Overall and complication‐free survival in a large cohort of patients with β ‐thalassemia major followed over 50 years

Autor: Gian Luca, Forni, Barbara, Gianesin, Khaled M, Musallam, Filomena, Longo, Rosamaria, Rosso, Roberto, Lisi, Maria Rita, Gamberini, Valeria Maria, Pinto, Giovanna, Graziadei, Angelantonio, Vitucci, Federico, Bonetti, Pellegrino, Musto, Antonio, Piga, Maria Domenica, Cappellini, Caterina, Borgna-Pignatti
Rok vydání: 2023
Předmět:
Zdroj: American Journal of Hematology. 98:381-387
ISSN: 1096-8652
0361-8609
DOI: 10.1002/ajh.26798
Popis: We report data on survival and complications for a longitudinal cohort of 709 transfusion-dependent β-thalassemia major patients (51.1% males) born between 1970 and 1997 and followed through 2020 at seven major centers in Italy. Overall survival probability at 30 years was 83.6% (95%CI: 78.5-89.1) in the oldest birth cohort (1970-1974) compared with 93.3% (95%CI: 88.6-98.3) in the youngest birth cohort (1985-1997) (p = 0.073). Females showed better survival than males (p = 0.022). There were a total of 93 deaths at a median age of 23.2 years with the most frequent disease-related causes being heart disease (n = 53), bone marrow transplant (BMT) complication (n = 10), infection (n = 8), liver disease (n = 4), cancer (n = 3), thromboembolism (n = 2) and severe anemia (n = 1). There was a steady decline in the number of deaths due to heart disease from the year 2000 onwards and no death from BMT was observed after the year 2010. A progressive decrease in the median age of BMT was observed in younger birth cohorts (p 0.001). A total of 480 (67.7%) patients developed ≥1 complication. Patients in younger birth cohorts demonstrated better complication-free survival (p 0.001) which was comparable between sexes (p = 0.230). Independent risk factors for death in multivariate analysis included heart disease (HR: 4.63, 95%CI: 1.78-12.1, p = 0.002), serum ferritin1000 ng/mL (HR: 15.5, 95%CI: 3.52-68.2, p 0.001), male sex (HR: 2.75, 95%CI: 0.89-8.45, p = 0.078), and splenectomy (HR: 6.97, 95%CI: 0.90-54.0, p 0.063). Survival in patients with β-thalassemia major continues to improve with adequate access to care, best practice sharing, continued research, and collaboration between centers.
Databáze: OpenAIRE
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