approaches, Old Issues, New Non-Curative Therapies, and Perspectives on Healing
2024-01-01 Origa, Raffaella; Issa, Layal
Differential effects of iron chelators on iron burden and long-term morbidity and mortality outcomes in a large cohort of transfusion-dependent β-thalassemia patients who remained on the same monotherapy over 10 years
2024-01-01 Musallam, Km; Barella, S; Origa, R; Ferrero, Gb; Lisi, R; Pasanisi, A; Longo, F; Gianesin, B; Forni, Gl
Phenoconversion’ in adult patients with β-thalassemia
2024-01-01 Musallam, Km; Barella, S; Origa, R; Ferrero, Gb; Lisi, R; Pasanisi, A; Longo, F; Gianesin, B; Forni, Gl
Revisiting iron overload status and change thresholds as predictors of mortality in transfusion-dependent β-thalassemia: a 10-year cohort study
2024-01-01 Musallam, Km; Barella, S; Origa, R; Ferrero, Gb; Lisi, R; Pasanisi, A; Longo, F; Gianesin, B; Forni, Gl
Pretransfusion hemoglobin level and mortality in adults with transfusiondependent β-thalassemia
2024-01-01 Musallam, Km; Barella, S; Origa, R; Ferrero, Gb; Lisi, R; Pasanisi, A; Longo, F; Gianesin, B; Forni, Gl.
Unplanned pregnancy in women with beta-thalassaemia treated with luspatercept
2024-01-01 Zaccheddu, E; Zappu, A; Barella, S; Clemente, Mg; Orecchia, V; Pilia, Mp; Piras, S; Pitturru, C; Scarano, M; Origa, R.
Deferasirox film-coated tablet-associated ulcerative colitis: An emerging pattern in thalassemia patients?
2024-01-01 Piolatto, A; Gaglioti, Cm; Tesio, N; Clemente, Mg; Culcasi, M; Matrone, A; Pila, Mp; Sambataro, A; Longo, F; Origa, R; Ferrero, Gb.
Luspatercept in the Treatment of Beta Thalassemia in Italy: Lights and Shadows in Clinical Practice
2023-01-01 Gianesin, Barbara; Rosso, Rosamaria; Lisi, Roberto; Zappu, Antonietta; Motta, Irene; Longo, Filomena; Pinto, Valeria; Maria Grazia Sanna, Paola; De Franceschi, Lucia; Battista Ruffo, Giovan; DI Maggio, Rosario; Bulla, Anna; Ann Di Giorgio, Mary; Rita Denotti, Anna; Lello Panzieri, Daniele; Culcasi, Martina; Quintino, Sabrina; Marchisio, Andrea; Mazzi, Filippo; Miciotto, Francesca; Barone, Rita; Di Raimondo, Francesco; Ferraresi, Marta; Marchetti, Beatrice; Origa, Raffaella
Atrial Fibrillation Prevalence and Its Management in Transfusion-Dependent Thalassemias: The Fathal Study
2023-01-01 Di Stefano, Valeria; Gianesin, Barbara; Bertini, Matteo; Culcasi, Martina; Rita Denotti, Anna; Derchi, Giorgio; Giuditta, Marianna; Longo, Filomena; Regina Manca, Maria; Cristina Martinoli, Maria; Orecchia, Valeria; Origa, Raffaella; Motta, Irene
Reply to "Hepatocellular carcinoma in thalassemia and other hemoglobinopathies"
2023-01-01 Origa, R; Gianesin, B; Longo, F; Di Maggio, R; Cassinerio, E; Gamberini, Mr; Pinto, Vm; Casale, M; La Nasa, G; Caocci, G; Piroddi, A; Piolatto, A; Di Mauro, A; Romano, C; Gigante, A; Barella, S; Maggio, A; Graziadei, G; Perrotta, S; Forni, Gl.
Treating Thalassemia Patients with Luspatercept: An Expert Opinion Based on Current Evidence
2023-01-01 Longo, F; Motta, I; Pinto, V; Piolatto, A; Ricchi, P; Tartaglione, I; Origa, R.
Hepatitis C and Thalassemia: A Story with (Almost) a Happy Ending
2023-01-01 Origa, Raffaella
Incidence of cancer and related deaths in hemoglobinopathies: A follow-up of 4631 patients between 1970 and 2021
2023-01-01 Origa, Raffaella; Gianesin, Barbara; Longo, Filomena; Di Maggio, Rosario; Cassinerio, Elena; Rita Gamberini, Maria; Maria Pinto, Valeria; Quarta, Antonella; Casale, Maddalena; LA NASA, Giorgio; Caocci, Giovanni; Piroddi, Antonio; Piolatto, Andrea; Di Mauro, Alessandra; Romano, Claudia; Gigante, Antonia; Barella, Susanna; Maggio, Aurelio; Graziadei, Giovanna; Perrotta, Silverio; Luca Forni, Gian
Management of the Sickle Cell Trait: An Opinion by Expert Panel Members.
2023-01-01 Pinto, Vm; De Franceschi, L; Gianesin, B; Gigante, A; Graziadei, G; Lombardini, L; Palazzi, G; Quota, A; Russo, R; Sainati, L; Venturelli, D; Forni, Gl; Origa, R.
Nephrological Complications in Hemoglobinopathies: SITE Good Practice
2023-01-01 Ruffo, Gb; Russo, R; Casini, T; Lombardini, L; Orecchia, V; Voi, V; Origa, R; Forni, Gl; Marchetti, M; Gigante, A; Garibotto, G; Maggio, A; De Franceschi, L.
The Management of Endocrine Complications in Patients with Haemoglobinopathies: Good Clinical Practice of the Italian Society of Thalassemia and Haemoglobinopathies (SITE)
2022-01-01 Casale, Maddalena; Baldini, Marina; Del Monte, Patrizia; Gigante, Antonia; Grandone, Anna; Origa, Raffaella; Poggi, Maurizio; Gadda, Franco; Lai, Rosalba; Marchetti, Monia; Luca Forni, Gian
2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia
2022-01-01 Farmakis, D.; Porter, J.; Taher, A.; Cappellini, M. D.; Angastiniotis, M.; Eleftheriou, A.; Alassaf, A.; Angastiniotis, M.; Angelucci, E.; Aydinok, Y.; Bou-Fakhredin Rayan, R.; Brunetta, L.; Cappellini, M. D.; Constantinou, G.; Daar, S.; De Sanctis, V.; Dusheiko, G.; Elbard, R.; Eleftheriou, A.; Eleftheriou, P.; Englezos, P.; Farmakis, D.; Haines, D.; Hattab, F. N.; Kaltsounis, G.; Kattamis, A.; Koskinas, J.; Kumar, N.; Kulozik, A.; Kyriakou, A.; Maggio, A.; Maharai, R.; Mednick, L.; Michalaki, E.; Murphy, W.; Oevermann, L.; Origa, R.; Papayanni, P. -G.; Politis, C.; Porter, J.; Shah, F.; Skafi, A.; Skordis, N.; Sodani, P.; Soliman, A.; Subair, S.; Taher, A.; Tampaki, M.; Trompeter, S.; Tuli, S.; Walker, M.; Yamashita, R.; Yannaki, E.; Yardumian, A.
Crushed deferasirox film-coated tablets in pediatric patients with transfusional hemosiderosis: Results from a single-arm, interventional phase 4 study (MIMAS)
2022-01-01 Wali, Y.; Hassan, T.; Charoenkwan, P.; Trompeter, S.; Tartaglione, I.; Origa, R.; Gamberini, M. R.; Viprakasit, V.; Izquierdo, M.; Opio, S.; Roy, T.; Taher, A.
Risk factors for endocrine complications in transfusion-dependent thalassemia patients on chelation therapy with deferasirox: a risk assessment study from a multicentre nation-wide cohort
2022-01-01 Casale, M; Forni, Gl; Cassinerio, E; Pasquali, D; Origa, R; Serra, M; Campisi, S; Peluso, A; Renni, R; Cattoni, A; De Michele, E; Allò, M; Poggi, M; Ferrara, F; Di Concilio, R; Sportelli, F; Quarta, A; Putti, Mc; Notarangelo, Ld; Sau, A; Ladogana, S; Tartaglione, I; Picariello, S; Marcon, A; Sturiale, P; Roberti, D; Lazzarino, Ai; Perrotta, S.
Tricuspid-valve regurgitant jet velocity as a risk factor for death in β-Thalassemia
2022-01-01 Derchi, Giorgio; Musallam, Khaled M.; Maria Pinto, Valeria; Graziadei, Giovanna; Giuditta, Marianna; Barella, Susanna; Origa, Raffaella; Casu, Gavino; Pasanisi, Annamaria; Longo, Filomena; Casale, Maddalena; Miceli, Roberta; Merella, Pierluigi; Gianesin, Barbara; Ameri, Pietro; Tartaglione, Immacolata; Perrotta, Silverio; Piga, Antonio; Domenica Cappellini, Maria; Gian Luca Forni, And
Mortality in β-thalassemia patients with confirmed pulmonary arterial hypertension on right heart catheterization
2022-01-01 Pinto, V. M.; Musallam, K. M.; Derchi, G.; Graziadei, G.; Giuditta, M.; Origa, R.; Barella, S.; Casu, G.; Pasanisi, A.; Longo, F.; Casale, M.; Miceli, R.; Merella, P.; Tartaglione, I.; Piga, A.; Cappellini, M. D.; Gianesin, B.; Forni, G. L.
Good Clinical Practice of the Italian Society of Thalassemia and Haemoglobinopathies (SITE) for the Management of Endocrine Complications in Patients with Haemoglobinopathies
2022-01-01 Casale, M; Baldini, Mi; Del Monte, P; Gigante, A; Grandone, A; Origa, R; Poggi, M; Gadda, F; Lai, R; Marchetti, M; Forni, Gl
Safety and Efficacy of the New Combination Iron Chelation Regimens in Patients with Transfusion-Dependent Thalassemia and Severe Iron Overload
2022-01-01 Origa, R.; Cinus, M.; Pilia, M. P.; Gianesin, B.; Zappu, A.; Orecchia, V.; Clemente, M. G.; Pitturru, C.; Denotti, A. R.; Corongiu, F.; Piras, S.; Barella, S.
Transfusional Approach in Multi-Ethnic Sickle Cell Patients: Real-World Practice Data From a Multicenter Survey in Italy
2022-01-01 Graziadei, G; De Franceschi, L; Sainati, L; Venturelli, D; Masera, N; Bonomo, P; Vassanelli, A; Casale, M; Lodi, G; Voi, V; Rigano, P; Pinto, Vm; Quota, A; Notarangelo, Ld; Russo, G; Allo, M; Rosso, R; D'Ascola, D; Facchini, E; Macchi, S; Arcioni, F; Bonetti, F; Rossi, E; Sau, A; Campisi, S; Colarusso, G; Giona, F; Lisi, R; Giordano, P; Boscarol, G; Filosa, A; Marktel, S; Maroni, P; Murgia, M; Origa, R; Longo, F; Bortolotti, M; Colombatti, R; Di Maggio, R; Mariani, R; Piperno, A; Corti, P; Fidone, C; Palazzi, G; Badalamenti, L; Gianesin, B; Piel, Fb; Forni, Gl
Changing patterns of thalassaemia in Italy: a WebThal perspective.
2021-01-01 Longo, F; Corrieri, P; Origa, R; Barella, S; Sanna, Pmg; Bitti, Pp; Zuccarelli, A; Commendatore, Fv; Vitucci, A; Quarta, A; Lisi, R; Cappellini, Md; Massei, F; Forni, Gl; Piga, A.
Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm
2021-01-01 Baronciani, D.; Casale, M.; De Franceschi, L.; Graziadei, G.; Longo, F.; Origa, R.; Rigano, P.; Pinto, V.; Marchetti, M.; Gigante, A.; Forni, G. L.
Hemoglobinopathies and Cancer: Preliminary Results of an Italian Multicenter Experience
2021-01-01 Origa, Raffaella; Longo, Filomena; Di Maggio, Rosario; Cassinerio, Elena; Maria Pinto, Valeria; Quarta, Antonella; Casale, Maddalena; Gianesin, Barbara; Barella, Susanna; Piolatto, Andrea; Maggio, Aurelio; Graziadei, Giovanna; Romano, Claudia; Perrotta, Silverio; Luca Forni, Gian
Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial
2020-01-01 Maggio, A; Kattamis, A; Felisi, M; Reggiardo, G; El-Beshlawy, A; Bejaoui, M; Sherief, L; Christou, S; Cosmi, C; Della Pasqua, O; Del Vecchio, Gc; Filosa, A; Cuccia, L; Hassab, H; Kreka, M; Origa, R; Putti, Mc; Spino, M; Telfer, P; Tempesta, B; Vitrano, A; Tsang, Yc; Zaka, A; Tricta, F; Bonifazi, D; Ceci, A.
Two-year long safety and efficacy of deferasirox film-coated tablets in patients with thalassemia or lower/intermediate risk MDS: phase 3 results from a subset of patients previously treated with deferasirox in the ECLIPSE study
2020-01-01 Tartaglione, I; Origa, R; Kattamis, A; Pfeilstöcker, M; Gunes, S; Crowe, S; Fagan, N; Vincenzi, B; Ruffo, Gb.
A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia
2020-01-01 Cappellini, Md; Viprakasit, V; Taher, At; Georgiev, P; Kuo, Khm; Coates, T; Voskaridou, E; Liew, Hk; Pazgal-Kobrowski, I; Forni, Gl; Perrotta, S; Khelif, A; Lal, A; Kattamis, A; Vlachaki, E; Origa, R; Aydinok, Y; Bejaoui, M; Ho, Pj; Chew, Lp; Bee, Pc; Lim, Sm; Lu, My; Tantiworawit, A; Ganeva, P; Gercheva, L; Shah, F; Neufeld, Ej; Thompson, A; Laadem, A; Shetty, Jk; Zou, J; Zhang, J; Miteva, D; Zinger, T; Linde, Pg; Sherman, Ml; Hermine, O; Porter, J; Piga, A; Believe, Investigators.
Selecting ß-Thalassemia Patients for Gene Therapy: A Decision-Making Algorithm
2019-01-01 Baronciani, D; Casale, M; De Franceschi, L; Graziadei, G; Longo, F; Origa, R; Pinto, Vm; Rigano, P; Marchetti, M; Gigante, A; Angelucci, E; Cappellini, Md; Iolascon, A; Piga, A; Forni, Gl.
Influence of patient-reported outcomes on the treatment effect of deferasirox film-coated and dispersible tablet formulations in the ECLIPSE trial: A post hoc mediation analysis
2019-01-01 Taher, At; Origa, R; Perrotta, S; Kouraklis, A; Belhoul, K; Huang, V; Han, J; Bruederle, A; Bobbili, P; Duh, Ms; Porter, Jb
Amustaline-glutathione pathogen-reduced red blood cell concentrates for transfusion-dependent thalassaemia
2019-01-01 Aydinok, Y; Piga, A; Origa, R; Mufti, N; Erickson, A; North, A; Waldhaus, K; Ernst, C; Lin, Js; Huang, N; Benjamin, Rj; Corash, L.
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia
2019-01-01 Marktel, S; Scaramuzza, S; Cicalese, Mp; Giglio, F; Galimberti, S; Lidonnici, Mr; Calbi, V; Assanelli, A; Bernardo, Me; Rossi, C; Calabria, A; Milani, R; Gattillo, S; Benedicenti, F; Spinozzi, G; Aprile, A; Bergami, A; Casiraghi, M; Consiglieri, G; Masera, N; D'Angelo, E; Mirra, N; Origa, R; Tartaglione, I; Perrotta, S; Winter, R; Coppola, M; Viarengo, G; Santoleri, L; Graziadei, G; Gabaldo, M; Valsecchi, Mg; Montini, E; Naldini, L; Cappellini, Md; Ciceri, F; Aiuti, A; Ferrari, G.
Risk factors for heart disease in transfusion-dependent thalassemia: serum ferritin revisited
2019-01-01 Derchi, G; Dessì, C; Bina, P; Cappellini, Md; Piga, A; Perrotta, S; Tartaglione, I; Giuditta, M; Longo, F; Origa, R; Quarta, A; Pinto, V; Forni, Gl
Impact of the direct-acting antiviral agents (DAAs) on chronic hepatitis C in Sardinian patients with transfusion-dependent Thalassemia major
2019-01-01 Ponti, Ml; Comitini, F; Murgia, D; Ganga, R; Canu, R; Dessì, C; Foschini, Ml; Leoni, G; Morittu, M; Perra, M; Pilia, Mp; Casini, Mr; Zappu, A; Origa, R.
Sotatercept, a novel transforming growth factor β ligand trap, improves anemia in β-thalassemia: a phase II, open-label, dose-finding study
2019-01-01 Cappellini, Md; Porter, J; Origa, R; Forni, Gl; Voskaridou, E; Galactéros, F; Taher, At; Arlet, Jb; Ribeil, Ja; Garbowski, M; Graziadei, G; Brouzes, C; Semeraro, M; Laadem, A; Miteva, D; Zou, J; Sung, V; Zinger, T; Attie, Km; Hermine, O.
| Titolo | Data di pubblicazione | Autore(i) | Rivista | Editore |
|---|---|---|---|---|
| The Emerging Role of Liver Stiffness Measurement in Transfusion Dependent Thalassemia. | 1-gen-2026 | Gianesin, B; Ferrero, Gb; Longo, F; Barella, S; Origa, R; Lisi, R; Pasanisi, A; Bacigalupo, L; De Franceschi, L; Forni, Gl. | AMERICAN JOURNAL OF HEMATOLOGY | - |
| Real-world evidence supports the safety and efficacy profile of luspatercept in clinically complex and heavily iron-overloaded patients | 1-gen-2026 | Gianesin, B; Zappu, A; Ferrero, Gb; Gaglioti, Cm; Santodirocco, M; Fania, G; Graziadei, G; Panzieri, Dl; Massa, A; Mina, T; Polese, F; Vitucci, A; Arcioni, F; Putti, Mc; Cecinati, V; Fotzi, I; Sanna, Pmg; Cappello, A; Longo, F; Lisi, R; Pasanisi, A; Pietrasanta, D; Bocchia, M; Bertoni, E; Cossu, A; Serra, M; Barella, S; Gigante, A; Forni, Gl; Origa, R. | BRITISH JOURNAL OF HAEMATOLOGY | - |
| A Combination of Plerixafor and Filgrasting Promotes Successful CD34+ Cell Collection for CRISPR/Cas9 Therapy in Sickle Cell Disease Patients With Insufficient Response to Plerixafor Alone. | 1-gen-2026 | Arcioni, F; Bonetti, M; Sau, A; Zecca, M; Alzahrani, M; Alahmari, B; Alsadi, H; Almozaini, A; Ahmed, M; Essa, M; Perotti, C; Del Fante, C; Passeri, C; Iuliani, O; Russo, Ac; Di Ianni, M; Marchesi, M; Pasqua, Bl; Onorato, M; Berchicci, L; Pierini, A; Zei, T; Iacucci, R; Cerri, C; Gurdo, G; Innocente, A; Capolsini, I; Gorello, P; Colombatti, R; Origa, R; Caniglia, M. | AMERICAN JOURNAL OF HEMATOLOGY | - |
| Atrial fibrillation prevalence and its management in aging, transfusion-dependent patients with thalassemia: the FATHAL study | 1-gen-2026 | Di Stefano, V; Gianesin, B; Orecchia, V; Longo, F; Barella, S; Barone, A; Culcasi, M; Denotti, Ar; Costantini, S; Ferrara, F; Foggetti, I; Gigante, A; Manca, Mr; Mazzi, F; Pasanisi, A; Pisaniello, M; Ricchi, P; Serra, M; Origa, R; Bertini, M; Motta, I. | BLOOD ADVANCES | - |
| Atrial fibrillation prevalence and its management in aging transfusion-dependent thalassemia patients: the FATHAL study | 1-gen-2026 | Di Stefano, V; Gianesin, B; Orecchia, V; Longo, F; Susanna, B; Barone, A; Culcasi, M; Denotti, Ar; Costantini, S; Ferrara, F; Foggetti, I; Gigante, A; Manca, Mr; Mazzi, F; Pasanisi, A; Pisaniello, M; Ricchi, P; Serra, M; Origa, R; Bertini, M; Motta, I. | BLOOD ADVANCES | - |
| Pregnancy and childbirth in women with thalassemia: past and present | 1-gen-2026 | Demurtas, A; Gianesin, B; Zappu, A; Cassinerio, E; Leoni, S; Ricchi, P; Fatigati, C; Ruffo, G; Miciotto, F; Roberti, D; Tartaglione, I; Pasanisi, A; Longo, F; Sorrentino, F; Salvo, A; De Franceschi, L; Lisi, R; Murgia, M; Pisano, V; Sanna, P; Testa, E; Atzeni, I; Mariani, R; Valentini, C; Arcioni, F; Barcella, L; Culcasi, M; Vitucci, A; Miulli, E; Pugliese, P; Fotzi, I; Paci, C; Barella, S; Gigante, A; Forni, G; Origa, R | HAEMATOLOGICA | - |
| Transfusion strategies in thalassemia and sickle cell disease SITE-SIMTI-SIdEM Good Practice | 1-gen-2025 | Forni Gian, Luca; Vassanelli, Aurora; De Franceschi, Lucia; Marson, Piero; Lisi, Roberto; Ostuni, Angelo; Gigante, Antonia; Origa, Raffaella; Fiorin, Francesco | BLOOD TRANSFUSION | - |
| Thyroid Nodule Surveillance in Transfusion-Dependent Thalassemia: A Comparative Ultrasonographic Study | 1-gen-2025 | Casale, M; Errico, M; Origa, R; Mureddu, P; Allosso, F; Digitale Selvaggio, L; Grande, G; Di Ludovico, C; Navarra, R; Roberti, D; Capellupo, Mc; Perrotta, S; Pasquali, D. | JOURNAL OF CLINICAL MEDICINE | - |
| Long-term efficacy and safety of luspatercept for the treatment of anaemia in patients with transfusion-dependent β-thalassaemia (BELIEVE): final results from a phase 3 randomised trial | 1-gen-2025 | Domenica Cappellini, Maria; Viprakasit, Vip; Georgiev, Pencho; D Coates, Thomas; Origa, Raffaella; Khelif, Abderrahim; Liew, Hong-Keng; Tantiworawit, Adisak; Chew, Lee-Ping; Khalil, Abdalla; Joy Ho, P; M Kuo, Kevin H; Holot, Natalia; Perin, Martina; Carolina Giuseppi, Ana; Kuo, Wen-Ling; Lai, Yinzhi; Felber Medlin, Loyse; Moro Bueno, Luciana; Kattamis, Antonis; T Taher, Ali | THE LANCET. HAEMATOLOGY | - |
| The novel SCD MED ALERT mHealth app: empowering patients with sickle cell disease | 1-gen-2025 | Villaboni, S; Pinto, Vm; Gianesin, B; Casale, M; Perrotta, S; Mazzi, F; Ceolan, J; Pirrone, R; Cavellini, P; Gigante, A; Calvillo, M; Origa, R; Forni, Gl; De Franceschi, L | Rare Disease and Orphan Drugs Journal | - |
| Luspatercept for Transfusion-Dependent Beta-Thalassemia: Real-World Experience in a Large Cohort of Patients From Italy | 1-gen-2025 | Origa, Raffaella; Gianesin, Barbara; Zappu, Antonietta; Denotti Anna, Rita; Di Giorgio Mary, Ann; Sciortino, Roberta; Motta, Irene; Panzieri Daniele, Lello; Rosso, Rosamaria; Bulla, Anna; Culcasi, Martina; Pasanisi Anna, Maria; De Franceschi, Lucia; Di Maggio, Rosario; Pinto Valeria, Maria; Sanna Paola Maria, Grazia; Ricchi, Paolo; Ruffo Giovan, Battista; Schieppati, Francesca; Roberti, Domenico; Ferrero Giovanni, Battista; De Michele, Elisa; Arcioni, Francesco; Fotzi, Ilaria; Marktel, Sarah; Poloni, Antonella; Soverini, Giulia; Testa Epifania, Rita; Cabiddu, Giusy; Fortugno, Carmelo; Gigante, Antonia; Polese, Francesca; Rapezzi, Davide; Sau, Antonella; Forni Gian, Luca; Cappellini Maria, Domenica; Longo, Filomena | AMERICAN JOURNAL OF HEMATOLOGY | - |
| Thalassemias and Sickle Cell Diseases in Pregnancy: SITE Good Practice. | 1-gen-2025 | Maria Pinto, Valeria; Cima, Rosanna; Di Maggio, Rosario; Livia Alga, Maria; Gigante, Antonia; Longo, Filomena; Maria Pasanisi, Anna; Venturelli, Donatella; Cassinerio, Elena; Casale, Maddalena; Origa, Raffaella; Zanconato, Giovanni; Luca Forni, Gian; De Franceschi, Lucia | JOURNAL OF CLINICAL MEDICINE | - |
| Prevalence and mortality trends of hemoglobinopathies in Italy: a nationwide study | 1-gen-2025 | Gianesin, B; Piel, Fb; Musallam, Km; Barella, S; Casale, M; Cassinerio, E; Di Maggio, R; Gigante, A; Gamberini, Mr; Graziadei, G; Lisi, R; Longo, F; Maggio, A; Origa, R; Pasanisi, A; Perrotta, S; Piga, Ag; Pinto, Vm; Rosso, R; Robello, G; Russo, G; Zecca, M; De Franceschi, L; Forni, Gl | HAEMATOLOGICA | - |
| Beta Thalassemia in Children: Established Approaches, Old Issues, New Non-Curative Therapies, and Perspectives on Healing | 1-gen-2024 | Origa, Raffaella; Issa, Layal | JOURNAL OF CLINICAL MEDICINE | - |
| Differential effects of iron chelators on iron burden and long-term morbidity and mortality outcomes in a large cohort of transfusion-dependent β-thalassemia patients who remained on the same monotherapy over 10 years | 1-gen-2024 | Musallam, Km; Barella, S; Origa, R; Ferrero, Gb; Lisi, R; Pasanisi, A; Longo, F; Gianesin, B; Forni, Gl | BLOOD CELLS, MOLECULES, & DISEASES | - |
| Phenoconversion’ in adult patients with β-thalassemia | 1-gen-2024 | Musallam, Km; Barella, S; Origa, R; Ferrero, Gb; Lisi, R; Pasanisi, A; Longo, F; Gianesin, B; Forni, Gl | AMERICAN JOURNAL OF HEMATOLOGY | - |
| Revisiting iron overload status and change thresholds as predictors of mortality in transfusion-dependent β-thalassemia: a 10-year cohort study | 1-gen-2024 | Musallam, Km; Barella, S; Origa, R; Ferrero, Gb; Lisi, R; Pasanisi, A; Longo, F; Gianesin, B; Forni, Gl | ANNALS OF HEMATOLOGY | - |
| Pretransfusion hemoglobin level and mortality in adults with transfusiondependent β-thalassemia | 1-gen-2024 | Musallam, Km; Barella, S; Origa, R; Ferrero, Gb; Lisi, R; Pasanisi, A; Longo, F; Gianesin, B; Forni, Gl. | BLOOD | - |
| Unplanned pregnancy in women with beta-thalassaemia treated with luspatercept | 1-gen-2024 | Zaccheddu, E; Zappu, A; Barella, S; Clemente, Mg; Orecchia, V; Pilia, Mp; Piras, S; Pitturru, C; Scarano, M; Origa, R. | BRITISH JOURNAL OF HAEMATOLOGY | - |
| Deferasirox film-coated tablet-associated ulcerative colitis: An emerging pattern in thalassemia patients? | 1-gen-2024 | Piolatto, A; Gaglioti, Cm; Tesio, N; Clemente, Mg; Culcasi, M; Matrone, A; Pila, Mp; Sambataro, A; Longo, F; Origa, R; Ferrero, Gb. | BRITISH JOURNAL OF HAEMATOLOGY | - |
| Luspatercept in the Treatment of Beta Thalassemia in Italy: Lights and Shadows in Clinical Practice | 1-gen-2023 | Gianesin, Barbara; Rosso, Rosamaria; Lisi, Roberto; Zappu, Antonietta; Motta, Irene; Longo, Filomena; Pinto, Valeria; Maria Grazia Sanna, Paola; De Franceschi, Lucia; Battista Ruffo, Giovan; DI Maggio, Rosario; Bulla, Anna; Ann Di Giorgio, Mary; Rita Denotti, Anna; Lello Panzieri, Daniele; Culcasi, Martina; Quintino, Sabrina; Marchisio, Andrea; Mazzi, Filippo; Miciotto, Francesca; Barone, Rita; Di Raimondo, Francesco; Ferraresi, Marta; Marchetti, Beatrice; Origa, Raffaella | BLOOD | - |
| Atrial Fibrillation Prevalence and Its Management in Transfusion-Dependent Thalassemias: The Fathal Study | 1-gen-2023 | Di Stefano, Valeria; Gianesin, Barbara; Bertini, Matteo; Culcasi, Martina; Rita Denotti, Anna; Derchi, Giorgio; Giuditta, Marianna; Longo, Filomena; Regina Manca, Maria; Cristina Martinoli, Maria; Orecchia, Valeria; Origa, Raffaella; Motta, Irene | BLOOD | - |
| Reply to "Hepatocellular carcinoma in thalassemia and other hemoglobinopathies" | 1-gen-2023 | Origa, R; Gianesin, B; Longo, F; Di Maggio, R; Cassinerio, E; Gamberini, Mr; Pinto, Vm; Casale, M; La Nasa, G; Caocci, G; Piroddi, A; Piolatto, A; Di Mauro, A; Romano, C; Gigante, A; Barella, S; Maggio, A; Graziadei, G; Perrotta, S; Forni, Gl. | CANCER | - |
| Treating Thalassemia Patients with Luspatercept: An Expert Opinion Based on Current Evidence | 1-gen-2023 | Longo, F; Motta, I; Pinto, V; Piolatto, A; Ricchi, P; Tartaglione, I; Origa, R. | JOURNAL OF CLINICAL MEDICINE | - |
| Hepatitis C and Thalassemia: A Story with (Almost) a Happy Ending | 1-gen-2023 | Origa, Raffaella | PATHOGENS | - |
| Incidence of cancer and related deaths in hemoglobinopathies: A follow-up of 4631 patients between 1970 and 2021 | 1-gen-2023 | Origa, Raffaella; Gianesin, Barbara; Longo, Filomena; Di Maggio, Rosario; Cassinerio, Elena; Rita Gamberini, Maria; Maria Pinto, Valeria; Quarta, Antonella; Casale, Maddalena; LA NASA, Giorgio; Caocci, Giovanni; Piroddi, Antonio; Piolatto, Andrea; Di Mauro, Alessandra; Romano, Claudia; Gigante, Antonia; Barella, Susanna; Maggio, Aurelio; Graziadei, Giovanna; Perrotta, Silverio; Luca Forni, Gian | CANCER | - |
| Management of the Sickle Cell Trait: An Opinion by Expert Panel Members. | 1-gen-2023 | Pinto, Vm; De Franceschi, L; Gianesin, B; Gigante, A; Graziadei, G; Lombardini, L; Palazzi, G; Quota, A; Russo, R; Sainati, L; Venturelli, D; Forni, Gl; Origa, R. | JOURNAL OF CLINICAL MEDICINE | - |
| Nephrological Complications in Hemoglobinopathies: SITE Good Practice | 1-gen-2023 | Ruffo, Gb; Russo, R; Casini, T; Lombardini, L; Orecchia, V; Voi, V; Origa, R; Forni, Gl; Marchetti, M; Gigante, A; Garibotto, G; Maggio, A; De Franceschi, L. | JOURNAL OF CLINICAL MEDICINE | - |
| The Management of Endocrine Complications in Patients with Haemoglobinopathies: Good Clinical Practice of the Italian Society of Thalassemia and Haemoglobinopathies (SITE) | 1-gen-2022 | Casale, Maddalena; Baldini, Marina; Del Monte, Patrizia; Gigante, Antonia; Grandone, Anna; Origa, Raffaella; Poggi, Maurizio; Gadda, Franco; Lai, Rosalba; Marchetti, Monia; Luca Forni, Gian | RECENTI PROGRESSI IN MEDICINA | - |
| 2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia | 1-gen-2022 | Farmakis, D.; Porter, J.; Taher, A.; Cappellini, M. D.; Angastiniotis, M.; Eleftheriou, A.; Alassaf, A.; Angastiniotis, M.; Angelucci, E.; Aydinok, Y.; Bou-Fakhredin Rayan, R.; Brunetta, L.; Cappellini, M. D.; Constantinou, G.; Daar, S.; De Sanctis, V.; Dusheiko, G.; Elbard, R.; Eleftheriou, A.; Eleftheriou, P.; Englezos, P.; Farmakis, D.; Haines, D.; Hattab, F. N.; Kaltsounis, G.; Kattamis, A.; Koskinas, J.; Kumar, N.; Kulozik, A.; Kyriakou, A.; Maggio, A.; Maharai, R.; Mednick, L.; Michalaki, E.; Murphy, W.; Oevermann, L.; Origa, R.; Papayanni, P. -G.; Politis, C.; Porter, J.; Shah, F.; Skafi, A.; Skordis, N.; Sodani, P.; Soliman, A.; Subair, S.; Taher, A.; Tampaki, M.; Trompeter, S.; Tuli, S.; Walker, M.; Yamashita, R.; Yannaki, E.; Yardumian, A. | HEMASPHERE | - |
| Crushed deferasirox film-coated tablets in pediatric patients with transfusional hemosiderosis: Results from a single-arm, interventional phase 4 study (MIMAS) | 1-gen-2022 | Wali, Y.; Hassan, T.; Charoenkwan, P.; Trompeter, S.; Tartaglione, I.; Origa, R.; Gamberini, M. R.; Viprakasit, V.; Izquierdo, M.; Opio, S.; Roy, T.; Taher, A. | AMERICAN JOURNAL OF HEMATOLOGY | - |
| Risk factors for endocrine complications in transfusion-dependent thalassemia patients on chelation therapy with deferasirox: a risk assessment study from a multicentre nation-wide cohort | 1-gen-2022 | Casale, M; Forni, Gl; Cassinerio, E; Pasquali, D; Origa, R; Serra, M; Campisi, S; Peluso, A; Renni, R; Cattoni, A; De Michele, E; Allò, M; Poggi, M; Ferrara, F; Di Concilio, R; Sportelli, F; Quarta, A; Putti, Mc; Notarangelo, Ld; Sau, A; Ladogana, S; Tartaglione, I; Picariello, S; Marcon, A; Sturiale, P; Roberti, D; Lazzarino, Ai; Perrotta, S. | HAEMATOLOGICA | - |
| Tricuspid-valve regurgitant jet velocity as a risk factor for death in β-Thalassemia | 1-gen-2022 | Derchi, Giorgio; Musallam, Khaled M.; Maria Pinto, Valeria; Graziadei, Giovanna; Giuditta, Marianna; Barella, Susanna; Origa, Raffaella; Casu, Gavino; Pasanisi, Annamaria; Longo, Filomena; Casale, Maddalena; Miceli, Roberta; Merella, Pierluigi; Gianesin, Barbara; Ameri, Pietro; Tartaglione, Immacolata; Perrotta, Silverio; Piga, Antonio; Domenica Cappellini, Maria; Gian Luca Forni, And | HAEMATOLOGICA | - |
| Mortality in β-thalassemia patients with confirmed pulmonary arterial hypertension on right heart catheterization | 1-gen-2022 | Pinto, V. M.; Musallam, K. M.; Derchi, G.; Graziadei, G.; Giuditta, M.; Origa, R.; Barella, S.; Casu, G.; Pasanisi, A.; Longo, F.; Casale, M.; Miceli, R.; Merella, P.; Tartaglione, I.; Piga, A.; Cappellini, M. D.; Gianesin, B.; Forni, G. L. | BLOOD | - |
| Good Clinical Practice of the Italian Society of Thalassemia and Haemoglobinopathies (SITE) for the Management of Endocrine Complications in Patients with Haemoglobinopathies | 1-gen-2022 | Casale, M; Baldini, Mi; Del Monte, P; Gigante, A; Grandone, A; Origa, R; Poggi, M; Gadda, F; Lai, R; Marchetti, M; Forni, Gl | JOURNAL OF CLINICAL MEDICINE | - |
| Safety and Efficacy of the New Combination Iron Chelation Regimens in Patients with Transfusion-Dependent Thalassemia and Severe Iron Overload | 1-gen-2022 | Origa, R.; Cinus, M.; Pilia, M. P.; Gianesin, B.; Zappu, A.; Orecchia, V.; Clemente, M. G.; Pitturru, C.; Denotti, A. R.; Corongiu, F.; Piras, S.; Barella, S. | JOURNAL OF CLINICAL MEDICINE | - |
| Transfusional Approach in Multi-Ethnic Sickle Cell Patients: Real-World Practice Data From a Multicenter Survey in Italy | 1-gen-2022 | Graziadei, G; De Franceschi, L; Sainati, L; Venturelli, D; Masera, N; Bonomo, P; Vassanelli, A; Casale, M; Lodi, G; Voi, V; Rigano, P; Pinto, Vm; Quota, A; Notarangelo, Ld; Russo, G; Allo, M; Rosso, R; D'Ascola, D; Facchini, E; Macchi, S; Arcioni, F; Bonetti, F; Rossi, E; Sau, A; Campisi, S; Colarusso, G; Giona, F; Lisi, R; Giordano, P; Boscarol, G; Filosa, A; Marktel, S; Maroni, P; Murgia, M; Origa, R; Longo, F; Bortolotti, M; Colombatti, R; Di Maggio, R; Mariani, R; Piperno, A; Corti, P; Fidone, C; Palazzi, G; Badalamenti, L; Gianesin, B; Piel, Fb; Forni, Gl | FRONTIERS IN MEDICINE | - |
| Changing patterns of thalassaemia in Italy: a WebThal perspective. | 1-gen-2021 | Longo, F; Corrieri, P; Origa, R; Barella, S; Sanna, Pmg; Bitti, Pp; Zuccarelli, A; Commendatore, Fv; Vitucci, A; Quarta, A; Lisi, R; Cappellini, Md; Massei, F; Forni, Gl; Piga, A. | BLOOD TRANSFUSION | - |
| Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm | 1-gen-2021 | Baronciani, D.; Casale, M.; De Franceschi, L.; Graziadei, G.; Longo, F.; Origa, R.; Rigano, P.; Pinto, V.; Marchetti, M.; Gigante, A.; Forni, G. L. | HEMASPHERE | - |
| Hemoglobinopathies and Cancer: Preliminary Results of an Italian Multicenter Experience | 1-gen-2021 | Origa, Raffaella; Longo, Filomena; Di Maggio, Rosario; Cassinerio, Elena; Maria Pinto, Valeria; Quarta, Antonella; Casale, Maddalena; Gianesin, Barbara; Barella, Susanna; Piolatto, Andrea; Maggio, Aurelio; Graziadei, Giovanna; Romano, Claudia; Perrotta, Silverio; Luca Forni, Gian | BLOOD | - |
| Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial | 1-gen-2020 | Maggio, A; Kattamis, A; Felisi, M; Reggiardo, G; El-Beshlawy, A; Bejaoui, M; Sherief, L; Christou, S; Cosmi, C; Della Pasqua, O; Del Vecchio, Gc; Filosa, A; Cuccia, L; Hassab, H; Kreka, M; Origa, R; Putti, Mc; Spino, M; Telfer, P; Tempesta, B; Vitrano, A; Tsang, Yc; Zaka, A; Tricta, F; Bonifazi, D; Ceci, A. | THE LANCET. HAEMATOLOGY | - |
| Two-year long safety and efficacy of deferasirox film-coated tablets in patients with thalassemia or lower/intermediate risk MDS: phase 3 results from a subset of patients previously treated with deferasirox in the ECLIPSE study | 1-gen-2020 | Tartaglione, I; Origa, R; Kattamis, A; Pfeilstöcker, M; Gunes, S; Crowe, S; Fagan, N; Vincenzi, B; Ruffo, Gb. | EXPERIMENTAL HEMATOLOGY & ONCOLOGY | - |
| A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia | 1-gen-2020 | Cappellini, Md; Viprakasit, V; Taher, At; Georgiev, P; Kuo, Khm; Coates, T; Voskaridou, E; Liew, Hk; Pazgal-Kobrowski, I; Forni, Gl; Perrotta, S; Khelif, A; Lal, A; Kattamis, A; Vlachaki, E; Origa, R; Aydinok, Y; Bejaoui, M; Ho, Pj; Chew, Lp; Bee, Pc; Lim, Sm; Lu, My; Tantiworawit, A; Ganeva, P; Gercheva, L; Shah, F; Neufeld, Ej; Thompson, A; Laadem, A; Shetty, Jk; Zou, J; Zhang, J; Miteva, D; Zinger, T; Linde, Pg; Sherman, Ml; Hermine, O; Porter, J; Piga, A; Believe, Investigators. | NEW ENGLAND JOURNAL OF MEDICINE | - |
| Selecting ß-Thalassemia Patients for Gene Therapy: A Decision-Making Algorithm | 1-gen-2019 | Baronciani, D; Casale, M; De Franceschi, L; Graziadei, G; Longo, F; Origa, R; Pinto, Vm; Rigano, P; Marchetti, M; Gigante, A; Angelucci, E; Cappellini, Md; Iolascon, A; Piga, A; Forni, Gl. | BLOOD | - |
| Influence of patient-reported outcomes on the treatment effect of deferasirox film-coated and dispersible tablet formulations in the ECLIPSE trial: A post hoc mediation analysis | 1-gen-2019 | Taher, At; Origa, R; Perrotta, S; Kouraklis, A; Belhoul, K; Huang, V; Han, J; Bruederle, A; Bobbili, P; Duh, Ms; Porter, Jb | AMERICAN JOURNAL OF HEMATOLOGY | - |
| Amustaline-glutathione pathogen-reduced red blood cell concentrates for transfusion-dependent thalassaemia | 1-gen-2019 | Aydinok, Y; Piga, A; Origa, R; Mufti, N; Erickson, A; North, A; Waldhaus, K; Ernst, C; Lin, Js; Huang, N; Benjamin, Rj; Corash, L. | BRITISH JOURNAL OF HAEMATOLOGY | - |
| Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia | 1-gen-2019 | Marktel, S; Scaramuzza, S; Cicalese, Mp; Giglio, F; Galimberti, S; Lidonnici, Mr; Calbi, V; Assanelli, A; Bernardo, Me; Rossi, C; Calabria, A; Milani, R; Gattillo, S; Benedicenti, F; Spinozzi, G; Aprile, A; Bergami, A; Casiraghi, M; Consiglieri, G; Masera, N; D'Angelo, E; Mirra, N; Origa, R; Tartaglione, I; Perrotta, S; Winter, R; Coppola, M; Viarengo, G; Santoleri, L; Graziadei, G; Gabaldo, M; Valsecchi, Mg; Montini, E; Naldini, L; Cappellini, Md; Ciceri, F; Aiuti, A; Ferrari, G. | NATURE MEDICINE | - |
| Risk factors for heart disease in transfusion-dependent thalassemia: serum ferritin revisited | 1-gen-2019 | Derchi, G; Dessì, C; Bina, P; Cappellini, Md; Piga, A; Perrotta, S; Tartaglione, I; Giuditta, M; Longo, F; Origa, R; Quarta, A; Pinto, V; Forni, Gl | INTERNAL AND EMERGENCY MEDICINE | - |
| Impact of the direct-acting antiviral agents (DAAs) on chronic hepatitis C in Sardinian patients with transfusion-dependent Thalassemia major | 1-gen-2019 | Ponti, Ml; Comitini, F; Murgia, D; Ganga, R; Canu, R; Dessì, C; Foschini, Ml; Leoni, G; Morittu, M; Perra, M; Pilia, Mp; Casini, Mr; Zappu, A; Origa, R. | DIGESTIVE AND LIVER DISEASE | - |
| Sotatercept, a novel transforming growth factor β ligand trap, improves anemia in β-thalassemia: a phase II, open-label, dose-finding study | 1-gen-2019 | Cappellini, Md; Porter, J; Origa, R; Forni, Gl; Voskaridou, E; Galactéros, F; Taher, At; Arlet, Jb; Ribeil, Ja; Garbowski, M; Graziadei, G; Brouzes, C; Semeraro, M; Laadem, A; Miteva, D; Zou, J; Sung, V; Zinger, T; Attie, Km; Hermine, O. | HAEMATOLOGICA | - |
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