Long-term survival rates of thalassemia patients following hematopoietic stem cell transplantation: A systematic review and meta-analysis.

BACKGROUND

Hematopoietic stem cell transplantation is the sole therapeutic approach that can provide a complete cure for thalassemia. However, this procedure is associated with complications that may have life-threatening consequences. To date, long-term survival outcomes after transplantation in thalassemia have not been systematically synthesized. This study aims to specifically evaluate long-term overall survival in patients with thalassemia undergoing hematopoietic stem cell transplantation.

METHODS

A comprehensive search was conducted across multiple databases, including PubMed, CENTRAL, Europe PMC (incorporating medRxiv and bioRxiv), EBSCOHost (Medline), and ProQuest. The search spanned from the inception of each database through July 10, 2024, using a combination of predefined keywords: 'Hematopoietic Stem Cell Transplantation', 'Thalassemia', 'Survival Rates', and synonyms. Risk of bias was assessed using the Quality in Prognosis Studies (QUIPS) tool. Heterogeneity was evaluated via I2 statistics, while pooled effect estimates were calculated using the DerSimonian-Laird inverse-variance random-effects model. Statistical significance was defined as p < 0.05. Additionally, a leave-one-out sensitivity analysis was performed to ensure the robustness of the findings.

RESULTS

Out of an initial 690 records identified, four articles involving a total of 616 transplanted thalassemia patients were included in this study. The survival rates were 86.8% (95% CI: 80.1%- 92.3%) in 15 years, 89.2% (95% CI: 82.2%-96.2%) in 20 years, 82.6% (95% CI: 79.9%-85.3%) in 30 years, and 81.4% (95% CI: 74.5%-88.9%) in 39 years. The pooled survival rate was 85% (95% CI: 81%-89%; I2 = 40%). The pooled survival rate showed no significant differences in leave-one-out sensitivity analysis.

CONCLUSION

The present meta-analysis shows relatively high long-term overall survival rates in patients with thalassemia after hematopoietic stem cell transplantation. Nonetheless, the relatively small patient population, heterogeneity in anti-thymocyte globulin implementation, and potential confounding risks intrinsic to the study necessitate cautious interpretation of the findings.