title: The era of comparable life expectancy between thalassaemia major and intermedia: Is it time to revisit the major-intermedia dichotomy?
abstract: In the last few decades, the life expectancy of regularly transfused β-thalassaemia major (TM) patients has dramatically improved following the introduction of safe transfusion practices, iron chelation therapy, aggressive treatment of infections and improved management of cardiac complications. How such changes, especially those attributed to the introduction of iron chelation therapy, improved the survival of TM patients to approach those with β-thalassaemia intermedia (TI) remains unknown. Three hundred and seventy-nine patients with TM (n = 284, dead 40) and TI (n = 95, dead 13) were followed retrospectively since birth until 30 June 2015 or death. Kaplan-Meir curves showed statistically significant differences in TM and TI survival (P < 0·0001) before the introduction of iron chelation in 1965, which were no longer apparent after that date (P = 0·086), reducing the Hazard Ratio of death in TM compared to TI from 6·8 [95% confidence interval (CI) 2·6-17·5] before 1965 to 2·8 (95% CI 0·8-9·2). These findings suggest that, in the era of iron chelation therapy and improved survival for TM, the major-intermedia dichotomy needs to be revisited alongside future directions in general management and prevention for both conditions.
authors.affiliation: Campus of Haematology Franco e Piera Cutino - A.O.O.R. "Villa Sofia-Cervello", Palermo, Italy.
authors.forename: Angela
authors.initials: A
authors.lastname: Vitrano
authors.affiliation: Campus of Haematology Franco e Piera Cutino - A.O.O.R. "Villa Sofia-Cervello", Palermo, Italy.
authors.forename: Giuseppina
authors.initials: G
authors.lastname: Calvaruso
authors.affiliation: Ospedale microcitemico, Centro Talassemici Adulti, University of Cagliari, Cagliari, Italy.
authors.forename: Eliana
authors.initials: E
authors.lastname: Lai
authors.affiliation: Azienda Ospedaliera Università Policlinico "Vittorio Emanuele - P.O. Ferrarotto", Catania, Italy.
authors.forename: Grazia
authors.initials: G
authors.lastname: Colletta
authors.affiliation: U.O.S Talassemia P.O. Vittorio Emanuele, Gela (CL), Italy.
authors.forename: Alessandra
authors.initials: A
authors.lastname: Quota
authors.affiliation: U.O.S. di Talassemia P.O. "Giovanni Paolo II" Sciacca, Sciacca, Italy.
authors.forename: Calogera
authors.initials: C
authors.lastname: Gerardi
authors.affiliation: U.O. Genetica ed Immunologia Pediatrica, Policlinico "G. Martino", Messina, Italy.
authors.forename: Luciana
authors.initials: L
authors.lastname: Concetta Rigoli
authors.affiliation: Campus of Haematology Franco e Piera Cutino - A.O.O.R. "Villa Sofia-Cervello", Palermo, Italy.
authors.forename: Lorella
authors.initials: L
authors.lastname: Pitrolo
authors.affiliation: U.O.C. 7a Pediatria per le Emopatie Ereditarie, Centro di Prevenzione Diagnosi e Cura della Talassemia, A.O. "G. Di Cristina", Palermo, Italy.
authors.forename: Liana
authors.initials: L
authors.lastname: Cuccia
authors.affiliation: U.O.C. 7a Pediatria per le Emopatie Ereditarie, Centro di Prevenzione Diagnosi e Cura della Talassemia, A.O. "G. Di Cristina", Palermo, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Gagliardotto
authors.affiliation: U.O.D. Malattie Rare del Globulo Rosso, A.O.R.N. "A. Cardarelli", Napoli, Italy.
authors.forename: Aldo
authors.initials: A
authors.lastname: Filosa
authors.affiliation: U.O. Talassemia Ospedale Garibaldi, Catania, Italy.
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Caruso
authors.affiliation: U.O.C. Medicina Trasfusionale, A.O. "S. Giovanni di Dio", Agrigento, Italy.
authors.forename: Crocetta
authors.initials: C
authors.lastname: Argento
authors.affiliation: U.O.S. Talassemia, A.O. Umberto I, Siracusa, Italy.
authors.forename: Saveria
authors.initials: S
authors.lastname: Campisi
authors.affiliation: U.O.C. Ematologia, A.O. "S. ELIA", Caltanissetta, Italy.
authors.forename: Michele
authors.initials: M
authors.lastname: Rizzo
authors.affiliation: Centro Microcitemia, A.O. B.M.M., Reggio Calabria, Italy.
authors.forename: Luciano
authors.initials: L
authors.lastname: Prossomariti
authors.affiliation: U.O.C. Centro Trasfusionale, A.O. "M. Paternò Arezzo", Ragusa, Italy.
authors.forename: Carmelo
authors.initials: C
authors.lastname: Fidone
authors.affiliation: Campus of Haematology Franco e Piera Cutino - A.O.O.R. "Villa Sofia-Cervello", Palermo, Italy.
authors.forename: Maria
authors.initials: M
authors.lastname: Fustaneo
authors.affiliation: Campus of Haematology Franco e Piera Cutino - A.O.O.R. "Villa Sofia-Cervello", Palermo, Italy.
authors.forename: Rosario
authors.initials: R
authors.lastname: Di Maggio
authors.affiliation: Campus of Haematology Franco e Piera Cutino - A.O.O.R. "Villa Sofia-Cervello", Palermo, Italy.
authors.forename: Aurelio
authors.initials: A
authors.lastname: Maggio
mesh_terms: D000328:Adult; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008017:Life Expectancy; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D015996:Survival Rate; D055815:Young Adult; D017086:beta-Thalassemia / Q000145:classification* / Q000453:epidemiology / Q000401:mortality* / Q000628:therapy
keywords: classification; severity; survival; thalassaemia