The present meta-analysis was conducted to determine the efficacy of
hydroxyurea in patients with transfusion dependent major β-
thalassemia. The present meta-analysis was conducted following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (
PRISMA) and Meta-analyses of Observational Studies in Epidemiology (MOOSE) guidelines. A systematic search was carried out to evaluate the efficacy of
hydroxyurea in patients with transfusion-dependent B-thalassaemia using electronic databases, including MEDLINE, Cochrane Central Register of Controlled Trials, and EMBASE. The keywords used to search for relevant studies included "
hydroxyurea", "
thalassemia", "transfusion-dependent", and "efficacy". Outcomes assessed in the present meta-analysis included transfusion in one year and intervals between transfusions (in days). Other outcomes assessed in the present meta-analysis were
fetal hemoglobin (%),
hemoglobin (%), and
ferritin levels (ng/dl). Total of five studies were included in the analysis enrolling 294 patients with major B-
thalassemia. The pooled analysis reported that the mean interval between transfusions was significantly higher in patients receiving
hydroxyurea compared to those not receiving
hydroxyurea (mean deviation {MD}: 10.07, 95% CI: 2.16, 17.99). Hemoglobin was significantly higher in patients receiving
hydroxyurea compared to its counterparts (MD: 1.71, 95% CI: 0.84, 2.57). Patients receiving
hydroxyurea had significantly lower
ferritin levels compared to those not receiving
hydroxyurea (MD: -299.65, 95% CI: -518.35, -80.96). These findings suggest that
hydroxyurea may be a promising and cost-effective alternative to
blood transfusions and
iron chelation therapies for
beta-thalassemia patients. However, the authors noted that further randomized controlled trials are needed to validate these findings and to determine the optimal dosages and treatment regimens for
hydroxyurea in this patient population.