Objective: Chronic blood transfusion remains the most feasible therapeutic option for lifelong transfusion-dependent β-thalassemia (lifelong TDβT). However, it is associated with serious risks and complications. Hydroxyurea (HU), an oral chemotherapeutic drug, is expected to increase hemoglobin levels, thereby minimizing the burden of blood transfusion and its complications. Growing literature over the last twenty years suggests promising results of the use HU in lifelong TDβT; however, its role and safety remain unanswered questions. The objective of this study was to evaluate the clinical efficacy and safety of HU in patients with lifelong TDβT.
Methods: MEDLINE, EMBASE, Cochrane databases, and major preceding conferences for studies that assessed HU in lifelong TDβT patients were searched. The effect size was estimated as a proportion (responder/sample size).
Results: Eleven observational studies, collectively involving 859 patients, fulfilled eligibility criteria. HU was associated with a significant decrease in transfusion need with complete and overall (≥50%) response rates of 26% [95% confidence interval (CI), 13-41%] and 60% (95% CI, 41-78%), respectively. No serious adverse effects were reported. All of the studies had several limitations, such as lack of a comparison group.
Conclusion: HU appears to be effective, well tolerated; however, large randomized clinical trials should be done to confirm such findings.
Keywords: Blood transfusion; hydroxyurea; lifelong transfusion-dependent β-thalassemia; meta-analysis; β-thalassemia major.