In:
American Journal of Hematology, Wiley, Vol. 98, No. 3 ( 2023-03), p. 381-387
Abstract:
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 ferritin 〉 1000 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.
Type of Medium:
Online Resource
ISSN:
0361-8609
,
1096-8652
Language:
English
Publisher:
Wiley
Publication Date:
2023
detail.hit.zdb_id:
1492749-4
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